Trial Profile
ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID
Status:
Completed
Phase of Trial:
Phase II
Latest Information Update: 02 Feb 2024
Price :
$35
*
At a glance
- Drugs GSK 2696273 (Primary) ; Busulfan
- Indications Adenosine deaminase deficiency
- Focus Therapeutic Use
- Sponsors GlaxoSmithKline; Orchard Therapeutics
- 07 Aug 2019 Status changed from active, no longer recruiting to completed.
- 30 May 2016 According to a MolMed media release, the European Commission approved GSK-2696273 (Strimvelis), the first ex-vivo stem cell gene therapy to treat patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID).
- 27 May 2016 Primary endpoint (Survival) has been met, according to a GlaxoSmithKline media release.