NCT03878069

Registry Study of Revcovi Treatment in Patients With ADA-SCID

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

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 32 (actual)
• Sponsor: Chiesi Farmaceutici S.p.A. · Industry
• Sex: All
• Age: 0 Months – 65 Years
• Healthy volunteers: Not accepted

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.

Detailed description

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 either until they are able to successfully undergo a stem cell transplant or stem cell gene therapy and thus no longer require ERT treatment, or until all ongoing participants have received a minimum of 24 months of Revcovi treatment. Note: Due to the nature of this study, all analyses are descriptive and no statistical hypotheses will be tested.

Conditions

• Adenosine Deaminase Severe Combined Immunodeficiency

Interventions

Type	Name	Description
BIOLOGICAL	elapegademase-lvlr	Revcovi is administered intramuscularly (i.m.). Weekly dosage is calculated in mg/kg of body weight, and can be adjusted over the course of the trial based on ADA activity and dAXP concentration as well as on clinical assessment by the treating physician.

Timeline

• Start date: 2019-09-30
• Primary completion: 2023-01-18
• Completion: 2023-01-18
• First posted: 2019-03-18
• Last updated: 2024-11-15
• Results posted: 2024-11-15

Locations

14 sites across 1 country: United States

Regulatory

• FDA-regulated drug study