A Phase 1/2 interventional study of CX 4945 in Medulloblastoma, Childhood, Medulloblastoma Recurrent and Medulloblastoma, sponsored by Pediatric Brain Tumor Consortium. Terminated at 12 sites in United States. Open to participants aged 3 Years and older. Per ClinicalTrials.gov, last updated 2026-10-19.
Sponsored by Pediatric Brain Tumor Consortium · Phase 1/2, Interventional, and Treatment
This is a multi center, Phase I, Phase II and surgical study of the CX-4945 drug (silmitasertib sodium) for patients with recurrent SHH (Sonic Hedgehog) medulloblastoma
PRIMARY OBJECTIVES:
I. To estimate the maximum tolerated dose (MTD) and recommend a Phase II dose of CX-4945 administered orally daily to skeletally-immature children with recurrent SHH (sonic hedgehog) medulloblastoma (Phase I) II. To describe the toxicity profile and define the dose-limiting toxicities associated with CX-4945 in children with recurrent SHH (Sonic hedgehog) medulloblastoma (Phase I) III. To characterize the pharmacokinetics of CX-4945 administered on this schedule in skeletally-immature children with recurrent SHH medulloblastoma (Phase I) IV. To characterize the concentrations of CX-4945 in tumor after administration of CX-4945 and surgical resection (Surgical Study). V. To establish the safety and characterize the toxicity of 1000mg BID continuous dosing of CX-4945 in skeletally-mature patients with recurrent SHH medulloblastoma (Phase II). VI. To estimate the objective response rate associated with CX-4945 in skeletally-mature patients with recurrent SHH medulloblastoma
SECONDARY OBJECTIVES:
I. To document preliminary antitumor activity of CX-4945 in skeletally-immature children with recurrent SHH medulloblastoma (Phase I). II. To perform a genomic analysis within the confines of a Phase I study to investigate correlation between response to treatment and the presence of specific genomic alterations. and/or specific subgroups of disease (Phase I). III. To explore the ability of CX-4945 at the MTD/ RP2D to inhibit CK2-mediated signaling in tumor (Surgical Study). IV. To characterize the pharmacokinetics of CX-4945 in skeletally-mature patients with recurrent SHH medulloblastoma (Phase II). V. To perform a genomic analysis within the confines of a Phase II study to investigate correlation between response to treatment and the presence of specific genomic alterations and/or specific subgroups of disease (Phase II).
OUTLINE: Phase I component is a dose-escalation study. The Phase II component is to establish the safety of 1000mg BID given continuously.
The study will open with a safety cohort of 3 subjects who are considered skeletally-mature. The initial 3 subjects will be administered CX-4945 twice a day at the adult RP2D of 1000 mg BID or at its BSA adjusted equivalent; however, the dose will be given continuously. If there are not excessive toxicities in this cohort, the study will proceed following the Phase II design for subjects who are skeletally-mature.
Following the safety lead in, the Phase 1 component of this trial will be initiated. Skeletally-immature children with refractory or recurrent medulloblastoma of the SHH subgroup, will be administered CX-4945 twice a day on a continuous basis at a starting dose of 600mg/m2 BID which corresponds approximately to the BSA adjusted recommended Phase 2 dose (RP2D) of 1000mg. The Phase 1 study will escalate doses to determine the maximum tolerated dose skeletally-immature children.
The surgical study will be initiated after the first 3 patients in the skeletally-mature cohort are treated for initial assessment of safety and did not experience excessive toxicity. Skeletally-mature subjects with recurrent or refractory SHH medulloblastoma will be eligible as soon the surgical study is initiated and will receive drug at 1000mg BID or its BSA adjusted equivalent depending upon age and BSA. Skeletally-immature subjects will only be eligible to enroll on the surgical trial once the MTD is defined in the Phase 1 component and will receive drug at the established MTD for this cohort.
After completion of study treatment, patients are followed up to 2 years.
317 studies on the registry are indexed under Medulloblastoma; 51 are open to participants now.
This study's enrollment of 21 is below the median of 31 across 255 interventional studies indexed under Medulloblastoma.
Browse Medulloblastoma 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.
Screening Criteria:
Tumor
a. Patient must have a diagnosis of medulloblastoma that is recurrent or progressive. All tumors must have histologic verification either at the time of diagnosis or recurrence.
Prior Therapy
a. Patient must have received and failed standard therapy for medulloblastoma.
Inclusion Criteria:
Phase I Skeletally-immature:
Dose Level 0 (400 mg/m2 BID): Minimum - 0.84m2; Maximum - 2.25m2 Dose Level 1 (600 mg/m2 BID): Minimum - 0.60m2; Maximum - 2.00m2 Dose Level 2 (800 mg/m2 BID): Minimum - 0.63m2; Maximum - 2.00m2
Phase II Skeletally-mature:
a. Patients must be skeletally-mature, defined as females with a bone age ≥14 years and males with a bone age ≥ 16 years OR have a chronological age >18 years.
b. Patients must have bi-dimensionally measurable disease, defined as at least one lesion that can be accurately measured in at least two dimensions. Patients with measurable extraneural disease only are also eligible.
Surgical Study:
All Phases:
Prior Therapy
If neurological deficits are present, must have been stable for a minimum of 1 week prior to enrollment.
C. Exclusion Criteria:
1. All Phases
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.
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
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.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
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.
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.
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.
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.
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.
Measurable disease
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
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.
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.
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.
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.
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.
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.
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.
Skeletally-immature children with refractory or recurrent medulloblastoma of the SHH group
Drug: CX 4945
Skeletally-mature subjects with refractory or recurrent medulloblastoma of the SHH group
Drug: CX 4945
Subjects who are eligible for the Phase I or Phase II arm of the trial and are candidates for surgery, may be enrolled in the surgical arm prior to initiation of the Phase I or Phase II treatment.
Drug: CX 4945
CX-4945 is supplied as 200 mg capsules delivered orally as a formulated API
Also known as: Silmitasertib sodium
Number of Participants Who Experienced Dose-Limiting Toxicities (DLTs)
DLT is any event occurring during the dose-finding period (Course 1) that is at least possibly related to CX-4945, regardless of expectedness. DLTs include: (1) any CX-4945-related AE leading to dose reduction, permanent treatment discontinuation, or treatment delay \>14 days (dose modifications per Sections 7.1 and 7.2); (2) non-hematologic toxicity: any Grade 4 event; any Grade 3 event except controllable Grade 3 nausea/vomiting, supportive care-responsive Grade 3 diarrhea, reversible Grade 3 AST/ALT returning to eligibility levels within 7 days of treatment interruption without recurrence on rechallenge, Grade 3 fever, Grade 3 infection, or enteral supplementation-responsive Grade 3 electrolyte abnormalities; and any non-hematologic toxicity persisting ≥14 days that is medically significant or intolerable enough to require treatment interruption; (3) hematologic toxicity: Grade 3/4 thrombocytopenia or Grade 4 neutropenia.
Time frame: up to 4 weeks for each dosing cohort
Maximum Tolerated Dose (MTD) or Recommended Phase 2 Dose (RP2D) of CX-4945 in Skeletally Immature Patients
The rolling-6 design was used to estimate the MTD in the Phase I group. The MTD was defined as the highest dose level at which six patients have been treated with at most one patient experiencing a dose limiting toxicity (DLT) and the next higher dose level has been determined to be too toxic.
Time frame: 4 weeks
Intratumoral PK Concentrations in Surgical Cohort Participants
Average tumor CX-4945 concentrations in surgical patients
Time frame: During surgery at the time of tissue collection
Sustained Objective Response (PR-CR) Rate in Skeletally-mature Patients
A sustained objective responder is defined as a patient who achieves a confirmed partial response or complete response, which also must be sustained for at least 8 weeks with central radiographic confirmation. Response rates will be calculated as the percent of evaluable patients who are responders. Tumor response criteria are determined by changes in size, measured as the longest tumor dimension and its perpendicular. FLAIR, T2 or post contrast T1 weighted images may be used - whichever gives the best estimate of tumor size.
Time frame: Up to 2 years from enrollment
Pharmacokinetic (PK) Parameters of CX4945, Area Under the Curve (AUC), in Skeletally Immature Patients
The mean of the AUC evaluated at day 1 and day 28 of course 1 in skeletally immature patients who had plasma sample available.
Time frame: Day 1 and day 28 of the course 1.
Progression Free-Survival (PFS)
The Kaplan-Meier method will be used to estimate the median survival time of PFS. The PFS is defined as the time between treatment initiation until progression or death for any reason for patients who experience an event and last follow-up for patients who are event-free at the time of analysis.
Time frame: up to 24 months from enrollment
Sustained Objective Response (PR-CR) Rate in Skeletally-immature Patients
A sustained objective responder is defined as a patient who achieves a confirmed partial response or complete response, which also must be sustained for at least 8 weeks with central radiographic confirmation. Response rates will be calculated as the percent of evaluable patients who are responders. Tumor response criteria are determined by changes in size, measured as the longest tumor dimension and its perpendicular. FLAIR, T2 or post contrast T1 weighted images may be used - whichever gives the best estimate of tumor size.
Time frame: Up to 2 years from enrollment
Pharmacokinetic (PK) Parameters of CX4945, Area Under the Curve (AUC), in Skeletally Mature Patients
The mean of the AUC evaluated on day 1 and day 28 of course 1 in skeletally mature patients with a plasma sample available.
Time frame: Day 1 and day 28 of the course 1.
Correlation Between Response to Treatment and the Presence of Specific Genomic Alterations
Estimated correlation coefficient between antitumor response and presence of genomic alterations.
Time frame: At time of enrollment
Reduction in CK2-mediated Signaling in Tumor for Surgical Participants
Change in CK2 activity in tumor tissue from patients on the surgical are at baseline and after treatment.
Time frame: 4 weeks
This is a multi-center, Phase I, Phase II and surgical study of the CX-4945 (silmitasertib sodium) for pediatric and adult patients with recurrent SHH (Sonic Hedgehog) medulloblastoma. Patients were enrolled at member institutions of the Pediatric Brain Tumor Consortium. The first patient was enrolled on 07/25/2019 and the last patient was enrolled on 02/07/2025.
| Milestone | Phase I Dose Level 1: 600 mg/m^2 | Phase I Dose Level 2: 800 mg/m^2 | Phase II Dose Level 1: 600 mg/m^2 or 1000 mg | Surgical Dose Level 1: 600 mg/m^2 or 1000 mg |
|---|---|---|---|---|
| Started | 0 | 0 | 3 | 0 |
| Completed | 0 | 0 | 3 | 0 |
| Not completed | 0 | 0 | 0 | 0 |
| Milestone | Phase I Dose Level 1: 600 mg/m^2 | Phase I Dose Level 2: 800 mg/m^2 | Phase II Dose Level 1: 600 mg/m^2 or 1000 mg | Surgical Dose Level 1: 600 mg/m^2 or 1000 mg |
|---|---|---|---|---|
| Started | 3 | 3 | 3 | 3 |
| Completed | 3 | 3 | 3 | 3 |
| Not completed | 0 | 0 | 0 | 0 |
| Milestone | Phase I Dose Level 1: 600 mg/m^2 | Phase I Dose Level 2: 800 mg/m^2 | Phase II Dose Level 1: 600 mg/m^2 or 1000 mg | Surgical Dose Level 1: 600 mg/m^2 or 1000 mg |
|---|---|---|---|---|
| Started | 0 | 0 | 6 | 0 |
| Completed | 0 | 0 | 5 | 0 |
| Not completed | 0 | 0 | 1 | 0 |
| Withdrew: Did not start treatment | 0 | 0 | 1 | 0 |
DLT is any event occurring during the dose-finding period (Course 1) that is at least possibly related to CX-4945, regardless of expectedness. DLTs include: (1) any CX-4945-related AE leading to dose reduction, permanent treatment discontinuation, or treatment delay \>14 days (dose modifications per Sections 7.1 and 7.2); (2) non-hematologic toxicity: any Grade 4 event; any Grade 3 event except controllable Grade 3 nausea/vomiting, supportive care-responsive Grade 3 diarrhea, reversible Grade 3 AST/ALT returning to eligibility levels within 7 days of treatment interruption without recurrence on rechallenge, Grade 3 fever, Grade 3 infection, or enteral supplementation-responsive Grade 3 electrolyte abnormalities; and any non-hematologic toxicity persisting ≥14 days that is medically significant or intolerable enough to require treatment interruption; (3) hematologic toxicity: Grade 3/4 thrombocytopenia or Grade 4 neutropenia.
| Participants | Phase I Dose Level 1: 600 mg/m^2 | Phase I Dose Level 2: 800 mg/m^2 | Phase II Dose Level 1: 600 mg/m^2 or 1000 mg |
|---|---|---|---|
| Number of Participants Who Experienced Dose-Limiting Toxicities (DLTs) | 0 | 0 | 1 |
The rolling-6 design was used to estimate the MTD in the Phase I group. The MTD was defined as the highest dose level at which six patients have been treated with at most one patient experiencing a dose limiting toxicity (DLT) and the next higher dose level has been determined to be too toxic.
| mg/m^2 twice a day | All Participants in Phase I |
|---|---|
| Maximum Tolerated Dose (MTD) or Recommended Phase 2 Dose (RP2D) of CX-4945 in Skeletally Immature Patients | NA |
Average tumor CX-4945 concentrations in surgical patients
| ng/mL | Surgical Dose Level 1: 600 mg/m^2 or 1000 mg |
|---|---|
| Intratumoral PK Concentrations in Surgical Cohort Participants | 92.5 (54 to 131) |
The Kaplan-Meier method will be used to estimate the median survival time of PFS. The PFS is defined as the time between treatment initiation until progression or death for any reason for patients who experience an event and last follow-up for patients who are event-free at the time of analysis.
| Months | Phase I Dose Level 1: 600 mg/m^2 | Phase I Dose Level 2: 800 mg/m^2 | Phase II Dose Level 1 + Surgical Dose Level 1 |
|---|---|---|---|
| Progression Free-Survival (PFS) | 1.8 (0.9 to NA) | 1.8 (1.5 to NA) | 1.8 (1.7 to 2.3) |
A sustained objective responder is defined as a patient who achieves a confirmed partial response or complete response, which also must be sustained for at least 8 weeks with central radiographic confirmation. Response rates will be calculated as the percent of evaluable patients who are responders. Tumor response criteria are determined by changes in size, measured as the longest tumor dimension and its perpendicular. FLAIR, T2 or post contrast T1 weighted images may be used - whichever gives the best estimate of tumor size.
No measurements were reported for this outcome.
The mean of the AUC evaluated on day 1 and day 28 of course 1 in skeletally mature patients with a plasma sample available.
| h·ng/mL | Phase II Dose Level 1 + Surgical Dose Level 1 |
|---|---|
| Day 1 of Course 1 | 5962 ± 5309 |
| Day 28 of Course 1 | 13649 ± 19644 |
Estimated correlation coefficient between antitumor response and presence of genomic alterations.
No measurements were reported for this outcome.
Change in CK2 activity in tumor tissue from patients on the surgical are at baseline and after treatment.
No measurements were reported for this outcome.
A sustained objective responder is defined as a patient who achieves a confirmed partial response or complete response, which also must be sustained for at least 8 weeks with central radiographic confirmation. Response rates will be calculated as the percent of evaluable patients who are responders. Tumor response criteria are determined by changes in size, measured as the longest tumor dimension and its perpendicular. FLAIR, T2 or post contrast T1 weighted images may be used - whichever gives the best estimate of tumor size.
| percentage of evaluable participants | Phase II Dose Level 1 + Surgical Dose Level 1 |
|---|---|
| Sustained Objective Response (PR-CR) Rate in Skeletally-mature Patients | 0 (0 to 28.5) |
The mean of the AUC evaluated at day 1 and day 28 of course 1 in skeletally immature patients who had plasma sample available.
| h·ng/mL | Phase I Dose Level 1: 600 mg/m^2 | Phase I Dose Level 2: 800 mg/m^2 |
|---|---|---|
| Day 1 of Course 1 | 10324 ± 10546 | 9993 ± 6518 |
| Day 28 of Course 1 | 17078 ± 6962 | 29273 ± 22149 |
Collected over Up to 2 years after starting treatment. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Phase I Dose Level 1: 600 mg/m^2 | 0/3 (0%) | 1/3 (33.3%) | 3/3 (100%) |
| Phase I Dose Level 2: 800 mg/m^2 | 0/3 (0%) | 0/3 (0%) | 3/3 (100%) |
| Phase II Dose Level 1: 600 mg/m^2 or 1000 mg | 1/12 (8.3%) | 3/12 (25%) | 11/12 (91.7%) |
| Surgical Dose Level 1: 600 mg/m^2 or 1000 mg | 0/3 (0%) | 2/3 (66.7%) | 3/3 (100%) |
| Event | Phase I Dose Level 1: 600 mg/m^2 | Phase I Dose Level 2: 800 mg/m^2 | Phase II Dose Level 1: 600 mg/m^2 or 1000 mg | Surgical Dose Level 1: 600 mg/m^2 or 1000 mg |
|---|---|---|---|---|
| FeverGeneral disorders | 0/3 | 0/3 | 0/12 | 1/3 |
| Cerebrospinal fluid leakageNervous system disorders | 0/3 | 0/3 | 0/12 | 1/3 |
| HeadacheNervous system disorders | 1/3 | 0/3 | 0/12 | 0/3 |
| HydrocephalusNervous system disorders | 0/3 | 0/3 | 0/12 | 1/3 |
| DiarrheaGastrointestinal disorders | 0/3 | 0/3 | 1/12 | 0/3 |
| NauseaGastrointestinal disorders | 0/3 | 0/3 | 1/12 | 0/3 |
| Disease progressionGeneral disorders | 0/3 | 0/3 | 1/12 | 0/3 |
| Tumor hemorrhageNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 0/3 | 0/3 | 1/12 | 0/3 |
| SeizureNervous system disorders | 0/3 | 0/3 | 1/12 | 0/3 |
| Event | Phase I Dose Level 1: 600 mg/m^2 | Phase I Dose Level 2: 800 mg/m^2 | Phase II Dose Level 1: 600 mg/m^2 or 1000 mg | Surgical Dose Level 1: 600 mg/m^2 or 1000 mg |
|---|---|---|---|---|
| NauseaGastrointestinal disorders | 2/3 | 3/3 | 4/12 | 2/3 |
| DiarrheaGastrointestinal disorders | 2/3 | 2/3 | 9/12 | 2/3 |
| VomitingGastrointestinal disorders | 2/3 | 1/3 | 4/12 | 1/3 |
| FeverGeneral disorders | 0/3 | 0/3 | 0/12 | 2/3 |
| Alanine aminotransferase increasedInvestigations | 0/3 | 2/3 | 4/12 | 1/3 |
| Aspartate aminotransferase increasedInvestigations | 0/3 | 2/3 | 3/12 | 1/3 |
| Blood bicarbonate decreasedInvestigations | 0/3 | 2/3 | 0/12 | 0/3 |
| Creatinine increasedInvestigations | 1/3 | 2/3 | 2/12 | 0/3 |
| AnemiaBlood and lymphatic system disorders | 1/3 | 0/3 | 2/12 | 0/3 |
| Abdominal painGastrointestinal disorders | 1/3 | 0/3 | 0/12 | 0/3 |
All enrolled patients who are eligible for the study.
| Age, Categorical(Participants) | Phase I Dose Level 1: 600 mg/m^2 | Phase I Dose Level 2: 800 mg/m^2 | Phase II Dose Level 1: 600 mg/m^2 or 1000 mg | Surgical Dose Level 1: 600 mg/m^2 or 1000 mg | Total |
|---|---|---|---|---|---|
| <=18 years | 3 | 3 | 4 | 0 | 10 |
| Between 18 and 65 years | 0 | 0 | 8 | 3 | 11 |
| >=65 years | 0 | 0 | 0 | 0 | 0 |
| Age, Continuous(Years) | Phase I Dose Level 1: 600 mg/m^2 | Phase I Dose Level 2: 800 mg/m^2 | Phase II Dose Level 1: 600 mg/m^2 or 1000 mg | Surgical Dose Level 1: 600 mg/m^2 or 1000 mg | Total |
|---|---|---|---|---|---|
| Median | 11.4 (8.6 to 13.9) | 10.7 (8.3 to 10.9) | 24.7 (13.3 to 42.0) | 24.7 (22.9 to 35.4) | 20.3 (8.3 to 42.0) |
| Sex: Female, Male(Participants) | Phase I Dose Level 1: 600 mg/m^2 | Phase I Dose Level 2: 800 mg/m^2 | Phase II Dose Level 1: 600 mg/m^2 or 1000 mg | Surgical Dose Level 1: 600 mg/m^2 or 1000 mg | Total |
|---|---|---|---|---|---|
| Female | 2 | 2 | 4 | 0 | 8 |
| Male | 1 | 1 | 8 | 3 | 13 |
| Race (NIH/OMB)(Participants) | Phase I Dose Level 1: 600 mg/m^2 | Phase I Dose Level 2: 800 mg/m^2 | Phase II Dose Level 1: 600 mg/m^2 or 1000 mg | Surgical Dose Level 1: 600 mg/m^2 or 1000 mg | Total |
|---|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 1 | 1 |
| Asian | 0 | 0 | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 |
| Black or African American | 0 | 1 | 1 | 0 | 2 |
| White | 2 | 0 | 7 | 0 | 9 |
| More than one race | 0 | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 1 | 2 | 4 | 2 | 9 |
| Ethnicity (NIH/OMB)(Participants) | Phase I Dose Level 1: 600 mg/m^2 | Phase I Dose Level 2: 800 mg/m^2 | Phase II Dose Level 1: 600 mg/m^2 or 1000 mg | Surgical Dose Level 1: 600 mg/m^2 or 1000 mg | Total |
|---|---|---|---|---|---|
| Hispanic or Latino | 1 | 2 | 2 | 1 | 6 |
| Not Hispanic or Latino | 2 | 1 | 6 | 1 | 10 |
| Unknown or Not Reported | 0 | 0 | 4 | 1 | 5 |
| Region of Enrollment(Participants) | Phase I Dose Level 1: 600 mg/m^2 | Phase I Dose Level 2: 800 mg/m^2 | Phase II Dose Level 1: 600 mg/m^2 or 1000 mg | Surgical Dose Level 1: 600 mg/m^2 or 1000 mg | Total |
|---|---|---|---|---|---|
| United States | 3 | 3 | 12 | 3 | 21 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Jul 2019 to primary completion in Aug 2025, 72 months.
Results were first posted in Oct 2026, 1 year 2 months after primary completion.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is terminated, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Silmitasertib
Pediatric Brain Tumor Consortium