Phase 3 Study of Human TH-SC01 Cell Injection for Treating Perianal Fistulas in Patients With Crohn's Disease

A Randomized, Double-blind, Placebo-controlled, Multi-center Phase 3 Clinical Trial to Evaluate the Efficacy and Safety of Human TH-SC01 Cell Injection in the Treatment of Anal Fistulas in Patients With Non-active/Mildly Active Crohn's Disease

The purpose of this study is to evaluate the efficacy of human TH-SC01 cell injection for the treatment of perianal fistulas in Crohn's Disease over 24 weeks.

Study Overview

• Status: Recruiting
• Conditions: Crohn's Disease
• Intervention / Treatment: Other: Human TH-SC01 cell injection; Other: Saline solution

• Study Type: Interventional
• Enrollment (Estimated): 228
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Jueyu Xia, Master; Phone Number: 025-52700103; Email: xiajueyu@topcelbio.com

Study Locations

• China
  • Jiangsu
    • Nanjing, Jiangsu, China
      • Recruiting
      • Jiangsu Topcel-KH Pharmaceutical Co., Ltd.
      • Contact: Jueyu Xia, Master; Phone Number: 025-52700103; Email: xiajueyu@topcelbio.com
      • Principal Investigator: Zhaoshen Li, Ph.D.
      • Principal Investigator: Donglin Ren, Ph.D.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Signed informed consent
2. Subjects with Crohn's disease diagnosed at least 6 months earlier according to the Chinese Consensus Opinion on the Diagnosis and Treatment of Inflammatory Bowel Diseases (Beijing, 2018)
3. Subjects with active perianal fistula and non active luminal CD defined by a CDAI ≤ 200.
4. Presence of perianal fistulas with a maximum of 2 fistulas (internal openings) and a maximum of 3 external openings, assessed by clinical assessment and MRI.
5. Eastern Cooperative Oncology Group (ECOG) performance status score of 0 to 1.
6. American Society of Anesthesiologists (ASA) physical status classification of grade I to grade II.
7. All subjects and their partners were not planning to have a child from screening to the end of the trial and agreed to use effective non-drug contraception during the trial.

Exclusion Criteria:

1. Subjects allergic to human serum albumin, human platelet lysate, gentamicin sulfate, anesthetic drugs or MRI contrast agents.
2. Subjects with active infection evaluated by the investigator
3. Subjects with active Crohn's disease before drug administration or have not completed the induction-phase treatment.
4. Subjects with abscess or collections >2 cm.
5. Rectal and/or anal stenosis , if this means a limitation for any surgical procedure
6. Subjects with concurrent active proctitis.
7. Subjects with other fistulas such as rectum/anal canal-vaginal fistulas or rectum-bladder fistulas.
8. Patients have undergone a diverting stoma operation in the past, except for patients have completed stoma closure at least one month before drug administration.
9. Patients plan to undergo surgery in the perianal area during the trial period due to other diseases other than anal fistula.
10. Subjects with abnormal laboratory results: liver function: total bilirubin >=1.5 × ULN, and aspartate aminotransferase (AST) and alanine aminotransferase (ALT) >=2 × ULN; renal function: creatinine clearance below 60 mL/minute calculated using Cockcroft-Gault formula or by serum creatinine >=1.5 × upper limit of normal (ULN).
11. Serum virology test (HBeAg, HCV antibody, HIV antibody, Treponema pallidum antibody) positive.
12. Subjects with malignant tumors or a history of malignant tumors.
13. Subjects with congenital or acquired immunodeficiency.
14. Contraindication to MRI scan
15. Current or recent history of abnormal, severe, progressive, uncontrolled hepatic, haematological, gastrointestinal (except CD), endocrine, pulmonary, cardiac, neurological, psychiatric, or cerebral disease.
16. Subjects received stem cell treatment within two years prior to screening.
17. Patient with ongoing steroid treatment or treated with steroids in the last 4 weeks.
18. Patient undergone major surgical operations (such as orthopedic surgery, abdominal surgery) within one month before drug administration, or Patient plan to receive surgeries during the study period (except for the surgeries required by the procedures of this trial).
19. Patients with a history of drug abuse or alcoholism (Criteria for alcoholism: having a long-term drinking history of more than 5 years, with the amount of ethanol equivalent to ≥40g/d for men and ≥20g/d for women, or having a history of heavy drinking within 2 weeks, with the amount of ethanol equivalent to >80g/d. The conversion formula for the amount of ethanol (g) = the amount of alcohol consumed (ml) × the ethanol content (%) × 0.8).
20. Pregnant or lactating women.
21. Patients with a history of diverting ostomy, except those who have undergone stoma reversal at least 1 month prior to drug administration.
22. Other diseases or conditions that the investigator deems inappropriate for participating in this clinical trial, such as poor subject compliance and the expectation of being unable to complete follow-up and evaluation.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Treatment Arm; 120 million cells administered by intralesional injection.	Other: Human TH-SC01 cell injection; Human TH-SC01 cell injection is a cell suspension in aseptic buffered solution containing human expanded umbilical cord-derived stem cells of allogeneic origin in disposable vials with no preservative agents. The cells will be given at a dose of 120 million cells (5 million cells / mL) for intralesional injection.
Placebo Comparator: Placebo-control group; 24 mL saline solution by intralesional injection	Other: Saline solution; Placebo (saline solution) will be given also for intralesional injection at the same quantity (volume, 24 mL) and following the same schedule.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The proportion of subjects achieved combined remission	Combined Remission of perianal fistulising Crohn's disease defined as the all treated external openings achieved clinical remission at week 24， and absence of collections > 2 cm of the treated perianal fistulas confirmed by centrally blinded MRI assessment by week 24.	24 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The proportion of subjects achieved clinical remission	Clinical Remission defined as closure of all treated external openings that were draining at baseline despite gentle finger compression, and no obvious fistulas can be felt by digital examination，as clinically assessed.	24 weeks ，52 weeks and104 weeks
The proportion of subjects achieved a clinical response	Clinical response defined as closure of at least 50% of all treated external openings that were draining at baseline, as clinically assessed	24 weeks，52 weeks and104 weeks
The proportion of subjects recurrence	Recurrence defined as the reopening of the closed external openings that had achieved clinical remission after treatment, the appearance of spontaneous fluid drainage, or the presence of fistulas with fluid collections > 2 cm as assessed by enhanced pelvic MRI	24 weeks，52 weeks and104 weeks
Time to achieve clinical remission, clinical response and recurrence	Time to achieve clinical remission, clinical response and recurrence within 24 weeks after drug administration.	24 weeks，52 weeks and104 weeks
The proportion of subjects achieved combined remission	Combined Remission of perianal fistulising Crohn's disease at week 52 and 104 (as defined for week 24)	52 weeks and104 weeks
VAS score	Change from baseline in pain score (VAS score).Total score ranges from 0 to 10. Higher score means more pain	24 weeks，52 weeks and104 weeks
Perianal Disease Activity Index (PDAI)	Change from baseline in Perianal Disease Activity Index (PDAI). Total score ranges from 0 to 20. Higher score means more severe disease	24 weeks，52 weeks and104 weeks
Crohn's Disease Activity Index (CDAI) score	Change from baseline in the Crohn's Disease Activity Index (CDAI) score. Higher score means more severe disease and especially severe disease was defined as a value of greater than 450	24 weeks，52 weeks and104 weeks
Inflammatory Bowel Disease Questionnaire（IBDQ）score	Change from baseline in IBDQ score. Total score ranges from 32 to 224. Higher score means better quality of life	24 weeks，52 weeks and104 weeks
Anal sphincter function：Wexner incontinence score	Change from baseline in anal sphincter function, Wexner incontinence score,Total score ranges from 0 to 20. Higher score means more severe disease	24 weeks，52 weeks and104 weeks
peripheral blood cytokines	Changes in the levels of peripheral blood cytokines compared to the baseline.	2 weeks，4 weeks ，8 weeks ，12weeks and 24 weeks

Collaborators and Investigators

• Sponsor: Jiangsu Topcel-KH Pharmaceutical Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): March 20, 2025
• Primary Completion (Estimated): December 28, 2026
• Study Completion (Estimated): June 28, 2028

Study Registration Dates

• First Submitted: April 7, 2025
• First Submitted That Met QC Criteria: April 11, 2025
• First Posted (Actual): April 13, 2025

Study Record Updates

• Last Update Posted (Actual): April 13, 2025
• Last Update Submitted That Met QC Criteria: April 11, 2025
• Last Verified: March 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathological Conditions, Anatomical; Intestinal Diseases; Digestive System Diseases; Gastrointestinal Diseases; Gastroenteritis; Inflammatory Bowel Diseases; Crohn Disease; Fistula
• Other Study ID Numbers: TH-SC01-CD-Ⅲ-01

Drug and device information, study documents

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