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Gene Therapy for Pyruvate Kinase Deficiency (PKD)

NCT04105166 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 4

Last updated 2025-09-05

No results posted yet for this study

Summary

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

Conditions

  • Pyruvate Kinase Deficiency

Interventions

BIOLOGICAL

RP-L301

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

Sponsors & Collaborators

  • Rocket Pharmaceuticals Inc.

    lead INDUSTRY

Principal Investigators

  • José Luis López Lorenzo, MD · Hospital Universitario Fundación Jiménez Díaz

  • Ami Shah, MD · Stanford University

  • Julián Sevilla Navarro, MD, PhD · Hospital Universitario Fundación Jiménez Díaz

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
8 Years
Max Age
50 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-07-06
Primary Completion
2025-06-09
Completion
2025-06-09
FDA Drug
Yes

Countries

  • United States
  • Spain

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04105166 on ClinicalTrials.gov