NCT04905407

Tamibarotene Plus Venetoclax/Azacitidine in Participants With Newly Diagnosed Acute Myeloid Leukemia (AML)

Tamibarotene in Combination With Venetoclax and Azacitidine in Previously Untreated Adult Patients Selected for RARA-positive AML Who Are Ineligible for Standard Induction Therapy

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 66 (actual)
• Sponsor: Syros Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Tamibarotene is being studied as a treatment for participants with a type of leukemia called acute myeloid leukemia, or AML for short. Tamibarotene is being studied as a treatment for participants with AML whose cancer has a specific genetic abnormality characterized by the overexpression of the retinoic acid receptor alpha (RARA) gene. This genetic profile is found in about 3 of every 10 people with AML. During the trial, tamibarotene will be given with 2 other drugs that are already used together to treat people who have AML and who cannot start treatment with standard chemotherapy.

Detailed description

This study consists of 3 parts. In Part 1, the safety, tolerability, and pharmacokinetic (PK) evaluation of tamibarotene/venetoclax/azacitidine combination will inform the appropriate tamibarotene dose to be combined with the standard of care (SOC) venetoclax/azacitidine in Part 2 and Part 3. In Part 2, participants will be randomized 1:1 to receive either tamibarotene/venetoclax/azacitidine or venetoclax/azacitidine to compare the clinical activity of the 2 combinations. In Part 3, tamibarotene will be added to the venetoclax/azacytidine regimen of a subset of Part 2 participants who experience progressive disease, relapse after initial complete remission (CR) or CR with incomplete blood count recovery (CRi) response, or treatment failure.

Conditions

• Acute Myeloid Leukemia

Interventions

Type	Name	Description
DRUG	Tamibarotene	Tamibarotene tablets will be administered per dose and schedule specified in the arm.
DRUG	Venetoclax	Venetoclax tablets will be administered per dose and schedule specified in the arm.
DRUG	Azacitidine	Azacitidine injection will be administered per dose and schedule specified in the arm.

Timeline

• Start date: 2021-08-26
• Primary completion: 2024-08-12
• Completion: 2024-08-12
• First posted: 2021-05-27
• Last updated: 2025-02-24
• Results posted: 2025-02-24

Locations

28 sites across 2 countries: United States, France

Regulatory

• FDA-regulated drug study