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Pyruvate Kinase Deficiency Global Longitudinal Registry

NCT03481738 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 500

Last updated 2026-04-23

No results posted yet for this study

Summary

This study is an observational (ie, noninterventional), longitudinal, multicenter, global registry for patients with pyruvate kinase (PK) deficiency, a rare nonspherocytic hemolytic anemia.

This Registry will be open for enrollment for 7 years and all enrolled participants will be followed prospectively for a minimum of 2 years, and up to 9 years.

Data will be collected from participating Registry Physicians, participants, and, where appropriate, parents/guardians who have provided informed consent or assent (where relevant) and authorization pursuant to applicable laws and regulations.

Data should include demographic, clinical, and treatment data; and other data of relevance to the management of patients with PK deficiency. Annual chart review and data entry are expected in order to enhance longitudinal understanding of PK deficiency; however, no specific protocol schedule of assessment is required by this Registry protocol.

Conditions

  • Pyruvate Kinase Deficiency

Sponsors & Collaborators

  • Agios Pharmaceuticals, Inc.

    lead INDUSTRY

Principal Investigators

  • Eva Gallagher, VP, Medical Affairs · Agios Pharmaceuticals, Inc.

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-04-23
Primary Completion
2027-05-31
Completion
2027-05-31

Countries

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

Study Locations

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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 NCT03481738 on ClinicalTrials.gov