Registry Study of Revcovi Treatment in Patients With ADA-SCID

Launched by CHIESI FARMACEUTICI S.P.A. · Mar 13, 2019

Keywords

ClinConnect Summary

Patients with ADA-SCID who require ERT will receive Revcovi on a dosage and schedule determined by the treating physician. They will be followed for safety throughout the study, and will be monitored for the efficacy markers of adenosine deaminase (ADA) activity and deoxyadenosine nucleotide (dAXP) concentration according to a suggested schedule. Some subjects will be new to ERT; some will have transitioned from Adagen, which was the ERT available before Revcovi; and some will have previously participated in an earlier Phase 3 trial of Revcovi (study STP-2279-002). Patients will be followed...

Gender

ALL

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.