Rare diseases
Investigational molecules
Locations
88 Sidney Street, 02139-4137, United States
The sponsor demonstrates significant commitment to advancing treatment options for patients with acute myeloid leukemia and myelodysplastic syndromes. Research initiatives focus on addressing relapsed or refractory disease in adult populations, with particular attention to patients who have exhausted standard therapeutic options. The investigational approaches target specific molecular pathways involved in hematologic malignancies, exploring novel mechanisms that may offer clinical benefit to patients with limited treatment alternatives.
The research portfolio includes trials designed to evaluate therapeutic efficacy in patients who have previously received multiple lines of therapy, addressing a critical unmet need in refractory hematologic conditions.
Agios Pharmaceuticals Inc. runs clinical trials in conditions such as Thalassaemia, Anaemia, Thalassaemia beta, Thalassaemia alpha, among others.
Clinical trials registered by this sponsor – none is enrolling right now.
Effect of mitapivat versus placebo on transfusion burden in patients with sickle cell disease
BelgiumPhase 3 study of mitapivat for effectiveness and safety in children with non‑transfusion‑dependent alpha or beta thalassemia
GreecePhase 3 Study of Mitapivat to Reduce Transfusions in Children with Alpha or Beta Transfusion‑Dependent Thalassemia
GreeceStudy on the Effects of Tebapivat and AG-946 in Patients with Sickle Cell Disease
BelgiumStudy on the Effectiveness and Safety of Mitapivat for Patients with Non-Transfusion-Dependent Alpha or Beta Thalassemia
BulgariaStudy on the Effects of Mitapivat for Patients with Transfusion-Dependent Alpha or Beta Thalassemia
BulgariaStudy on the Effectiveness and Safety of Mitapivat for Children with Pyruvate Kinase Deficiency Receiving Regular Blood Transfusions
CzechiaStudy on the Effectiveness and Safety of Mitapivat for Patients with Sickle Cell Disease
BelgiumStudy of Mitapivat for Patients with Sickle Cell Disease and Kidney Disease
FranceStudy on the Effectiveness and Safety of Mitapivat in Children with Pyruvate Kinase Deficiency Not Receiving Regular Blood Transfusions
FranceStudy of AG-946 for Treating Anemia in Patients with Lower-Risk Myelodysplastic Syndromes
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