NCT04536792

A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Participants With Sickle Cell Disease

A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Subjects With Sickle Cell Disease

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 122 (actual)
• Sponsor: Agios Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Accepted

Summary

The purpose of the study is to assess the safety and tolerability of AG-946 in healthy volunteers after oral administration of single ascending doses (SAD) and multiple ascending doses (MAD) of AG-946 over 14 or up to 28 days of dosing, and to identify a range of doses that are safe and pharmacologically active in participants with sickle cell disease. The SAD and MAD parts of the study will be randomized and double-blinded, and will assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of AG-946 as well as the effect of food (SAD only) on the pharmacokinetics (PK) of AG-946. The sickle cell disease (SCD) part of the study will be non-randomized and open-label, and is designed to identify 1 or more safe and tolerable dose(s) of AG-946 with potential activity in the treatment of participants with sickle cell disease (SCD).

Conditions

• Healthy Volunteers
• Anemia, Sickle Cell

Interventions

Type	Name	Description
DRUG	AG-946	AG-946, oral encapsulated coated-granules or tablets.
DRUG	AG-946 Matched Placebo	AG-946 matched-placebo, oral encapsulated sugar spheres or tablets.

Timeline

• Start date: 2020-07-10
• Primary completion: 2023-12-19
• Completion: 2023-12-19
• First posted: 2020-09-03
• Last updated: 2024-04-16

Locations

10 sites across 3 countries: United States, Denmark, Spain

Regulatory

• FDA-regulated drug study