A Phase 1 Study of the EZH2 Inhibitor Tazemetostat in Pediatric Subjects With Relapsed or Refractory INI1-Negative Tumors or Synovial Sarcoma

• Status: Active, not recruiting
• Trial end date: 2022-11-01

Summary

This is a Phase I, open-label, dose escalation and dose expansion study with BID (suspension) and TID (tablet) oral dose of tazemetostat. Subjects will be screened for eligibility within 14 days of the planned first dose of tazemetostat. A treatment cycle will be 28 days. Response assessment will be evaluated after 8 weeks of treatment and subsequently every 8 weeks while on study.

The study has two parts: Dose Escalation and Dose Expansion.

Dose escalation for subjects with the following relapsed/refractory malignancies:

• Rhabdoid tumors:

• Atypical teratoid rhabdoid tumor (ATRT)

• Malignant rhabdoid tumor (MRT)

• Rhabdoid tumor of kidney (RTK)

• Selected tumors with rhabdoid features

• INI1-negative tumors:

• Epithelioid sarcoma

• Epithelioid malignant peripheral nerve sheath tumor

• Extraskeletal myxoid chondrosarcoma

• Myoepithelial carcinoma

• Renal medullary carcinoma

• Other INI1-negative malignant tumors (e.g., dedifferentiated chordoma) (with Sponsor approval)

• Synovial Sarcoma with a SS18-SSX rearrangement Dose Escalation cohorts are closed to enrollment.

Dose Expansion at the MTD or the RP2D

• Cohort 1 - ATRT (closed to enrollment)

• Cohort 2 - MRT/RTK/selected tumors with rhabdoid features (closed to enrollment)

• Cohort 3 - INI-negative tumors:

• Epithelioid sarcoma

• Epithelioid malignant peripheral nerve sheath tumor

• Extraskeletal myxoid chondrosarcoma

• Myoepithelial carcinoma

• Renal medullary carcinoma

• Chordoma (poorly differentiated or de-differentiated)

• Other INI1-negative malignant tumors (e.g., dedifferentiated chordoma) with Sponsor approval

• Cohort 4 - Tumor types eligible for Cohorts 1 through 3 or synovial sarcoma with SS18-SSX rearrangement (closed to enrollment)

• Phase: Phase 1

Details

• Lead Sponsor: Epizyme, Inc.