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Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome

NCT02333760 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2021-06-03

No results posted yet for this study

Summary

An open follow up study of patients enrolled in the Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome and treated with autologous CD34+ cells transduced with the w1.6\_hWASP\_WPRE (VSVg) lentiviral vector.

Conditions

  • Wiskott-Aldrich Syndrome

Interventions

GENETIC

Autologous CD34+ cells transduced with WASP lentiviral vector

Follow up of ex vivo gene therapy transplantation of patient's autologous CD34+ cells transduced with lentiviral vector containing human WASP gene

Sponsors & Collaborators

  • Genethon

    lead OTHER

Study Design

Allocation
NA
Purpose
OTHER
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-09-30
Primary Completion
2032-10-31
Completion
2032-10-31

Countries

  • France
  • 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 NCT02333760 on ClinicalTrials.gov