Adenosine Deaminase Deficiency (DBCOND0051206)

Identifiers

Synonyms
ADA Deficiency / Adenosine deaminase deficiency / Adenosine deaminase deficiency, unspecified / Adenosine deaminase [ADA] deficiency / Adenosine deaminase deficiency (disorder) / Combined immunodeficiency, unspecified / Combined immunodeficiency / Combined immunodeficiency syndrome / Combined immunity deficiency / Combined immunodeficiencies / Combined immunodeficiency disease (disorder)

Associated Data

Indicated Drugs and Targets
DrugDescriptionTargets
Pegademase
A modified form of bovine adenosine deaminase used to treat adenosine deaminase deficiency, a condition which leads to the formation of adenosine metabolites which are cytotoxic to lymphocytes.
Clinical Trials
IdentifierTitleDrug(s)PurposePhaseStatus
NCT01380990
Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiencytreatment1 / 2completed
NCT04049084
An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCIDNot AvailableNot Availableenrolling_by_invitation
NCT05300334
Investigation of ADA Enzyme DeficiencyNo drug interventionsNot AvailableNot Availableunknown_status
NCT02022696
Treatment of SCID Due to ADA Deficiency With Autologous Transplantation of Cord Blood or Hematopoietic CD 34+ Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral VectorNo drug interventionstreatment1completed
NCT01279720
Gene Therapy ADA DeficiencyNo drug interventionstreatment1 / 2completed
NCT05300373
Evaluation of Adenosine Deaminase (ADA) Enzyme Deficiency in Patients With Lymphopenia and/or Elevated Immunoglobulin ENo drug interventionsNot AvailableNot Availableunknown_status
NCT03878069
Registry Study of Revcovi Treatment in Patients With ADA-SCIDNot AvailableNot Availablecompleted
NCT05300347
Observational Study Evaluating the Prevalence of Enzyme Deficiency in Pulmonology Clinics (ADA)No drug interventionsNot AvailableNot Availableunknown_status
NCT01420627
EZN-2279 in Patients With ADA-SCIDtreatment3completed
NCT00008450
Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplanttreatment1completed