Pyruvate Kinase Deficiency Global Longitudinal Registry (NCT03481738)
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: Phoenix Childrens Hospital — Phoenix, Arizona
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (52 Sites)
Showing the first 25 of 52 sites.
- Phoenix Childrens Hospital — Phoenix, Arizona
- Arkansas Children's Hospital — Little Rock, Arkansas
- University of Arkansas for Medical Sciences — Little Rock, Arkansas
- Children's Hospital of Orange County — Orange, California
- Stanford University Medical Center — Palo Alto, California
- Children's Healthcare of Atlanta — Atlanta, Georgia
- Massachusetts General Hospital — Boston, Massachusetts
- Boston Children's Hospital — Boston, Massachusetts
- UMass Memorial Medical Center — Worcester, Massachusetts
- Children's Hospital of Michigan — Detroit, Michigan
- Duke University Medical Center — Durham, North Carolina
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
- St Jude Children's Research Hospital — Memphis, Tennessee
- Primary Children's Hospital — Salt Lake City, Utah
- University of Vermont Medical Center — Burlington, Vermont
- Saint Josephs Healthcare System — Hamilton, Ontario
- Toronto General Hospital — Toronto, Ontario
- St. Justine Hospital — Montreal, Quebec
- Fakultni nemocnice Olomouc — Olomouc,
- Ustav hematologie a krevni transfuze — Prague,
- Fakultni nemocnice v Motole — Prague,
- Copenhagen University Hospital — Herlev,
- Hopital Necker — Paris,
- Charite - Universitatsmedizin Berlin — Berlin,
- Evangelisches Krankenhaus Bielefeld gGmbH — Bielefeld,
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
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Explore by condition: All Pyruvate Kinase Deficiency clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.