Condition category
Nutritional, Metabolic, Endocrine
Date applied
27/11/2006
Date assigned
26/01/2007
Last edited
10/03/2015
Prospective/Retrospective
Prospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

Contact information

Type

Scientific

Primary contact

Dr Julian Gillmore

ORCID ID

Contact details

National Amyloidosis Centre
Royal Free & University College Medical School
Royal Free Hospital
London
NW3 2PF
United Kingdom
+44 (0)207 433 2726
j.gillmore@medsch.ucl.ac.uk

Additional identifiers

EudraCT number

ClinicalTrials.gov number

Protocol/serial number

BRD/06/055

Study information

Scientific title

A randomised, multi-centre trial to assess the feasibility of conducting a future phase III randomised trial in primary amyloidosis, comparing cyclophosphamide, thalidomide and dexamethasone with stem cell transplantation in patients with low risk of treatment related mortality and cyclophosphamide, thalidomide and dexamethasone with Mel-Dex in patients with high risk of treatment related mortality

Acronym

UKATT

Study hypothesis

This trial is intended to test the feasibility of a phase III study to address issues in patients with newly diagnosed primary (AL) amyloidosis at all stages of disease. The aim is to compare different chemotherapeutic regimens as an initial therapy with respect to rate of clonal response, safety and treatment related mortality and organ response.

In addition, quality of life before and after chemotherapy will be investigated to assess the validity of the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ C30) and Multiple Myeloma (MY20) questionnaire in this patient population.

Ethics approval

Not provided at time of registration – pending

Study design

Randomised multi-centre feasibility study

Primary study design

Interventional

Secondary study design

Randomised controlled trial

Trial setting

Hospitals

Trial type

Treatment

Patient information sheet

Not available in web format, please use the contact details below to request a patient information sheet

Condition

AL Amyloidosis

Intervention

Patients will enter one of two treatment pathways (high or low intensity) on the basis of their disease and will be randomised within each pathway to one of two chemotherapy regimens on a 1:1 basis.

Patients entering the high intensity pathway will be randomised to Stem Cell Transplantation (SCT) or Cyclophosphamide, Thalidomide, Dexamethasone (CTD) and those randomised to the low intensity pathway will be randomised to receive either CTD or Melphalan and Dexamethasone (Mel Dex).

Intervention type

Drug

Phase

Phase III

Drug names

Cyclophosphamide, thalidomide, dexamethasone and melphalan

Primary outcome measures

1. Clonal response
2. Toxicity and safety (including treatment related mortality)
3. Recruitment rate and feasibility

Secondary outcome measures

1. Acceptability of randomisations in each pathway
2. Quality of life questionnaire validity
3. Amyloidotic organ function

Overall trial start date

31/01/2007

Overall trial end date

31/01/2008

Reason abandoned

Eligibility

Participant inclusion criteria

1. Aged 18 years or greater
2. Newly diagnosed as having systemic AL amyloidosis who have:
2.1. Diagnostic Congo red histology confirming amyloid deposits
2.2. Immunohistochemical exclusion of Systemic (AA) and Transthyretin (TTR) amyloidosis
2.3. Exclusion of genetic mutations associated with hereditary amyloidosis whenever doubt about the diagnosis exists, according to Network Advisory Committee (NAC) current practice
2.4. 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 more than or equal to 100 mg/l associated with an abnormal kappa/lambda ratio
2.5. Amyloid-related organ dysfunction or organ syndrome
3. Capable of providing written, informed consent
4. Estimated life expectancy of at least six months
5. Prepared to use contraception in accordance with (and consent to) the Pharmion Risk Management Programme
6. Women of Child-Bearing Potential (WCBP) must agree to use TWO methods of contraception beginning two weeks prior to the start of thalidomide, while on thalidomide and four weeks after the last dose of thalidomide. The two methods of contraception must include one highly effective method and one additional effective (barrier) method, as outlined in the Pharmion Risk Management Programme
7. Male patients (including those who have had a vasectomy) must use condoms when engaging in heterosexual activity with WCBP while on thalidomide and four weeks after the last dose of thalidomide, as outlined in the Pharmion Risk Management Programme

Participant type

Patient

Age group

Adult

Gender

Both

Target number of participants

48

Participant exclusion criteria

1. Overt symptomatic multiple myeloma
2. Bone marrow plasmacytosis more than 10%
3. Underlying Immunoglobulin M (IgM) paraproteinaemia
4. Amyloidosis of unknown or non AL type
5. 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)
6. Trivial or incidental AL amyloid deposits in the absence of a significant amyloid related organ syndrome (e.g., isolated carpal tunnel syndrome)
7. Isolated soft tissue involvement
8. Severe peripheral neuropathy causing significant functional impairment
9. New York Heart Association (NYHA) class IV heart failure
10. Liver involvement by amyloid causing bilirubin more than 1.5 times upper limit of normal
11. Previous treatment for systemic AL amyloidosis
12. Previous or concurrent active malignancies, except surgically removed basal cell carcinoma of the skin or other in situ carcinomas
13. Pregnant, lactating or unwilling to use adequate contraception
14. Intolerance/sensitivity to any of the study drugs

Recruitment start date

31/01/2007

Recruitment end date

31/01/2008

Locations

Countries of recruitment

United Kingdom

Trial participating centre

National Amyloidosis Centre
London
NW3 2PF
United Kingdom

Sponsor information

Organisation

University College London (UK)

Sponsor details

Joint UCLH and UCL Biomedical Research Unit
Ground Floor
Rosenheim Wing
25 Grafton Way
London
WC1E 5DB
United Kingdom
+44 (0)845 155 5000
y.enever@medsch.ucl.ac.uk

Sponsor type

University/education

Website

http://www.ucl.ac.uk/biomed-r-d

Funders

Funder type

Research organisation

Funder name

Clinical Trials Advisory and Awards Committee (CTAAC) (UK) (ref: C23723/A7726)

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Results and Publications

Publication and dissemination plan

Not provided at time of registration

Intention to publish date

Participant level data

Not provided at time of registration

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes