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

NCT04797260 · Status: RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2024-04-18

No results posted yet for this study

Summary

This study is a prospective, non-randomized, open-label, two-centre phase I/II intervention study designed to treat children up to 24 months of age with RAG1-deficient SCID with an indication for allogeneic hematopoietic stem cell transplantation but lacking an HLA-matched donor. The study involves infusion of autologous CD34+ cells transduced with the pCCL.MND.coRAG1.wpre lentiviral vector (hereafter called RAG1 LV CD34+ cells) in five patients with RAG1-deficient SCID.

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

  • ZonMw: The Netherlands Organisation for Health Research and Development

    collaborator OTHER

  • Horizon 2020 - European Commission

    collaborator OTHER

  • Leiden University Medical Center

    lead OTHER

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
2029-12-31
Completion
2029-12-31

Countries

  • Australia
  • Italy
  • Netherlands
  • Poland
  • Spain
  • Turkey (Türkiye)
  • United Kingdom

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 NCT04797260 on ClinicalTrials.gov