Mitapivat for Children With Non-Transfusion-Dependent Thalassemia
Treatments studied
Part of Blood & lymphatic clinical trials.
This study tests a medicine called mitapivat in children ages 1–17 with thalassemia who don't need frequent blood transfusions. The medicine may help their bodies make more healthy red blood cells and reduce anemia symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Child is between 1 and 18 years old and weighs at least 7 kilograms (about 15 pounds)
- Confirmed diagnosis of thalassemia (alpha or beta type) from blood tests or genetic testing
- Low hemoglobin level (≤10 g/dL) measured at least twice, 7 days apart
- Received 5 or fewer blood transfusions in the past 6 months, and none in the past 8 weeks or during screening
- No history of gene therapy, bone marrow/stem cell transplant, or certain blood-boosting medications in past 18 weeks
- No serious heart, lung, liver, or kidney disease; no active infections or hepatitis
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This trial studies an investigational medicine in adults with beta thalassemia intermedia (including non-transfusion dependent thalassemia). The goal is to see how well the treatment works and how safe it is for people whose hemoglobin runs in a certain range.
This study tests a study pill called mitapivat for children aged 1 to 17 with transfusion-dependent thalassemia. The goal is to see if mitapivat can reduce the need for blood transfusions and improve blood counts.
This study tests whether a daily pill called mitapivat can reduce the number of blood transfusions you need. It is for people with sickle cell disease who have had at least one transfusion in the past year and are not on regular transfusion therapy.
This trial tests a drug called mitapivat (taken as a pill) to see if it can safely improve red blood cell health and reduce anemia symptoms in people with inherited red blood cell membrane disorders. It may help increase hemoglobin levels and relieve fatigue.
This study provides continued access to the drug mitapivat for people who have already been in a mitapativ study and are benefiting from it.
This study tests a medicine called luspatercept to see if it safely helps children with beta-thalassemia need fewer blood transfusions or keep their hemoglobin levels stable. It is for children aged 6 to 17 who either get regular transfusions or have low hemoglobin and few transfusions.
Hear when a new Non-Transfusion-dependent Alpha-Thalassemia trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.