Non-Transfusion Dependent Thalassemia
Non-Transfusion Dependent Thalassemia (NTDT) is a genetic blood disorder characterized by reduced production of hemoglobin, leading to mild to moderate anemia without the need for regular blood transfusions.
Non-Transfusion-Dependent Thalassemia: Mitapivat Study
We are studying whether Mitapivat can improve anemia in people with non-transfusion-dependent alpha or beta thalassemia. This trial compares the medication to a placebo to evaluate its effects.
What diseases are being studied?
Health conditions and diseases that the clinical trial is designed to study and treat.
Who can participate in the clinical trial?
These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..
- Are you at least 18 years old?
- Do you have thalassemia (a blood disorder that affects hemoglobin)?
- Are you currently pregnant or breastfeeding?
- Are you taking any medications that are strong inhibitors or inducers of CYP3A4, like certain antifungals or antibiotics?
- Have you had any major surgery in the last 16 weeks?
What experimental substances are being tested?
Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.
Mitapivat
Where is the clinical trial being conducted?
Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.
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Multiprofile Hospital For Active Treatment Dr Nikola Vasiliev AD
Department of Transfusion Hematology
Pernik, Bulgaria
National Specialised Hospital For Active Treatment Of Haematological Diseases
Department of Hematopoietic Stem Cell Transplantation
Sofia, Bulgaria
Rigshospitalet
Department of Heamatology
Copenhagen, Denmark
Sponsor: Agios Pharmaceuticals Inc.
Last updated: Nov 27, 2025
Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.