Status:
ACTIVE_NOT_RECRUITING
Pyruvate Kinase Deficiency Global Longitudinal Registry
Lead Sponsor:
Agios Pharmaceuticals, Inc.
Conditions:
Pyruvate Kinase Deficiency
Eligibility:
All Genders
Brief 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 Regi...
Detailed Description
Data will be submitted to the Registry via electronic case report forms (eCRFs). Relevant datasets, such as historical trial data, claims, medical records, or central lab data will be electronically i...
Eligibility Criteria
Inclusion
- Inclusion Criteria:
- Participants of all ages with a confirmed diagnosis of PK deficiency via genetic testing are eligible to enroll;
- Participants will be considered for enrollment on the basis of clinical features consistent with PK deficiency together with the presence of 2 or more PKLR gene mutations. For novel or indeterminate PKLR gene mutations, participants will be deemed eligible if, in the opinion of the investigator, the reported PKLR gene mutations are sufficient to support a diagnosis of PK deficiency;
- The participant or the parent/guardian of the participant must be willing and able to give written informed consent and/or assent. E-consent or remote consent may be utilized where permissible as applicable if country regulations and site policies allow.
Exclusion
Key Trial Info
Start Date :
April 23 2018
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
May 31 2027
Estimated Enrollment :
500 Patients enrolled
Trial Details
Trial ID
NCT03481738
Start Date
April 23 2018
End Date
May 31 2027
Last Update
December 31 2025
Active Locations (52)
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1
Phoenix Childrens Hospital
Phoenix, Arizona, United States, 85016
2
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
3
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States, 72205
4
Children's Hospital of Orange County
Orange, California, United States, 92868