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

Pyruvate Kinase Deficiency Clinical Trial

Official title:

Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008: Cognition in Participants With Pyruvate Kinase Deficiency

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04995315
• Other study ID #: AG348-C-016
• Status: Completed
• Start date: July 2, 2021
• Est. completion date: September 22, 2023

Study information

• Verified date: October 2023
• Source: Agios Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 13
• Est. completion date: September 22, 2023
• Est. primary completion date: September 22, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Participant must be =18 years of age;

• Participant must be enrolled in Study AG348-C-008.

Related Conditions & MeSH terms

• Pyruvate Kinase Deficiency

Locations

Country	Name	City	State
Italy	Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico	Milan
Netherlands	Universitair Medisch Centrum Utrecht	Utrecht
United States	Massachusetts General Hospital	Boston	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Agios Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Italy,  Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change Over Time in Cogstate Brief Battery (CBB) Scores	CBB is a series of brief computerized assessments used to evaluate 4 major cognitive domains: psychomotor speed, attention/vigilance, visual learning, and working memory. If the CBB score falls between -10 to +10, it is considered normal. In Cogstate scoring 0 could be considered as performing in the average range, a negative score means below-average performance, and a positive score means above-average performance. A positive change in scores indicates slightly better performance.	Day 0, Day 90
Secondary	CBB Scores of Participants as Compared to the Scores of Age-Matched Controls	CBB is a series of brief computerized assessments used to evaluate 4 major cognitive domains: psychomotor speed, attention/vigilance, visual learning, and working memory. An age-standardized score of =-1 on a test will be defined as abnormal. Cognitive impairment on the day of testing will be defined as =2 tests with abnormal performance.	Day 0, Day 90
Secondary	Percentage of Participants With Blood Transfusions Affecting Cognition, as Assessed by the Investigator		Day 90