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Trial registered on ANZCTR


Registration number
ACTRN12624000778583
Ethics application status
Approved
Date submitted
29/05/2024
Date registered
25/06/2024
Date last updated
19/10/2024
Date data sharing statement initially provided
25/06/2024
Type of registration
Prospectively registered

Titles & IDs
Public title
Amnion cell derivatives for fistulising perianal Crohn’s disease
Scientific title
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
Secondary ID [1] 312251 0
Nil known
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Crohn's perianal fistulas 333954 0
Condition category
Condition code
Oral and Gastrointestinal 330627 330627 0 0
Crohn's disease

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
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.
Intervention code [1] 328700 0
Treatment: Other
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 338373 0
Safety
Timepoint [1] 338373 0
The primary outcome timepoint is assessed at week 24 after administration of intervention.
Secondary outcome [1] 435667 0
Feasibility of route of administration
Timepoint [1] 435667 0
The secondary outcome timepoint is assessed at week 24 after administration of intervention.
Secondary outcome [2] 435668 0
Efficacy of treatment
Timepoint [2] 435668 0
The secondary outcome timepoint is assessed at week 24 after administration of intervention compared with baseline assessment of symptoms and MRI fistula morphology assessment.
Secondary outcome [3] 435669 0
Health-related quality of life
Timepoint [3] 435669 0
The secondary outcome timepoint is assessed at week 24 after administration of intervention.

Eligibility
Key inclusion criteria
1. Adult participant
2. Non-active or mildly active luminal Crohn's for over 6 months defined by a Crohn's Disease Activity Index (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. Active fistula defined as drainage and symptoms (PDAI score > 4)
7. 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).
Minimum age
18 Years
Maximum age
80 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Rectal or anal stenosis
2. Active severe proctitis (presence of superficial or deep ulcers)
3. Abscess or collection more than 2 cm size that were not properly drained
4. Corticosteroid use within the previous 4 weeks
5. Inability to tolerate or comply with trial requirements
6. Current pregnancy

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
Not Applicable
Type of endpoint/s
Safety
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
1/08/2024
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
15
Accrual to date
0
Final
Recruitment in Australia
Recruitment state(s)
VIC

Funding & Sponsors
Funding source category [1] 316627 0
Government body
Name [1] 316627 0
Department of Health and Aged Care, Medical Research Future Fund, Stem Cell Missions Grant
Country [1] 316627 0
Australia
Funding source category [2] 316630 0
Government body
Name [2] 316630 0
Brandon Biocatalyst, Department of Health and Aged Care, Medical Research Future Fund, CUREator
Country [2] 316630 0
Australia
Primary sponsor type
Hospital
Name
Monash Health
Address
Country
Australia
Secondary sponsor category [1] 318814 0
Other
Name [1] 318814 0
Hudson Institute of Medical Research
Address [1] 318814 0
Country [1] 318814 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 315410 0
Monash Health Human Research Ethics Committee A
Ethics committee address [1] 315410 0
https://monashhealth.org/research/resources/resource-library/
Ethics committee country [1] 315410 0
Australia
Date submitted for ethics approval [1] 315410 0
02/03/2023
Approval date [1] 315410 0
04/12/2023
Ethics approval number [1] 315410 0

Summary
Brief 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.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 134622 0
A/Prof Gregory Moore
Address 134622 0
Level 3, Department of Gastroenterology, Monash Medical Centre, 246 Clayton Road, Clayton, Vic 3168
Country 134622 0
Australia
Phone 134622 0
+61 3 9594 3577
Fax 134622 0
Email 134622 0
[email protected]
Contact person for public queries
Name 134623 0
Dr Charlotte Keung
Address 134623 0
Level 3, Department of Gastroenterology, Monash Medical Centre, 246 Clayton Road, Clayton, Vic 3168
Country 134623 0
Australia
Phone 134623 0
+61 3 9594 3577
Fax 134623 0
Email 134623 0
[email protected]
Contact person for scientific queries
Name 134624 0
Dr Charlotte Keung
Address 134624 0
Level 3, Department of Gastroenterology, Monash Medical Centre, 246 Clayton Road, Clayton, Vic 3168
Country 134624 0
Australia
Phone 134624 0
+61 3 9594 3577
Fax 134624 0
Email 134624 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


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.