Sobi.PEGCET-101
Research type
Research Study
Full title
A Phase 3, Randomized, Double-blind, Placebo-controlled Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients with Cold Agglutinin Disease (CAD)
IRAS ID
1004834
Contact name
Anke Arnold
Contact email
Sponsor organisation
Swedish Orphan Biovitrum AB
Eudract number
2021-003160-27
Research summary
Cold agglutinin disease(CAD) is a disease that makes the body’s immune system attack the red blood cells and destroy them. It is triggered by cold temperatures, and it can cause problems that range from dizziness to heart failure. It’s also called cold antibody hemolytic anemia.
Pegcetacoplan is a SST2 agonist drug that prevents C3-mediated extravascular and intravascular hemolysis (premature destruction of red blood cells). As a new study drug, Pegcetacoplan, has the potential to deliver a benefit to CAD patients by displaying a safety and efficacy (how well it works) profile.
This is a Phase 3, double-blinded study (where both the participant and the investigator doesn't know what the participant is receiving) will be conducted to evaluate the efficacy and safety profile.
The primary aim of this research study is to see how well pegcetacoplan works on its own by comparing with a placebo (a dummy treatment), The study will also look at how well people tolerate taking pegcetacoplan.
If the participants decides to be in this study, and the study doctor says they can be in the study, their participation could last up to 102 weeks (approximately 2 years). This study has five parts, which are described below:
1. Screening (to see if they are eligible for the study, up to 4 weeks)
2. Double-blind treatment period (24 weeks, during which they will receive either pegcetacoplan or placebo)
3. Open-label treatment period (24 weeks, during which all patients will receive pegcetacoplan)
4. Open-label maintenance period (up to 48 weeks or until the product is commercially available, during which all patients will receive pegcetacoplan)
5. Follow-up period (6 weeks, to check on their health status after treatment).
Several tests and procedures are planned for this study. The vast majority of them are the same as those for regular CAD care, although they may be performed more frequently than done as per standard of care.
Up to 57 adult participant are to be treated.REC name
South West - Central Bristol Research Ethics Committee
REC reference
22/SW/0021
Date of REC Opinion
20 May 2022
REC opinion
Further Information Favourable Opinion