Registry Study of Revcovi Treatment in Patients With ADA-SCID

Severe Combined Immunodeficiency Clinical Trial

Official title: Single Arm, Open-Label, Multicenter, Registry Study of Revcovi (Elapegademase-lvlr) Treatment in ADA-SCID Patients Requiring Enzyme Replacement Therapy

Status Completed

Clinical Trial 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.

Clinical Trial Description

Patients with ADA-SCID who require treatment with Revcovi as Enzyme Replacement Therapy (ERT) will be followed until the last enrolled patient has reached a minimum of 24 months of Revcovi treatment or until undergoing HSCT or HSCGT, whichever occurs first.

Patients undergoing HSCT or HSC-GT will be followed one month after last Revcovi dose and again at six months to assess adverse events (AEs) and survival. Throughout the duration of the study, patients will be assessed continually for AEs.

Patients/Parents/Caregivers will self-administer weekly intramuscular (IM) dose(s) of Revcovi and will be followed according to the Suggested Schedule of Assessments for trough dAXP and ADA activity. Treatment dosing and monitoring will be individualized per provider and patient characteristics in adherence with each study sites' standards of care.

Patients in the Phase 3 Revcovi™ study (STP-2279-002) will be given the opportunity to enroll in this registry study and proceed to the Treatment Month 6 Visit per the Suggested Schedule of Assessments for Adagen-Transitioning Patients. ;

Related Conditions & MeSH terms

• Adenosine Deaminase Deficiency
• Severe Combined Immunodeficiency

• NCT number: NCT03878069
• Study type: Observational [Patient Registry]
• Source: Chiesi Farmaceutici S.p.A.
• Status: Completed
• Start date: June 25, 2019
• Completion date: January 18, 2023