Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12609000886280
Ethics application status
Not yet submitted
Date submitted
27/09/2009
Date registered
12/10/2009
Date last updated
28/09/2018
Type of registration
Prospectively registered

Titles & IDs
Public title
An observational case series of the use of anti-Immunoglobulin E antibody for the management of treatment-resistant systemic mastocytosis.
Scientific title
An observational case series of the use of anti-Immunoglobulin E antibody for the management of treatment-resistant systemic mastocytosis.
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Mastocytosis 251924 0
Condition category
Condition code
Inflammatory and Immune System 252109 252109 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Anti-immunoglobulin E antibody will be administered to participants as a subcutaneous injection, at a dose of 375mg (which may be altered during the course of treatment, depending on individual patient response), every fortnight, for a total of 24 weeks, equivalent to twelve doses.
Intervention code [1] 241347 0
Treatment: Drugs
Comparator / control treatment
No comparator or control
Control group
Uncontrolled

Outcomes
Primary outcome [1] 252989 0
The primary outcome of this trial will be the effect of treatment with anti-immunoglobulin E antibody on the symptoms of mastocytosis. Symptoms of mastocytosis include a flaring rash, anaphylaxis (a severe life-threatening episode that resembles severe allergic reaction), nausea, vomiting, diarrhoea and headaches. This will be assessed using a variety of data collection tools including questionnaires for the patients to complete on a regular basis, photographic doumentation of the rash before and after treatment, assessment of symptoms and rash by a doctor, and independent assessment of rash by a dermatologist. Blood tests measuring serum tryptase (a marker of mastocytosis activity) will also be collected several times during the trial.
Timepoint [1] 252989 0
The primary outcome will be assessed at multiple time points - on a fortnightly basis during the 24 weeks of treatment, at the end of 24 weeks of treatment, and every month after treatment has been completed for 6 months.
Secondary outcome [1] 257756 0
The secondary outcome of this trial is physiological evidence of reduction of mast cell burden (mast cells being the cells that are increased in mastocytosis and are responsible for the symptoms). Mast cell burden is assessed on bone marrow and a blood test (tryptase). A bone marrow is done as part of standard care in mastocytosis and so this trial will collect the results of this test, done any time in the 12 months leading up to the start of the trial. A second bone marrow will be repeated 6 months after therapy has been completed. Bone marrow assessment will follow published guidelines for mastocytosis. Blood levels of Immunoglobulin E will also be performed to assess the effect of treatment.
Timepoint [1] 257756 0
Again, this secondary outcome will be assessed by blood test (tryptase and total Immunogloblin E) at several points in the trial - at the end of treatment and 6 months after treatment.
Bone marrow will only be performed twice for trial purposes.

Eligibility
Key inclusion criteria
Adults with symptomatic systemic mastocytosis that is resistant to other conventional therapies
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Associated haematological clonal non-mast cell lineage disease or mast cell leukaemia;
Pregnancy.

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)
Identification of patients with suitable diagnosis via outpatients clinic
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Not randomised
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 4
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
1/11/2009
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
15
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Funding source category [1] 243803 0
Self funded/Unfunded
Name [1] 243803 0
Department of Clinical Immunology and Allergy
Country [1] 243803 0
Australia
Primary sponsor type
Individual
Name
Dr Karl Bleasel
Address
Department of Clinical Immunology and Allergy
Royal Melbourne Hospital
Parkville 3050
Country
Australia
Secondary sponsor category [1] 237155 0
Hospital
Name [1] 237155 0
The Royal Melbourne Hospital
Address [1] 237155 0
Grattan Street
Parkville VIC 3050
Country [1] 237155 0
Australia

Ethics approval
Ethics application status
Not yet submitted
Ethics committee name [1] 243933 0
Ethics committee address [1] 243933 0
Ethics committee country [1] 243933 0
Date submitted for ethics approval [1] 243933 0
02/10/2009
Approval date [1] 243933 0
Ethics approval number [1] 243933 0

Summary
Brief summary
The primary purpose of the study is to observe the effect of anti-immunoglobulin E antibody in the treatment of a small number of people with symptomatic mastocytosis.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 30333 0
Address 30333 0
Country 30333 0
Phone 30333 0
Fax 30333 0
Email 30333 0
Contact person for public queries
Name 13580 0
Dr Karl Bleasel
Address 13580 0
Department of Clinical Immunology and Allergy
The Royal Melbourne Hospital
Parkville VIC 3050
Country 13580 0
Australia
Phone 13580 0
+61 3 9342 7191
Fax 13580 0
+61 3 9349 3199
Email 13580 0
[email protected]
Contact person for scientific queries
Name 4508 0
Dr Karl Bleasel
Address 4508 0
Department of Clinical Immunology and Allergy
The Royal Melbourne Hospital
Parkville VIC 3050
Country 4508 0
Australia
Phone 4508 0
+61 3 9342 7191
Fax 4508 0
Email 4508 0
[email protected]

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.