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Gene Therapy for Wiskott-Aldrich Syndrome (WAS)

NCT01347242 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2021-06-03

No results posted yet for this study

Summary

This is a phase I/II study to evaluate the safety and efficacy of Hematopoietic Stem Cell genetherapy for the Wiskott-Aldrich Syndrome.

Conditions

  • Wiskott-Aldrich Syndrome

Interventions

GENETIC

Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene

transplantation of patient's autologous CD34+ cells transduced with lentiviral vector containing human WAS gene

Sponsors & Collaborators

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    collaborator OTHER

  • Institute of Child Health

    collaborator OTHER

  • Genethon

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-03-31
Primary Completion
2019-11-13
Completion
2019-11-13

Countries

  • 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 NCT01347242 on ClinicalTrials.gov