ALXN1820-ANCA-201 - Phase 2 - Tarperprumig in Adult Pts. with ANCA-Associated Vasculitis
Research type
Research Study
Full title
Phase 2, Randomized, Double-Blind, Placebo-controlled, Parallel-Group, Multicenter Study to Evaluate the Safety and Efficacy of Tarperprumig in Adult Participants with Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
IRAS ID
1012367
Contact name
Youssef Farag
Contact email
Sponsor organisation
Alexion Pharmaceuticals, Inc.
Clinicaltrials.gov Identifier
Research summary
The purpose of this Phase 2 study is to see if tarperprumig, the study drug, is safe and works better compared to placebo in adult patients with a disease called ANCA- associated vasculitis. The study aims to assess but not limited to:
- Side effects of Tarperprumig and how severe the side effects are compared to placebo.
- What the body does to tarperprumig (pharmacokinetics) and what tarperprumig does to the body (pharmacodynamics).
- Changes in safety assessments from the start of the study (vital sign measurements, physical examination, clinical laboratory tests, and ECG results).
Tarperprumig works by blocking properdin, a protein in the body that helps the immune system function. When the body’s natural defence system (know as the complement system) works properly, it is a powerful tool that protects the body against harmful invaders. But when the complement system is activated in patients with this disease, the proteins participate in ANCA-associated vasculitis.
The study has a screening period of up to 2 weeks, treatment period of 52 weeks and safety follow-up period of 18 weeks. Participants will enter the treatment period if they meet the screening eligibility requirements and will be split to one of three study arms; Traperprumig, alternating weeks for Tarperprumig or placebo, placebo). The participants will have a 1 in 3 chance of randomly being assigned one of these study arms. The treatment period is blinded. Tarperprumig and/or placebo will be given to participants via weekly injection for the first 6 months followed by once every other week for the last 6 months. In each of the three arms, participants will receive the standard of care (which is a treatment that is given even if not taking part in a clinical trial). The standard of care is Rituximab and Glucocorticoid.
After completion of the treatment period, participants will have a safety follow-up period. The total study duration is 72 weeks. 75 participants worldwide will be enrolled.REC name
Wales REC 5
REC reference
25/WA/0312
Date of REC Opinion
3 Dec 2025
REC opinion
Further Information Favourable Opinion