Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency
Phase I/II, Historical Controlled, Open-label, Non-randomised, Single-centre Trial to Assess the Safety and Efficacy of EF1αS-ADA Lentiviral Vector Mediated Gene Modification of Autologous CD34+ Cells From ADA-deficient Individuals
Sponsor: Great Ormond Street Hospital for Children NHS Foundation Trust
This PHASE1/PHASE2 trial investigates Adenosine Deaminase Deficiency and Severe Combined Immunodeficiencies (SCID) and is currently completed. Great Ormond Street Hospital for Children NHS Foundation Trust leads this study, which shows 8 recorded versions since 2012 — indicating limited longitudinal coverage. The change history captured here reflects the iterative nature of clinical trial conduct.
Status Flow
Change History
8 versions recorded-
Sep 2024 — Present [monthly]
Completed PHASE1/PHASE2
-
Jul 2024 — Sep 2024 [monthly]
Completed PHASE1/PHASE2
Phase: PHASE1_PHASE2 → PHASE1/PHASE2
-
Oct 2021 — Jul 2024 [monthly]
Completed PHASE1_PHASE2
-
Jan 2021 — Oct 2021 [monthly]
Completed PHASE1_PHASE2
-
May 2020 — Jan 2021 [monthly]
Completed PHASE1_PHASE2
Status: Active Not Recruiting → Completed
▶ Show 3 earlier versions
-
Sep 2018 — May 2020 [monthly]
Active Not Recruiting PHASE1_PHASE2
Status: Recruiting → Active Not Recruiting
-
Jun 2018 — Sep 2018 [monthly]
Recruiting PHASE1_PHASE2
-
Jan 2017 — Jun 2018 [monthly]
Recruiting PHASE1_PHASE2
First recorded
Nov 2012
Trial started
Per CT.gov start date — pre-dates our first snapshot
Eligibility Summary
No eligibility information available.
Contact Information
- Great Ormond Street Hospital for Children NHS Foundation Trust
- Orchard Therapeutics
For direct contact, visit the study record on ClinicalTrials.gov .