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

Phase 1

Recruiting

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

• Registration Number: NCT05039411
• Lead Sponsor: CryoCord Sdn Bhd

Brief Summary

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).

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: 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 visits, with a 6 - 12 weeks 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 Timeline

• Study First Submitted: 2021-09-01
• Study First Submitted QC: 2021-09-02
• Study First Posted: 2021-09-09
• Study Start: 2022-03-01
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-07
• Last Update Posted: 2025-05-11
• Primary Completion: 2026-12-31
• Study Completion: 2026-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 5

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
PF2020-CELL (UC-MSCs) - Allogeneic Human Umbilical Cord Mesenchymal Stem Cells	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.
PF2020-CELL (UC-MSCs) - Allogeneic Human Umbilical Cord Mesenchymal Stem Cells	PF2020-CELL (UC-MSCs) - Allogeneic Human Umbilical Cord Mesenchymal Stem Cells	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 visits, with a 6 - 12 weeks 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.

Primary Outcome Measures

Name	Time	Method
No incidence of treatment-emergent adverse events (TEAEs)	Throughout study completion, an average of 1.5 year	example of TEAEs are pyrexia (non-persistent fever, \<38.5°C), infection, perianal abscess, enlargement of fistula \& allergic reaction.

Secondary Outcome Measures

Name	Time	Method
Clinical healing	Visits 5, 9, 13, 17, 21 (approximately 6 to 12 weeks between visits)	Mainly on re-epithelialization of external opening

Trial Locations

Locations (1)

University of Malaya Medical Centre; 🇲🇾 Kuala Lumpur, Malaysia