Browse Studies — Arctuva

A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301

The purpose of this study is to evaluate the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion-dep…

Sickle Cell DiseaseTransfusion-dependent Beta-ThalassemiaHemoglobinopathies

Estimated

~$10 – $1,050

Actual pay is set by the research site.

Editas Medicine, Inc.NCT06363760

Phase 3

A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Subjects With α- or β- Transfusion-Dependent Thalassemia

The primary objective of this study is to compare the effect of mitapivat versus placebo on transfusion burden in pediatric participants with α- or β-transfusi…

Transfusion-dependent Alpha-ThalassemiaTransfusion-dependent Beta-Thalassemia

Estimated

~$50 – $2,100

Actual pay is set by the research site.

Agios Pharmaceuticals, Inc.NCT07506863

Phase 3

A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Subjects With α- or β-Non-Transfusion-Dependent Thalassemia

The primary objective of this study is to compare the effect of mitapivat versus placebo on anemia in pediatric participants with alpha- or beta-non-transfusio…

Non-Transfusion-dependent Alpha-ThalassemiaNon-Transfusion-dependent Beta-Thalassemia

Estimated

~$50 – $2,100

Actual pay is set by the research site.

Agios Pharmaceuticals, Inc.NCT07517133