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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT01631708




Registration number
NCT01631708
Ethics application status
Date submitted
24/06/2012
Date registered
29/06/2012
Date last updated
27/09/2016

Titles & IDs
Public title
Mi-iron - Moderately Increased Iron - is Reducing Iron Overload Necessary?
Scientific title
Mi-Iron - Moderately Increased Iron - is Reducing Iron Overload Necessary?
Secondary ID [1] 0 0
04609
Universal Trial Number (UTN)
Trial acronym
Mi-iron
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hereditary Haemochromatosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Diet and Nutrition 0 0 0 0
Other diet and nutrition disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Surgery - Erythrocytapheresis
Treatment: Surgery - Plasmapheresis

Active Comparator: Erythrocytapheresis - Erythrocytapheresis is a procedure whereby whole blood is drawn from an individual and all elements except erythrocytes are returned to the donor. An automated filtration process removes the erythrocytes.
Those in arm 1 will have third weekly erythrocytapheresis until their SF is returned to the normal range.

Sham Comparator: Plasmapheresis - In plasmapheresis, the plasma is removed by the automated filtration process whilst other blood elements including erythrocytes are returned to the subject.
Those in arm 2 will have plasmapheresis with the approximate number of episodes of apheresis that would be required to reduce their SF to normal had they been randomised to the true treatment arm.


Treatment: Surgery: Erythrocytapheresis
To achieve a blinded randomised trial, apheresis treatment will be used. Those in arm 1 will have erythrocytapheresis reducing iron levels and those in arm 2 will have plasmapheresis and their iron levels will not be reduced.
An apheresis machine will be used to remove red blood cells only from the erythrocytapheresis group. Subjects will have third weekly treatments until SF levels are reduced to ~100 ug/L in accordance with current guidelines.

Treatment: Surgery: Plasmapheresis
An apheresis machine will be used to remove blood plasma only from the plasmapheresis group. Those in arm 2 will have the approximate number of episodes of apheresis that would be required to reduce their SF to normal had they been randomised to the true treatment arm. Those in the sham arm will be offered to have venesection at their choice of venue or to have their SF normalised by erythrocytapheresis after the initial blinded part of the study. This will be done because it will not be known for some time if there is benefit from normalisation of SF and therefore leaving people with elevated SF that may be harmful.

Intervention code [1] 0 0
Treatment: Surgery
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Fatigue
Timepoint [1] 0 0
Clinically and statistically significant change in measures taken at baseline and at the end of treatment will be compared. Patients will have approximately 6 third weekly treatments however this will vary depending on initial SF.
Secondary outcome [1] 0 0
Change in markers of liver fibrosis
Timepoint [1] 0 0
Clinically and statistically significant change in measures taken at baseline and at the end of treatment will be compared. Patients will have on average 6 third weekly treatments (15 weeks).
Secondary outcome [2] 0 0
Quality of life
Timepoint [2] 0 0
Clinically and statistically significant change in measures taken at baseline and at the end of treatment will be compared. Patients will have on average 6 third weekly treatments (15 weeks).
Secondary outcome [3] 0 0
Depression and Anxiety
Timepoint [3] 0 0
Clinically and statistically significant change in measures taken at baseline and at the end of treatment will be compared. Patients will have on average 6 third weekly treatments (15 weeks).
Secondary outcome [4] 0 0
Arthritis
Timepoint [4] 0 0
Clinically and statistically significant change in measures taken at baseline and at the end of treatment will be compared. Patients will have on average 6 third weekly treatments (15 weeks).
Secondary outcome [5] 0 0
Markers of oxidative stress
Timepoint [5] 0 0
Clinically and statistically significant change in measures taken at baseline and at the end of treatment will be compared. Patients will have on average 6 third weekly treatments (15 weeks).

Eligibility
Key inclusion criteria
1. HFE C282Y homozygous.

2. Aged 18 - 70 years .

3. SF above the upper limit of the normal range of 300µg/L but less than 1000µg/L with a
currently or previously raised TS (>greater than the upper limit of normal for the
testing laboratory).
Minimum age
18 Years
Maximum age
70 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
1. HH due to genotypes other than HFE C282Y homozygosity.

2. Normal SF, SF > 1000µg/L.

3. Other major risk factor(s) for liver toxicity or other significant co-morbidities
including positivity for hepatitis B or C, excess alcohol consumption (> 60g/day in
males and 40g/day in females) or body mass index > 35.

4. Has had venesection therapy for HH in the last two years.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled 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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


Intervention assignment
Single group
Other design features
Phase
Not Applicable
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
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)
QLD,VIC
Recruitment hospital [1] 0 0
Royal Brisbane and Woman's Hospital - Brisbane
Recruitment hospital [2] 0 0
Austin Health - Melbourne
Recruitment hospital [3] 0 0
Royal Melbourne Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
4072 - Brisbane
Recruitment postcode(s) [2] 0 0
3081 - Melbourne
Recruitment postcode(s) [3] 0 0
- Melbourne

Funding & Sponsors
Primary sponsor type
Other
Name
Murdoch Childrens Research Institute
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Austin Health
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
Royal Brisbane and Women's Hospital
Address [2] 0 0
Country [2] 0 0
Other collaborator category [3] 0 0
Other
Name [3] 0 0
Fremantle Hospital and Health Service
Address [3] 0 0
Country [3] 0 0
Other collaborator category [4] 0 0
Other
Name [4] 0 0
Melbourne Health
Address [4] 0 0
Country [4] 0 0
Other collaborator category [5] 0 0
Other
Name [5] 0 0
The University of Queensland
Address [5] 0 0
Country [5] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Haemochromatosis is a preventable genetic iron overload disorder. Untreated, it can shorten
life due mainly to liver cirrhosis and cancer. It can be prevented by blood donation to
maintain normal iron levels. It is unclear, however, whether treatment is necessary when
individuals have moderate elevation of iron in the body. This research project will study the
effects of treatment in this group by assessing a number of scans, questionnaires and blood
tests in treated and untreated individuals.
Trial website
https://clinicaltrials.gov/ct2/show/NCT01631708
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Martin B Delatycki
Address 0 0
Austin Health/Murdoch Childrens Research Institute
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT01631708