Safety of Allogeneic Human Umbilical Cord Mesenchymal Stem Cells (UC-MSCs) to Treat Perianal Fistulas Patients With Crohn's Disease

A Phase I Study of the Safety of Allogeneic Human Umbilical Cord Mesenchymal Stem Cells (UC-MSCs) for Perianal Fistulas in Patients With Crohn's Disease

Objectives:

Primary: To demonstrate the safety of allogeneic UC-MSCs administered by injection for complex perianal fistulas in patients with Crohn's disease

Secondary: To determine the efficacy of a single/multiple allogeneic UC-MSCs injection in improving complex perianal fistula complications and (re-epithelialization of the external openings).

Study Overview

• Status: Recruiting
• Conditions: Fistula in Ano; Perianal Fistula Due to Crohn's Disease
• Intervention / Treatment: Biological: PF2020-CELL (UC-MSCs) - Allogeneic Human Umbilical Cord Mesenchymal Stem Cells

Detailed Description

Design and Investigation:

Number of Patients: 5 (including a consideration of 20% [1 patient] dropout from the trial) Dosage and mode of administration

Dosage: Two million cells per milliliter (2M/mL) with a total of 60 million PF2020-CELL (UC-MSCs) will be injected locally around the fistula tract, with a separation of 1 cm between injections. All patients will receive a total of 60 million cells per visit for 5 consecutive visits, with a 4-week interval between the injections. If the patient is completely healed during any course of stem cell injections, subsequent stem cell treatments can be discontinued. However, patients will still be required to undergo regular follow-up examinations, including physical examinations and other medical tests, until the study is completed.

Mode of administration: The procedure involves localizing the fistulous tract, performing curettage and closing the internal opening, and the injection of PF2020-CELL (UC-MSCs). The indicated dose of PF2020-CELL (UC-MSCs) will be injected starting from the tissues around the internal orifice or orifices and then progressing towards the external opening.

• Study Type: Interventional
• Enrollment (Estimated): 5
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: April Camilla Roslani, Prof. Dr.; Phone Number: +603-7949 2050; Email: april@ummc.edu.my

Study Locations

• Malaysia
  • Kuala Lumpur, Malaysia, 59100
    • Recruiting
    • University of Malaya Medical Centre
    • Contact: April Camilla Roslani, Prof. Dr.; Phone Number: +603-7949 2050; Email: april@ummc.edu.my
    • Principal Investigator: April Camilla Roslani, Prof. Dr.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Adult men and women age 18 years and above.
• Diagnosis of perianal fistulae associated with Crohn's disease refractory to medical therapy.
• Presence of perianal fistula(s) with a maximum of 2 internal openings and a maximum of 3 external openings based on clinical assessment.
• Fit for surgery.

Exclusion Criteria:

• Informed consent refusal.
• Pregnancy or breastfeeding women.
• Current diagnosis of active cancer or remission for less than 5 years.
• Evidence of active sepsis or significant localised infection.
• Patients with HIV, HBV, HCV or treponema infection, whether active or latent.
• Patients with documented allergies.
• Patients who have received infliximab or any other biologics in the 4 weeks before the cell treatment administration.
• Patients currently receiving, or having received within 1 month prior to enrollment into this clinical trial, any investigational drug.
• Patients with any other co-morbidity/ co-pathologies which is deemed as contraindication to stem cells infusion (infection, administration of steroids).

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: PF2020-CELL (UC-MSCs) - Allogeneic Human Umbilical Cord Mesenchymal Stem Cells; Two million cells per milliliter (2M/mL) with a total of 60 million PF2020-CELL (UC-MSCs) will be injected locally around the fistula tract, with a separation of 1 cm between injections. All patients to receive allogeneic PF2020-CELL (UC-MSCs) via intralesional injection for 5 consecutive visits, with a 4-week interval between the injections. If the patient is completely healed during any course of stem cell injections, subsequent stem cell treatments can be discontinued. However, patients will still be required to undergo regular follow-up examinations, including physical examinations and other medical tests, until the study is completed.

Biological: PF2020-CELL (UC-MSCs) - Allogeneic Human Umbilical Cord Mesenchymal Stem Cells; All patients will receive a total of 60 million PF2020-CELL (UC-MSCs) cells per treatment visit.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
No incidence of treatment-emergent adverse events (TEAEs)	TEAEs in this study are defined as adverse events other than: pyrexia (non-persistent fever, <38.5°C), anal abscess, pain in the rectum/anus & procedural pain (pain after fistula cleaning).	Throughout study completion, an average of 1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Clinical healing	Mainly on re-epithelialization of external opening	Week 2, 6, 10, 14, 18, 20, 24, 32, 48

Collaborators and Investigators

• Sponsor: CryoCord Sdn Bhd
• Collaborators: University of Malaya
• Investigators: Principal Investigator: April Camilla Roslani, Prof. Dr., Faculty of Medicine, University of Malaya

Study record dates

Study Major Dates

• Study Start (Actual): March 1, 2022
• Primary Completion (Estimated): December 31, 2024
• Study Completion (Estimated): January 27, 2025

Study Registration Dates

• First Submitted: September 1, 2021
• First Submitted That Met QC Criteria: September 2, 2021
• First Posted (Actual): September 9, 2021

Study Record Updates

• Last Update Posted (Actual): October 10, 2023
• Last Update Submitted That Met QC Criteria: October 9, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Keywords: Crohn's Disease; Perianal fistula; Umbilical cord mesenchymal stem cells (UC-MSCs); Allogeneic stem cells
• Additional Relevant MeSH Terms: Digestive System Diseases; Gastrointestinal Diseases; Gastroenteritis; Intestinal Diseases; Pathological Conditions, Anatomical; Rectal Diseases; Inflammatory Bowel Diseases; Intestinal Fistula; Digestive System Fistula; Fistula; Crohn Disease; Rectal Fistula
• Other Study ID Numbers: CCSB-CT-PF-01-2021 (Ver3_2023)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No