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4 recruiting studies matching your filters
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
~$10 – $8,750
Agios Pharmaceuticals, Inc.NCT07517133
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
~$10 – $8,750
Agios Pharmaceuticals, Inc.NCT07506863
International Prospective Registry of Patients With Alpha Thalassemia
This is an international prospective registry of patients with Alpha thalassemia to understand the natural history of the disease and the outcomes of fetal the…
Alpha-ThalassemiaAlpha Thalassemia MajorAlpha Thalassemia Minor
Estimated
~$10 – $675
University of California, San FranciscoNCT04872179
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patie…
Alpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more
Estimated
~$10 – $1,125
xCuresNCT06539169