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Nucleoside Analog

Tamibarotene + Azacitidine for Myelodysplastic Syndrome

Phase 3

Recruiting

Research Sponsored by Syros Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Participants must have Eastern Cooperative Oncology Group (ECOG) Performance Status of ≤2.

Diagnosis of MDS according to the World Health Organization (WHO) classification (Arber 2016) and classified by the Revised International Prognostic Scoring System (IPSS R) risk category as very high, high, or intermediate risk.

Must not have

Participants are suitable for and agree to undergo allogeneic HSCT at the time of Screening.

Participants who received prior treatment for MDS with any hypomethylating agent, chemotherapy or allogeneic HSCT.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Pivotal Trial

Summary

This trial compares two treatments for MDS patients who are positive for the RARA gene. The goal is to see if one treatment results in more complete remissions than the other.

Who is the study for?

This trial is for adults over 18 with newly diagnosed higher-risk myelodysplastic syndrome (HR-MDS) who test positive for RARA and haven't been treated before. They should be able to perform daily activities with some limitations (ECOG ≤2). Those planning a stem cell transplant or who've had certain MDS treatments can't join.

What is being tested?

The study tests Tamibarotene combined with Azacitidine against Azacitidine plus a placebo in patients with HR-MDS. The main focus is to see which group has more complete remission of the disease.

What are the potential side effects?

Possible side effects include reactions related to the immune system, blood abnormalities, gastrointestinal issues, fatigue, and skin changes. Each patient's experience may vary.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I can take care of myself and am up and about more than half of my waking hours.

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My MDS is classified as very high, high, or intermediate risk according to WHO.

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I am 18 years or older.

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My cancer is RARA-positive according to a specific test.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I agree to have a stem cell transplant from a donor.

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I have received treatment for MDS with specific drugs or a stem cell transplant.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Tamibarotene + AzacitidineExperimental Treatment2 Interventions

Tamibarotene: 6 mg administered orally twice per day (BID) on Days 8 through 28 of each 28-day treatment cycle. Azacitidine: 75 mg/m\^2 administered intravenously or subcutaneously each day on Days 1 through 7 of each 28-day treatment cycle.

Group II: Tamibarotene Matched Placebo + AzacitidinePlacebo Group2 Interventions

Placebo: Tamibarotene-matching tablets administered orally BID on Days 8 through 28 of each 28-day treatment cycle. Azacitidine: 75 mg/m\^2 administered intravenously or subcutaneously each day on Days 1 through 7 of each 28-day treatment cycle.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Tamibarotene

2016

Completed Phase 2

~160

Azacitidine

2012

Completed Phase 3

~1440