Clinical trials

4

Search and review clinical trials. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Not yet recruiting

A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)

The primary objective of this study is to determine the effect of mitapivat versus placebo on the need for transfusions in subjects with SCD.

Participants needed: 159
Trial details
Phase: Phase 3Age: 12+Biological sex: AllType: InterventionalSponsor: Agios Pharmaceuticals, Inc.Updated: Jun 23, 2026
Eligibility criteria

Age ≥12 years. [+12]

Pregnant, breastfeeding, or parturient. [+32]

Status: Not yet recruiting

A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)

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-transfusion-dependent thalassemia.

Participants needed: 54
Trial details
Phase: Phase 3Age: 1-17Biological sex: AllType: InterventionalSponsor: Agios Pharmaceuticals, Inc.Updated: Jun 23, 2026
Eligibility criteria

Written informed consent/assent from the participant (or their legally authorize... [+5]

Pregnant or breastfeeding. [+23]

Status: Not yet recruiting

A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants 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-transfusion-dependent thalassemia.

Participants needed: 45
Trial details
Phase: Phase 3Age: 1-17Biological sex: AllType: InterventionalSponsor: Agios Pharmaceuticals, Inc.Updated: Jun 23, 2026
Eligibility criteria

Written informed consent/assent from the participant (or their legally authorize... [+6]

Pregnant or breastfeeding. [+23]

Status: Recruiting

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria

The primary purpose of this study is to assess the safety and tolerability of AG-181 in subjects with Phenylketonuria (PKU).

Participants needed: 20
Trial details
Phase: Phase 1Age: 18-69Biological sex: AllType: InterventionalSponsor: Agios Pharmaceuticals, Inc.Updated: Jun 23, 2026Locations: 4
Eligibility criteria

Diagnosis of PKU, defined as documented presence of 2 mutant alleles in the phen... [+4]

Prior exposure to AG-181. [+7]