Browse Studies — Arctuva

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

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

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