Phase 2 Rare Disease Trial, Not Yet Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• You have been diagnosed with one of these rare inherited blood conditions: a type of inherited sideroblastic anemia (CSA), a congenital dyserythropoietic anemia (CDA type I or II), or a form of Diamond-Blackfan anemia (DBA) that does not require regular blood transfusions
• Your diagnosis has been confirmed by a genetic test showing a specific type of genetic change (classified as ACMG class 4 or 5) (confirm with trial site)
• You are 18 years old or older at the time of your first screening appointment
• If you have CSA or CDA, you may qualify whether or not you need regular blood transfusions — transfusion-dependent patients must have received 6 to 20 units of red blood cells in the past 24 weeks with no break longer than 56 days; non-transfusion-dependent patients must have a low hemoglobin level (below 10.5 g/dl on average) and have not had a transfusion in the 8 weeks before joining
• Your kidneys are working well enough, based on standard blood test results (confirm with trial site)
• Your liver is working well enough, based on standard blood test results (confirm with trial site)
• You are generally able to carry out everyday activities, with at most some limitations (based on a standard health scale called ECOG 0–2) (confirm with trial site)
• You are willing and able to sign a consent form and follow all study procedures for the full duration of the trial
• If you are a woman who could become pregnant, you must have two negative pregnancy tests before starting, agree to monthly pregnancy testing during the study, and use highly effective contraception (such as hormonal contraception, an intrauterine device, or tubal ligation) starting 5 weeks before treatment and for 12 weeks after finishing
• If you are a man, you must agree to use a condom during any sexual contact with a pregnant woman or a woman who could become pregnant, throughout the study and for at least 12 weeks after finishing treatment

Who may not be able to join:

• DBA patients who need regular blood transfusions, or whose genetic change does not fall within specific gene types listed in the trial (RPS19, RPS26, RPL5, or RPL11), or who have no confirmed genetic diagnosis
• CSA patients without a confirmed genetic diagnosis if they may actually have an acquired (non-inherited) form of the condition (confirm with trial site)
• People with a serious, uncontrolled infection or other severe uncontrolled health condition
• People with uncontrolled high blood pressure
• People with significant heart disease (classified as NYHA Class III or IV), or who have had a heart attack in the past 6 months
• People who have used an experimental drug in the past 30 days, or any cancer treatment in the past 2 weeks
• People who have used a medication called EPO (erythropoietin) within the past 4 weeks before joining
• People with an active cancer diagnosis, or who have had cancer in the past year (except for certain very limited skin or cervical/breast conditions — confirm with trial site)
• People already taking part in another clinical trial involving an experimental drug
• People with a known HIV infection, or active hepatitis B or C
• Women who are pregnant, could become pregnant, or are currently breastfeeding
• Anyone who has a medical or mental health condition that would prevent them from understanding and signing the consent form
• People who may be eligible for a bone marrow transplant in the near or medium-term future
• People with a known allergy to the study drug (luspatercept) or any of its ingredients
• People who do not have health insurance coverage
• Men or women able to have children who are unwilling to use contraception or abstain from sex during the trial
• People whose freedom is restricted by a court or government decision
• People under a legal protection order such as guardianship or curatorship

Important: Always verify eligibility with the trial site directly before applying.