NCT02141828

A Phase 1 Dose Escalation and Expanded Cohort Study of EPZ-5676 in the Treatment of Pediatric Patients With Relapsed/Refractory Leukemias Bearing a Rearrangement of the MLL Gene

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 18 (actual)
• Sponsor: Epizyme, Inc. · Industry
• Sex: All
• Age: 3 Months – 18 Years
• Healthy volunteers: Not accepted

Summary

A subset of patients with acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL) harbor rearrangements of the MLL gene, which are detected either by cytogenetic or fluorescent in situ hybridization evaluation at the time of diagnosis. A protein called DOT1L plays an important role in the malignant process in these leukemias. EPZ-5676 is a molecule that blocks the activity of DOT1L, and is therefore being evaluated in the treatment of patients with MLL-rearranged leukemias.

Detailed description

This is a Phase 1b study of EPZ-5676 in pediatric patients. The study will have two phases. The first phase will assess escalating doses of EPZ-5676 in order to determine the maximally tolerated dose (MTD) or recommended phase 2 dose (RP2D) of EPZ-5676 as a 28-day continuous IV infusion. Once the MTD and/or RP2D is established, a second phase of the study will further evaluate the safety of EPZ-5676 and assess the anti-leukemia activity.

Conditions

• Leukemia
• Acute Myeloid Leukemia
• Acute Lymphocytic Leukemia
• Acute Leukemias

Interventions

Type	Name	Description
DRUG	EPZ-5676	28-day continuous IV infusion of each 28-day cycle, given until disease progression or unacceptable toxicity develops.

Timeline

• Start date: 2014-05-01
• Primary completion: 2016-05-01
• Completion: 2016-06-01
• First posted: 2014-05-20
• Last updated: 2023-06-18

Locations

9 sites across 2 countries: United States, Canada