Not applicableUnknownNCT05300334What this trial is testingInvestigation of ADA Enzyme DeficiencyWho this might be right forAdenosine Deaminase Deficiency TRPHARM 800
Testing effectiveness (Phase 2)Study completedNCT01279720What this trial is testingGene Therapy ADA DeficiencyWho this might be right forAdenosine Deaminase Deficiency Great Ormond Street Hospital for Children NHS Foundation Trust 8
Not applicableEnrolling By InvitationNCT01186913What this trial is testingNatural History Study of SCID DisordersWho this might be right forSevere Combined Immunodeficiency (SCID)Leaky SCIDOmenn Syndrome+3 more National Institute of Allergy and Infectious Diseases (NIAID) 690
Testing effectiveness (Phase 2)Study completedNCT02999984What this trial is testingEfficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCIDWho this might be right forSevere Combined Immunodeficiency Due to ADA Deficiency University of California, Los Angeles 10
Not applicableWithdrawnNCT01182857What this trial is testingQuality of Life and Neuropsychiatric Sequelae in Patients Treated With Gene Therapy for ADA-SCID and in Their ParentsWho this might be right forADA-SCID National Human Genome Research Institute (NHGRI)
Not applicableUnknownNCT01346150What this trial is testingPatients Treated for SCID (1968-Present)Who this might be right forSCIDADA-SCIDXSCID+3 more National Institute of Allergy and Infectious Diseases (NIAID) 1,007
Early research (Phase 1)Study completedNCT00008450What this trial is testingTotal-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow TransplantWho this might be right forAdenosine Deaminase DeficiencyAutosomal Recessive DisorderImmune System Disorder+4 more Fred Hutchinson Cancer Center 6
Early research (Phase 1)Study completedNCT00018018What this trial is testingGene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) DeficiencyWho this might be right forSevere Combined Immunodeficiency Syndrome National Human Genome Research Institute (NHGRI) 8
Testing effectiveness (Phase 2)Study completedNCT01852071What this trial is testingAutologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA GeneWho this might be right forADA-SCID University of California, Los Angeles 46
Testing effectiveness (Phase 2)Study completedNCT01380990What this trial is testingLentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) DeficiencyWho this might be right forAdenosine Deaminase DeficiencySevere Combined Immunodeficiencies (SCID) Great Ormond Street Hospital for Children NHS Foundation Trust 36
Not applicableStudy completedNCT00001255What this trial is testingGene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History StudyWho this might be right forSevere Combined Immunodeficiency National Human Genome Research Institute (NHGRI) 10
Not applicableNot Yet RecruitingNCT03645460What this trial is testingGene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)Who this might be right forAdenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID) Shenzhen Geno-Immune Medical Institute 10
Testing effectiveness (Phase 2)Study completedNCT03765632What this trial is testingEfficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCIDWho this might be right forSevere Combined Immunodeficiency Due to ADA Deficiency Great Ormond Street Hospital for Children NHS Foundation Trust 13
Large-scale testing (Phase 3)WithdrawnNCT04140539What this trial is testingA Clinical Study to Enable Process Validation of Commercial Grade OTL-101Who this might be right forSevere Combined Immunodeficiency Due to ADA Deficiency University of California, Los Angeles
Not applicableUnknownNCT04959890What this trial is testingMethodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene TherapyWho this might be right forSevere Combined Immunodeficiency Due to ADA Deficiency Fondazione Telethon 15
Testing effectiveness (Phase 2)Study completedNCT00794508What this trial is testingMND-ADA Transduction of CD34+ Cells From Children With ADA-SCIDWho this might be right forSevere Combined Immunodeficiency Donald B. Kohn, M.D. 10
Not applicableEnrolling By InvitationNCT04049084What this trial is testingAn Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCIDWho this might be right forAdenosine Deaminase DeficiencySevere Combined Immunodeficiency (SCID) University of California, Los Angeles 70
Not applicableStudy completedNCT03878069What this trial is testingRegistry Study of Revcovi Treatment in Patients With ADA-SCIDWho this might be right forAdenosine Deaminase Severe Combined Immunodeficiency Chiesi Farmaceutici S.p.A. 32
Large-scale testing (Phase 3)Study completedNCT01420627What this trial is testingEZN-2279 in Patients With ADA-SCIDWho this might be right forADA-SCIDAdenosine Deaminase DeficiencySevere Combined Immunodeficiency Leadiant Biosciences, Inc. 7
Testing effectiveness (Phase 2)Active Not RecruitingNCT05432310What this trial is testingGene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA VectorWho this might be right forAdenosine Deaminase Severe Combined Immune Deficiency University of California, Los Angeles 20