- Active not recruitingPHASE3
A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension Period
This study is for children aged 1 to 17 with a blood condition called Pyruvate Kinase Deficiency (PKD) who don't regularly need blood transfusions. It's testing a new medicine called mitapivat to see if it's safe and effective, compared to a dummy medicine.
For: Pediatric Pyruvate Kinase Deficiency · Pediatric Hemolytic AnemiaUnited States · Canada · France - Active not recruitingPHASE3
A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period
This study is looking at a new medicine called Mitapivat for children with a rare blood disorder called Pyruvate Kinase Deficiency (PKD). It aims to see if Mitapivat can help children who need regular blood transfusions, compared to a dummy medicine.
For: Pediatric Pyruvate Kinase Deficiency · Pediatric Hemolytic AnemiaUnited States · Canada · Czechia - Enrolling by invitationPHASE4
A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study
This study allows patients who are already benefiting from a drug called mitapivat in a previous trial to continue taking it. It's for those with conditions like Pyruvate Kinase Deficiency, Sickle Cell Disease, or Thalassemia, to ensure they can keep receiving the treatment if their doctor believes it's helping them.
For: Pyruvate Kinase Deficiency · Anemia, Hemolytic · Sickle Cell DiseaseBelgium · Brazil · Canada - Active not recruitingPHASE3
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
This study looked at a new drug, mitapivat, for people aged 18 and over with a type of blood disorder called thalassemia that doesn't need regular blood transfusions. It aimed to see if mitapivat could help reduce anaemia compared to a dummy pill.
For: Non-Transfusion-dependent Alpha-Thalassemia · Non-Transfusion-dependent Beta-ThalassemiaUnited States · Brazil · Bulgaria - Active not recruitingPHASE3
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
This study looks at a new medicine called mitapivat for people with severe types of thalassemia who need regular blood transfusions. It aims to see if mitapivat can reduce how often they need transfusions compared to a dummy treatment.
For: Transfusion-dependent Alpha-Thalassemia · Transfusion-dependent Beta-ThalassemiaUnited States · Brazil · Bulgaria - Ongoing, recruitingTherapeutic exploratory (Phase II)
A Phase 2, Open-label, Multicenter Study of Mitapivat in Subjects With Sickle Cell Disease and Nephropathy
This study is testing a new medicine called Mitapivat for people with Sickle Cell Disease and kidney problems. Researchers want to see if it can help reduce kidney damage and improve health, checking for how safe and effective it is over six months.
For: Sickle Cell Disease (SCD) and NephropathyFrance · Ireland - Active not recruitingPHASE2, PHASE3
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
This study is looking at a new medicine called mitapivat for sickle cell disease. It aims to find the right dose and see if it helps increase healthy red blood cells and reduce painful crises.
For: Sickle Cell DiseaseUnited States · Belgium · Brazil - AuthorisedTherapeutic confirmatory (Phase III)
The Effect of Mitapivat on Cerebral Perfusion and Oxygen Metabolism in Sickle Cell Disease
This study looks at a new medicine called mitapivat for people with sickle cell anaemia. It wants to see how this medicine affects blood flow to the brain and how the brain uses oxygen, using special MRI scans over 12 months.
For: Sickle cell anaemiaNetherlands