Pyruvate Kinase Deficiency Natural History Study
NCT02053480 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 254
Last updated 2020-05-22
Summary
The purpose of this study is to describe the range and incidence of symptoms, treatments, and complications related to pyruvate kinase deficiency (PKD). Eligible patients are those of all ages with known PKD or with a hemolytic anemia and a family member with PKD. The study will collect retrospective medical history, routine clinical care data, and quality of life measures at baseline and annually for patients with PKD.
Conditions
- Pyruvate Kinase Deficiency
- Congenital Non-Spherocytic Hemolytic Anemia
Sponsors & Collaborators
-
Agios Pharmaceuticals, Inc.
collaborator INDUSTRY - lead OTHER
Eligibility
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2013-12-31
- Primary Completion
- 2019-12-31
- Completion
- 2020-05-31
Countries
- United States
- Canada
- Czechia
- Germany
- Italy
- Netherlands
Study Locations
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