Birtamimab plus SoC or Placebo plus SoC in Light Chain AL
Research type
Research Study
Full title
A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled, Efficacy and Safety Study of Birtamimab Plus Standard of Care vs. Placebo Plus Standard of Care in Mayo Stage IV Subjects with Light Chain (AL) Amyloidosis
IRAS ID
1003764
Contact name
Rory Graham
Contact email
Sponsor organisation
Prothena Biosciences Limited
Eudract number
2021-000037-14
Research summary
The purpose of this study is to evaluate whether birtamimab will improve survival in participants with Mayo Stage IV AL amyloidosis. This is a relatively rare blood disease in which deposits of abnormal proteins, called amyloid, can build up in organs and cause progressive damage so that they no longer work properly.
People who are 18 years of age and older who qualify for this study will receive either birtamimab plus the standard treatment for amyloidosis (called standard of care) or placebo (saline) plus the standard of care. The placebo does not have any active ingredients in it. The term “study medication” refers to both birtamimab and placebo. Participants will be assigned randomly to one of two groups. They will have a 2 in 3 (67%) of chance receiving birtamimab plus standard of care and a 1 in 3 (33%) chance of receiving placebo plus standard of care.
Participants will receive either placebo or birtamimab about every 28 days depending on which group they are in. Every participant in this study will receive the same first-line chemotherapy, which is the first chemotherapy that they will receive for their AL amyloidosis which includes Bortezomib and will be injected under the skin. The study doctor will make decisions about the chemotherapy that participants receive after that. The study is expected to last for approximately 2.5 years, this includes screening, enrolment, and treatment periods.
The study is double-blinded, which means that participants, the study doctor, and the study staff will not know if participants are getting birtamimab, but the study doctor can find out if there is an emergency or if it is necessary to know. This study design is the best way to measure the effect of birtamimab.
This study will be conducted at approximately 80 study sites in North America, Europe, Israel, South America, Asia, and Australia. Overall, approximately 150 participants are expected to enroll in this study.REC name
South West - Central Bristol Research Ethics Committee
REC reference
21/SW/0067
Date of REC Opinion
23 Jun 2021
REC opinion
Further Information Favourable Opinion