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Trial registered on ANZCTR


Registration number
ACTRN12605000795695
Ethics application status
Approved
Date submitted
9/12/2005
Date registered
16/12/2005
Date last updated
8/08/2017
Type of registration
Prospectively registered

Titles & IDs
Public title
A Phase II Study of Risk-Adapted IV Melphalan in Patients with AL (Primary systemic) Amyloidosis
Scientific title
Impact of a risk-adapted IV Melphalan schedule on the six month serum free light chain response in patients with AL (Primary systemic) amyloidosis
Secondary ID [1] 227 0
Australasian Leukaemia and Lymphoma Group (ALLG): ALLG MM8
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
AL amyloidosis 954 0
Condition category
Condition code
Other 1023 1023 0 0
Conditions of unknown or disputed aetiology (such as chronic fatigue syndrome/myalgic encephalomyelitis)

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
IV Melphalan will be dosed according to a risk-adapted schedule. Those who can safely receive high-dose IV Melphalan (=Transplant candidates) must have minimal cardiac disease (BNP<300, BP >90, no CCF/arrhythmias, O2 sats >94%, EF >40%), age < 65, ECOG performance status < 2, organs involved < 2 and bilirubin < 30mmol/L). Patients unsuitable for high-dose Melphalan (Non-transplant candidates) will receive intermediate-dose IV Melphalan and dexamethasone.Transplant candidates:Peripheral blood stem cell collection with filgrastim alone (10µg/kg)Autologous stem cell transplantation with melphalan (140mg/m2 if CrCl < 50 mls/min or age > 60 yrs or ECOG performance status=2, otherwise 200mg/m2). Non-transplant candidates:Melphalan 20mg/m2 iv + dexamethasone 40mg d1-4 every 4 weeks for 2 cycles beyond free light chain plateau
Intervention code [1] 794 0
Treatment: Drugs
Comparator / control treatment
Uncontrolled study- no control
Control group
Uncontrolled

Outcomes
Primary outcome [1] 1372 0
To determine the proprotion of patients who achieve a 50% reduction serum free lighty chains at 6 months following therapy with intravenous melphalan
Timepoint [1] 1372 0
6 months
Secondary outcome [1] 2430 0
To determine the treatment related mortality as defined by death within 100 days of completion of therapy.
Timepoint [1] 2430 0
100 days following completion of therapy
Secondary outcome [2] 2431 0
To determine the duration of serum free light chain response.
Timepoint [2] 2431 0
time from first achievement of a 50% reduction in the monoclonal light chain until the time of free light chain increase of 50% from nadir and an increase to > 100mg/L
Secondary outcome [3] 2432 0
To determine the organ response rates as defined by disease specific criteria.
Timepoint [3] 2432 0
2 years following start of therapy
Secondary outcome [4] 2433 0
To determine overall survival.
Timepoint [4] 2433 0
Study closeout date

Eligibility
Key inclusion criteria
1. Confirmed diagnosis of AL amyloidosis (must have biopsy-proven amyloidosis and identification of a monoclonal light chain in the serum, urine or in a bone marrow where a monoclonal light chain is identified by immunohistochemical staining or flow cytometry). Positive immunohistochemistry of amyloid deposits or exclusion of hereditary mutations is required if there is uncertainty as to the type of amyloid. 2. Evidence of cardiac, renal, liver or neurologic involvement or other symptomatic organ involvement (asymptomatic gastrointestinal or marrow involvement alone are not indications for study inclusion). 3. ECOG performance status 0 to 3 inclusive. 4. Written informed consent prior to study registration.
Minimum age
18 Years
Maximum age
75 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Clinically overt multiple myeloma with > 30% plasma cells in the bone marrow or the presence of lytic bone lesions or hypercalaemia2. B-cell lymphoproliferative disease3. Prior intravenous melphalan4. Therapy to reduce plasma cell dyscrasia within the previous four weeksNote: Prior stem cell mobilisation with G-CSF is not an exclusion criteria5. Absolute neutrophil count < 1.5 x 109 /L and platelet count < 100 x 109/L6. Concurrent or previous malignancy except adequately treated non-melanoma skin cancer, curatively treated in-situ cancer of the cervix or other solid tumors treated for cure with no evidence of disease for at least 2 years7. Known HIV positivity8. Likely inability of the patient to comply with treatment assessments9. Pregnancy and lactation. Adults of reproductive potential must agree to use an effective method of birth control during treatment and for at least 3 months thereafter.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint(s)
Efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Funding source category [1] 1125 0
Charities/Societies/Foundations
Name [1] 1125 0
Leukaemia Foundation of Queensland
Address [1] 1125 0
P O Box 6577, Cairns, QLD 4870
Country [1] 1125 0
Australia
Funding source category [2] 1126 0
Commercial sector/Industry
Name [2] 1126 0
Amgen Australia
Address [2] 1126 0
Amgen Australia Pty Ltd
Level 1, 801 Glenferrie Road
Hawthorn VIC 3122
Country [2] 1126 0
Australia
Primary sponsor type
Other Collaborative groups
Name
Australasian Leukaemia and Lymphoma Group
Address
Level 2/ 10 St Andrews Place
East Melbourne, Australia, 3002
Country
Australia
Secondary sponsor category [1] 983 0
None
Name [1] 983 0
n/a
Address [1] 983 0
Country [1] 983 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 298364 0
Princess Alexandra Hospital
Ethics committee address [1] 298364 0
Woolloongabba, QLD 4102
Ethics committee country [1] 298364 0
Australia
Date submitted for ethics approval [1] 298364 0
Approval date [1] 298364 0
31/05/2006
Ethics approval number [1] 298364 0

Summary
Brief summary
Trial website
Trial related presentations / publications
Mollee P, Tiley C, Cunningham I, Moore J, Prince HM, Cannell P, Gibbons S, Tate J, Paul S, Mar Fan H, Gill DS. A phase II study of risk-adapted intravenous melphalan in patients with AL amyloidosis. British journal of haematology. 2012;157(6):766-769
Public notes

Contacts
Principal investigator
Name 35690 0
Address 35690 0
Country 35690 0
Phone 35690 0
Fax 35690 0
Email 35690 0
Contact person for public queries
Name 9983 0
A/Prof. Sanjoy Paul
Address 9983 0
Queensland Clinical Trials & Biostatistics Centre
Princess Alexandra Hospital, Ipswich Road, Woolloongabba QLD 4102
Country 9983 0
Australia
Phone 9983 0
+61 7 3176 5809
Fax 9983 0
Email 9983 0
s.paul@sph.uq.edu.au
Contact person for scientific queries
Name 911 0
Dr Peter Mollee
Address 911 0
Haematology Department
Princess Alexandra Hospital
Ipswich Road
Woollongabba QLD 4102
Country 911 0
Australia
Phone 911 0
+61 7 32406739
Fax 911 0
+61 7 32407042
Email 911 0
peter.mollee@health.qld.gov.au

No information has been provided regarding IPD availability
Summary results
Have study results been published in a peer-reviewed journal?
Other publications
Have study results been made publicly available in another format?
Results – basic reporting
Results – plain English summary