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
Drug Description Targets 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.- Adenosinetarget
- Growth factor receptor-bound protein 2target
- Clinical Trials
Identifier Title Drug(s) Purpose Phase Status NCT01380990 Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency treatment 1 / 2 completed NCT04049084 An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID Not Available Not Available enrolling_by_invitation NCT05300334 Investigation of ADA Enzyme Deficiency No drug interventions Not Available Not Available unknown_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 Vector No drug interventions treatment 1 completed NCT01279720 Gene Therapy ADA Deficiency No drug interventions treatment 1 / 2 completed NCT05300373 Evaluation of Adenosine Deaminase (ADA) Enzyme Deficiency in Patients With Lymphopenia and/or Elevated Immunoglobulin E No drug interventions Not Available Not Available unknown_status NCT03878069 Registry Study of Revcovi Treatment in Patients With ADA-SCID Not Available Not Available completed NCT05300347 Observational Study Evaluating the Prevalence of Enzyme Deficiency in Pulmonology Clinics (ADA) No drug interventions Not Available Not Available unknown_status NCT01420627 EZN-2279 in Patients With ADA-SCID treatment 3 completed NCT00008450 Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant treatment 1 completed