Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008

Agios Pharmaceuticals

Status: Completed

Conditions: Pyruvate Kinase Deficiency

Study type: Observational

Funder types: Industry

Identifiers

NCT04995315

AG348-C-016

Details and patient eligibility

About

This is a substudy of Study AG348-C-008, an observational, longitudinal, multicenter, global registry that aims to better understand the longitudinal clinical implications of pyruvate kinase deficiency (PK deficiency). The primary purpose of this study is to describe the cognition of participants with PK deficiency, as objectively measured by the Cogstate Brief Battery (CBB).

Enrollment: 13 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria:

• Participant must be ≥18 years of age;
• Participant must be enrolled in Study AG348-C-008.

Trial design

13 participants in 1 patient group

PK Deficiency Diagnosed Participants

Description:

Participants will receive online user access to the CBB. Participants will complete 2 CBB assessments on Study Day 0 and Study Day 90.