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

A Phase I Study to Evaluate the Safety and Tolerability of Human TH-SC01 Cell Injection for the Treatment of Perianal Fistulas in Crohn's Disease

The purpose of this study is to evaluate the safety and tolerability of human TH-SC01 cell injection for the treatment of perianal fistulas in Crohn's Disease

Study Overview

• Status: Completed
• Conditions: Crohn's Disease; Perianal Fistula
• Intervention / Treatment: Drug: Human TH-SC01 cell injection

Detailed Description

This study is a phase 1, single-arm, safety and tolerability study of human TH-SC01 cell injection for perianal fistulas in Crohn's Disease.

• Study Type: Interventional
• Enrollment (Actual): 9
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• China
  • Shanghai
    • Shanghai, Shanghai, China
      • Shanghai

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (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. Subjects with perianal fistula confirmed by clinical assessment and MRI.
5. Subjects aged between 18 and 70, both male and female.
6. 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.
7. Subjects failed to respond to adequate treatment with any of the conventional antibiotics, immunomodulatory drugs (including steroids), anti-tumor necrosis factor-α (TNF-α) monoclonal antibodies and other biological agents.

Exclusion Criteria:

1. Subjects with active infection evaluated by the investigator.
2. Subjects with Crohn's disease requiring immediate therapy.
3. Subjects with abscess or collections >2 cm.
4. Subjects with rectal and/or anal stenosis and/or active proctitis.
5. Subjects who treated with systemic steroids in the 4 weeks prior to stem cells administration.
6. 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).
7. Subjects with malignant tumors or a history of malignant tumors.
8. Subjects with severe, progressive, uncontrolled hepatic, hematological, gastrointestinal (except Crohn's disease), endocrine, pulmonary, cardiac, neurological, psychiatric, or cerebral diseases.
9. Serum virology test (HBeAg, HCV antibody, HIV antibody, Treponema pallidum antibody) positive.
10. Subjects allergic to gentamicin sulfate, anesthetics or contrast media.
11. Subjects allergic to MRI contrast.
12. Subjects who has received stem cells in a previous clinical study or as a therapeutic agent.
13. Subjects who has major surgery or severe trauma within 6 months prior to the screening period.
14. Subjects who has received any investigational drug within 3 months prior to the screening.
15. Subjects deemed inappropriate by the investigator to participate in this clinical trial.
16. The female participant who is pregnant, or is lactating.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Human TH-SC01 cell injection; Single injection of 0.6×10^7, 1.2×10^8, 1.8×10^8 cells/kg	Drug: Human TH-SC01 cell injection; Single injection of human TH-SC01 cell injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Severity and incidence of adverse events	Severity and incidence of adverse events within 28 days after adiministration	28 days
Dose-limiting toxicity (DLT)	Exploration of dose-limiting toxicity (DLT)	84 days
Maximum tolerated dose (MTD)	Exploration of maximum tolerated dose (MTD)	84 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Perianal fistula healing	Change from baseline in Van Assche Score by MRI test. The score range was from 0 to 25. Higher score means more severe disease.	84 days
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.	84 days
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.	84 days
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.	84 days
VAS score	Change from baseline in pain score (VAS score).Total score ranges from 0 to 10. Higher score means more pain.	84 days
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.	84 days
Anal sphincter function： rectal pressure	Change from baseline in rectal pressure，In healthy people, the pressure ranges from 60 to 150mmHg, with the internal sphincter pressure accounts for about 80% and external sphincter pressure accounts for 20%.	84 days

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Actual): January 30, 2022
• Primary Completion (Actual): August 30, 2023
• Study Completion (Actual): February 28, 2025

Study Registration Dates

• First Submitted: October 26, 2022
• First Submitted That Met QC Criteria: November 15, 2022
• First Posted (Actual): November 23, 2022

Study Record Updates

• Last Update Posted (Actual): April 1, 2025
• Last Update Submitted That Met QC Criteria: March 27, 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; Rectal Diseases; Gastroenteritis; Inflammatory Bowel Diseases; Digestive System Fistula; Intestinal Fistula; Crohn Disease; Fistula; Rectal Fistula
• Other Study ID Numbers: TH-SC 2.0

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
• product manufactured in and exported from the U.S.: No