CATALYST
Research type
Research Study
Full title
A single arm open labeled multicentre phase 1b dose escalation study of carfilzomib taken in combination with Thalidomide and Dexamethasone in relapsed AL amyloidosis (CATALYST trial).
IRAS ID
183136
Contact name
Ashutosh Wechalekar
Contact email
Sponsor organisation
University College London
Eudract number
2015-000954-40
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 6 months, days
Research summary
Patients with AL amyloidosis have an abnormal line of cells (called plasma or B cells) which are usually in the bone marrow, and which produce the amyloid forming protein. AL amyloidosis occurs in about 10 per million population, and the frequency increases with age. Most patients with AL amyloidosis are aged over 45 years, but it occasionally occurs in young adults. The amyloid deposits tend to be laid down in many parts of the body, although usually one or two organs are predominantly affected
The treatment of systemic AL amyloidosis has evolved to a risk adapted approach based on the end organ damage, particularly cardiac involvement, and the functional status of the patient. The majority of patients with AL amyloidosis will generally be treated with combination chemotherapy.
The purpose of this study is to determine the maximum tolerated dose (MTD) and recommended dose (RD) of carfilzomib when used in combination with thalidomide and dexamethasone in patients with relapsed or refractory AL amyloidosis. Patients will receive treatment with carfilzomib in escalating dose cohorts along with a fixed dose of dexamethasone and thalidomide. Safety and tolerability of KTD will be further assessed at the RD of Carfilzomib identified in the dose escalation phase.
REC name
London - Brent Research Ethics Committee
REC reference
16/LO/0087
Date of REC Opinion
29 Mar 2016
REC opinion
Further Information Favourable Opinion