NCT04105166View on ClinicalTrials.gov

Gene Therapy for Pyruvate Kinase Deficiency (PKD)

PHASE1CompletedINTERVENTIONAL
Enrollment

4

Participants

Timeline

Start Date

July 6, 2020

Primary Completion Date

June 9, 2025

Study Completion Date

June 9, 2025

Conditions
Pyruvate Kinase Deficiency
Interventions
BIOLOGICAL

RP-L301

Autologous genetically modified CD34+ hematopoietic stem cells containing the corrected PKD gene

Trial Locations (3)

28009

Hospital Infantil Universitario Niño Jesús, Madrid

94304

Stanford University, Stanford

Unknown

Hospital Universitario Fundación Jiménez Díaz, Madrid

All Listed Sponsors
lead

Rocket Pharmaceuticals Inc.

INDUSTRY

NCT04105166 - Gene Therapy for Pyruvate Kinase Deficiency (PKD) | Biotech Hunter | Biotech Hunter