None listed
Conditions
Brief summary
Effective treatments for complex perianal Crohn's disease remains an important unmet area of need in inflammatory bowel disease. Perianal fistulas affect over one third of Crohn's disease with severe cases requiring surgery and long-term immune-modifying treatments with side effects. Local injection of mesenchymal stem cells has been the only effective new treatment since the anti-tumour necrosis factor drugs over a decade ago. Fistula healing effects of stem cells have been shown last even at 1 year after treatment. Human amnion epithelial cells are a non-embryonic adult stem cell which is easily obtained from healthy human placentas (after delivery of a healthy baby) which would otherwise be discarded. Compared with mesenchymal stem cells, amnion stem cells are obtained much faster, produce much greater amounts and theoretically should be safer. This gives them a greater advantage. This study is a pilot study aiming to recruit 10 adults with complex perianal Crohn's fistulas who have previously failed one conventional treatment. Participants will receive one injection of amnion stem cells while undergoing examination under anaesthesia, which is a common procedure performed for those suffering this condition. The primary purpose of this study is to establish safety of the amnion stem cells with secondary goals to assess feasibility of treatment, effectiveness in fistula healing and improvement in quality of life. These will be measured at 24 weeks after treatment with stem cells. The total duration of the trial is approximately 28 weeks, however we would like to assess longer term with a final visit at 1 year. We hypothesise that human amnion epithelial cell therapy will be a safe, tolerable and effective treatment.
Interventions
Update (made prior to enrolment commencement)
This intervention requires a single local injection of human amnion epithelial cells into perianal fistula sites. This will occur during the examination under anaesthesia surgical procedure performed by a specialist colorectal surgeon in an operating theatre. The dose of the human amnion epithelial cells is 40 million cells per fistula, with up to 3 fistulas treated per participant (maximum 120 million cells). Prior to fistula injection, all perianal
Update (made prior to enrolment commencement) This intervention requires a single local injection of human amnion epithelial cells into perianal fistula sites. This will occur during the examination under anaesthesia surgical procedure performed by a specialist colorectal surgeon in an operating theatre. The dose of the human amnion epithelial cells is 40 million cells per fistula, with up to 3 fistulas treated per participant (maximum 120 million cells). Prior to fistula injection, all perianal setons must be removed and the internal opening of the fistula sutured closed to allow healing.
Sponsors
Monash Health
Study design
Allocation
Non-randomised trial
Primary purpose
Treatment
Eligibility
Sex/Gender
All
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
1. Adult participant 2. Non-active or mildly active luminal Crohn's for over 6 months (defined by CDAI less than 220) 3. Presence of complex perianal fistulas as classified by the American Gastroenterological Association (AGA) 4. More than 1 of the following: high intersphincteric, high trans-sphincteric, extra-sphincteric, supra-sphincteric, more than 2 external openings or associated collections 5. Fistulas with a maximum of 2 internal and 3 external openings 6. Fistulas draining for more than 6 weeks prior to inclusion 7. Refractory to more than 1 of the following treatments: antibiotics with no response after 1 month, immunomodulators with no response after 3 months, anti-TNF induction or maintenance. Definition of lack of response will assessed as per the Perianal Disease Activity Index (PDAI).
Exclusion criteria
1. Rectal or anal stenosis 2. Active severe proctitis (presence of superficial or deep ulcers) 3. Diverting stomas 4. Abscess or collection more than 2 cm size that were not properly drained 5. Corticosteroid use within the previous 4 weeks 6. Inability to tolerate or comply with trial requirements 7. Current pregnancy
Outcome results
None listed