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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT01090817




Registration number
NCT01090817
Ethics application status
Date submitted
22/03/2010
Date registered
23/03/2010
Date last updated
9/06/2015

Titles & IDs
Public title
An Australian Study of Mesenchymal Stromal Cells for Crohn's Disease
Scientific title
A Multicentre Australian Phase 2 Study to Evaluate Safety and Efficacy of Mesenchymal Stromal Cells for Treating Biologic Refractory Crohn's Disease
Secondary ID [1] 0 0
CTN2010/0098
Secondary ID [2] 0 0
EC2009/123
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Crohn Disease 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Inflammatory bowel disease
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders
Oral and Gastrointestinal 0 0 0 0
Crohn's disease

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Mesenchymal stromal cells (MSC) for infusion

Experimental: Mesenchymal stromal cells - Mesenchymal stromal cells administered weekly for 4 weeks


Treatment: Drugs: Mesenchymal stromal cells (MSC) for infusion
MSC 2X10E6/kg recipient weight are infused over 15 minutes intravenously weekly for 4 weeks

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

Outcomes
Primary outcome [1] 0 0
Clinical response to MSC: Reduction of Crohn's disease Activity score by 100 points or more at six weeks post start of therapy - Colonoscopy and biopsy as well as clinical parameters used for the Crohn's disease acivity (www.ibdaustralia.org/cdai/) will be undertaken at screening pre-therapy and at 6 weeks after start of therapy.
Timepoint [1] 0 0
Six weeks
Secondary outcome [1] 0 0
Incidence of infusional toxicity - Subjects will receive mesenchymal stromal cell therapy, 2X10E6/kg weekly by IV infusion for 4 weeks and will be assessed for 4 hours post infusion
Timepoint [1] 0 0
Six weeks
Secondary outcome [2] 0 0
Induction of remission - Crohn's disease activity index assessed as below 150
Timepoint [2] 0 0
Six weeks
Secondary outcome [3] 0 0
Improved quality of life - Increase in IBDQ and SF-36 scores measured at six weeks
Timepoint [3] 0 0
Six weeks
Secondary outcome [4] 0 0
Endoscopic improvement. - Crohn's disease endoscopic improvement score will be measured at repeat endoscopy six weeks after start of treatment
Timepoint [4] 0 0
Six weeks

Eligibility
Key inclusion criteria
- Colonic or small bowel Crohn's disease based on endoscopic appearances and histology

- Refractory to induction with infliximab or adalimumab; or have lost response to these
agents; or have had side effects precluding their further use

- Where there has been loss of response to one of these agents, the other must be tried
before being eligible

- Crohn's disease activity score (CDAI) 250 or more.

- C-reactive protein >10mg/L

- Surgery must have been offered to the subject (if appropriate) and declined

- Signed informed consent
Minimum age
18 Years
Maximum age
55 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Active sepsis, perforating disease. Coexistent perianal fistulous disease is
permitted, providing no co-existent infection within previous 4 weeks

- Chronic stricturing disease in isolation

- Coexistent CMV disease

- Prior history of malignancy

- Pregnant or unwilling to practice contraceptive therapy or breast feeding females

- Last biologic therapy must be greater than 4 weeks prior, must be on stable
corticosteroid dose for 14 days prior, during therapy and for 14 days after therapy,
must be on stable immunomodulator dose (eg, azathioprine) for 14 days prior, during
therapy and for 14 days after.

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)
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)
WA
Recruitment hospital [1] 0 0
Department of Gastroenterology and Hepatology, Royal Perth Hospital - Perth
Recruitment postcode(s) [1] 0 0
6000 - Perth

Funding & Sponsors
Primary sponsor type
Other
Name
R.P.Herrmann
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
The Queen Elizabeth Hospital
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
Concord Hospital
Address [2] 0 0
Country [2] 0 0
Other collaborator category [3] 0 0
Other
Name [3] 0 0
Sir Charles Gairdner Hospital
Address [3] 0 0
Country [3] 0 0
Other collaborator category [4] 0 0
Other
Name [4] 0 0
The Alfred
Address [4] 0 0
Country [4] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Despite the advent of newer biologic therapies such as infliximab for Crohn's disease, a form
of autoimmune inflammatory bowel disease, a proportion of patients are refractory to such
therapy and require surgery. The hypothesis is that mesenchymal stromal cell therapy using
third party human cultured cells will be safe and effective
Trial website
https://clinicaltrials.gov/show/NCT01090817
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Geoff Forbes, MD
Address 0 0
Royal Perth Hospital
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
Other publications