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A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

NCT03548220 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 80

Last updated 2022-05-24

Study results available
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Summary

Study AG348-C-006 evaluated the efficacy and safety of orally administered AG-348 as compared with placebo in participants with pyruvate kinase (PK) deficiency, who were not regularly receiving blood transfusions. Participants were randomized 1:1 to receive either AG-348 or a matching placebo.

Conditions

  • Pyruvate Kinase Deficiency
  • Anemia, Hemolytic

Interventions

DRUG

Placebo

Placebo matching AG-348 tablets, administered to maintain the blind.

DRUG

AG-348

AG-348 tablets.

Sponsors & Collaborators

  • Agios Pharmaceuticals, Inc.

    lead INDUSTRY

Principal Investigators

  • Medical Affairs · Agios Pharmaceuticals, Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-08-09
Primary Completion
2020-10-09
Completion
2020-10-09
FDA Drug
Yes

Countries

  • United States
  • Brazil
  • Canada
  • Czechia
  • Denmark
  • France
  • Germany
  • Italy
  • Japan
  • Netherlands
  • South Korea
  • Spain
  • Switzerland
  • Thailand
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03548220 on ClinicalTrials.gov