MedTrace Logo

Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCID

NCT04797260 · Status: SUSPENDED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2026-07-16

No results posted yet for this study

Summary

This trial is a prospective, non-randomized, open-label, multicentre single-arm phase I/II intervention trial in children up to 24 months of age with RAG1-deficient SCID and an indication for allogeneic hematopoietic stem cell transplantation but lacking an HLA-matched donor. The trial involves infusion of autologous CD34+ cells transduced with the pCCL.MND.coRAG1.wpre lentiviral vector (hereafter called RAG1 LV CD34+ cells) in up to 10 patients with RAG1-deficient SCID. Patients will be regularly monitored for 5 years after infusion. Follow up as part of routine clinical care for post-transplant patients will be annual after this, for at least 15 years after IMP infusion.

Conditions

  • Severe Combined Immunodeficiency Due to RAG1 Deficiency

Interventions

GENETIC

Gene therapy

Patients will be infused with autologous CD34+ cells transduced with the pCCL.MND.coRAG1.wpre lentiviral vector (RAG1 LV CD34+ cells).

Sponsors & Collaborators

  • CR2O B.V.

    collaborator UNKNOWN

  • Videja B.V.

    lead INDUSTRY

Principal Investigators

  • Arjan C Lankester, Prof.dr. · Leiden University Medical Center

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
8 Weeks
Max Age
24 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-07-23
Primary Completion
2031-12-31
Completion
2031-12-31

Countries

  • Netherlands
  • Poland
  • Spain
  • Turkey (Türkiye)

Study Locations

More Related Trials

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 NCT04797260 on ClinicalTrials.gov