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NCT Number: NCT04995315

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

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).

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Key information

Conditions

Pyruvate Kinase Deficiency Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Hemolytic, Congenital Nonspherocytic Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Pyruvate Kinase Deficiency of Red Cells

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Milan, Italy

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Treatment and study plan

Primary outcomes

  1. Change Over Time in Cogstate Brief Battery (CBB) Scores

    Time frame: 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 outcomes

  1. CBB Scores of Participants as Compared to the Scores of Age-Matched Controls

    Time frame: 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.

  2. Percentage of Participants With Blood Transfusions Affecting Cognition, as Assessed by the Investigator

    Time frame: Day 90

Sponsors and collaborators

Lead sponsor

Agios Pharmaceuticals, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2021
Primary completion
2023
Study completion
2023
First posted
Aug 6, 2021
Registry last updated
Oct 16, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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