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Long-Term Safety Study of Azacitidine for Patients with Blood Disorders Who Previously Participated in Azacitidine Clinical Trials

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the long-term safety of a medication called azacitidine, which is used to treat certain blood disorders. Blood disorders can affect the way blood cells function, leading to various health issues. The study involves participants who have previously taken part in other clinical trials with azacitidine and have been receiving this treatment. The purpose of the study is to ensure that continuing the treatment is safe for these participants over a longer period.

Participants in this study will continue to take azacitidine in the form of a tablet. The study will monitor the safety of the treatment by observing any side effects that may occur. These side effects are known as adverse events, which are any unwanted effects that happen during the treatment. The study will also look at how long participants live after starting the treatment, which is referred to as survival. The study is designed to provide valuable information about the safety of azacitidine when used over an extended time.

The study is open-label, meaning that both the participants and the researchers know which treatment is being given. It is a single-arm study, which means all participants receive the same treatment without a comparison group. The study will continue until 2026, allowing researchers to gather comprehensive data on the long-term effects of azacitidine in treating blood disorders. Participants will take the medication orally, and the study will track their health and any changes that occur during the treatment period.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Participation begins after previous involvement in a study with CC-486 (oral azacitidine).

    Eligibility requires continued clinical benefit from CC-486 as determined by the investigator.

  2. Step 2

    Treatment phase

    Receive azacitidine in tablet form, available in 100 mg, 150 mg, 200 mg, and 300 mg dosages.

    The medication is taken orally. The specific dosage and frequency are determined by the healthcare provider based on individual needs.

  3. Step 3

    Monitoring and safety evaluation

    Regular monitoring for any side effects or adverse events related to the treatment.

    The focus is on the type, frequency, and severity of any side effects experienced.

  4. Step 4

    Follow-up phase

    Participants may enter a follow-up phase if they were part of a previous study requiring survival monitoring.

    Continued use of contraception is required for both men and women of childbearing potential during and after the study as specified.

  5. Step 5

    Study completion

    The study is estimated to conclude by March 1, 2026.

    The primary focus is on long-term safety and survival outcomes.

Who can join the trial?

4 criteria

  • Must have previously participated in a study with the medication called CC-486 and continue to meet the requirements of that original study.
  • Must still be benefiting from the treatment with CC-486, according to the doctor's opinion.
  • If participating in the follow-up phase for survival, must have been in a previous CC-486 study where survival monitoring was necessary.
  • Both women who can have children and men who are sexually active with such women must use a reliable method of birth control after the last dose of CC-486 in the previous study, before starting this study, during the study, and for a certain time after finishing the treatment, as specified in the study guidelines.

Who cannot join the trial?

2 criteria

  • Participants who have not received CC-486 as a single treatment in previous related studies.
  • Participants who the study doctors do not believe will benefit from continuing treatment with CC-486.
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Investigated drugs

CC-486 (oral Azacitidine) is a medication being studied for its long-term safety in patients with blood disorders. It is used as a monotherapy, meaning it is given alone without combining it with other treatments. The trial aims to see if patients who have already been treated with CC-486 in previous studies can continue to benefit from it.

What is already known about the treatment

Azacitidine – This medication is administered orally and is currently being studied in clinical trials for its long-term safety in treating hematological disorders. It is primarily used for conditions affecting the blood and bone marrow. Azacitidine works by incorporating into the DNA and RNA of cells, leading to the reactivation of genes that suppress tumor growth. It belongs to the pharmacological class of antineoplastic agents, specifically DNA methyltransferase inhibitors.

Investigated diseases

Hematological Disorders – These are conditions that affect the blood, bone marrow, and lymphatic system. They can involve problems with blood cells, such as red blood cells, white blood cells, or platelets. Common symptoms may include fatigue, weakness, shortness of breath, and unusual bleeding or bruising. The progression of these disorders varies widely depending on the specific type and underlying cause. Some hematological disorders may lead to anemia, clotting issues, or immune system dysfunction. They can be chronic or acute, with some conditions developing slowly over time and others appearing suddenly.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-503272-25-00Protocol codeCC-486-GEN-001Estimated enrolment3 patientsSponsorCelgene Corp.

sourced from the EU Clinical Trials Register and site verification

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).