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Trial registered on ANZCTR


Registration number
ACTRN12624000537550
Ethics application status
Approved
Date submitted
15/04/2024
Date registered
30/04/2024
Date last updated
1/12/2024
Date data sharing statement initially provided
30/04/2024
Type of registration
Prospectively registered

Titles & IDs
Public title
Amnion cell therapy for ulcerative proctitis
Scientific title
A human pilot study evaluating the safety of locally administered human amnion epithelial cells for the treatment of refractory ulcerative proctitis in adults
Secondary ID [1] 311961 0
Nil
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Ulcerative proctitis 333560 0
Condition category
Condition code
Oral and Gastrointestinal 330244 330244 0 0
Inflammatory bowel disease

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
This intervention requires a single procedure of allogeneic human amnion epithelial cells (hAEC) obtained from the placentas of consenting healthy donors delivering at-term healthy babies at Monash Health, obtained under Good Manufacturing Practice-like conditions for clinical trial use. 20 million to 60 million cells will be administered per patient in 4-12 injections into the rectum (based on the baseline length of disease) during colonoscopy by a specialist gastroenterologist in our endoscopy suite at our quaternary Australian hospital. The overall estimated duration of injection will be approximately 30 minutes. The anaesthesia administered will be deep sedation as per routine general colonoscopy. The entire endoscopic procedure will be recorded and read by the endoscopist and a central reader as well as recorded in a procedural report.
Intervention code [1] 328419 0
Treatment: Other
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 337991 0
The primary outcome is safety with an adverse event defined as any of the following 1) worsening (change in nature, severity or frequency) of ulcerative proctitis present at the time of the study based on the Modified Mayo Score, 2) intercurrent illness based on patient self-reporting, 3) abnormal laboratory values (FBE, UEC, LFT, CRP) defined as clinically significant shifts from baseline within the range of normal that the investigator considers to be clinically significant or 4) clinically significant abnormalities in physical examination, vital signs, weight and drainage for the perianal fistulas. A causality assessment will be performed regarding adverse events and defined as one of the following: unrelated, unlikely, possible or probable.

The severity of AEs will be graded using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE), v5.0. A serious adverse event (SAE) is any untoward medical occurrence that occurs at any dose. An AE in a clinical trial is designated to be serious if it results in death, is life-threatening, requires inpatient hospitalisation, or prolongs existing hospitalisation, results in persistent or significant disability or incapacity.
Timepoint [1] 337991 0
The primary outcome timepoint is assessed at week 12 after administration of intervention.
Secondary outcome [1] 434038 0
Feasibility of route of stem-like cell administration based on a specifically designed questionnaire for investigator completion.
Timepoint [1] 434038 0
The secondary outcome timepoint is assessed at week 12 after administration of intervention.
Secondary outcome [2] 434039 0
Efficacy of treatment
This will be assessed based on symptoms (stool frequency, rectal bleeding) using the Modified Mayo Score and the baseline and week 12 Mayo endoscopic subscore as a composite outcome.
Timepoint [2] 434039 0
The secondary outcome timepoint is assessed at week 12 after administration of intervention compared with baseline assessment of symptoms and endoscopic subscore.
Secondary outcome [3] 434040 0
Health-related quality of life assessed by the Short Inflammatory Bowel Disease Questionnaire
Timepoint [3] 434040 0
The secondary outcome timepoint is assessed at week 12 after administration of intervention.

Eligibility
Key inclusion criteria
1. Adult participant
2. Ulcerative proctitis defined by less than 15 cm length of active inflammation from anus
3. At least 6 months disease duration
4. Refractory to at least one of the following treatments: topical aminosalicylate or prednisolone or budesonide. Definition of lack of response will assessed as per Modified Mayo Score of 5 or more, endoscopic subscore of 2 or more.
Minimum age
18 Years
Maximum age
80 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Acute severe ulcerative colitis
2. Rectal or anal stenosis
3. Diverting stomas
4. Perianal or rectovaginal fistula
5. Active infection
6. Any previous colonic dysplasia or malignancy
7. Inability to tolerate or comply with trial requirements eg. high anaesthetic risk unsuitable for endoscopy
8. Current pregnancy/breast feeding

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
Who is / are masked / blinded?



Intervention assignment
Other design features
Phase
Not Applicable
Type of endpoint/s
Safety
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
1/05/2024
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
5
Accrual to date
0
Final
Recruitment in Australia
Recruitment state(s)
VIC

Funding & Sponsors
Funding source category [1] 299589 0
Charities/Societies/Foundations
Name [1] 299589 0
The Gutsy Group
Country [1] 299589 0
Australia
Primary sponsor type
Hospital
Name
Monash Health
Address
Country
Australia
Secondary sponsor category [1] 318488 0
None
Name [1] 318488 0
Nil
Address [1] 318488 0
Country [1] 318488 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 300490 0
Monash Health Human Research Ethics Committee A
Ethics committee address [1] 300490 0
Ethics committee country [1] 300490 0
Australia
Date submitted for ethics approval [1] 300490 0
07/09/2023
Approval date [1] 300490 0
19/12/2023
Ethics approval number [1] 300490 0

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

Contacts
Principal investigator
Name 83826 0
A/Prof Gregory Moore
Address 83826 0
Level 3, Department of Gastroenterology, Monash Medical Centre, 246 Clayton Road, Clayton, VIC 3168
Country 83826 0
Australia
Phone 83826 0
+61 3 9594 3577
Fax 83826 0
Email 83826 0
Gregory.Moore@monashhealth.org
Contact person for public queries
Name 83827 0
Dr Charlotte Keung
Address 83827 0
Level 3, Department of Gastroenterology, Monash Medical Centre, 246 Clayton Road, Clayton, VIC 3168
Country 83827 0
Australia
Phone 83827 0
+61 3 9594 3577
Fax 83827 0
Email 83827 0
Charlotte.Keung@monashhealth.org
Contact person for scientific queries
Name 83828 0
Dr Charlotte Keung
Address 83828 0
Level 3, Department of Gastroenterology, Monash Medical Centre, 246 Clayton Road, Clayton, VIC 3168
Country 83828 0
Australia
Phone 83828 0
+61 3 9594 3577
Fax 83828 0
Email 83828 0
Charlotte.Keung@monashhealth.org

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.