Clinical trial NCT05677672: 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
Official title: Phase I/Ⅱ Study to Evaluate the Safety and Efficacy of Human TH-SC01 Cell Injection in the Treatment of Complex Perianal Fistula
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Complex Perianal Fistulas
Lead sponsor: Jiangsu Topcel-KH Pharmaceutical Co., Ltd.
Sponsor class: INDUSTRY
Collaborators: name: Xiangya Hospital of Central South University
First posted: 2023-01-10
Enrollment: 84
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Phase 1 was a non-randomized, single-arm, multicenter, dose-escalation clinical trial design.

Phase 2 was a randomized, double-masking , multicenter, blank control, dose-extended clinical trial design.
Primary purpose: TREATMENT

Brief summary: 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

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

Eligibility criteria: 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.

Primary outcomes:
measure: Severity and incidence of study drug-related adverse-events
timeFrame: Day 28 (Week 4)
measure: Dose-limiting toxicity (DLT)
timeFrame: Day 28
measure: maximum tolerated dose (MTD)
timeFrame: Day 28
measure: Percentage of effectiveness
timeFrame: Day 84
Secondary outcomes:
measure: Severity and incidence of study drug-related adverse-events
timeFrame: Day 28
Other outcomes:
measure: Patient quality of life score
timeFrame: Week 1-Week 52
measure: Rate of recurrence
timeFrame: Week 24-Week 104
measure: Closure time of fistulas
timeFrame: Day 1
measure: SF-36 score
timeFrame: Week 1-Week 52
measure: Van Assche Score
timeFrame: Day 0-Week 52
measure: Wexner incontinence Score
timeFrame: Week 4-Week 52
measure: Anal sphincter function： rectal pressure
timeFrame: Week 4-Week 52
Locations:
city: Changsha
country: China
facility: Xiangya Hospital Central University
state: Hunan
Study officials:
References: