Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at Oakland, California and other locations
Dates
study started
completion around

Description

Summary

This is a multicenter, open-label, extension study to evaluate the long-term safety, tolerability, and efficacy of treatment with AG-348 in participants who were previously enrolled in Study AG348-C-006 or Study AG348-C-007.

Official Title

An Open-Label, Multicenter, Extension Study of AG-348 in Adult Subjects With Pyruvate Kinase Deficiency Previously Enrolled in AG-348 Studies

Keywords

Pyruvate Kinase Deficiency, Anemia, Hemolytic, Congenital Nonspherocytic, Inborn Errors Pyruvate Metabolism, Mitapivat

Eligibility

You can join if…

Open to people ages 18 years and up

  • Be willing and able to comply with study visits and procedures;
  • Have signed written informed consent prior to participating in this extension study;
  • Have completed either antecedent study AG348-C-006 or AG348-C-007 through the Part 2 Week 24 Visit;
  • Cohorts 2 and 3: Have demonstrated clinical benefit from AG-348 treatment in the antecedent study, in the opinion of the Investigator;
  • For women of reproductive potential, have a negative pregnancy test during screening;
  • For women of reproductive potential as well as men with partners who are women of reproductive potential, be abstinent as part of their usual lifestyle, or agree to use 2 forms of contraception, 1 of which must be considered highly effective, from the time of giving informed consent, during the study, and for 28 days following the last dose of study drug for women and 90 days following the last dose of study drug for men.

You CAN'T join if...

  • Have a significant medical condition (including clinically significant laboratory abnormality) that developed during his/her antecedent AG- 348 study that confers an unacceptable risk to participating in this extension study, that could confound the interpretation of the study data, and/or that compromises the ability of the participant to complete study visits and procedures.
  • Are currently pregnant or breastfeeding.
  • Have a splenectomy scheduled during the study treatment period.
  • Meet the withdrawal criteria of his/her antecedent AG-348 study during screening of this extension study.
  • Are currently receiving medications that are strong inhibitors of cytochrome P450 (CYP)3A4 that have not been stopped for a duration of at least 5 days or a time frame equivalent to 5 half-lives (whichever is longer) before start of study drug; or strong inducers of CYP3A4 that have not been stopped for a duration of at least 28 days or a time frame equivalent to 5 half-lives (whichever is longer) before start of study drug on this extension study.
  • Have received anabolic steroids, including testosterone preparations, within 28 days prior to start of study drug on this extension study.
  • Have received hematopoietic stimulating agents (eg, erythropoietins, granulocyte colony stimulating factors, thrombopoietins) within 28 days prior to start of study drug on this extension study.
  • Have exposure to any investigational drug other than AG-348, device, or procedure within 3 months prior to start of study drug on this extension study.

Locations

  • UCSF Benioff Children's Hospital, Oakland
    Oakland California 95609 United States
  • Phoenix Children's Hospital
    Phoenix Arizona 85016 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Agios Pharmaceuticals, Inc.
ID
NCT03853798
Phase
Phase 3 research study
Study Type
Interventional
Participants
About 90 people participating
Last Updated