Multi-Year Registry Study of Elapegademase Treatment in Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) Requiring Enzyme Replacement Therapy.

ABSTRACT:

Purpose

The safety and tolerability of elapegademase (elapegademase-lvlr; Revcovi^®) a PEGylated recombinant adenosine deaminase (ADA), were demonstrated in two Phase 3 clinical trials in the U.S. and Japan in patients with ADA-deficient severe combined immunodeficiency (ADA-SCID). Elapegademase replaced Adagen^® (pegademase, a PEGylated bovine ADA) in 2018. This registry study (NCT03878069) was conducted as a post-marketing requirement to bolster the limited safety and effectiveness data on elapegademase in patients with ADA-SCID and to study patients starting on enzyme replacement therapy (ERT) de novo.

Methods

Patients were managed by routine clinical care and treating physicians' judgement from September 2019 to January 2023. Primary endpoints include

SUBMITTER: Dorsey MJ 

PROVIDER: S-EPMC11947034 | biostudies-literature | 2025 Mar

REPOSITORIES: biostudies-literature