A Phase 1/2 interventional study of PLX3397 and sirolimus in Sarcoma and Malignant Peripheral Nerve Sheath Tumors, sponsored by Gulam Manji. Terminated at 5 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-03-21.
Sponsored by Gulam Manji · Phase 1/2, Interventional, and Treatment
The purpose of this study is to determine if treatment with PLX3397 and Sirolimus will be tolerated and result in shrinking of the cancer or stopping the cancer from growing. In the phase I portion, the maximum tolerate dose of the study drug will be determined. In the Phase II portion, progression free survival will be assessed at the dose level found in Phase I. Participants will continue to take the study drug until they experience an unacceptable side effect or their disease progresses. Funding Source - FDA OOPD
Malignant peripheral nerve sheath tumors (MPNSTs) represent up to 10% of adult soft tissue sarcomas. Due to its rarity, few MPNST-specific prospective trials exist, and treatments are largely based on extrapolation from results from other sarcoma subtypes. Since the molecular pathways driving pathogenesis within sarcoma subtypes are distinct, these treatment options are likely suboptimal at best. Targeted therapies that block key pathways known to drive MPNST will likely result in superior tumor responses with limited toxicities.
98 studies on the registry are indexed under Neurofibrosarcoma; 23 are open to participants now.
This study's enrollment of 39 is below the median of 46 across 84 interventional studies indexed under Neurofibrosarcoma.
Browse Neurofibrosarcoma studies →Gulam Manji is the lead sponsor of 4 studies on the registry; 1 is open to participants now.
Compared within Neurofibrosarcoma, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Sarcoma. The counts are of registry records indexed under each term, refreshed daily. How we classify →
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.
Disease site/type with pathologic confirmation of diagnosis at participating cancer site.
Allowable prior therapy
Allowable laboratory values with date range
Exclusion Criteria:
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.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
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.
The standard rules for measuring tumours on CT or MRI scans, to tell whether a cancer is shrinking, stable or growing during treatment.
“Measurable disease per RECIST 1.1” usually means at least one tumour 10 mm or more at its longest on a CT scan, or a lymph node 15 mm or more across its short axis.
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.
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).
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.
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.
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.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
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.
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.
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.
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
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.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
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.
Cancer deposits in the brain from a cancer that started elsewhere in the body (CNS metastases include the spinal cord).
Studies differ: some leave out any brain metastases, some accept ones treated and stable for some weeks. Check what this study allows.
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.
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.
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
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.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
RECIST
The standard rules for measuring tumours on CT or MRI scans, to tell whether a cancer is shrinking, stable or growing during treatment.
“Measurable disease per RECIST 1.1” usually means at least one tumour 10 mm or more at its longest on a CT scan, or a lymph node 15 mm or more across its short axis.
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.
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.
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.
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.
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.
Metastases
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
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.
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.
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.
Postmenopausal
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
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.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CTCAE grade
The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
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.
Brain metastases
Cancer deposits in the brain from a cancer that started elsewhere in the body (CNS metastases include the spinal cord).
Studies differ: some leave out any brain metastases, some accept ones treated and stable for some weeks. Check what this study allows.
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.
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.
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.
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.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CYP3A (CYP3A4) inhibitors and inducers
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Subjects with unresectable or metastatic sarcoma will take orally PLX3397 (600 - 1000mg) in combination with Sirolimus (2-6mg) daily.
Drug: PLX3397 · Drug: sirolimus
Subjects with unresectable or metastatic Malignant Peripheral Nerve Sheath Tumors (MPNSTs) will take PLX3397 and Sirolimus at the recommended Phase 2 dose (RP2D).
Drug: PLX3397 · Drug: sirolimus
PLX3397 is a small molecule that potently and selectively inhibits macrophage colony-stimulating factor receptor (FMS), Kit, and FMS-like tyrosine kinase 3 (Flt3)-internal tandem duplication (ITD) kinases, which regulate key components of the tumor microenvironment and oncogenic variants of these kinases that drive certain tumors.
Also known as: pexidartinib
Sirolimus is a macrocyclic lactone that binds to tacrolimus (FK506) binding protein 12 and inhibits mammalian target of rapamycin (mTOR) resulting in cell-cycle arrest and apoptosis. Sirolimus is currently approved as an immunosuppressive agent for organ transplantation and more recently, as a component of cardiac arterial stents because of its potent antiproliferative effects on fibroblasts responsible for restenosis after such a procedure (26) Sirolimus is commonly administered orally on a daily basis, in doses ranging from 2 to 40 mg/day.
Also known as: Rapamune
Maximum Tolerated Dose (MTD) - Phase 1
The highest dose of a drug or treatment that does not cause unacceptable side effects, which will be used in Phase 2.
Time frame: Up to 3 years
Progression Free Survival (PFS) Rate - Phase 2
The time from the start of treatment until disease progression or death from any cause, calculated in weeks.
Time frame: Up to 3 years
Overall Survival Rate
The time from the start of treatment until death, estimated using the Kaplan Meier method.
Time frame: Up to 3 years
| Milestone | Cohort 1, Phase 1 | Cohort 2, Phase 1 | Cohort 3, Phase 1 | Cohort 4, Phase 1 | Phase 2 Cohort |
|---|---|---|---|---|---|
| Started | 0 | 0 | 0 | 0 | 0 |
| Completed | 0 | 0 | 0 | 0 | 0 |
| Not completed | 0 | 0 | 0 | 0 | 0 |
| Milestone | Cohort 1, Phase 1 | Cohort 2, Phase 1 | Cohort 3, Phase 1 | Cohort 4, Phase 1 | Phase 2 Cohort |
|---|---|---|---|---|---|
| Started | 2 | 0 | 0 | 0 | 0 |
| Completed | 2 | 0 | 0 | 0 | 0 |
| Not completed | 0 | 0 | 0 | 0 | 0 |
| Milestone | Cohort 1, Phase 1 | Cohort 2, Phase 1 | Cohort 3, Phase 1 | Cohort 4, Phase 1 | Phase 2 Cohort |
|---|---|---|---|---|---|
| Started | 0 | 12 | 0 | 0 | 0 |
| Completed | 0 | 9 | 0 | 0 | 0 |
| Not completed | 0 | 3 | 0 | 0 | 0 |
| Milestone | Cohort 1, Phase 1 | Cohort 2, Phase 1 | Cohort 3, Phase 1 | Cohort 4, Phase 1 | Phase 2 Cohort |
|---|---|---|---|---|---|
| Started | 0 | 0 | 9 | 0 | 0 |
| Completed | 0 | 0 | 6 | 0 | 0 |
| Not completed | 0 | 0 | 3 | 0 | 0 |
| Milestone | Cohort 1, Phase 1 | Cohort 2, Phase 1 | Cohort 3, Phase 1 | Cohort 4, Phase 1 | Phase 2 Cohort |
|---|---|---|---|---|---|
| Started | 0 | 0 | 0 | 1 | 0 |
| Completed | 0 | 0 | 0 | 1 | 0 |
| Not completed | 0 | 0 | 0 | 0 | 0 |
| Milestone | Cohort 1, Phase 1 | Cohort 2, Phase 1 | Cohort 3, Phase 1 | Cohort 4, Phase 1 | Phase 2 Cohort |
|---|---|---|---|---|---|
| Started | 0 | 0 | 0 | 0 | 14 |
| Completed | 0 | 0 | 0 | 0 | 14 |
| Not completed | 0 | 0 | 0 | 0 | 0 |
The highest dose of a drug or treatment that does not cause unacceptable side effects, which will be used in Phase 2.
| mg | Phase 1 |
|---|---|
| Pexidartinib | 1000 |
| Sirolimus | 2 |
The time from the start of treatment until disease progression or death from any cause, calculated in weeks.
| weeks | Phase 2 |
|---|---|
| Progression Free Survival (PFS) Rate - Phase 2 | 6 (6 to 19.1) |
The time from the start of treatment until death, estimated using the Kaplan Meier method.
| weeks | Phase 2 |
|---|---|
| Overall Survival Rate | 17.9 (13.7 to NA) |
Collected over Up to 4 years. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cohort 1, Phase 1 | 0/2 (0%) | 1/2 (50%) | 2/2 (100%) |
| Cohort 2, Phase 1 | 0/12 (0%) | 6/12 (50%) | 12/12 (100%) |
| Cohort 3, Phase 1 | 1/9 (11.1%) | 7/9 (77.8%) | 9/9 (100%) |
| Cohort 4, Phase 1 | 0/1 (0%) | 0/1 (0%) | 1/1 (100%) |
| Phase 2 Cohort | 1/14 (7.1%) | 5/14 (35.7%) | 14/14 (100%) |
| Event | Cohort 1, Phase 1 | Cohort 2, Phase 1 | Cohort 3, Phase 1 | Cohort 4, Phase 1 | Phase 2 Cohort |
|---|---|---|---|---|---|
| NeoplasmsNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 1/2 | 0/12 | 0/9 | — | 0/14 |
| Pleural effusionRespiratory, thoracic and mediastinal disorders | 0/2 | 0/12 | 1/9 | — | 3/14 |
| PainGeneral disorders | 0/2 | 1/12 | 1/9 | — | 1/14 |
| VomitingGeneral disorders | 0/2 | 1/12 | 1/9 | — | 0/14 |
| DyspneaRespiratory, thoracic and mediastinal disorders | 0/2 | 0/12 | 1/9 | — | 1/14 |
| HyperkalemiaBlood and lymphatic system disorders | 0/2 | 0/12 | 1/9 | — | 0/14 |
| Administration site conditionGeneral disorders | 0/2 | 0/12 | 1/9 | — | 0/14 |
| NauseaGeneral disorders | 0/2 | 0/12 | 1/9 | — | 0/14 |
| Obstruction gastricGastrointestinal disorders | 0/2 | 0/12 | 1/9 | — | 1/14 |
| Wound infectionInfections and infestations | 0/2 | 0/12 | 1/9 | — | 0/14 |
| Event | Cohort 1, Phase 1 | Cohort 2, Phase 1 | Cohort 3, Phase 1 | Cohort 4, Phase 1 | Phase 2 Cohort |
|---|---|---|---|---|---|
| Alanine aminotransferase increasedHepatobiliary disorders | 2/2 | 3/12 | 2/9 | 1/1 | 3/14 |
| Alkaline phosphatase increasedHepatobiliary disorders | 2/2 | 4/12 | 4/9 | 1/1 | 3/14 |
| AnemiaBlood and lymphatic system disorders | 0/2 | 10/12 | 5/9 | 1/1 | 3/14 |
| AnorexiaGeneral disorders | 0/2 | 5/12 | 6/9 | 1/1 | 6/14 |
| Aspartate aminotransferase increasedGeneral disorders | 2/2 | 9/12 | 5/9 | 1/1 | 3/14 |
| Blurred visionGeneral disorders | 0/2 | 0/12 | 1/9 | 1/1 | 0/14 |
| CoughRespiratory, thoracic and mediastinal disorders | 2/2 | 3/12 | 1/9 | 0/1 | 2/14 |
| Creatinine increasedGeneral disorders | 2/2 | 4/12 | 4/9 | 0/1 | 0/14 |
| DyspneaRespiratory, thoracic and mediastinal disorders | 0/2 | 2/12 | 4/9 | 1/1 | 2/14 |
| FatigueGeneral disorders | 0/2 | 7/12 | 7/9 | 1/1 | 6/14 |
| Age, Continuous(years) | Cohort 1, Phase 1 | Cohort 2, Phase 1 | Cohort 3, Phase 1 | Cohort 4, Phase 1 | Phase 2 Cohort | Total |
|---|---|---|---|---|---|---|
| Median | 52.5 (52 to 53) | 47.0 (22 to 70) | 60 (21 to 72) | 44 (44 to 44) | 36.5 (19 to 72) | 49.4 (19 to 72) |
| Sex: Female, Male(Participants) | Cohort 1, Phase 1 | Cohort 2, Phase 1 | Cohort 3, Phase 1 | Cohort 4, Phase 1 | Phase 2 Cohort | Total |
|---|---|---|---|---|---|---|
| Female | 1 | 8 | 2 | 1 | 4 | 16 |
| Male | 1 | 4 | 7 | 0 | 10 | 22 |
| Ethnicity (NIH/OMB)(Participants) | Cohort 1, Phase 1 | Cohort 2, Phase 1 | Cohort 3, Phase 1 | Cohort 4, Phase 1 | Phase 2 Cohort | Total |
|---|---|---|---|---|---|---|
| Hispanic or Latino | 1 | 2 | 0 | 1 | 2 | 6 |
| Not Hispanic or Latino | 1 | 10 | 9 | 0 | 12 | 32 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Cohort 1, Phase 1 | Cohort 2, Phase 1 | Cohort 3, Phase 1 | Cohort 4, Phase 1 | Phase 2 Cohort | Total |
|---|---|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 | 0 |
| Asian | 0 | 1 | 1 | 0 | 0 | 2 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 | 0 |
| Black or African American | 0 | 3 | 1 | 0 | 1 | 5 |
| White | 1 | 6 | 7 | 0 | 12 | 26 |
| More than one race | 0 | 0 | 0 | 0 | 0 | 0 |
| Unknown or Not Reported | 1 | 2 | 0 | 1 | 1 | 5 |
| Region of Enrollment(Participants) | Cohort 1, Phase 1 | Cohort 2, Phase 1 | Cohort 3, Phase 1 | Cohort 4, Phase 1 | Phase 2 Cohort | Total |
|---|---|---|---|---|---|---|
| United States | 2 | 12 | 9 | 1 | 14 | 38 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Nov 2015 to primary completion in Jul 2023, 92 months.
Results were first posted in Mar 2025, 1 year 8 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is terminated, as verified in Mar 2025. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
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Studies of the drug tested here: Pexidartinib
Gulam Manji