Trial · NCT02601937
EZH2 Inhibitor Tazemetostat in Pediatric Subjects With Relapsed or Refractory INI1-Negative Tumors or Synovial Sarcoma
NCT02601937, Phase 1, Completed, Epizyme, Inc.; results first posted 2024-10-03
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. EZH2 Inhibitor Tazemetostat in Pediatric Subjects With Relapsed or Refractory INI1-Negative Tumors or Synovial Sarcoma. Trials, record NCT02601937. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02601937
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- EZH2 Inhibitor Tazemetostat in Pediatric Subjects With Relapsed or Refractory INI1-Negative Tumors or Synovial Sarcoma
- NCT number
- NCT02601937
- Status
- Completed
- Phases
- Phase 1
- Enrollment
- 109 participants, actual
- Lead sponsor
- Epizyme, Inc.
- Conditions
- Rhabdoid Tumors, INI1-negative Tumors, Synovial Sarcoma, Malignant Rhabdoid Tumor of Ovary
- Interventions
- Tazemetostat
- First posted
- 2015-11-11
- Last updated
- 2024-10-03
- Other registries' IDs
- eudract:2015-002468-18
- Results first posted
- 2024-10-03
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Recommended Phase 2 Dose (RP2D) (Dose Escalation Only) | Cycle 1, from the start of study treatment (Day 1) until the end of the first Cycle (Day 28) of treatment | The incidence and severity of treatment-emergent adverse events qualifying as protocol-defined dose-limiting toxicities that occurred during the first month of treatment was used to determine the RP2D and/or maximum tolerated dose (MTD) in pediatric patients treated with tazemetostat. |
| Primary | Number of Dose-limiting Toxicities (Dose Escalation Only) | Cycle 1, from the start of study treatment (Day 1) until the end of the first Cycle (Day 28) of treatment | The RP2D in pediatric patients treated with tazemetostat as determined by the incidence and severity of treatment-emergent adverse events qualifying as protocol-defined dose-limiting toxicities that occurred during the first month of treatment. |
| Primary | Overall Response Rate (ORR) (Dose Expansion Only) | RECIST assessments performed at screening (within 14 days before start of study intervention) and every 8 weeks post start of dosing, approximately up to 302 weeks | ORR is defined as the percentage of patients who achieved a confirmed complete response (CR) and/or partial response (PR) defined by response evaluation criteria in solid tumors (RECIST) or response assessment in neuro-oncology (RANO) criteria from the start of tazemetostat treatment until disease progression or the start of subsequent anticancer therapy, whichever occurs first. CR is defined as disappearance of all target and non-target lesions (with all lymph nodes must be non-pathological in size or under 10 millimeters \[mm\] in short axis) and PR is defined as at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. ORR= CR+PR. |
| Secondary | ORR (Dose Escalation Only) | RECIST assessments performed at screening (within 14 days before start of study intervention) and every 8 weeks post start of dosing, approximately up to 302 weeks | ORR is defined as the percentage of patients who achieved a confirmed CR and/or PR defined by RECIST or RANO criteria from the start of tazemetostat treatment until disease progression or the start of subsequent anticancer therapy, whichever occurs first. CR is defined as disappearance of all target and non-target lesions (with all lymph nodes must be non-pathological in size or under 10 mm in short axis) and PR is defined as at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. ORR= CR+PR. |
| Secondary | Progression Free Survival (PFS) (Dose Expansion Only) | RECIST assessments performed at screening (within 14 days before start of study intervention) and every 8 weeks post start of dosing, approximately up to 302 weeks | PFS was defined as the interval of time (in weeks) from the date of first dose of study drug and the earliest date of disease progression or death, from any cause defined by RECIST or RANO criteria. Disease progression is defined as at least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study (including at least a 5 mm absolute increase). The presence of new lesions also constitutes to disease progression. |
| Secondary | Overall Survival (Dose Expansion Only) | RECIST assessments performed at screening (within 14 days before start of study intervention) and every 8 weeks post start of dosing, approximately up to 302 weeks | Overall survival was defined as the time (in weeks) from the first dose of study drug to the date of death due to any cause. |
Source ClinicalTrials.gov
Outcomes6
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Recommended Phase 2 Dose (RP2D) (Dose Escalation Only) | Cycle 1, from the start of study treatment (Day 1) until the end of the first Cycle (Day 28) of treatment | The ITT population included all patients who received at least one dose of tazemetostat. | mg/m^2 BID | NUMBER |
| PRIMARY | Number of Dose-limiting Toxicities (Dose Escalation Only) | Cycle 1, from the start of study treatment (Day 1) until the end of the first Cycle (Day 28) of treatment | Dose-Limiting Toxicity (DLT) population included all patients in the safety population set who experienced a DLT during Cycle 1 or were not removed from Cycle 1 for reasons other than toxicity and did not have an interruption in study treatment for more than 14 days during Cycle 1. Only those patients with data collected at Cycle 1 are reported. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Overall Response Rate (ORR) (Dose Expansion Only) | RECIST assessments performed at screening (within 14 days before start of study intervention) and every 8 weeks post start of dosing, approximately up to 302 weeks | The ITT population included all patients who received at least one dose of tazemetostat. | Percentage of participants | NUMBER |
| SECONDARY | ORR (Dose Escalation Only) | RECIST assessments performed at screening (within 14 days before start of study intervention) and every 8 weeks post start of dosing, approximately up to 302 weeks | The ITT population included all patients who received at least one dose of tazemetostat. | Percentage of participants | NUMBER |
| SECONDARY | Progression Free Survival (PFS) (Dose Expansion Only) | RECIST assessments performed at screening (within 14 days before start of study intervention) and every 8 weeks post start of dosing, approximately up to 302 weeks | The ITT population included all patients who received at least one dose of tazemetostat. | weeks | MEDIAN |
| SECONDARY | Overall Survival (Dose Expansion Only) | RECIST assessments performed at screening (within 14 days before start of study intervention) and every 8 weeks post start of dosing, approximately up to 302 weeks | The ITT population included all patients who received at least one dose of tazemetostat. | weeks | MEDIAN |
Source ClinicalTrials.gov
Results by group28
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Recommended Phase 2 Dose (RP2D) (Dose Escalation Only) | Overall Dose Escalation | RP2D for patients with ATRT or CNS involvement | 1200 | mg/m^2 BID | |
| Recommended Phase 2 Dose (RP2D) (Dose Escalation Only) | Overall Dose Escalation | RP2D for patients without CNS involvement | 520 | mg/m^2 BID | |
| Number of Dose-limiting Toxicities (Dose Escalation Only) | Dose Escalation Level 1 | 0 | Participants | ||
| Number of Dose-limiting Toxicities (Dose Escalation Only) | Dose Escalation Level 2 | 1 | Participants | ||
| Number of Dose-limiting Toxicities (Dose Escalation Only) | Dose Escalation Level 3 | 0 | Participants | ||
| Number of Dose-limiting Toxicities (Dose Escalation Only) | Dose Escalation Level 4 | 0 | Participants | ||
| Number of Dose-limiting Toxicities (Dose Escalation Only) | Dose Escalation Level 5 | 0 | Participants | ||
| Number of Dose-limiting Toxicities (Dose Escalation Only) | Dose Escalation Level 6 | 1 | Participants | ||
| Number of Dose-limiting Toxicities (Dose Escalation Only) | Dose Escalation Level 7 | 0 | Participants | ||
| Overall Response Rate (ORR) (Dose Expansion Only) | Dose Expansion Cohort 1 | 26.3 | Percentage of participants |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 results by groupParticipant flow66
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Period 1: Dose Escalation Phase | STARTED | Dose Escalation Level 1 | 8 | |
| Period 1: Dose Escalation Phase | STARTED | Dose Escalation Level 2 | 6 | |
| Period 1: Dose Escalation Phase | STARTED | Dose Escalation Level 3 | 6 | |
| Period 1: Dose Escalation Phase | STARTED | Dose Escalation Level 4 | 7 | |
| Period 1: Dose Escalation Phase | STARTED | Dose Escalation Level 5 | 6 | |
| Period 1: Dose Escalation Phase | STARTED | Dose Escalation Level 6 | 6 | |
| Period 1: Dose Escalation Phase | STARTED | Dose Escalation Level 7 | 7 | |
| Period 1: Dose Escalation Phase | STARTED | Dose Expansion Cohort 1 | 0 | |
| Period 1: Dose Escalation Phase | STARTED | Dose Expansion Cohort 2 | 0 | |
| Period 1: Dose Escalation Phase | STARTED | Dose Expansion Cohort 3 | 0 |
1 to 10 of 66Source ClinicalTrials.gov
See all 66 participant flowAdverse events4,026
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 | Otitis media | Infections and infestations | Dose Escalation Level 1 | 1 | 8 | 1 |
| other | Otitis media | Infections and infestations | Dose Escalation Level 2 | 1 | 6 | 2 |
| other | Otitis media | Infections and infestations | Dose Escalation Level 3 | 0 | 6 | 0 |
| other | Otitis media | Infections and infestations | Dose Escalation Level 4 | 1 | 7 | 2 |
| other | Otitis media | Infections and infestations | Dose Escalation Level 5 | 1 | 6 | 1 |
| other | Otitis media | Infections and infestations | Dose Escalation Level 6 | 1 | 6 | 1 |
| other | Otitis media | Infections and infestations | Dose Escalation Level 7 | 0 | 7 | 0 |
| other | Otitis media | Infections and infestations | Dose Expansion Cohort 1 | 3 | 19 | 3 |
| other | Otitis media | Infections and infestations | Dose Expansion Cohort 2 | 0 | 20 | 0 |
| other | Otitis media | Infections and infestations | Dose Expansion Cohort 3 | 0 | 18 | 0 |
1 to 10 of 4,026Source ClinicalTrials.gov
See all 4,026 adverse eventsAdverse event totals33
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Dose Escalation Level 1 | 7 | 8 |
| Total, all-cause mortality | Dose Escalation Level 2 | 6 | 6 |
| Total, all-cause mortality | Dose Escalation Level 3 | 4 | 6 |
| Total, all-cause mortality | Dose Escalation Level 4 | 4 | 7 |
| Total, all-cause mortality | Dose Escalation Level 5 | 5 | 6 |
| Total, all-cause mortality | Dose Escalation Level 6 | 4 | 6 |
| Total, all-cause mortality | Dose Escalation Level 7 | 7 | 7 |
| Total, all-cause mortality | Dose Expansion Cohort 1 | 15 | 19 |
| Total, all-cause mortality | Dose Expansion Cohort 2 | 18 | 20 |
| Total, all-cause mortality | Dose Expansion Cohort 3 | 16 | 18 |
1 to 10 of 33Source ClinicalTrials.gov
See all 33 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
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.
None in this release.
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/NCT02601937
GET https://conductscience.org/api/trials/v1/NCT02601937/results?page=2