Trial Profile
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
Status:
Completed
Phase of Trial:
Phase I/II
Latest Information Update: 21 Sep 2021
Price :
$35
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At a glance
- Drugs Simoladagene autotemcel (Primary) ; Busulfan; Elapegademase
- Indications Adenosine deaminase deficiency
- Focus First in man; Therapeutic Use
- 11 May 2021 Results (n=50) from NCT01852071, NCT02999984, and NCT01380990 assessing overall and event-free survival in autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency, published in the New England Journal of Medicine
- 15 Apr 2020 Status changed from active, no longer recruiting to completed.
- 08 Aug 2018 Status changed from recruiting to active, no longer recruiting.