NCT06422351View on ClinicalTrials.gov

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

PHASE2Not yet recruitingINTERVENTIONAL
Enrollment

10

Participants

Timeline

Start Date

August 31, 2024

Primary Completion Date

November 30, 2026

Study Completion Date

January 31, 2029

Conditions
Pyruvate Kinase Deficiency
Interventions
BIOLOGICAL

RP-L301

Autologous genetically modified CD34+ hematopoietic stem cells containing the corrected PKLR (Pyruvate Kinase L/R) gene

Trial Locations (3)

28009

Hospital Infantil Universitario Niño Jesús, Madrid

94305

Stanford University, Palo Alto

Unknown

Hospital Universitario Fundación Jiménez Díaz, Madrid

All Listed Sponsors
lead

Rocket Pharmaceuticals Inc.

INDUSTRY