In short
There is currently 1 ongoing clinical trial for bone marrow failure. This trial is focused on evaluating the long-term safety of azacitidine, a medication used to treat blood disorders, in patients who have previously participated in azacitidine clinical trials. The trial is taking place in Finland and is expected to continue until 2026.
Clinical trial locations
Long-Term Safety Study of Azacitidine for Patients with Blood Disorders Who Previously Participated in Azacitidine Clinical Trials
This clinical trial is designed to study the long-term safety of azacitidine, also known as CC-486, in patients with blood disorders. The study is being conducted in Finland and involves participants who have already been taking part in previous azacitidine clinical trials and continue to benefit from the treatment.
Inclusion criteria: To participate in this study, patients must have previously taken part in a clinical trial involving CC-486 and continue to meet the requirements of that original study. They must still be benefiting from the treatment according to their doctor's assessment. If participants are in the follow-up phase for survival monitoring, they must have been in a previous CC-486 study where such monitoring was required. Both women of childbearing potential and men who are sexually active with such women must use reliable birth control methods during and after the treatment period as specified in the study guidelines.
Exclusion criteria: The study excludes participants who have not received CC-486 as a single treatment in previous related studies. Additionally, participants whom the study doctors do not believe will benefit from continuing treatment with CC-486 are not eligible.
Focus and goal: The main goal of this trial is to monitor the long-term safety of azacitidine when used over an extended period. Researchers will carefully observe any side effects or adverse events that occur during treatment. The study will also track survival outcomes, providing valuable information about how participants respond to continued treatment. This is an open-label, single-arm study, meaning all participants receive the same treatment and both patients and researchers know which medication is being given.
Investigational drug: The medication being studied is CC-486, which is an oral form of azacitidine. It is available in tablet form in various dosages: 100 mg, 150 mg, 200 mg, and 300 mg. Azacitidine works by affecting the DNA and RNA of cells, which helps reactivate genes that suppress abnormal cell growth. It belongs to a class of medications called DNA methyltransferase inhibitors, which are used to treat certain blood and bone marrow conditions. In this study, azacitidine is used as a single treatment without being combined with other medications.
Participants will continue taking azacitidine tablets orally, with the specific dosage and frequency determined by their healthcare provider based on their individual needs. Throughout the study, regular monitoring will track any side effects, including their type, frequency, and severity. The study is expected to conclude by March 2026, allowing researchers to gather comprehensive data on the long-term effects of this treatment.
Summary
Currently, there is only one ongoing clinical trial for bone marrow failure, taking place in Finland. This trial focuses exclusively on the long-term safety of azacitidine (CC-486) in patients who have already participated in previous azacitidine studies and continue to benefit from the treatment. The study represents an important continuation of care for patients who are responding well to this medication, providing valuable safety data over an extended treatment period. The trial is expected to run until March 2026 and will contribute to our understanding of azacitidine's safety profile when used as a long-term treatment option for blood disorders.
