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NCT04964323 · ClinicalTrials.gov registry record
Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)
A clinical trial, sponsored by Agios Pharmaceuticals.
- Terminated
- Registry status
- 15
- Enrollment target
NCT04964323: Clinical Trial study, sponsored by Agios Pharmaceuticals.
NCT04964323 is a clinical trial that was terminated before completion, run by Agios Pharmaceuticals. The registered enrollment target is 15 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04964323 was terminated before completion, sponsored by Agios Pharmaceuticals.
- TERMINATED
- Registry status
- 15 participants
- Enrollment target
Study Summary
The primary objective of this study is to understand and characterize the health-related quality of life (HRQoL) and disease burden of adult participants with PK deficiency receiving routine clinical care. This study is an observational (i.e., noninterventional), longitudinal, multicenter, global registry for participants with PK deficiency, a rare nonspherocytic hemolytic anemia. This study will be open for enrollment for 2 years and all enrolled participants will be followed prospectively for up to 96 weeks. Data will be collected from participants who have provided informed consent and authorization pursuant to applicable laws and regulations.
Primary Outcome
The PKDD is a 7-item measure of the core signs and symptoms of PK deficiency. The score range is 25 to 76, with higher scores indicating a higher disease burden.
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 15 participants |
| Start Date | 2021-07-02 |
| Est. Completion | 2023-06-30 |
Why NCT04964323 stopped before completion
NCT04964323 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 15 participants, a relatively small participant target.
The record links to 0 conditions, and to 0 interventions.
NCT04964323 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04964323 about?
NCT04964323 is a clinical study titled "Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)". The primary objective of this study is to understand and characterize the health-related quality of life (HRQoL) and disease burden of adult participants with PK deficiency receiving routine clinical care. This study is an observational (i.e., noninterventional), longitudinal, multicenter, global re...
What is the current status of trial NCT04964323?
This trial is currently terminated. The enrollment target is 15 participants. The study started on 2021-07-02. Estimated completion is 2023-06-30.
Who is sponsoring clinical trial NCT04964323?
This trial is sponsored by Agios Pharmaceuticals, which has 44 total clinical trials registered on ClinicalTrials.gov.
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