Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)

  • End date
    Aug 1, 2025
  • participants needed
  • sponsor
    Agios Pharmaceuticals, Inc.
Updated on 15 September 2021
Medical Affairs Agios Pharmaceuticals, Inc.
Primary Contact
Massachusetts General Hospital (4.5 mi away) Contact


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.


Participants aged 18 years and previously enrolled in Study AG348-C-008 (NCT03481738) will be eligible to participate in this study. Data will be submitted via electronic patient-reported outcomes (ePRO) application.

All data collection efforts will abide by this protocol and be prospectively disclosed in the study informed consent. This study, with the appropriate participant (and or parent/guardian) consent/assent, may incorporate retrospective data from other properly consented studies done for the purpose of examining the longitudinal natural history of PK deficiency.

Condition Blood disorder, Anemia, Hemolytic anemia, Pyruvate Kinase Deficiency, anaemia, Pyruvate Kinase Deficiency Anemia
Clinical Study IdentifierNCT04964323
SponsorAgios Pharmaceuticals, Inc.
Last Modified on15 September 2021


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Inclusion Criteria

Participant must be greater than or equal to ()18 years of age
Participant must be enrolled in Study AG348-C-008

Exclusion Criteria

Participant is enrolled in an Agios-sponsored clinical study involving treatment with a pyruvate kinase activator
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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