Amnion cell derivatives for fistulising perianal Crohn’s disease
A human pilot study evaluating the safety of locally administered derivatives from allogeneic human amnion epithelial cells for the treatment of complex refractory perianal fistulising Crohn’s disease
Monash Health
15 participants
Aug 1, 2024
Interventional
Conditions
Summary
Complex perianal Crohn's fistulas are common with poor healing rates and high relapse rates despite long-term immune medications and repeated surgical procedures. Local fistula injection of stem cells derived from fat tissue is a new effective treatment.. We have completed a Phase I study using local injection of amniotic epithelial cells from the placenta in 10 adults with refractory complex Crohn's and reported the treatment as safe, well-tolerated and there was fistula improvement in 8 of 10 patients. In this study, we are using stem cell products produced from the amniotic cells instead as they also have medicinal properties. Participants will receive one or two injections of stem cell products into the fistula during examination under anaesthesia and undergo assessment using routine colonoscopy, blood tests, stool tests and MRI scans. We hypothesise that these stem cell products will be safe, well-tolerated and feasible to inject.
Eligibility
Inclusion Criteria7
- Adult participant
- Non-active or mildly active luminal Crohn's for over 6 months defined by a Crohn's Disease Activity Index (CDAI) less than 220
- Presence of complex perianal fistulas as classified by the American Gastroenterological Association (AGA)
- More than 1 of the following: high intersphincteric, high trans-sphincteric, extra-sphincteric, supra-sphincteric, more than 2 external openings or associated collections
- Fistulas with a maximum of 2 internal and 3 external openings
- Active fistula defined as drainage and symptoms (PDAI score > 4)
- Refractory to more than 1 treatment modalities such as antibiotics or immunotherapies. Definition of lack of response will assessed as per the Perianal Disease Activity Index (PDAI).
Exclusion Criteria6
- Rectal or anal stenosis
- Active severe proctitis (presence of superficial or deep ulcers)
- Abscess or collection more than 2 cm size that were not properly drained
- Corticosteroid use within the previous 4 weeks
- Inability to tolerate or comply with trial requirements
- Current pregnancy
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Interventions
This intervention requires one or two injections (six to eight weeks apart) of derivatives from 40 million human amnion epithelial cells per fistula into perianal fistulas. The human amnion epithelial cells were obtained from the placentas of healthy consenting donors delivering at-term healthy babies at Monash Health obtained under Good-Manufacturing Practice-like conditions for clinical trial use. After the initial treatment injection, if fistula healing is not achieved by week 6 (from intervention) based on clinical examination, then a second dose of stem cell derivative will be injected. For all injections, the dosage administered is 6 ml of derivative solution per fistula (up to a maximum of 3 fistulas per patient i.e. 18 ml solution) which will be injected along the fistula tract. These injections will be undertaken during an examination under anaesthesia surgical procedure performed by a specialist colorectal surgeon in an operating theatre at our quaternary Australian hospital and take approximately 1 hour. Prior to fistula injection, all setons must be removed and the internal fistula opening closed with sutures to allow for healing. There anaesthesia administered will be a general anaesthesia which is routine for perianal fistula examination under anaesthesia. The procedure will be recorded in a procedural report.
Locations(1)
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ACTRN12624000778583