Found 10 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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EZN-2279 in Patients With ADA-SCID PHASE3 The purpose of this study is to evaluate the safety, efficacy, and pharmaco...
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Children's Hospital Los Angeles
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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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Registry Study of Revcovi Treatment in Patients With ADA-SCID N/A This registry study is being conducted in patients with adenosine deaminase...
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University of California Los Angeles
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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
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University of Alabama at Birmingham
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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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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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Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID PHASE1 This is a prospective, non-randomized, single-cohort, longitudinal, single-...
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Great Ormond Street Hospital for Children NHS Foundation Trust
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Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID PHASE1 This is a prospective, non-randomized, single-cohort, longitudinal, single-...
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University of California, Los Angeles
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