Tazemetostat for Chordoma

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Boston Children's Hospital, Boston, MA
Chordoma+10 More
Tazemetostat - Drug
Eligibility
< 65
All Sexes
What conditions do you have?
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Study Summary

This research study involves a combination of three drugs given together as a possible treatment for malignant rhabdoid tumor, atypical teratoid rhabdoid tumor, epithelioid sarcoma, chordoma or other tumors that are deficient in one of two possible proteins, either INI-1 (SMARCB1) or SMARCA4. The names of the study drugs involved in this study are: Tazemetostat (TAZVERIK) Nivolumab (OPDIVO) Ipilimumab (YERVOY)

Eligible Conditions

  • Chordoma
  • Rhabdoid Tumor of the Kidney (RTK)
  • INI1 (SMARCB1)-Deficient Primary CNS Malignant Tumors
  • Malignant Rhabdoid Tumor (MRT)
  • Atypical Teratoid Rhabdoid Tumors (ATRT)
  • SMARCA4-deficient Primary CNS Malignant Tumors
  • Epithelioid Sarcoma (ES)

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

2 Primary · 4 Secondary · Reporting Duration: Treatment duration is a median of N cycles range (t1 - t2). Treatment continues until disease progression or unacceptable toxicity up to 5.5 years

Year 5
Incidence of Grade 3 or Higher Treatment-Related Toxicity
Year 5
Overall Response Rate (ORR)
Year 3
Median Progression-free survival (PFS)
Year 5
Maximal Tolerated Dose (MTD)
Recommended Pediatric Phase 2 Dose (RP2D)
Up through 5.5 years
Median Overall Survival (OS)

Trial Safety

Safety Progress

1 of 3

Trial Design

8 Treatment Groups

Phase I b: DOSE ESCALATION (STRATUM B, NON-ATRT, NON-CNS)
1 of 8
EXP A1: TAZEMETOSTAT + NIVOLUMAB + IPILIMUMAB DOSE EXPANSION (SUBSTRATA A1)
1 of 8
EXP B1: TAZEMETOSTAT + NIVOLUMAB + IPILIMUMAB DOSE EXPANSION (SUBSTRATA B1)
1 of 8
Phase I a: DOSE ESCALATION (STRATUM A, ATRT and primary CNS malignant tumor, INI...
1 of 8
EXP B2: TAZEMETOSTAT + NIVOLUMAB + IPILIMUMAB DOSE EXPANSION (SUBSTRATA B2)
1 of 8
EXP A2: TAZEMETOSTAT + NIVOLUMAB + IPILIMUMAB DOSE EXPANSION (SUBSTRATA A2)
1 of 8
EXP A3: TAZEMETOSTAT + NIVOLUMAB + IPILIMUMAB DOSE EXPANSION (SUBSTRATA A3)
1 of 8
EXP B3: TAZEMETOSTAT + NIVOLUMAB + IPILIMUMAB DOSE EXPANSION (SUBSTRATA B3)
1 of 8
Experimental Treatment

49 Total Participants · 8 Treatment Groups

Primary Treatment: Tazemetostat · No Placebo Group · Phase 1 & 2

Phase I b: DOSE ESCALATION (STRATUM B, NON-ATRT, NON-CNS)Experimental Group · 3 Interventions: Tazemetostat, Ipilimumab, Nivolumab · Intervention Types: Drug, Drug, Drug
EXP A1: TAZEMETOSTAT + NIVOLUMAB + IPILIMUMAB DOSE EXPANSION (SUBSTRATA A1)Experimental Group · 3 Interventions: Tazemetostat, Ipilimumab, Nivolumab · Intervention Types: Drug, Drug, Drug
EXP B1: TAZEMETOSTAT + NIVOLUMAB + IPILIMUMAB DOSE EXPANSION (SUBSTRATA B1)Experimental Group · 3 Interventions: Tazemetostat, Ipilimumab, Nivolumab · Intervention Types: Drug, Drug, Drug
Phase I a: DOSE ESCALATION (STRATUM A, ATRT and primary CNS malignant tumor, INI/SMARCA4-deficient)Experimental Group · 3 Interventions: Tazemetostat, Ipilimumab, Nivolumab · Intervention Types: Drug, Drug, Drug
EXP B2: TAZEMETOSTAT + NIVOLUMAB + IPILIMUMAB DOSE EXPANSION (SUBSTRATA B2)Experimental Group · 3 Interventions: Tazemetostat, Ipilimumab, Nivolumab · Intervention Types: Drug, Drug, Drug
EXP A2: TAZEMETOSTAT + NIVOLUMAB + IPILIMUMAB DOSE EXPANSION (SUBSTRATA A2)Experimental Group · 3 Interventions: Tazemetostat, Ipilimumab, Nivolumab · Intervention Types: Drug, Drug, Drug
EXP A3: TAZEMETOSTAT + NIVOLUMAB + IPILIMUMAB DOSE EXPANSION (SUBSTRATA A3)Experimental Group · 3 Interventions: Tazemetostat, Ipilimumab, Nivolumab · Intervention Types: Drug, Drug, Drug
EXP B3: TAZEMETOSTAT + NIVOLUMAB + IPILIMUMAB DOSE EXPANSION (SUBSTRATA B3)Experimental Group · 3 Interventions: Tazemetostat, Ipilimumab, Nivolumab · Intervention Types: Drug, Drug, Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Tazemetostat
2016
Completed Phase 2
~760
Ipilimumab
2014
Completed Phase 3
~3350
Nivolumab
2014
Completed Phase 3
~5540

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: treatment duration is a median of n cycles range (t1 - t2). treatment continues until disease progression or unacceptable toxicity up to 5.5 years
Closest Location: Boston Children's Hospital · Boston, MA
Photo of Boston  1Photo of Boston  2Photo of Boston  3
2020First Recorded Clinical Trial
2 TrialsResearching Chordoma
349 CompletedClinical Trials

Eligibility Criteria

Age < 65 · All Participants · 10 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
Atypical teratoid rhabdoid tumor (ATRT) is a rare tumor of the central nervous system.
You have epithelioid sarcoma.
You have a poorly differentiated or de-differentiated chordoma.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.