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

NCT06422351 · Status: SUSPENDED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2026-04-16

No results posted yet for this study

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).

Conditions

  • Pyruvate Kinase Deficiency

Interventions

BIOLOGICAL

RP-L301

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

Sponsors & Collaborators

  • Rocket Pharmaceuticals Inc.

    lead INDUSTRY

Principal Investigators

  • Ami Shah, MD · Stanford University

  • Julian Sevilla Navarro, MD, PhD · Hospital Infantil Universitario Niño Jesús

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

  • Maria Chitty-Lopez, MD · Rocket Pharmaceuticals Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

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

Timeline & Regulatory

Start
2026-04-30
Primary Completion
2029-01-31
Completion
2029-01-31
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 NCT06422351 on ClinicalTrials.gov