Found 21 matching trials
Patients Treated for SCID (1968-Present) N/A Individuals with a past diagnosis of severe combined immune deficiency (inc...
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University of Alabama at Birmingham
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Retroviral Insertion Site Methodology Study PHASE4 Adenosine deaminase (ADA) is an enzyme involved in the development and func...
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Multiple Locations
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MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID PHASE2 Severe combined immune deficiency (SCID) may result from inherited deficien...
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University of California, Los Angeles
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ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID PHASE2 This is a phase I/II protocol to evaluate the safety and efficacy of ADA ge...
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Investigational Site
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Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency PHASE1 This is a historically controlled, non-randomized Phase I/II clinical trial...
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Great Ormond Street Hospital for Children NHS Foundation Trust
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Natural History Study of SCID Disorders N/A This study is a prospective evaluation of children with Severe Combined Imm...
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Enrolling By Invitation
Location
University of Alabama at Birmingham
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A Clinical Study to Enable Process Validation of Commercial Grade OTL-101 PHASE2 The purpose of the current study is to treat at least 3 ADA-SCID patients w...
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University of California, Los Angeles
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Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study N/A This study will monitor the long-term effects of gene therapy in patients w...
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National Human Genome Research Institute (NHGRI)
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Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) N/A Adenosine deaminase (ADA) enzyme deficiency results in severe combined immu...
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Enrolling By Invitation
Location
Ospedale San Raffaele
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Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene PHASE1 The aim of this study is to assess the safety and efficacy of autologous tr...
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Mattel Children's Hospital, UCLA
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