At a glance
ClinicalIndex Comparison RecordStandardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
Gene Therapy Via Intravenous Injection of Lentiviral Vector (Ivlv-ADA) for the Treatment of Adenosine Deaminase-severe Combined Immunodeficiency (ADA-SCID)
In Brief
A clinical study evaluating Direct intravenous injection of ivlv-ADA lentiviral vector for Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID). Currently recruiting, targeting 10 participants across 1 site.
Detailed Summary
This is a Phase I/II trial of in vivo lentiviral gene therapy for treating adenosine deaminase severe combined immunodeficiency (ADA-SCID) using a self-inactivating lentiviral vector (LV) ivlv-ADA to functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the direct intravenous (iv) LV gene therapy protocol.
Study Details
Timeline
Arms & Interventions
Direct intravenous injection of ivlv-ADA lentiviral vector
Interventions
Injection of ivlv-ADA lentiviral vector at \~1x10\^9 per kg body weight