Trial Review

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial registered on ANZCTR


Registration number
ACTRN12610000068066
Ethics application status
Approved
Date submitted
13/01/2010
Date registered
20/01/2010
Date last updated
10/07/2012
Type of registration
Retrospectively registered

Titles & IDs
Public title
Mesenchymal Stromal Cells to Treat Steroid-refractory Graft Versus Host Disease after Bone Marrow Transplantation
Scientific title
A phase 1 study to assess the safety of mesenchymal stromal cells to treat steroid-refractory graft versus host disease after allogeneic haemopoietic stem cell tranplantation.
Secondary ID [1] 1286 0
Nil
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Graft Versus Host Disease 256537 0
Condition category
Condition code
Inflammatory and Immune System 256693 256693 0 0
Other inflammatory or immune system disorders
Blood 256710 256710 0 0
Haematological diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Mesenchymal stromal cells 2 X 10E6/kg administered intrvenously over 15 minutes weekly for two weeks
Intervention code [1] 255800 0
Other interventions
Intervention code [2] 255814 0
Treatment: Other
Comparator / control treatment
None
Control group
Uncontrolled

Outcomes
Primary outcome [1] 257583 0
Safety assessed clinically and by blood tests
weekly for 4 weeks, then monthly for 12 months after therapy
Timepoint [1] 257583 0
12 months after therapy finished, as assessed weekly for 4 weeks after therapy finished, then monthly to 12 months
Secondary outcome [1] 262836 0
Overall Survival as measured by actuarial survival from data linkage to medical records
Timepoint [1] 262836 0
12 months following completion of mesenchymal stromal cell therapy

Eligibility
Key inclusion criteria
Age 18-60
Steroid-refractory graft versus host disease post allogeneic haemopoietic stem cell transplant
Informed consent given
Minimum age
18 Years
Maximum age
60 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
No informed consent

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)
Discussion of risks, benefits, explanation of patient information document.
No allocation concealment, open label treatment
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
N/A
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Mesenchymal cells may be Human Leucocyte Antigen (HLA) identical, haploidentical or third party
Phase
Phase 1
Type of endpoint/s
Safety
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
1/12/2007
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
20
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Funding source category [1] 256287 0
Charities/Societies/Foundations
Name [1] 256287 0
Ray and Bill Dobney Trust
Country [1] 256287 0
Australia
Primary sponsor type
Individual
Name
R.P.Herrmann
Address
Royal Perth Hospital
Wellington St
Perth 6000
Western Auistralia
Country
Australia
Secondary sponsor category [1] 251612 0
None
Name [1] 251612 0
None
Address [1] 251612 0
N/A
Country [1] 251612 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 258381 0
ethics committee, royal perth hospital
Ethics committee address [1] 258381 0
Royal Perth Hospital
wellington St
Perth
Western Australia 6000
Ethics committee country [1] 258381 0
Australia
Date submitted for ethics approval [1] 258381 0
Approval date [1] 258381 0
11/04/2007
Ethics approval number [1] 258381 0
2007/090

Summary
Brief summary
Study hypothesis is that mesenchymal cells are safe and will be effective in treating steroid refractory graft versus host disease. Trial subjects will receive 2 X 10 E6/kg mesenchymal cells weekly for 2 weeks, are then reassessed weekly. If inadequate response may receive two further doses at weekly intervals. May be retreated if they subsequently relapse
Trial website
Trial related presentations / publications
Mesenchymal Stromal Cells: A Transplant Tale. Australian Tissue Banking Forum May 2009, programme page 5.
Public notes

Contacts
Principal investigator
Name 30693 0
Address 30693 0
Country 30693 0
Phone 30693 0
Fax 30693 0
Email 30693 0
Contact person for public queries
Name 13940 0
Prof R.P.Herrmann
Address 13940 0
Cell and Tissue Therapies W.A.
C/- Haematology Dept
Royal Perth Hospital
Wellington St
Perth 6000
Western Australia
Country 13940 0
Australia
Phone 13940 0
+61 892243967
Fax 13940 0
+61 892243449
Email 13940 0
[email protected]
Contact person for scientific queries
Name 4868 0
r.p.herrmann
Address 4868 0
Cell and Tissue Therapies W.A.
C/- Haematology Dept
Royal Perth Hospital
Wellington St
Perth 6000
Western Australia
Country 4868 0
Australia
Phone 4868 0
+61 892243967
Fax 4868 0
+61892243449
Email 4868 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.