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Clinical trial

Efficacy and Safety of a Cryopreserved Formulation of Autologous CD34+ Haematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1α Short Form (EFS) Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency (SCID) Due to Adenosine Deaminase Deficiency

NCT03765632 is registered in ClinicalTrials.gov with Great Ormond Street Hospital for Children NHS Foundation Trust as lead sponsor.

NCT NCT03765632
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Registry record

As filed in ClinicalTrials.gov.

Title
Efficacy and Safety of a Cryopreserved Formulation of Autologous CD34+ Haematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1α Short Form (EFS) Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency (SCID) Due to Adenosine Deaminase Deficiency
Lead sponsor
Great Ormond Street Hospital for Children NHS Foundation Trust
Phase
Phase 1/2
Overall status
Completed
Start date
January 3, 2018
Completion date
September 28, 2022
Enrollment
13 (actual)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

severe combined immunodeficiency due to ada deficiency