Clinical trial NCT01090817: An Australian Study of Mesenchymal Stromal Cells for Crohn's Disease
Official title: A Multicentre Australian Phase 2 Study to Evaluate Safety and Efficacy of Mesenchymal Stromal Cells for Treating Biologic Refractory Crohn's Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Crohn Disease
Keywords: mesenchymal stromal cells
ileitis
colitis
Lead sponsor: Royal Perth Hospital
Sponsor class: OTHER
Collaborators: name: The Queen Elizabeth Hospital
name: Concord Hospital
name: Sir Charles Gairdner Hospital
name: The Alfred
First posted: 2010-03-23
Enrollment: 21
Sex: ALL
Minimum age (years): 18
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


Eligibility criteria: 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

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.

Primary outcomes:
measure: Clinical response to MSC: Reduction of Crohn's disease Activity score by 100 points or more at six weeks post start of therapy
timeFrame: Six weeks
Secondary outcomes:
measure: Incidence of infusional toxicity
timeFrame: Six weeks
measure: Induction of remission
timeFrame: Six weeks
measure: Improved quality of life
timeFrame: Six weeks
measure: Endoscopic improvement.
timeFrame: Six weeks
Other outcomes:
Locations:
city: Perth
country: Australia
facility: Department of Gastroenterology and Hepatology, Royal Perth Hospital
state: Western Australia
Study officials:
affiliation: Royal Perth Hospital
name: Geoff Forbes, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Forbes GM, Sturm MJ, Leong RW, Sparrow MP, Segarajasingam D, Cummins AG, Phillips M, Herrmann RP. A phase 2 study of allogeneic mesenchymal stromal cells for luminal Crohn's disease refractory to biologic therapy. Clin Gastroenterol Hepatol. 2014 Jan;12(1):64-71. doi: 10.1016/j.cgh.2013.06.021. Epub 2013 Jul 19.