Open
Actively Recruiting
Registry Study of Revcovi Treatment in Patients With ADA-SCID
About
Brief Summary
This registry is conducted in patients with adenosine deaminase severe combined immune deficiency (ADA-SCID) treated with Revcovi™ to collect periodic clinical and biochemical data on safety and dose adjustment.
Study Type
Phase
Eligibility
Gender
Healthy Volunteers
Minimum Age
Maximum Age
Inclusion Criteria:
- Patients currently receiving chronic ERT with Adagen® and transitioned/transitioning to Revcovi;
- Infants diagnosed via newborn screening and definitive testing for ADA deficiency prescribed Revcovi;
- Patients receiving Revcovi while preparing for Hematopoietic Stem Cell Transplant (HSCT) or Hematopoietic Stem Cell Gene Therapy (HSCGT)
- Patients who decline, are ineligible or do not respond to HSCT or HSC-GT and resume/start Revcovi.
Exclusion Criteria:
- Any condition that, in the opinion of the Investigator, makes the patient unsuitable for the study.
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Study Stats
Protocol No.
19-000797
Category
Pediatrics
Principal Investigator
Contact
Location
- UCLA Westwood