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NCT03481738 · ClinicalTrials.gov registry record
Pyruvate Kinase Deficiency Global Longitudinal Registry
A clinical trial of Pyruvate Kinase Deficiency, sponsored by Agios Pharmaceuticals.
- Active
- Registry status
- 500
- Enrollment target
- 20
- Study locations
NCT03481738: Active study of Pyruvate Kinase Deficiency, sponsored by Agios Pharmaceuticals.
NCT03481738 is a study of Pyruvate Kinase Deficiency that is active but no longer recruiting, run by Agios Pharmaceuticals. The registered enrollment target is 500 participants. The trial reports 20 study locations across 14 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03481738, a study of Pyruvate Kinase Deficiency, is active but no longer recruiting, sponsored by Agios Pharmaceuticals.
- ACTIVE NOT RECRUITING
- Registry status
- 500 participants
- Enrollment target
- 20
- Study locations
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.
Primary Outcome
To develop an understanding of the longitudinal clinical implications of PK deficiency, including disease natural history, treatments and outcomes, and variability in clinical care and disease burden.
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 |
What the registry record for NCT03481738 still lists
NCT03481738 is an observational study that tracks outcomes without assigning an intervention. The registered 500 participants enrollment target is mid-sized for trials with a published cap.
The record links to 1 condition, with Pyruvate Kinase Deficiency appearing as the primary indexed condition, and to 0 interventions.
NCT03481738 names 20 study sites across 14 states, led by Massachusetts, Arkansas, California.
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.
Who is sponsoring clinical trial NCT03481738?
This trial is sponsored by Agios Pharmaceuticals, which has 44 total clinical trials registered on ClinicalTrials.gov.
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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