Peg-Ada

DRUG2 trials

Sponsors

Great Ormond Street Hospital for Children NHS Foundation Trust

Conditions

Adenosine Deaminase DeficiencySevere Combined Immunodeficiencies (SCID)Severe Combined Immunodeficiency Due to ADA Deficiency

Phase 1

Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency

CompletedNCT01380990

Great Ormond Street Hospital for Children NHS Foundation TrustAdenosine Deaminase Deficiency, Severe Combined Immunodeficiencies (SCID)

Start: 2012-11-15End: 2019-12-23Updated: 2021-09-16

Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID

CompletedNCT03765632

Great Ormond Street Hospital for Children NHS Foundation TrustSevere Combined Immunodeficiency Due to ADA Deficiency

Start: 2018-01-03End: 2022-09-28Updated: 2023-02-01

Related Papers

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency

New England Journal of Medicine2021-05-11204 citations