N/A
N=32
Registry Study of Revcovi Treatment in Patients With ADA-SCID
Adenosine Deaminase Severe Combined Immunodeficiency
Bottom Line
View on ClinicalTrials.gov: NCT03878069 ↗Enrolled (actual)
32
Serious AEs
0.0%
Results posted
Nov 2024
Primary outcome: Primary: Number of Subjects Meeting the Toxicity Threshold for Deoxyadenosine Nucleotide (dAXP) Concentration at the Last Measurement — 6; 17; 4 Participants
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- elapegademase-lvlr (Biological)
- Age
- Pediatric, Adult, Older Adult · 0+ yrs
- Sex
- All
- Sponsor
- Chiesi Farmaceutici S.p.A.
- Primary completion
- Jan 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Subjects Meeting the Toxicity Threshold for Deoxyadenosine Nucleotide (dAXP) Concentration at the Last Measurement |
6; 17; 4 | — |
| PRIMARY Number of Subjects Meeting the Optimal Threshold for ADA Activity at the Last Measurement |
7; 14; 4 | — |
| PRIMARY Safety of Revcovi |
3; 1; 0 | — |
Summary
This registry study is being conducted in patients with adenosine deaminase severe combined immune deficiency (ADA-SCID) who require enzyme replacement therapy (ERT) treatment with Revcovi. Data on safety and on measures of efficacy are collected.
Eligibility Criteria
Inclusion Criteria
- Male or female, aged newborn to adult
- In need of ERT treatment due to one of the following circumstances:
- Waiting to receive a stem cell transplant
- Previously declined, had been found to be ineligible for, or did not respond to a stem cell transplant
- Waiting to enrol in a clinical trial on gene therapy, or had been found to be ineligible for or had failed such a trial
- One of the following histories of ERT treatment:
- Revcovi only
- Previously on Adagen but had transitioned to Revcovi
- Not yet on any ERT but about to start on Revcovi
Exclusion Criteria
- Any condition that, in the opinion of the Investigator, makes the patient unsuitable for the study.
Data sourced from ClinicalTrials.gov (NCT03878069). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.