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MND-ADA Gene Therapy for ADA-SCID
NCT00794508Phase 2Completed
- Start
- Nov 2008
- Primary completion
- Dec 2014
- Modality
- Gene Editing, Gene Silencing, Stem Cell Therapies
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of autologous CD34+ cells transduced with MND-ADA retroviral vector in severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID). Primary endpoint: Number of Participants With Adverse Events at 2 years. Started Nov 2008; status: completed.
02
Drugs
autologous CD34+ cells transduced with MND-ADA retroviral vector
03
Conditions
severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID)
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