Open
Actively Recruiting
Registry Study of Revcovi Treatment in Patients With ADA-SCID
About
Brief Summary
The objective of this study is to develop a registry of patients with adenosine deaminase severe combined immune deficiency (ADA-SCID) treated with Revcovi™ that contains clinical and biochemical assessments for safety and dose adjustment based on adenosine deaminase (ADA) activity and erythrocyte deoxyadenosine nucleotide (dAXP) levels as well as immunologic monitoring.
Study Type
Phase
Eligibility
Gender
Healthy Volunteers
Minimum Age
Maximum Age
Inclusion Criteria:
- Patients with diagnosis of ADA-SCID who require ERT with ADA as judged by the treating physicians, based on the medical history, biochemical test or genotyping.
- Understanding and willing to comply with the Registry recommendations via signed and dated written informed consent/assent.
- ADA-SCID patient requiring Revcovi as an ERT.
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