Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency

Summary

This is an open-label Phase II trial to evaluate the efficacy of a hematopoietic cell-based gene therapy for patients with Pyruvate Kinase Deficiency (PKD).

Timeline

Start

2026-04

Primary completion

2029-01

Completion

2029-01

Outcome

Outcome not reported

Stopped: “Initiation of study has been paused, but may re-start in the future.”

Drugs

Indications