A randomised, multi-centre feasibility trial in AL Amyloidosis, comparing CTD with SCT in patients with low risk of Treatment Related Mortality and CTD with Mel-Dex in patients in whom SCT would not be considered appropriate as first line therapy - UK Amyloidosis Treatment Trial (UKATT)
- Conditions
- The medical condition to be investigated is systemic AL Amyloidosis, a disorder of protein folding in which normally soluble proteins are deposited as abnormal, insoluble fibrils that progressively disrupt tissue structure and impair function.MedDRA version: 9.1 Level: LLT Classification code 10002022 Term: Amyloidosis
- Registration Number
- EUCTR2006-006395-37-GB
- Lead Sponsor
- Joint UCLH and UCL Biomedical Research Unit
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 48
Patients with the following characteristics are eligible for this study
•Aged 18 years or greater
•Newly diagnosed as having systemic AL amyloidosis who have:
-Diagnostic Congo red histology confirming amyloid deposits
-Immunohistochemical exclusion of AA and TTR amyloidosis, whenever doubt about the diagnosis exists, according to NAC standard practice
-Exclusion of genetic mutations associated with hereditary amyloidosis whenever doubt about the diagnosis exists, according to NAC current practice
-Underlying plasma cell dyscrasia that can be identified and monitored by Freelite serum free light chain assay as follows: absolute serum free light chain concentration = 50mg/l associated with an abnormal kappa / lambda ratio. Among patients with a creatinine clearance of <50mls/min, inclusion requires the kappa / lambda ratio to be either <0.26 or >2.0
-Amyloid related organ dysfunction or organ syndrome
•Capable of providing written, informed consent
•Estimated life expectancy of at least 6 months
•Prepared to use contraception in accordance with (and consent to) the Celgene approved process for Thalidomide Risk Management and Pregnancy Prevention or commit to absolute and continuous abstinence.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
Patients with the following characteristics are ineligible for this study
•Overt symptomatic multiple myeloma as evidenced by myeloma related end organ impaitment (ROTI) according to the international myeloma working group criteria 2003 but excluding any organ dysfunction caused by AL amyloidosis.
•Underlying IgM paraproteinaemia
•Amyloidosis of unknown or non AL type
•Localised AL amyloidosis (in which amyloid deposits are limited to a typical single organ, for example the bladder or larynx, in association with a clonal proliferative disorder within that organ
•Trivial or incidental AL amyloid deposits in the absence of a significant amyloid related organ syndrome (e.g., isolated carpal tunnel syndrome)
•Isolated soft tissue involvement
•Severe peripheral neuropathy causing significant functional impairment
•NYHA Class IV heart failure
•Liver involvement by amyloid causing bilirubin >1.5 times upper limit of normal
•Previous treatment for systemic AL amyloidosis
•Previous or concurrent active malignancies, except surgically removed basal cell carcinoma of the skin or other in situ carcinomas
•Pregnant, lactating or unwilling to use adequate contraception
•Intolerance / sensitivity to any of the study drugs
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method