NCT03478670View on ClinicalTrials.gov

Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)

Enrolling by invitationOBSERVATIONAL
Enrollment

50

Participants

Timeline

Start Date

March 28, 2017

Primary Completion Date

May 31, 2037

Study Completion Date

May 31, 2037

Conditions
Immunologic Deficiency Syndromes
Interventions
GENETIC

Strimvelis

"Strimvelis is a CD34+ cell enriched dispersion of human autologous bone marrow derived hematopoietic stem/progenitor cells transduced with a retroviral vector containing the human ADA gene. It will be administered as an intravenous infusion once only.~This is an observational registry that includes all patients who have previously received Strimvelis™ or GSK2696273."

Trial Locations (1)

20132

Ospedale San Raffaele, Milan

All Listed Sponsors
lead

Fondazione Telethon

OTHER