ADA-SCID (DBCOND0048585)
Identifiers
- Synonyms
- ADA SCID
Associated Data
- Indicated Drugs and Targets
- Not Available
- Clinical Trials
Identifier Title Drug(s) Purpose Phase Status NCT01186913 Natural History Study of SCID Disorders No drug interventions Not Available Not Available enrolling_by_invitation NCT01852071 Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene treatment 1 / 2 completed 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 NCT01182857 Quality of Life and Neuropsychiatric Sequelae in Patients Treated With Gene Therapy for ADA-SCID and in Their Parents No drug interventions Not Available Not Available withdrawn NCT01420627 EZN-2279 in Patients With ADA-SCID treatment 3 completed NCT01346150 Patients Treated for SCID (1968-Present) No drug interventions Not Available Not Available unknown_status