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

Clinical Trial
Study Details
Enrollment
122
Interventions
Description

AG-946, oral encapsulated coated-granules or tablets.

Name
AG-946
Type
Drug
Description

AG-946 matched-placebo, oral encapsulated sugar spheres or tablets.

Name
AG-946 Matched Placebo
Type
Drug
Phase
Phase 1
Status
COMPLETED
Study ID #
NCT04536792
Study Type
Interventional
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).

Study Overview
Contacts and Locations
Locations
Facility

University of California San Diego
La Jolla, California 92037
United States

GeoPoint
32.84727, -117.2742
Facility

Massachusetts General Hospital
Boston, Massachusetts 02114
United States

GeoPoint
42.35843, -71.05977
Facility

Boston Medical Center
Boston, Massachusetts 02118
United States

GeoPoint
42.35843, -71.05977
Facility

New York Presbyterian Hospital - Weill Cornell Medicine
New York, New York 10065
United States

GeoPoint
40.71427, -74.00597
Facility

University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania 15213
United States

GeoPoint
40.44062, -79.99589
Facility

PPD Development, LP
Austin, Texas 78744
United States

GeoPoint
30.26715, -97.74306
Facility

Texas Oncology-Baylor Charles A. Sammons Cancer Center - USOR
Dallas, Texas 75246
United States

GeoPoint
32.78306, -96.80667
Facility

University of Texas Health Science Center of Houston
Houston, Texas 77030
United States

GeoPoint
29.76328, -95.36327
Facility

Rigshospitalet, Department of Hematology
2100 Copenhagen
Denmark

GeoPoint
55.67594, 12.56553
Facility

Hospital Universitario Vall d'Hebron - PPDS
08035 Barcelona
Spain

GeoPoint
41.38879, 2.15899