NEOD001 versus placebo in AL Amyloidosis with cardiac dysfunction
Research type
Research Study
Full title
NEOD001-201: A Phase 2b, Randomized, Double-blind, Placebo-controlled Study of NEOD001 in Previously Treated Subjects with Light Chain (AL) Amyloidosis who have Persistent Cardiac Dysfunction
IRAS ID
195272
Contact name
Ashutosh Wechalekar
Contact email
Sponsor organisation
Prothena Therapeutics Limited
Eudract number
2015-004318-14
Duration of Study in the UK
2 years, 2 months, 29 days
Research summary
This is an international, Phase 2, randomized, double-blind, study to investigate the efficacy and safety of NEOD001 versus placebo in adults with Light Chain (AL) amyloidosis who had a response to previous treatment (e.g.., chemotherapy, autologous stem cell transplant [ASCT]) for their disease and have persistent cardiac dysfunction.
100 people with Light Chain (AL) amyloidosis and persistent cardiac dysfunction will participate in the study.
Study patients will be asked to visit their study doctor regularly for up to 14 months, consisting of a Screening Phase (1 month), Treatment Phase (monthly for up to 12 months), and an end of study visit (30 days after the last dose of study drug).
Eligible patients will be randomly assigned to receive either NEOD001 or placebo (normal saline) administered once monthly as an intravenous infusion over 1-2 hours.
A number of assessments and procedures will be performed during the study including physical examinations, blood tests, study drug/placebo administration, assessment questionnaires, urine sample assessments, echocardiograms (ECHO) and electrocardiograms (ECG). These assessments are performed as part of the evaluation to determine if the study drug is effective and safe.
The study will be run at up to 30 sites worldwide, and approximately 6 centres in the UK and is sponsored by Prothena Therapeutics Limited.
REC name
London - Riverside Research Ethics Committee
REC reference
16/LO/0021
Date of REC Opinion
5 Feb 2016
REC opinion
Further Information Favourable Opinion