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

Stopped early · Phase 2

Conditions studied: Acute Myeloid Leukemia

In brief

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.

Key facts

Study ID

NCT04905407

Run by

Syros Pharmaceuticals

People needed

66

Starts

2021-08-26

Expected to finish

2024-08-12

Last updated by the study team

2025-02-24

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• All participants must have obtained a blood sample for RARA biomarker investigational assay testing prior to starting treatment on Cycle 1 Day 1. The results of the investigational biomarker assay for all participants must be confirmed as RARA-positive by Cycle 1 Day 8 to enroll (Part 1) or to be randomized (Part 2) in the study.
• Participants must have newly diagnosed, previously untreated non-acute promyelocytic leukemia (APL) AML with a bone marrow or peripheral blood blast count ≥20% and must be unlikely to tolerate standard intensive chemotherapy at the time of Cycle 1 Day 1 Visit due to age, performance status, or comorbidities based on at least one of the following criteria:
• age ≥75 years old, or
• age <75 years old, with at least one of the following:
• Eastern Cooperative Oncology Group (ECOG) performance status of 3
• cardiac history of congestive heart failure (CHF) or documented ejection fraction (EF) ≤50%
• pulmonary disease with diffusing capacity of the lungs for carbon monoxide (DLCO) ≤65% or forced expiratory volume in one second (FEV1) ≤65%
• creatinine clearance ≥30 milliliters (mL)/minute (min) to <45 mL/min based on the Cockcroft-Gault glomerular filtration rate estimation
• hepatic impairment with total bilirubin >1.5 to ≤3.0 * upper limit of normal (ULN)
• any other comorbidity that the investigator judges to be incompatible with intensive chemotherapy, and reviewed and approved by the sponsor.

You may not qualify if…

• Participants have APL.
• Participants have known active central nervous system involvement with AML.
• Prior treatment (before Cycle 1 Day 1) for the diagnosis of AML, myelodysplastic syndromes (MDS), or antecedent hematologic malignancy with any hypomethylating agent, venetoclax, chemotherapy, or hematopoietic stem cell transplantation (HSCT), with the exception of prior treatment with hydroxyurea.

Where it is running

• City of Hope — Duarte, California, United States
• UCLA Medical Center Division of Hematology/Oncology — Los Angeles, California, United States
• University of Colorado — Denver, Colorado, United States
• Sarah Cannon Research Institute at Colorado Blood Cancer Institute — Denver, Colorado, United States
• Hartford HealthCare — Hartford, Connecticut, United States
• Northside — Atlanta, Georgia, United States
• University of Mississippi — Jackson, Mississippi, United States
• HCA Midwest Research Medical Center — Kansas City, Missouri, United States
• Atlantic Health — Morristown, New Jersey, United States
• Roswell Park Comprehensive Cancer Center — Buffalo, New York, United States
• Icahn School of Medicine at Mount Sinai — New York, New York, United States
• Memorial Sloan Kettering Cancer Center — New York, New York, United States
• Novant Health — Charlotte, North Carolina, United States
• The Ohio State University James Cancer Hospital — Columbus, Ohio, United States
• Oregon Health & Science University — Portland, Oregon, United States
• CHU Lyon Sud — Pierre-Bénite, Pierre Benite, France
• CHU Angers — Angers, France
• CH de la Côte Basque — Bayonne, France
• Hôpital Avicenne Hématologie Clinique — Bobigny, France
• CHU de Caen Normandie — Caen, France
• CHU Limoges — Limoges, France
• CHU de Nantes - Hôtel Dieu — Nantes, France
• Hôpital l'Archet- CHU de Nice — Nice, France
• CHU de Bordeaux - Hôpital Haut-Lévèque — Pessac, France
• CHU de Poitiers — Poitiers, France

Full record on ClinicalTrials.gov

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