A Phase 1 interventional study of APX005M treatment for recurrent or refractory primary malignant CNS tumor patients and APX005M treatment for newly diagnosed DIPG patients in Glioblastoma Multiforme, High-grade Astrocytoma Not Otherwise Specified (NOS) and CNS Primary Tumor, Not Otherwise Specified (NOS), sponsored by Pediatric Brain Tumor Consortium. Completed at 11 sites in United States. Open to participants aged 1 Year to 21 Years. Per ClinicalTrials.gov, last updated 2026-08-13.
Sponsored by Pediatric Brain Tumor Consortium · Phase 1, Interventional, and Treatment
This phase I trial studies the side effects and best dose of APX005M in treating younger patients with primary malignant central nervous system tumor that is growing, spreading, or getting worse (progressive), or newly diagnosed diffuse intrinsic pontine glioma. APX005M can trigger activation of B cells, monocytes, and dendritic cells and stimulate cytokine release from lymphocytes and monocytes. APX005M can mediate a direct cytotoxic effect on CD40+ tumor cells.
This is a multicenter phase I trial of APX005M in patients with recurrent or refractory primary malignant central nervous system tumor, or newly diagnosed diffuse intrinsic pontine glioma.
APX005M is a humanized IgG1κ mAb that binds to CD40. APX005M binds to both human and cynomolgus monkey CD40 with high affinity, triggering activation of B cells, monocytes, and dendritic cells and stimulating cytokine release from both human and monkey lymphocytes and monocytes. APX005M does not bind to mouse or rat CD40. CD40 is also expressed on many human tumor cells, and APX005M can mediate a direct cytotoxic effect on CD40+ tumor cells.
Activation of CD40 on tumor cells results in tumor cell apoptosis and inhibition of tumor growth. CD40 agonistic antibodies have demonstrated potent antitumor immune response stimulation in both animal models and cancer patients. Due to its action on both immune and tumor cells, CD40 has been studied as a target for novel cancer immunotherapy.
Apexigen has declared the adult recommended phase 2 dose to be 0.3 mg/kg because no dose limiting toxicities were encountered at that dose and the pharmacodynamic profile was similar to the 1 mg/kg maximally tolerated dose. This phase 1 clinical trial is to study APX005M in children with central nervous system tumors.
2,132 studies on the registry are indexed under Glioblastoma; 464 are open to participants now.
This study's enrollment of 32 is below the median of 36 across 1,753 interventional studies indexed under Glioblastoma.
Browse Glioblastoma studies →Pediatric Brain Tumor Consortium is the lead sponsor of 31 studies on the registry; none are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 5 (100%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Stratum 2: Newly diagnosed DIPG patients (on-hold until pediatric RP2D has been established in Stratum 1) Patients with diffuse intrinsic pontine gliomas (DIPGs) will be eligible 6 to 14 weeks post-completion of radiation therapy if they do not have any evidence of progression. Patients with newly diagnosed DIPGs, defined as tumors with a pontine epicenter and diffuse involvement of 2/3 or more of the pons, are eligible without histologic confirmation. Patients with pontine tumors that do not meet these criteria or not considered to be typical intrinsic pontine gliomas will only be eligible if the tumors have been biopsied and (1) are proven to be an anaplastic astrocytoma, glioblastoma multiforme, gliosarcoma, anaplastic mixed glioma or fibrillary astrocytoma or (2) have a histone mutation typically seen in DIPG. Patients with disseminated disease are not eligible, and MRI of spine must be performed if disseminated disease is suspected by the treating physician.
Stratum 2: Patients with DIPG who have pre-trial tumor tissue available are requested to submit tissue; however, this is not required for eligibility.
Refractory/Recurrent patients Patients must have recovered from the acute treatment related toxicities (defined as \< grade 1) of all prior chemotherapy, immunotherapy, radiotherapy or any other treatment modality prior to entering this study.
Myelosuppressive chemotherapy -- Patients must have received their last dose of known myelosuppressive anticancer therapy at least 21 days prior to enrollment or at least 42 days if nitrosourea.
Biological agent: Patient must have recovered from any acute toxicity potentially related to the agent and received their last dose of the biologic agent ≥ 7 days prior to study enrollment.
For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur.
Monoclonal antibody treatment and agents with known prolonged half-lives: At least three half-lives must have elapsed prior to enrollment.
Radiation --
Patients must have had their last fraction of:
Craniospinal irradiation (>24Gy) or total body irradiation or radiation to greater than 50% of pelvis > 3 months prior to enrollment.
Focal irradiation >6 weeks prior to enrollment Local palliative irradiation (small port) ≥4 weeks
Stem Cell Transplant -- Patient must be ≥ 6 months since autologous bone marrow/stem cell transplant prior to enrollment and have above 200/mm3.
Surgery -- Patients must be at least 4 weeks (28 days) from major surgery and fully recovered from all acute effects of prior surgical intervention.
Patients with seizure disorders may be enrolled if seizures are well controlled.
Patients must have adequate organ and bone marrow function as defined below:
(ANC) ≥ 1.0 x 109 cells/ L ≥ 100 x 109 cells/L (unsupported, defined as no platelet transfusion within 7 days) ≥ 8 g/dL (may receive transfusions) Total ≤1.5 times institutional (ULN) (SGOT)/ALT(SGPT) ≤ 3 x institutional upper limit of normal (ULN) Albumin ≥ 3 g/dl Serum creatinine based on age/gender as noted below. Patients that do not meet the criteria below but have a 24 hour or (radioisotope or iothalamate) ≥ 70 mL/min/1.73 m2 are eligible.
Age Maximum Serum Creatinine (mg/dL) 1 to \< 2 years 0.6, 0.6 (M, F); 2 to \< 6 years 0.8, 0.8 (M, F); 6 to \< 10 years 1, 1 (M, F); 10 to \< 13 years 1.2, 1.2 (M, F); 13 to \< 16 years 1.5, 1.4 (M, F); ≥ 16 years 1.7, 1.4 (M, F).
Exclusion Criteria:
Patients with a history of any other , except patients with a secondary brain tumor if the patient's first malignancy has been in remission for at least 5 years from the end of treatment.
Patients requiring systemic treatment with either (greater than physiologic replacement, defined as dexamethasone 0.75 mg/m2/day) or other medications within 14 days of study drug administration will be excluded. However, patients who require intermittent use of bronchodilators or local steroid injections will not be excluded from the study. Please see section 5.3 for a list of acceptable and unacceptable concomitant medications as well as reporting requirements.
Patients with bulky tumor on imaging are ineligible. Bulky tumor is defined as:
Tumor with any evidence of uncal herniation or midline shift Tumor that in the opinion of the site investigator, shows significant mass effect
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
The number of CD4 cells, a type of white blood cell, in a given amount of blood. It is used to follow how well the immune system is doing in HIV.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Relapsed and refractory
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Histologically or cytologically confirmed
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CD4 count
The number of CD4 cells, a type of white blood cell, in a given amount of blood. It is used to follow how well the immune system is doing in HIV.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The recurrent, progressive, or refractory primary malignant non-brainstem CNS tumor patients will be treated with APX005M.
Biological: APX005M treatment for recurrent or refractory primary malignant CNS tumor patients
The newly diagnosed diffuse intrinsic pontine gliomas (DIPGs) patients will be treated with APX005M.
Biological: APX005M treatment for newly diagnosed DIPG patients
APX005M dosing will begin at 0.1 mg/kg, the APX005M dose may be increased (0.3, 0.45, 0.6 mg/kg) or decreased (0.03 mg/kg) in subsequent cohorts until the maximum tolerated dose (MTD) is reached or until dose level 3 (0.6 mg/kg) is complete without the MTD being defined. APX005M will be administered at the assigned dose level every 21 days (3 weeks). Patients may continue to receive APX005M for 36 courses (approximately 2 years) or until disease progression, unacceptable toxicity or death, whichever occurs first.
The starting dose of APX005M for the DIPG patients will be one dose level below the recommended phase II dose (RP2D) determined in Stratum 1 patients. The dose may be decreased or increased to the RP2D established in Stratum 1. APX005M will be administered at the assigned dose level every 21 days (3 weeks). Patients may continue to receive APX005M for 36 courses (approximately 2 years) or until disease progression, unacceptable toxicity or death, whichever occurs first.
Number of Patients Who Experienced Dose-limiting Toxicities (DLTs)
DLTs were defined as adverse events (AE) at least possibly attributed to APX005M that occurred during the first 2 courses (6 weeks) following APX005M administration. DLTs included any APX005M-related AE that led to dose reduction or permanent cessation of therapy or resulted in a treatment delay \>2 weeks. Hematologic DLTs included grade 3 neutropenia with fever, any grade 4 hematologic toxicity except lymphopenia, and grade 3 thrombocytopenia on 2 separate days or requiring platelet transfusion on 2 days within a 7-day period. Non-hematologic DLTs included any grade 4 non-hematologic toxicity, grade 3 or higher cytokine release syndrome, or any grade 3 non-hematologic toxicity with some exceptions such as grade 3 nausea/vomiting \<5 days or grade 3 diarrhea that responded to treatment within 5 days.
Time frame: 6 weeks
Maximum Tolerated Dose (MTD) and/or Recommended Phase II Dose (RP2D) of APX005M in Stratum 1
Based on the 3+3 design, the MTD of APX005M was empirically defined as the highest dose level at which six patients were treated with at most one patient experiencing a DLT, and the next higher dose level was determined to be too toxic. A total of 12 subjects were to be treated at the MTD/RP2D to further define the toxicity profile. Stratum 1 consisted of patients with recurrent or refractory primary malignant central nervous system tumors.
Time frame: 6 weeks (first 2 courses of treatment)
Maximum Tolerated Dose (MTD) and/or Recommended Phase II Dose (RP2D) of APX005M in Stratum 2
The starting dose level for Stratum 2 was one dose level below the RP2D determined in Stratum 1. If there were no dose-limiting toxicities in the first 3 patients enrolled on Stratum 2, then we escalated to the Stratum 1 RP2D and could treat 6 diffuse intrinsic pontine glioma (DIPG) patients simultaneously. The RP2D was defined as the dose level at which 6 patients were treated with no more than one dose-limiting toxicity. Stratum 2 patients were those with newly diagnosed diffuse intrinsic pontine glioma (DIPG).
Time frame: 6 weeks (first 2 courses of treatment)
Serum Concentration of APX005M
Serial blood samples for APX005M pharmacokinetic studies were collected during courses 1 and 2 at pre-dose, at the end of infusion, and at 4, 24 ± 1 (day 2), and 168 ± 4 hours (day 8) from the start of infusion in that course. During courses 3 and 4, samples were obtained pre-dose and end of induction. Serum concentrations of sotigalimab were measured using a validated electrochemiluminescent (ECL) immunoassay (Method ICD 853 v1.00). Mean serum concentrations with standard deviations were calculated using Phoenix® WinNonlin® v.8.4. Concentrations below the limit of quantitation (0.010 μg/mL) were replaced with 0 to calculate means and SDs.
Time frame: Up to 12 weeks from start of study drug
Overall Survival for Stratum 2 (DIPG) Patients
Overall survival was defined as the time interval from treatment initiation to death from any cause or to date of last follow-up for survivors. Survival was estimated using the method of Kaplan and Meier. The 1-year estimate of survival is reported with a 95% confidence interval; estimates are reported by dose level. Stratum 2 patients were those with newly diagnosed diffuse intrinsic pontine glioma (DIPG).
Time frame: 1 year
Progression-free Survival for Stratum 2 (DIPG) Patients
Progression-free survival (PFS) was defined as the time interval from treatment initiation to date of first event (relapsed or progressive disease based on imaging or clinical progression or death from any cause) or to the date of last follow-up for patients without events. PFS was estimated using the method of Kaplan and Meier. The 1-year estimate of PFS is reported with a 95% confidence interval. PFS estimates were reported separately by dose level. Stratum 2 patients were those with newly diagnosed diffuse intrinsic pontine glioma (DIPG).
Time frame: 1 year
Overall Response Rate for Stratum 2 (DIPG) Patients
Complete or partial responses were considered responses. Response was evaluated by imaging or clinical progression. The response rate (percentage of participants with responses) is reported with a 95% Blyth-Still-Casella confidence interval. Response rates are reported separately by dose level.
Time frame: Up to 2 years
Duration of Response for Stratum 2 (DIPG) Patients
Complete or partial responses were considered responses. Response was evaluated by imaging or clinical progression. Duration of response was measured from the time measurement criteria are met for complete or partial response until the first date that recurrent or progressive disease was objectively documented.
Time frame: Up to 2 years
Progression-free Survival for Stratum 1 (Recurrent/Refractory) Patients
Progression-free survival (PFS) was defined as the time interval from treatment initiation to date of first event (relapsed or progressive disease based on imaging or clinical progression or death from any cause) or to the date of last follow-up for patients without events. PFS was estimated using the method of Kaplan and Meier. The 1-year estimate of PFS is reported with a 95% confidence interval. PFS estimates were reported separately by dose level. Stratum 1 patients were those with recurrent or refractory primary malignant CNS tumors.
Time frame: 1 year
Incidence of Anti-APX005M Antibodies
Serial blood samples for anti-drug-antibodies (ADA) were to be collected prior to dosing on courses 1, 2, 3, and 4, then every third course (courses 7 and 10), and then every 4 courses (courses 14, 18, 22, 26, 30, 34) until the end of therapy visit and collected into serum tubes. Percentages of patients with positive anti-drug antibodies with a 95% Blyth-Still-Casella confidence interval are reported by time point.
Time frame: Prior to dosing on courses 1, 2, 3, 4, 7, 10, 14, 18, 22, 26, 30, and 34 and at the end of therapy (up to end of course 36)
Concentration of the Cytokine Tumor Necrosis Factor-alpha (TNF-alpha)
The cytokine was quantitated using V-PLEX Human Proinflammatory Panel, a multiplex immunoassay kit developed by Meso Scale Diagnostics (MSD). A four-parameter logistic fit calibration curve was generated and used to calculate concentrations in tested samples. Analyses were performed at pre-treatment and 1 week following the first dose of drug and additionally at 2, 3, 6, and 9 weeks following the course 1 drug dose if feasible. The mean concentration of TNF-alpha in pg/ml was reported with a standard deviation. When only one patient was analyzed, the standard deviation was reported as 0 (rather than undefined).
Time frame: Pre-treatment (week 0), 1 week, 2 week, 3 weeks, 6 weeks and 9 weeks post-treatment
Concentration of the Cytokine Interleukin-8 (IL-8)
The cytokine was quantitated using V-PLEX Human Proinflammatory Panel, a multiplex immunoassay kit developed by Meso Scale Diagnostics (MSD). A four-parameter logistic fit calibration curve was generated and used to calculate concentrations in tested samples. Analyses were performed at pre-treatment and 1 week following the first dose of drug and additionally at 2, 3, 6, and 9 weeks following the course 1 drug dose if feasible. The mean concentration of IL-8 in pg/ml was reported with a standard deviation. When only 1 participant analyzed, the standard deviation was reported as 0 (rather than undefined).
Time frame: Pre-treatment (week 0), 1 week, 2 week, 3 weeks, 6 weeks and 9 weeks post-treatment
Tumor Mutation Burden Based on Whole Exome Sequencing of Tumor Tissue
Tumor mutation burden was defined as the number of somatic mutations per megabase. Average tumor mutation burden was reported with a standard deviation. When only one patient was analyzed, the standard deviation was reported as 0 rather than undefined.
Time frame: Day 0 of treatment
Tumor Mutation Burden Based on RNA Sequencing of Tumor Tissue
Tumor mutation burden was defined as the number of somatic mutations per megabase. Average tumor mutation burden was reported with a standard deviation. When only one patient was analyzed, the standard deviation was reported as 0 rather than undefined.
Time frame: Day 0 of treatment
Mutation Burden Based on TCR Sequencing
Mutation burden as detected by comparing the TCR sequencing of tumor tissue and/or PBMC. Mean numbers of clonotypes were reported by time point with standard deviations.
Time frame: Day 0 and Days 7, 14, and 21 after the start of treatment
Patients ≥1 and ≤21 years of age with recurrent or refractory primary malignant central nervous system (CNS) tumors (Stratum 1) or newly diagnosed diffuse intrinsic pontine glioma (DIPG) (Stratum 2) were enrolled at Pediatric Brain Tumor Consortium (PBTC) member institutions. The first patient was enrolled on 3/1/2018 and the last patient was enrolled on 1/20/2023. Accrual was closed after the maximum tolerated dose/recommended phase II dose was determined for both strata.
| Milestone | Stratum 1, Dose Level 1 | Stratum 1, Dose Level 2 | Stratum 1, Dose Level 3 | Stratum 2, Dose Level 2 | Stratum 2, Dose Level 3 |
|---|---|---|---|---|---|
| Started | 3 | 3 | 15 | 6 | 5 |
| Completed | 0 | 0 | 0 | 0 | 0 |
| Not completed | 3 | 3 | 15 | 6 | 5 |
| Withdrew: Lack of efficacy | 3 | 3 | 13 | 6 | 1 |
| Withdrew: Adverse event | 0 | 0 | 1 | 0 | 4 |
| Withdrew: Subject deemed ineligible after enrollment | 0 | 0 | 1 | 0 | 0 |
DLTs were defined as adverse events (AE) at least possibly attributed to APX005M that occurred during the first 2 courses (6 weeks) following APX005M administration. DLTs included any APX005M-related AE that led to dose reduction or permanent cessation of therapy or resulted in a treatment delay \>2 weeks. Hematologic DLTs included grade 3 neutropenia with fever, any grade 4 hematologic toxicity except lymphopenia, and grade 3 thrombocytopenia on 2 separate days or requiring platelet transfusion on 2 days within a 7-day period. Non-hematologic DLTs included any grade 4 non-hematologic toxicity, grade 3 or higher cytokine release syndrome, or any grade 3 non-hematologic toxicity with some exceptions such as grade 3 nausea/vomiting \<5 days or grade 3 diarrhea that responded to treatment within 5 days.
| Participants | Stratum 1, Dose Level 1 | Stratum 1, Dose Level 2 | Stratum 1, Dose Level 3 | Stratum 2, Dose Level 2 | Stratum 2, Dose Level 3 |
|---|---|---|---|---|---|
| Number of Patients Who Experienced Dose-limiting Toxicities (DLTs) | 0 | 0 | 2 | 0 | 3 |
Based on the 3+3 design, the MTD of APX005M was empirically defined as the highest dose level at which six patients were treated with at most one patient experiencing a DLT, and the next higher dose level was determined to be too toxic. A total of 12 subjects were to be treated at the MTD/RP2D to further define the toxicity profile. Stratum 1 consisted of patients with recurrent or refractory primary malignant central nervous system tumors.
| mg/kg | Stratum 1 |
|---|---|
| Maximum Tolerated Dose (MTD) and/or Recommended Phase II Dose (RP2D) of APX005M in Stratum 1 | 0.6 |
The starting dose level for Stratum 2 was one dose level below the RP2D determined in Stratum 1. If there were no dose-limiting toxicities in the first 3 patients enrolled on Stratum 2, then we escalated to the Stratum 1 RP2D and could treat 6 diffuse intrinsic pontine glioma (DIPG) patients simultaneously. The RP2D was defined as the dose level at which 6 patients were treated with no more than one dose-limiting toxicity. Stratum 2 patients were those with newly diagnosed diffuse intrinsic pontine glioma (DIPG).
| mg/kg | Stratum 2 |
|---|---|
| Maximum Tolerated Dose (MTD) and/or Recommended Phase II Dose (RP2D) of APX005M in Stratum 2 | 0.3 |
Serial blood samples for APX005M pharmacokinetic studies were collected during courses 1 and 2 at pre-dose, at the end of infusion, and at 4, 24 ± 1 (day 2), and 168 ± 4 hours (day 8) from the start of infusion in that course. During courses 3 and 4, samples were obtained pre-dose and end of induction. Serum concentrations of sotigalimab were measured using a validated electrochemiluminescent (ECL) immunoassay (Method ICD 853 v1.00). Mean serum concentrations with standard deviations were calculated using Phoenix® WinNonlin® v.8.4. Concentrations below the limit of quantitation (0.010 μg/mL) were replaced with 0 to calculate means and SDs.
| μg/mL | Stratum 1, Dose Level 1 | Stratum 1, Dose Level 2 | Stratum 1, Dose Level 3 | Stratum 2, Dose Level 2 | Stratum 2, Dose Level 3 |
|---|---|---|---|---|---|
| Course 1, pre-dose | 0 ± 0 | 0 ± 0 | 0 ± 0 | 0 ± 0 | 0 ± 0 |
| Course 1, 1 hour | 0.120 ± 0.115 | 3.95 ± 0.526 | 9.15 ± 2.98 | 3.49 ± 0.984 | 9.87 ± 1.95 |
| Course 1, 4 hours | 0.0357 ± 0 | 2.29 ± 0.349 | 6.64 ± 1.93 | 2.12 ± 1.06 | 7.88 ± 0.280 |
| Course 1, 24 hours | 0 ± 0 | 0 ± 0 | 0.851 ± 1.44 | 0 ± 0 | 0.0136 ± 0.0137 |
| Course 1, 168 hours | 0 ± 0 | 0 ± 0 | 0 ± 0 | 0 ± 0 | 0 ± 0 |
| Course 2, pre-dose | 0 ± 0 | 1.26 ± 2.19 | 0 ± 0 | 0 ± 0 | 0 ± 0 |
| Course 2, 1 hour | 0.148 ± 0.135 | 4.10 ± 0.247 | 8.47 ± 2.27 | 3.23 ± 1.48 | 9.63 ± 2.22 |
| Course 2, 4 hours | 0.032 ± 0.0253 | 2.66 ± 0.140 | 6.13 ± 1.87 | 1.55 ± 0.959 | 7.41 ± 3.15 |
| Course 2, 24 hours | 0 ± 0 | 0 ± 0 | 0.593 ± 0.872 | 0 ± 0 | 0.0317 ± 0.0448 |
| Course 2, 168 hours | 0 ± 0 | 0 ± 0 | 0 ± 0 | 0 ± 0 | 0 ± 0 |
| Course 3, pre-dose | 0 ± 0 | 0 ± 0 | 0 ± 0 | 0 ± 0 | 0 ± 0 |
| Course 3, 1 hour | 0.198 ± 0 | 3.54 ± 0.721 | 7.33 ± 1.98 | 1.89 ± 1 | 9.53 ± 1.80 |
| Course 4, pre-dose | 0 ± 0 | 0 ± 0 | 0 ± 0 | 0 ± 0 | 0 ± 0 |
| Course 4, 1 hour | 0.0114 ± 0 | 3.98 ± 0 | 8.82 ± 3.11 | 1.26 ± 1.17 | 1.84 ± 0 |
Overall survival was defined as the time interval from treatment initiation to death from any cause or to date of last follow-up for survivors. Survival was estimated using the method of Kaplan and Meier. The 1-year estimate of survival is reported with a 95% confidence interval; estimates are reported by dose level. Stratum 2 patients were those with newly diagnosed diffuse intrinsic pontine glioma (DIPG).
| Percent probability | Stratum 2, Dose Level 2 | Stratum 2, Dose Level 3 |
|---|---|---|
| Overall Survival for Stratum 2 (DIPG) Patients | 40.0 (4.9 to 75.1) | 25.0 (0.0 to 55.0) |
Progression-free survival (PFS) was defined as the time interval from treatment initiation to date of first event (relapsed or progressive disease based on imaging or clinical progression or death from any cause) or to the date of last follow-up for patients without events. PFS was estimated using the method of Kaplan and Meier. The 1-year estimate of PFS is reported with a 95% confidence interval. PFS estimates were reported separately by dose level. Stratum 2 patients were those with newly diagnosed diffuse intrinsic pontine glioma (DIPG).
| Percent probability | Stratum 2, Dose Level 2 | Stratum 2, Dose Level 3 |
|---|---|---|
| Progression-free Survival for Stratum 2 (DIPG) Patients | 16.7 (0.0 to 37.9) | 26.7 (0.0 to 58.3) |
Complete or partial responses were considered responses. Response was evaluated by imaging or clinical progression. The response rate (percentage of participants with responses) is reported with a 95% Blyth-Still-Casella confidence interval. Response rates are reported separately by dose level.
| percentage | Stratum 2, Dose Level 2 | Stratum 2, Dose Level 3 |
|---|---|---|
| Overall Response Rate for Stratum 2 (DIPG) Patients | 0 (0.0 to 40.2) | 0 (0.0 to 63.2) |
Complete or partial responses were considered responses. Response was evaluated by imaging or clinical progression. Duration of response was measured from the time measurement criteria are met for complete or partial response until the first date that recurrent or progressive disease was objectively documented.
No measurements were reported for this outcome.
Progression-free survival (PFS) was defined as the time interval from treatment initiation to date of first event (relapsed or progressive disease based on imaging or clinical progression or death from any cause) or to the date of last follow-up for patients without events. PFS was estimated using the method of Kaplan and Meier. The 1-year estimate of PFS is reported with a 95% confidence interval. PFS estimates were reported separately by dose level. Stratum 1 patients were those with recurrent or refractory primary malignant CNS tumors.
| Percent probability | Stratum 1, Dose Level 1 | Stratum 1, Dose Level 2 | Stratum 1, Dose Level 3 |
|---|---|---|---|
| Progression-free Survival for Stratum 1 (Recurrent/Refractory) Patients | 0.00 (0 to 0) | 0 (0 to 0) | 10.7 (0.0 to 59.9) |
Serial blood samples for anti-drug-antibodies (ADA) were to be collected prior to dosing on courses 1, 2, 3, and 4, then every third course (courses 7 and 10), and then every 4 courses (courses 14, 18, 22, 26, 30, 34) until the end of therapy visit and collected into serum tubes. Percentages of patients with positive anti-drug antibodies with a 95% Blyth-Still-Casella confidence interval are reported by time point.
| percentage of patients with positive ADA | Stratum 1, Dose Level 1 | Stratum 1, Dose Level 2 | Stratum 1, Dose Level 3 | Stratum 2, Dose Level 2 | Stratum 2, Dose Level 3 |
|---|---|---|---|---|---|
| Prior to Course 1 | 0 (0 to 77.64) | 0 (0 to 63.16) | 8.33 (0.43 to 34.89) | 0 (0 to 40.19) | 0 (0 to 52.71) |
| Prior to Course 2 | 33.33 (1.70 to 86.46) | 0 (0 to 63.16) | 0 (0 to 24.95) | 16.67 (0.85 to 59.81) | 0 (0 to 77.64) |
| Prior to Course 3 | 100 (50.0 to 100.0) | 0 (0 to 63.16) | 50.0 (9.76 to 90.24) | 50.0 (9.76 to 90.24) | 0 (0 to 77.64) |
| Prior to Course 4 | 100 (50.0 to 100.0) | 100 (50.0 to 100.0) | 50.0 (9.76 to 90.24) | 75 (24.86 to 98.73) | 0 (0 to 95.0) |
| Prior to Course 7 | — | 100 (50.0 to 100.0) | 50 (2.52 to 97.47) | 100 (50.0 to 100.0) | 0 (0 to 95.0) |
| Prior to Course 10 | — | — | 50 (2.52 to 97.47) | 100 (50.0 to 100.0) | — |
| Prior to Course 14 | — | — | 100 (50.0 to 100.0) | 100 (50.0 to 100.0) | — |
| Prior to Course 18 | — | — | 100 (50.0 to 100.0) | — | — |
| Prior to Course 22 | — | — | 0 (0 to 95.0) | — | — |
| Prior to Course 26 | — | — | 0 (0 to 95.0) | — | — |
| End of Treatment | 100 (22.36 to 100) | — | 33.33 (6.29 to 72.87) | 66.67 (13.54 to 98.30) | 0 (0 to 52.71) |
The cytokine was quantitated using V-PLEX Human Proinflammatory Panel, a multiplex immunoassay kit developed by Meso Scale Diagnostics (MSD). A four-parameter logistic fit calibration curve was generated and used to calculate concentrations in tested samples. Analyses were performed at pre-treatment and 1 week following the first dose of drug and additionally at 2, 3, 6, and 9 weeks following the course 1 drug dose if feasible. The mean concentration of TNF-alpha in pg/ml was reported with a standard deviation. When only one patient was analyzed, the standard deviation was reported as 0 (rather than undefined).
| pg/ml | Stratum 1, Dose Level 1 | Stratum 1, Dose Level 2 | Stratum 1, Dose Level 3 | Stratum 2, Dose Level 2 | Stratum 2, Dose Level 3 |
|---|---|---|---|---|---|
| Week 0 | 0.95 ± 0 | 1.24 ± 0 | 0.71 ± 0.28 | 0.87 ± 0.48 | 0.41 ± 0.18 |
| Week 1 | 1.02 ± 0.22 | 1.31 ± 0.49 | 0.75 ± 0.19 | 0.92 ± 0.65 | 0.44 ± 0.10 |
| Week 2 | 0.92 ± 0.35 | 1.43 ± 0.01 | 0.97 ± 0.38 | 0.79 ± 0.33 | 1.03 ± 0 |
| Week 3 | 1.15 ± 0 | 1.42 ± 0.34 | 0.83 ± 0.35 | 1.12 ± 0.74 | 26.07 ± 35.73 |
| Week 6 | 1.07 ± 0 | 1.37 ± 0.54 | 0.79 ± 0.34 | 0.84 ± 0.40 | — |
| Week 9 | 1.57 ± 0 | 1.81 ± 0.83 | 0.83 ± 0.35 | 1.26 ± 0.60 | 0.35 ± 0 |
The cytokine was quantitated using V-PLEX Human Proinflammatory Panel, a multiplex immunoassay kit developed by Meso Scale Diagnostics (MSD). A four-parameter logistic fit calibration curve was generated and used to calculate concentrations in tested samples. Analyses were performed at pre-treatment and 1 week following the first dose of drug and additionally at 2, 3, 6, and 9 weeks following the course 1 drug dose if feasible. The mean concentration of IL-8 in pg/ml was reported with a standard deviation. When only 1 participant analyzed, the standard deviation was reported as 0 (rather than undefined).
| pg/ml | Stratum 1, Dose Level 1 | Stratum 1, Dose Level 2 | Stratum 1, Dose Level 3 | Stratum 2, Dose Level 2 | Stratum 2, Dose Level 3 |
|---|---|---|---|---|---|
| Week 0 | 1.76 ± 0 | 5.88 ± 0 | 3.03 ± 0.80 | 4.91 ± 5.30 | 2.52 ± 1.18 |
| Week 1 | 1.29 ± 0.61 | 5.86 ± 4.17 | 4.67 ± 1.99 | 4.40 ± 2.34 | 1.52 ± 1.01 |
| Week 2 | 3.96 ± 0.60 | 4.27 ± 1.36 | 3.72 ± 1.30 | 2.74 ± 0.84 | 1.80 ± 0 |
| Week 3 | 3.82 ± 0 | 3.66 ± 1.91 | 3.73 ± 1.42 | 3.23 ± 0.99 | 217.42 ± 301.13 |
| Week 6 | 3.30 ± 0 | 3.94 ± 1.46 | 3.39 ± 2.06 | 2.54 ± 0.87 | — |
| Week 9 | 3.86 ± 0 | 10.71 ± 11.42 | 2.87 ± 0.70 | 3.03 ± 1.56 | 0.66 ± 0 |
Tumor mutation burden was defined as the number of somatic mutations per megabase. Average tumor mutation burden was reported with a standard deviation. When only one patient was analyzed, the standard deviation was reported as 0 rather than undefined.
| mutations per megabase | Stratum 1, Dose Level 1 | Stratum 1, Dose Level 2 | Stratum 1, Dose Level 3 | Stratum 2, Dose Level 2 | Stratum 2, Dose Level 3 |
|---|---|---|---|---|---|
| Tumor Mutation Burden Based on Whole Exome Sequencing of Tumor Tissue | 2.62 ± 1.03 | 3.82 ± 2.79 | 3.95 ± 2.34 | 0.97 ± 0.0 | 4.83 ± 5.94 |
Tumor mutation burden was defined as the number of somatic mutations per megabase. Average tumor mutation burden was reported with a standard deviation. When only one patient was analyzed, the standard deviation was reported as 0 rather than undefined.
| mutations per megabase | Stratum 1, Dose Level 1 | Stratum 1, Dose Level 2 | Stratum 1, Dose Level 3 | Stratum 2, Dose Level 2 | Stratum 2, Dose Level 3 |
|---|---|---|---|---|---|
| Tumor Mutation Burden Based on RNA Sequencing of Tumor Tissue | 3.15 ± 0.92 | 3.33 ± 0.32 | 3.26 ± 1.35 | — | — |
Mutation burden as detected by comparing the TCR sequencing of tumor tissue and/or PBMC. Mean numbers of clonotypes were reported by time point with standard deviations.
| Clonotyes | Stratum 1, Dose Level 1 | Stratum 1, Dose Level 2 | Stratum 1, Dose Level 3 | Stratum 2, Dose Level 2 | Stratum 2, Dose Level 3 |
|---|---|---|---|---|---|
| Day 0 | — | — | — | 54688.25 ± 48633.55 | — |
| Day 7 | — | — | — | 159239.75 ± 47953.53 | — |
| Day 14 | — | — | — | 55328.25 ± 43692.86 | — |
| Day 21 | — | — | — | 127911.25 ± 135922.69 | — |
Collected over Approximately 3 years after start of treatment. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Stratum 1, Dose Level 1 | 0/3 (0%) | 0/3 (0%) | 3/3 (100%) |
| Stratum 1, Dose Level 2 | 1/3 (33.3%) | 0/3 (0%) | 3/3 (100%) |
| Stratum 1, Dose Level 3 | 5/14 (35.7%) | 4/14 (28.6%) | 14/14 (100%) |
| Stratum 2, Dose Level 2 | 4/6 (66.7%) | 1/6 (16.7%) | 6/6 (100%) |
| Stratum 2, Dose Level 3 | 4/5 (80%) | 5/5 (100%) | 5/5 (100%) |
| Event | Stratum 1, Dose Level 1 | Stratum 1, Dose Level 2 | Stratum 1, Dose Level 3 | Stratum 2, Dose Level 2 | Stratum 2, Dose Level 3 |
|---|---|---|---|---|---|
| Cytokine release syndromeImmune system disorders | 0/3 | 0/3 | 1/14 | 0/6 | 2/5 |
| Infusion related reactionInjury, poisoning and procedural complications | 0/3 | 0/3 | 3/14 | 0/6 | 0/5 |
| DysphagiaGastrointestinal disorders | 0/3 | 0/3 | 0/14 | 0/6 | 1/5 |
| FatigueGeneral disorders | 0/3 | 0/3 | 1/14 | 0/6 | 1/5 |
| Hepatic failureHepatobiliary disorders | 0/3 | 0/3 | 0/14 | 0/6 | 1/5 |
| Allergic reactionImmune system disorders | 0/3 | 0/3 | 0/14 | 0/6 | 1/5 |
| SepsisInfections and infestations | 0/3 | 0/3 | 0/14 | 0/6 | 1/5 |
| Upper respiratory infectionInfections and infestations | 0/3 | 0/3 | 0/14 | 0/6 | 1/5 |
| Aspartate aminotransferase increasedInvestigations | 0/3 | 0/3 | 1/14 | 0/6 | 1/5 |
| Neutrophil count decreasedInvestigations | 0/3 | 0/3 | 0/14 | 0/6 | 1/5 |
| Event | Stratum 1, Dose Level 1 | Stratum 1, Dose Level 2 | Stratum 1, Dose Level 3 | Stratum 2, Dose Level 2 | Stratum 2, Dose Level 3 |
|---|---|---|---|---|---|
| White blood cell decreasedInvestigations | 1/3 | 3/3 | 9/14 | 3/6 | 3/5 |
| Alanine aminotransferase increasedInvestigations | 1/3 | 2/3 | 12/14 | 5/6 | 5/5 |
| Aspartate aminotransferase increasedInvestigations | 0/3 | 2/3 | 11/14 | 2/6 | 5/5 |
| Lymphocyte count decreasedInvestigations | 2/3 | 2/3 | 7/14 | 2/6 | 4/5 |
| AnorexiaMetabolism and nutrition disorders | 0/3 | 1/3 | 5/14 | 1/6 | 4/5 |
| Neutrophil count decreasedInvestigations | 1/3 | 2/3 | 5/14 | 2/6 | 2/5 |
| Platelet count decreasedInvestigations | 0/3 | 1/3 | 9/14 | 1/6 | 2/5 |
| HypertensionVascular disorders | 0/3 | 0/3 | 0/14 | 0/6 | 3/5 |
| Blood bilirubin increasedInvestigations | 0/3 | 0/3 | 3/14 | 0/6 | 3/5 |
| HypophosphatemiaMetabolism and nutrition disorders | 0/3 | 0/3 | 4/14 | 3/6 | 3/5 |
The one stratum 1, dose level 3 participant found to be ineligible after enrollment was excluded. Summary statistics for baseline characteristics are shown for eligible patients only (n=31).
| Age, Continuous(Year) | Stratum 1, Dose Level 1 | Stratum 1, Dose Level 2 | Stratum 1, Dose Level 3 | Stratum 2, Dose Level 2 | Stratum 2, Dose Level 3 | Total |
|---|---|---|---|---|---|---|
| Median | 4.2 (3.4 to 14.6) | 13.2 (8.9 to 21.9) | 9.7 (2.2 to 20.6) | 6.6 (1.4 to 10.4) | 8.8 (2.8 to 10.6) | 8.9 (1.4 to 21.9) |
| Sex: Female, Male(Participants) | Stratum 1, Dose Level 1 | Stratum 1, Dose Level 2 | Stratum 1, Dose Level 3 | Stratum 2, Dose Level 2 | Stratum 2, Dose Level 3 | Total |
|---|---|---|---|---|---|---|
| Female | 1 | 1 | 7 | 4 | 5 | 18 |
| Male | 2 | 2 | 7 | 2 | 0 | 13 |
| Ethnicity (NIH/OMB)(Participants) | Stratum 1, Dose Level 1 | Stratum 1, Dose Level 2 | Stratum 1, Dose Level 3 | Stratum 2, Dose Level 2 | Stratum 2, Dose Level 3 | Total |
|---|---|---|---|---|---|---|
| Hispanic or Latino | 1 | 1 | 4 | 3 | 2 | 11 |
| Not Hispanic or Latino | 2 | 1 | 9 | 3 | 2 | 17 |
| Unknown or Not Reported | 0 | 1 | 1 | 0 | 1 | 3 |
| Race (NIH/OMB)(Participants) | Stratum 1, Dose Level 1 | Stratum 1, Dose Level 2 | Stratum 1, Dose Level 3 | Stratum 2, Dose Level 2 | Stratum 2, Dose Level 3 | Total |
|---|---|---|---|---|---|---|
| American Indian or Alaska Native | 1 | 0 | 0 | 0 | 1 | 2 |
| Asian | 0 | 0 | 1 | 0 | 0 | 1 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 1 | 0 | 1 | 2 |
| White | 2 | 3 | 10 | 4 | 0 | 19 |
| More than one race | 0 | 0 | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 2 | 2 | 3 | 7 |
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Studies of the drug tested here: Sotigalimab
Pediatric Brain Tumor Consortium