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Clinical trials

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Status: Not yet recruiting

A Study to Investigate AG-236 in Subjects ≥18 Years of Age With Polycythemia Vera

This clinical trial is a Phase 2/3 study that will determine the recommended dose and dose regimen of AG-236 and evaluate the efficacy, safety, and pharmacokinetics of AG-236 in polycythemia vera (PV) by testing how well AG-236 works compared to placebo to increase the number of subjects with no phlebotomy and absence of phlebotomy eligibility over a 13-week period. In addition, the long-term effect of AG-236 on efficacy and safety will be explored in an open-label extension portion.

Participants needed: 245
Trial details
Phase: Phase 2, Phase 3Age: 18+Biological sex: AllType: InterventionalSponsor: Agios Pharmaceuticals, Inc.Updated: Oct 13, 2026
Eligibility criteria

PV diagnosis according to 2022 World Health Organization (WHO) criteria. [+5]

Use of any investigational or marketed N-acetylgalactosamine (GalNAc) targeting... [+6]

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Status: 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: Sep 28, 2026Locations: 3
Eligibility criteria

Age ≥12 years. [+12]

Pregnant, breastfeeding, or parturient. [+32]

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Status: 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: Sep 28, 2026Locations: 5
Eligibility criteria

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

Pregnant or breastfeeding. [+23]

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Status: 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: Sep 28, 2026Locations: 4
Eligibility criteria

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

Pregnant or breastfeeding. [+23]

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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: Sep 28, 2026Locations: 5
Eligibility criteria

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

Prior exposure to AG-181. [+7]

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