The Purpose of This Study is to Evaluate the Safety and Efficacy of Human TH-SC01 Cell Injection in the Treatment of Complex Perianal Fistula

Phase I/Ⅱ Study to Evaluate the Safety and Efficacy of Human TH-SC01 Cell Injection in the Treatment of Complex Perianal Fistula

The purpose of this study is to evaluate the safety and efficacy of human TH-SC01 cell injection for the treatment of complex perianal fistulas

Study Overview

• Status: Not yet recruiting
• Conditions: Complex Perianal Fistulas
• Intervention / Treatment: Biological: Human TH-SC01 Cell Injection; Other: Saline solution

Detailed Description

This study is a phase Ⅰ/Ⅱ study, Phase 1 was the dose increment phase, and phase 2 was the expansion phase

• Study Type: Interventional
• Enrollment (Anticipated): 84
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• China
  • Hunan
    • Changsha, Hunan, China, 410000
      • Xiangya Hospital Central University
      • Contact: Weidong Zhang, Dr.; Phone Number: 073189753568; Email: davidcsu@foxmail.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Signed informed consent.
2. Subjects aged between 18 and 70, both male and female.
3. The subject was clinically diagnosed as complex anal fistula. The diagnostic criteria for complex anal fistula were adopted by the 2016 American Association of Colorectal Surgeons Guidelines for the Treatment of Perianal Abscess, Anal Fistula and Rectovaginal Fistula.
4. The subjects had received conventional treatment for anal fistulas
5. 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 with anal fistula in the acute infection period.
2. Subjects with abscess or collections >2 cm.
3. 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).
4. Subjects with malignant tumors or a history of malignant tumors.
5. Subjects with severe, progressive, uncontrolled hepatic, hematological, gastrointestinal (except Crohn's disease), endocrine, pulmonary, cardiac, neurological, psychiatric, or cerebral diseases.
6. Serum virology test (HBeAg, HCV antibody, HIV antibody, Treponema pallidum antibody) positive.
7. Subjects allergic to gentamicin sulfate, anesthetics or contrast media.
8. Subjects allergic to MRI contrast.
9. Subjects who has major surgery or severe trauma within 6 months prior to the screening period.
10. Subjects who has received any investigational drug within 3 months prior to the screening.
11. Subjects deemed inappropriate by the investigator to participate in this clinical trial.
12. The female participant who is pregnant, or is lactating.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Human TH-SC01 cell injection; Human TH-SC01 Cell Injection is a human expanded umbilical cord mesenchymal stem cells suspension	Biological: Human TH-SC01 Cell Injection; Single injection of 0.6×10^7,1.2×10^8, 1.8×10^8 cells/kg
Placebo Comparator: Placebo-control group; Saline solution	Other: Saline solution; saline solution will be given at the same quantity and following the same schedule

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Severity and incidence of study drug-related adverse-events	Severity and incidence of study drug-related adverse-events within D28(W4) after administration.	Day 28 (Week 4)
Dose-limiting toxicity (DLT)	Exploration of dose-limiting toxicity (DLT)	Day 28
maximum tolerated dose (MTD)	Exploration of maximum tolerated dose(MTD)	Day 28
Percentage of effectiveness	Percentage of effectiveness at 84 days of administration by MRI imaging evaluated fistula healing with no or minimal effusion	Day 84

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Severity and incidence of study drug-related adverse-events	Severity and incidence of study drug-related adverse-events after D28(W4) after administration.	Day 28

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Patient quality of life score	Change from baseline in Patient quality of life score from W1-W52	Week 1-Week 52
Rate of recurrence	The recurrence rate after administration	Week 24-Week 104
Closure time of fistulas	Closure time of wound and fistula at the anal fistula surgical site after drug administration: Starting from the next day after administration (D1).	Day 1
SF-36 score	Change From Baseline in SF-36 score after administration，	Week 1-Week 52
Van Assche Score	Change From Baseline in Van Assche Score.Total score ranges from 0 to 10. Higher score means more pain.	Day 0-Week 52
Wexner incontinence Score	Change From Baseline in Wexner incontinence Score .Total score ranges from 0 to 20. Higher score means more severe disease.	Week 4-Week 52
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%.	Week 4-Week 52

Collaborators and Investigators

• Sponsor: Jiangsu Topcel-KH Pharmaceutical Co., Ltd.
• Collaborators: Xiangya Hospital of Central South University

Study record dates

Study Major Dates

• Study Start (Anticipated): December 30, 2022
• Primary Completion (Anticipated): August 30, 2023
• Study Completion (Anticipated): December 30, 2023

Study Registration Dates

• First Submitted: December 14, 2022
• First Submitted That Met QC Criteria: December 28, 2022
• First Posted (Estimate): January 10, 2023

Study Record Updates

• Last Update Posted (Estimate): January 10, 2023
• Last Update Submitted That Met QC Criteria: December 28, 2022
• Last Verified: December 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Gastrointestinal Diseases; Intestinal Diseases; Pathological Conditions, Anatomical; Rectal Diseases; Intestinal Fistula; Digestive System Fistula; Fistula; Rectal Fistula
• Other Study ID Numbers: TH-SC01-CAF-Ⅰ/Ⅱ 1.0

Drug and device information, study documents

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