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

NCT ID: NCT04995315

Last Updated: 2023-10-16

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 13 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2021-07-02
• Study Completion Date: 2023-09-22

Brief Summary

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).

Conditions

Pyruvate Kinase Deficiency

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

PK Deficiency Diagnosed Participants

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

No interventions assigned to this group

Eligibility Criteria

Inclusion Criteria

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Agios Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Affairs

Role: STUDY_CHAIR

Agios Pharmaceuticals, Inc.

Locations

Massachusetts General Hospital

Boston, Massachusetts, United States

Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

Milan, , Italy

Universitair Medisch Centrum Utrecht

Utrecht, , Netherlands

Countries

United States; Italy; Netherlands

Other Identifiers

AG348-C-016

Identifier Type: -

Identifier Source: org_study_id