SarcomaPhase 1/2PLX3397NCT02584647
PLX3397 Plus Sirolimus in Unresectable Sarcoma and Malignant Peripheral Nerve Sheath Tumors
A Phase 1/2 study testing PLX3397 and sirolimus in people with Sarcoma and Malignant Peripheral Nerve Sheath Tumors, run by Gulam Manji at 5 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sarcoma, Malignant Peripheral Nerve Sheath Tumors
- What is being tested?
- PLX3397 and sirolimus
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 39 participants
- Where
- 5 sitesUnited States
- Study dates
- 4 Nov 2015to 1 Oct 2024
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Disease site/type with pathologic confirmation of diagnosis at participating cancer site.
- Phase 1: Advanced, unresectable sarcoma (any subtype)
- Phase 2: Advanced, unresectable malignant peripheral nerve sheath tumors (MPNSTs)
- Extent of disease: Unresectable
- Allowable prior therapy
- Phase 1: Progressed on standard of care therapy with up to three prior treatments
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- Phase 2: MPNST with 0-3 prior systemic treatments (no prior radiotherapy is necessary).
- Eastern Cooperative Oncology Group (ECOG) performance status: 0, 1, or 2
- Age greater or equal to 18 years. Because no dosing or adverse event data are currently available on the use of PLX3397 in combination with sirolimus in patients <18 years of age, children are excluded from this study, but will be eligible for future pediatric trials.
- Presence of measurable lesions by Response Evaluation Criteria in Solid Tumors (RECIST) v1.1
- Allowable laboratory values with date range
- Absolute neutrophil count (ANC) ≥1.5 x 10^9/L, hemoglobin (Hgb) >9 g/dL, and platelet count ≥100 X 10^9/L
- Aspartate aminotransferase (AST)/alanine aminotransferase (ALT) ≤ upper limit of normal (ULN) or < 2.5 x ULN in the presence of liver metastases, bilirubin ≤ 1.5 x ULN, albumin ≥ 3.0g/dL.
- Bilirubin ≤ ULN; patients with hyperbilirubinemia clinically consistent with an inherited disorder of bilirubin metabolism (e.g., Gilbert syndrome) will be eligible at the discretion of the principal investigator.
- Albumin ≥ 3.0g/dL.
- Creatinine ≤ 1.5 x ULN or calculated creatinine clearance (CrCl) > 60 mL/min using the Cockcroft-Gault formula less than eight days pior to start of treatment.
- Women of child-bearing potential must have a negative serum pregnancy test at screening and must agree to use an effective form of contraception from the time of the negative pregnancy test and for a minimum of 3 months after the last dose of study drug. Effective forms of contraception include abstinence, hormonal contraceptive (injectable or implantable) in conjunction with a barrier method, or a double barrier method. Women of non-child-bearing potential must have been postmenopausal for ≥ 1 year or surgically sterile. The effects of PLX3397 and sirolimus on the developing human fetus are unknown. For this reason women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 3 months after completion of PLX3397 and sirolimus administration.
- Fertile men must agree to use an effective method of birth control during the study and for up to 3 months after the last dose of study drug.
- Willingness and ability to provide written informed consent prior to any study-related procedures and to comply with all study requirements.
- Agree to pre and post-treatment tumor biopsies.
- Prior treatment-related Adverse Events must be ≤ grade 1 (CTCAE v4.0), except alopecia, at time of initiating study drug.
Who cannot join
- Patients who have had chemotherapy or radiotherapy within 4 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier or within 14 days from cycle 1 day 1 of PLX3397 and sirolimus.
- Patients who are receiving any other investigational agents concurrently.
- Concomitant treatment with other anti-neoplastic agents (hormonal therapy acceptable).
- Patients with symptomatic brain metastases. Subjects with untreated brain metastasis ≤ 1 cm can be considered eligible if deemed asymptomatic by the investigator upon consultation with the medical monitor and do not require immediate radiation or steroids. Subjects with brain metastasis that is treated and stable for 1 month may be considered eligible if they are asymptomatic and on stable dose of steroids or if they do not require steroids following successful local therapy.
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to PLX3397 or sirolimus.
- For Phase 2 - Prior exposure to a receptor tyrosine kinase or mammalian target of Rapamycin inhibitor.
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- Pregnant women are excluded from this study because PLX3397 and sirolimus are agents with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with PLX3397 and sirolimus, breastfeeding should be discontinued if the mother is treated with PLX3397 and sirolimus.
- Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, active liver disease, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
- Active secondary malignancy unless the malignancy is not expected to interfere with the evaluation of safety and is approved by the Sponsor. Examples of the latter include basal or squamous cell carcinoma of the skin, in-situ carcinoma of the cervix, and isolated elevation of prostate-specific antigen. Subjects with a completely treated prior malignancy and no evidence of disease for ≥ 2 years are eligible.
- Major surgical procedure or significant traumatic injury within 14 days of initiating study drug or anticipation of the need for major surgery during the study.
- Previous radiotherapy to 25% or more of the bone marrow and/or radiation therapy within 28 days prior to study entry.
- Inability to swallow capsules, or refractory nausea and vomiting, malabsorption, an external biliary shunt, or significant bowel resection that would preclude adequate absorption.
- Congestive heart failure (CHF) New York (NY) Heart Association class III or IV; unstable coronary artery disease (myocardial infarction (MI) more than 6 months prior to study entry is permitted); or serious cardiac arrhythmia.
- Baseline QTc corrected by Fridericia's formula (QTcF) ≥ 450 ms (males) or ≥ 470 ms (females)
- HIV-positive patients on combination antiretroviral therapy are ineligible because of the potential for pharmacokinetic interactions with PLX3397. In addition, these patients are at increased risk of lethal infections when treated with marrow suppressive therapy. Similarly, patients with chronic or acute hepatitis C virus (HCV) or hepatitis B virus (HBV) infection are also ineligible.
- Of the five major cytochrome P450 (CYP) isoforms, 3A4 (BFC) may be involved in Phase I metabolism of PLX3397, with possibly CYP1A2 playing a minor role. Until information regarding exposure toxicity and exposure-response relationships are available with PLX3397, concomitant strong CYP3A4 inhibitors and inducers are not permitted in the event they alter the systemic exposure to PLX3397 (see Attachment 1 for a list of common CYP3A4 inhibitors and inducers). These include anticonvulsants, mycin antimicrobials, and antiretrovirals. Some common examples include inhibitors such as erythromycin, fluoxetine, gemfibrozil, and inducers such as rifampicin, carbamazepine, phenytoin, efavirenz, and nevirapine. Concomitant treatment is permitted if the medication is not expected to interfere with the evaluation of safety or efficacy of the study drug. Sirolimus undergoes extensive hepatic and intestinal metabolism via CYP3A4 and CYP3A5, as well as excretion by P-glycoprotein. Strong CYP3A inhibitors such as ketoconazole or grapefruit juice are not permitted. Patients should be monitored for supratherapeutic toxic levels of sirolimus and PLX3397. As bone marrow suppression including anemia, neutropenia, and thrombocytopenia have been reported in patients receiving sirolimus monotherapy, these adverse effects may be exacerbated in combination with PLX3397 for which patients will be closely monitored.
- Any patients on warfarin therapy
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
University of Michigan
Ann Arbor, Michigan, 48109-5848
Early Drug Development Center
Boston, Massachusetts, 02215
University of Iowa
Iowa City, Iowa, 52242
Columbia University
New York, New York, 10032
Washington University in St. Louis
St Louis, Missouri, 63110
About this study
Official title: Phase I Study Evaluating Combination Therapy With the Receptor Tyrosine Kinase Inhibitor PLX3397 and Sirolimus in Patients With Unresectable Sarcoma and Phase II Study in Malignant Peripheral Nerve Sheath Tumors
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
Read the detailed description
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.
- Conditions
- Sarcoma, Malignant Peripheral Nerve Sheath Tumors
- Being tested
- PLX3397, sirolimus
- Keywords
- Unresectable
- Medical subject headings
- Sarcoma, Neurofibrosarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PLX3397Drug
Also called pexidartinib
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.
sirolimusDrug
Also called Rapamune
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.
| Study group | Role | What they receive |
|---|---|---|
| Phase 1: PLX3397 and Sirolimus | Experimental | Subjects with unresectable or metastatic sarcoma will take orally PLX3397 (600 - 1000mg) in combination with Sirolimus (2-6mg) daily. Receives: Drug: PLX3397; Drug: sirolimus |
| Phase 2: PLX3397 and Sirolimus | Experimental | Subjects with unresectable or metastatic Malignant Peripheral Nerve Sheath Tumors (MPNSTs) will take PLX3397 and Sirolimus at the recommended Phase 2 dose (RP2D). Receives: Drug: PLX3397; Drug: sirolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: Up to 3 years
Secondary outcomes (1)
Overall Survival Rate
The time from the start of treatment until death, estimated using the Kaplan Meier method.
Measured: Up to 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Oct 2015 (estimated)
- Study start4 Nov 2015
- Primary completionJul 2023
- Study completion1 Oct 2024
Why it stopped: Poor enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for SarcomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gulam Manji
- Sponsor type
- University, hospital or other
- Collaborators
- Daiichi Sankyo
- Responsible party
- Sponsor-investigator, Gulam Manji, Columbia University
- Investigators
- Gulam A. Manji, MD, Principal investigator, Columbia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02584647
- Sponsor's protocol ID
- AAAO6059
- Other IDs
- R01FD005745
- Acronym
- PLX3397
- Status last verified
- Mar 2025
- First submitted
- 21 Oct 2015
- Last updated
- 21 Mar 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Manji GA, Van Tine BA, Lee SM, Raufi AG, Pellicciotta I, Hirbe AC, Pradhan J, Chen A, Rabadan R, Schwartz GK. A Phase I Study of the Combination of Pexidartinib and Sirolimus to Target Tumor-Associated Macrophages in Unresectable Sarcoma and Malignant Peripheral Nerve Sheath Tumors. Clin Cancer Res. 2021 Oct 15;27(20):5519-5527. doi: 10.1158/1078-0432.CCR-21-1779. Epub 2021 Jul 28.
PubMed 34321280
Study at a glance
From the registry record, last updated by the sponsor on 21 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study Evaluating Combination Therapy With the Receptor Tyrosine Kinase Inhibitor PLX3397 and Sirolimus in Patients With Unresectable Sarcoma and Phase II Study in Malignant Peripheral Nerve Sheath Tumors
- ClinicalTrials.gov ID
- NCT02584647
- Acronym / sponsor ID
- PLX3397 / AAAO6059
- Conditions
- Sarcoma, Malignant Peripheral Nerve Sheath Tumors
- Keywords
- Unresectable
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Poor enrollment
- Enrolment
- 39 participants (actual)
- Lead sponsor
- Gulam ManjiUniversity, hospital or other
- Collaborators
- Daiichi Sankyo
- Study start
- 4 Nov 2015 (actual)
- Primary completion
- Jul 2023 (actual)
- Study completion
- 1 Oct 2024 (actual)
- First posted
- 22 Oct 2015 (estimated)
- Results
- Posted 21 Mar 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Phase 1: PLX3397 and Sirolimus | Experimental | Drug: PLX3397, Drug: sirolimusSubjects with unresectable or metastatic sarcoma will take orally PLX3397 (600 - 1000mg) in combination with Sirolimus (2-6mg) daily. |
| Phase 2: PLX3397 and Sirolimus | Experimental | Drug: PLX3397, Drug: sirolimusSubjects with unresectable or metastatic Malignant Peripheral Nerve Sheath Tumors (MPNSTs) will take PLX3397 and Sirolimus at the recommended Phase 2 dose (RP2D). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (21 listed)
- Disease site/type with pathologic confirmation of diagnosis at participating cancer site.
- Extent of disease: Unresectable
- Allowable prior therapy
- Eastern Cooperative Oncology Group (ECOG) performance status: 0, 1, or 2
- Age greater or equal to 18 years. Because no dosing or adverse event data are currently available on the use of PLX3397 in combination with sirolimus in patients <18 years of age, children are excluded from this study, but will be eligible for future pediatric trials.
Key exclusion criteria (17 listed)
- Patients who have had chemotherapy or radiotherapy within 4 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier or within 14 days from cycle 1 day 1 of PLX3397 and sirolimus.
- Patients who are receiving any other investigational agents concurrently.
- Concomitant treatment with other anti-neoplastic agents (hormonal therapy acceptable).
- Patients with symptomatic brain metastases. Subjects with untreated brain metastasis ≤ 1 cm can be considered eligible if deemed asymptomatic by the investigator upon consultation with the medical monitor and do not require immediate radiation or steroids. Subjects with brain metastasis that is treated and stable for 1 month may be considered eligible if they are asymptomatic and on stable dose of steroids or if they do not require steroids following successful local therapy.
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to PLX3397 or sirolimus.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PLX3397Also: pexidartinibPLX3397 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. | Drug | Phase 1: PLX3397 and Sirolimus, Phase 2: PLX3397 and Sirolimus |
| sirolimusAlso: RapamuneSirolimus 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. | Drug | Phase 1: PLX3397 and Sirolimus, Phase 2: PLX3397 and Sirolimus |
Primary outcome
Maximum Tolerated Dose (MTD) - Phase 1
Time frame: Up to 3 years
The highest dose of a drug or treatment that does not cause unacceptable side effects, which will be used in Phase 2.
Progression Free Survival (PFS) Rate - Phase 2
Time frame: Up to 3 years
The time from the start of treatment until disease progression or death from any cause, calculated in weeks.
1 secondary outcome
Overall Survival Rate
Time frame: Up to 3 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Mar 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 34321280Manji GA, Van Tine BA, Lee SM, Raufi AG, Pellicciotta I, Hirbe AC, Pradhan J, Chen A, Rabadan R, Schwartz GK. A Phase I Study of the Combination of Pexidartinib and Sirolimus to Target Tumor-Associated Macrophages in Unresectable Sarcoma and Malignant Peripheral Nerve Sheath Tumors. Clin Cancer Res. 2021 Oct 15;27(20):5519-5527. doi: 10.1158/1078-0432.CCR-21-1779. Epub 2021 Jul 28.
- Herbert Irving Comprehensive Cancer CenterLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Gulam A. Manji, MD principal investigator
Columbia University
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing PLX3397 and sirolimus in people with Sarcoma and Malignant Peripheral Nerve Sheath Tumors, run by Gulam Manji at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 21 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 21 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 21 Mar 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum Tolerated Dose (MTD) - Phase 1 (time frame: Up to 3 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02584647, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Mar 2025.