CompletedPhase 2

A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency

United States52 participantsStarted 2015-06-26

Plain-language summary

Study AG348-C-003 is a multicenter study designed to evaluate the safety and efficacy of different dose levels of AG-348 (mitapivat) in participants with PK deficiency.

Who can participate

Age range18 Years

SexALL

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Inclusion criteria

✓. Informed consent
✓. Male or female, aged 18 years and older
✓. Known medical history of PK deficiency
✓. PK deficiency confirmed by enzymatic assay at Screening
✓. Genotypic characterization of PKR gene at Screening
✓. Genotypic characterization of uridine-5'-diphosphate-glucuronyltransferase-A1 (UGTA1) gene to document underlying Gilbert's disease (Gilbert's disease patients are eligible)
✓. Males Hb ≤ 12.0 g/dL, females Hb ≤ 11 g/dL
✓. Transfusion independent, defined as no more than 3 units of red blood cells (RBC) transfused in 12 months prior to the first day of study dosing and no transfusions within 4 months of first day of study dosing

Exclusion criteria

✕. Hb ˃ 12.0 g/dL if male, Hb ˃11.0 g/dL if female
✕. Additional diagnosis of other congenital or acquired blood disorder
✕. Iron overload sufficiently severe to result in cardiac, hepatic or pancreatic insufficiency
✕. Bone marrow or stem cell transplant
✕. Clinically symptomatic cholelithiasis or cholecystitis
✕. Currently enrolled in any other investigational trial. Participation in the PK Deficiency Natural History Study (NCT02053480) is permitted
✕. Exposure to any investigational drug, device or procedure within 28 days prior to screening or during trial participation
✕. Concurrent medical condition such as poorly controlled hypertension, heart failure, active infection, frequent post-splenectomy sepsis, Hepatitis B or C, Human Immunodeficiency Virus type 1 (HIV1) or Human Immunodeficiency Virus type 2 (HIV2) infection, poorly controlled diabetes mellitus, history of primary malignancy with the exception of curatively treated nonmelanomatous skin cancer, cervical cancer of breast cancer in situ

What they're measuring

Percentage of Participants Experiencing at Least One Adverse Event (AEs) in the Core Period

Timeframe: Up to Week 24

Trial details

NCT IDNCT02476916

SponsorAgios Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2017-05-08

Results submitted2020-05-21

View on ClinicalTrials.gov More Pyruvate Kinase Deficiency trials