ADA-SCID (DBCOND0048585)

Identifiers

Synonyms
ADA SCID

Associated Data

Indicated Drugs and Targets
Not Available
Clinical Trials
IdentifierTitleDrug(s)PurposePhaseStatus
NCT01186913
Natural History Study of SCID DisordersNo drug interventionsNot AvailableNot Availableenrolling_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 Genetreatment1 / 2completed
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
NCT01182857
Quality of Life and Neuropsychiatric Sequelae in Patients Treated With Gene Therapy for ADA-SCID and in Their ParentsNo drug interventionsNot AvailableNot Availablewithdrawn
NCT01420627
EZN-2279 in Patients With ADA-SCIDtreatment3completed
NCT01346150
Patients Treated for SCID (1968-Present)No drug interventionsNot AvailableNot Availableunknown_status