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Pyruvate Kinase Deficiency Global Longitudinal Registry
NCT03481738 · View on ClinicalTrials.gov ↗
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 Studied
Study Locations (20)
Massachusetts
- Massachusetts General Hospital — Boston
- Boston Children's Hospital — Boston
- UMass Memorial Medical Center — Worcester
Arkansas
- Arkansas Children's Hospital — Little Rock
- University of Arkansas for Medical Sciences — Little Rock
California
- Children's Hospital of Orange County — Orange
- Stanford University Medical Center — Palo Alto
Ontario
- Saint Josephs Healthcare System — Hamilton
- Toronto General Hospital — Toronto
Other
- Fakultni nemocnice Olomouc — Olomouc
- Ustav hematologie a krevni transfuze — Prague
Arizona
- Phoenix Childrens Hospital — Phoenix
Georgia
- Children's Healthcare of Atlanta — Atlanta
Michigan
- Children's Hospital of Michigan — Detroit
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 500 participants |
| Start Date | 2018-04-23 |
| Est. Completion | 2027-05-31 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03481738
The ClinicalTrials.gov registry entry for NCT03481738 describes a study currently listed as active not recruiting. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 500 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Agios Pharmaceuticals, which has 15 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Pyruvate Kinase Deficiency appearing as the primary indexed condition, and to 0 interventions. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT03481738 reports 20 study locations spanning 14 distinct geographic areas — top geographies include Massachusetts, Arkansas, California. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT03481738 about?
NCT03481738 is a clinical study titled "Pyruvate Kinase Deficiency Global Longitudinal Registry". 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 prospect...
What is the current status of trial NCT03481738?
This trial is currently active not recruiting. The enrollment target is 500 participants. The study started on 2018-04-23. Estimated completion is 2027-05-31.
What conditions does trial NCT03481738 study?
This clinical trial studies the following conditions: Pyruvate Kinase Deficiency. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
Who is sponsoring clinical trial NCT03481738?
This trial is sponsored by Agios Pharmaceuticals, which has 15 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT03481738 being conducted?
This trial has 20 study locations across Arizona, Arkansas, California, Georgia, Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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