Evaluation of the ex vivo effect of AG-348 treatment on pyruvate kinase-deficient cell metabolism and erythroid development

Completed

• Conditions: anemia; 10005330; red blood cell disease

• Registration Number: NL-OMON42151
• Lead Sponsor: Agios Pharmaceuticals Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 23 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 30

Inclusion Criteria

1. Adult patients with biochemically and genetically diagnosed PK deficiency, i.e. patients should be compound heterozygous or homozygous for mutations in PKLR, the gene that encodes red blood cell PK.
2. The participant is willing and able to give written informed consent.

Exclusion Criteria

Patients who have been recently transfused with packed red blood cells (*Recently* is defined as less than 3 months before blood collection).

Study & Design

• Study Type: Observational invasive
• Study Design: Not specified