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Clinical trial

Efficacy and Safety of Cryopreserved Autologous Mobilized Peripheral Blood CD34+ HSPCs Transduced Ex Vivo With the EFS-ADA Lentiviral Vector in Patients With Severe Combined Immune Deficiency Due To Adenosine Deaminase Deficiency

NCT05432310 is registered in ClinicalTrials.gov with University of California, Los Angeles as lead sponsor.

NCT NCT05432310
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Registry record

As filed in ClinicalTrials.gov.

Title
Efficacy and Safety of Cryopreserved Autologous Mobilized Peripheral Blood CD34+ HSPCs Transduced Ex Vivo With the EFS-ADA Lentiviral Vector in Patients With Severe Combined Immune Deficiency Due To Adenosine Deaminase Deficiency
Lead sponsor
University of California, Los Angeles
Phase
Phase 1/2
Overall status
Active, not recruiting
Start date
January 4, 2023
Completion date
December 31, 2027
Enrollment
20 (estimated)
Sites
1

Conditions

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

adenosine deaminase severe combined immune deficiency