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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT04995315
Other study ID # AG348-C-016
Secondary ID
Status Completed
Phase
First received
Last updated
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
Health authority
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.

Study Design


Related Conditions & MeSH terms


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
See also
  Status Clinical Trial Phase
Active, not recruiting NCT03853798 - Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007 Phase 3
Completed NCT03559699 - A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD) Phase 3
Active, not recruiting NCT02476916 - A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency Phase 2
Active, not recruiting NCT04105166 - Gene Therapy for Pyruvate Kinase Deficiency (PKD) Phase 1
Enrolling by invitation NCT05777993 - A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study Phase 4
Terminated NCT04964323 - Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)
Completed NCT03866590 - Pyruvate Kinase Deficiency Epidemiological Study (PIECE)
Recruiting NCT04902833 - Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms
Completed NCT02053480 - Pyruvate Kinase Deficiency Natural History Study
Completed NCT03548220 - A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD) Phase 3
Not yet recruiting NCT06422351 - Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency Phase 2
Recruiting NCT03481738 - Pyruvate Kinase Deficiency Global Longitudinal Registry