Efficacy and safety of CAEL-101 in AL Amyloidosis stage IIIb patients
Research type
Research Study
Full title
A Phase 3, Double-Blind, Multicenter Study to Evaluate the Efficacy and Safety of CAEL-101 and Plasma Cell Dyscrasia Treatment Versus Placebo and Plasma Cell Dyscrasia Treatment in Plasma Cell Dyscrasia Treatment-Naïve Patients with Mayo Stage IIIb AL Amyloidosis.
IRAS ID
282333
Contact name
Ashutosh Wechalekar
Contact email
Eudract number
2019-004254-28
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
2 years, 0 months, 1 days
Research summary
Cardiac amyloidosis is a disorder caused by deposits of an abnormal protein (amyloid) in the heart tissue. These deposits make it hard for the heart to work properly.
The purpose of the study is to determine if CAEL-101 helps people with AL amyloidosis that has affected their hearts to live longer.
CAEL-101 is an unlicensed drug and its therapeutic benefit is not yet known. Participants will be informed of this both verbally and in the participant information sheet. In addition safety monitoring of all participants will be conducted throughout the study.
If the patient chooses to participate, they will be given CAEL-101 or a placebo (saltwater) at the clinic by IV infusion (drip into the blood vessel through a plastic tube and needle) every week for 4 weeks then every 2 weeks after that for at least 50 weeks. They will be given CAEL-101 or the placebo in addition to the anti-AL amyloidosis medicines.
Placebo
This study is a placebo-controlled study so a proportion of participants will receive no active ingredient other than the anti-AL amyloidosis medicines (standard care). This is necessary in order to provide a comparison for participants treated with the study medication. Participants will be advised of this in the participant information sheet. Participants will be reminded they are free to withdraw from the study at any time.REC name
South Central - Oxford C Research Ethics Committee
REC reference
20/SC/0354
Date of REC Opinion
30 Nov 2020
REC opinion
Further Information Favourable Opinion