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

Stopped early

Conditions studied: Pyruvate Kinase Deficiency, Pyruvate Kinase Deficiency Anemia, Anemia, Anemia, Hemolytic

In brief

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.

Key facts

Study ID

NCT04964323

Run by

Agios Pharmaceuticals, Inc.

People needed

15

Starts

2021-07-02

Expected to finish

2023-06-30

Last updated by the study team

2023-09-07

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify if…

• Participant is enrolled in an Agios-sponsored clinical study involving treatment with a pyruvate kinase activator.

Where it is running

• Massachusetts General Hospital — Boston, Massachusetts, United States
• Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico — Milan, Italy
• Universitair Medisch Centrum Utrecht — Utrecht, Netherlands
• Hospital Infantil Universitario Niño Jesus - PIN — Madrid, Spain
• Hammersmith Hospital — London, United Kingdom

Full record on ClinicalTrials.gov

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