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

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

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT04995315

Enrollment

Registered

2021-08-06

Start date

2021-07-02

Completion date

2023-09-22

Last updated

2023-10-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Pyruvate Kinase Deficiency

Keywords

Glycolytic enzymopathy, Congenital Nonspherocytic hemolytic anemia (CNSA), PKD

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).

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

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

Design outcomes

Primary

Measure	Time frame	Description
Change Over Time in Cogstate Brief Battery (CBB) Scores	Day 0, Day 90	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.

Secondary

Measure	Time frame	Description
CBB Scores of Participants as Compared to the Scores of Age-Matched Controls	Day 0, Day 90	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.
Percentage of Participants With Blood Transfusions Affecting Cognition, as Assessed by the Investigator	Day 90	—

Countries

Italy, Netherlands, United States

Outcome results