Trial · NCT02584647
PLX3397 Plus Sirolimus in Unresectable Sarcoma and Malignant Peripheral Nerve Sheath Tumors
NCT02584647, Phase 1, Phase 2, Terminated, Gulam Manji; results first posted 2025-03-21
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. PLX3397 Plus Sirolimus in Unresectable Sarcoma and Malignant Peripheral Nerve Sheath Tumors. Trials, record NCT02584647. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02584647
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- PLX3397 Plus Sirolimus in Unresectable Sarcoma and Malignant Peripheral Nerve Sheath Tumors
- NCT number
- NCT02584647
- Status
- Terminated
- Phases
- Phase 1, Phase 2
- Enrollment
- 39 participants, actual
- Lead sponsor
- Gulam Manji
- Conditions
- Sarcoma, Malignant Peripheral Nerve Sheath Tumors
- Interventions
- PLX3397, sirolimus
- First posted
- 2015-10-22
- Last updated
- 2025-03-21
- Results first posted
- 2025-03-21
- Limitations and caveats
- In the present study, the combination of pexidartinib and sirolimus failed to meet the primary endpoint in a study that was stopped due to poor enrollment due to COVID-19.
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Maximum Tolerated Dose (MTD) - Phase 1 | 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. |
| Primary | Progression Free Survival (PFS) Rate - Phase 2 | Up to 3 years | The time from the start of treatment until disease progression or death from any cause, calculated in weeks. |
| Secondary | Overall Survival Rate | Up to 3 years | The time from the start of treatment until death, estimated using the Kaplan Meier method. |
Source ClinicalTrials.gov
Outcomes3
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Maximum Tolerated Dose (MTD) - Phase 1 | Up to 3 years | mg | NUMBER | |
| PRIMARY | Progression Free Survival (PFS) Rate - Phase 2 | Up to 3 years | weeks | MEDIAN | |
| SECONDARY | Overall Survival Rate | Up to 3 years | Only assessed for Phase 2 | weeks | MEDIAN |
Source ClinicalTrials.gov
Results by group4
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Maximum Tolerated Dose (MTD) - Phase 1 | Phase 1 | Pexidartinib | 1000 | mg | |
| Maximum Tolerated Dose (MTD) - Phase 1 | Phase 1 | Sirolimus | 2 | mg | |
| Progression Free Survival (PFS) Rate - Phase 2 | Phase 2 | 6 | 95% Confidence Interval 6 to 19.1 | weeks | |
| Overall Survival Rate | Phase 2 | 17.9 | 95% Confidence Interval 13.7 to NA | weeks |
Source ClinicalTrials.gov
Participant flow90
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Dose Level 1 (S: 2mg, P: 600mg) | STARTED | Cohort 1, Phase 1 | 0 | |
| Dose Level 1 (S: 2mg, P: 600mg) | STARTED | Cohort 2, Phase 1 | 0 | |
| Dose Level 1 (S: 2mg, P: 600mg) | STARTED | Cohort 3, Phase 1 | 0 | |
| Dose Level 1 (S: 2mg, P: 600mg) | STARTED | Cohort 4, Phase 1 | 0 | |
| Dose Level 1 (S: 2mg, P: 600mg) | STARTED | Phase 2 Cohort | 0 | |
| Dose Level 1 (S: 2mg, P: 600mg) | COMPLETED | Cohort 1, Phase 1 | 0 | |
| Dose Level 1 (S: 2mg, P: 600mg) | COMPLETED | Cohort 2, Phase 1 | 0 | |
| Dose Level 1 (S: 2mg, P: 600mg) | COMPLETED | Cohort 3, Phase 1 | 0 | |
| Dose Level 1 (S: 2mg, P: 600mg) | COMPLETED | Cohort 4, Phase 1 | 0 | |
| Dose Level 1 (S: 2mg, P: 600mg) | COMPLETED | Phase 2 Cohort | 0 |
1 to 10 of 90Source ClinicalTrials.gov
See all 90 participant flowAdverse events485
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Alanine aminotransferase increased | Hepatobiliary disorders | Cohort 1, Phase 1 | 2 | 2 | |
| other | Alanine aminotransferase increased | Hepatobiliary disorders | Cohort 2, Phase 1 | 3 | 12 | |
| other | Alanine aminotransferase increased | Hepatobiliary disorders | Cohort 3, Phase 1 | 2 | 9 | |
| other | Alanine aminotransferase increased | Hepatobiliary disorders | Cohort 4, Phase 1 | 1 | 1 | |
| other | Alanine aminotransferase increased | Hepatobiliary disorders | Phase 2 Cohort | 3 | 14 | |
| other | Alkaline phosphatase increased | Hepatobiliary disorders | Cohort 1, Phase 1 | 2 | 2 | |
| other | Alkaline phosphatase increased | Hepatobiliary disorders | Cohort 2, Phase 1 | 4 | 12 | |
| other | Alkaline phosphatase increased | Hepatobiliary disorders | Cohort 3, Phase 1 | 4 | 9 | |
| other | Alkaline phosphatase increased | Hepatobiliary disorders | Cohort 4, Phase 1 | 1 | 1 | |
| other | Alkaline phosphatase increased | Hepatobiliary disorders | Phase 2 Cohort | 3 | 14 |
1 to 10 of 485Source ClinicalTrials.gov
See all 485 adverse eventsAdverse event totals15
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1, Phase 1 | 0 | 2 |
| Total, all-cause mortality | Cohort 2, Phase 1 | 0 | 12 |
| Total, all-cause mortality | Cohort 3, Phase 1 | 1 | 9 |
| Total, all-cause mortality | Cohort 4, Phase 1 | 0 | 1 |
| Total, all-cause mortality | Phase 2 Cohort | 1 | 14 |
| Total, other adverse events | Cohort 1, Phase 1 | 2 | 2 |
| Total, other adverse events | Cohort 2, Phase 1 | 12 | 12 |
| Total, other adverse events | Cohort 3, Phase 1 | 9 | 9 |
| Total, other adverse events | Cohort 4, Phase 1 | 1 | 1 |
| Total, other adverse events | Phase 2 Cohort | 14 | 14 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 34321280 | DERIVED |
Source PubMed
Grants1
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| R01FD005745 | Phase 2 study of combination therapy with PLX3397 and sirolimus to target tumor-associated macrophages in malignant peripheral nerve sheath tumors | MANJI, GULAM ABBAS; SCHWARTZ, GARY K | COLUMBIA UNIVERSITY HEALTH SCIENCES | FOOD AND DRUG ADMINISTRATION | 2017 to 2023 |
Source NIH RePORTER
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02584647
GET https://conductscience.org/api/trials/v1/NCT02584647/flow?page=2