Clinical trial NCT05626023: A Study of Human TH-SC01 Cell Injection for Treating Perianal Fistulas in Patients With Crohn's Disease
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Perianal Fistula
Crohn's Disease
Lead sponsor: Jiangsu Topcel-KH Pharmaceutical Co., Ltd.
Sponsor class: INDUSTRY
Collaborators: name: Changhai Hospital
First posted: 2022-11-23
Enrollment: 9
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: 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

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Severity and incidence of adverse events
timeFrame: 28 days
measure: Dose-limiting toxicity (DLT)
timeFrame: 84 days
measure: Maximum tolerated dose (MTD)
timeFrame: 84 days
Secondary outcomes:
measure: Perianal fistula healing
timeFrame: 84 days
measure: Crohn's Disease Activity Index (CDAI) score
timeFrame: 84 days
measure: Perianal Disease Activity Index (PDAI)
timeFrame: 84 days
measure: Inflammatory Bowel Disease Questionnaire（IBDQ）score
timeFrame: 84 days
measure: VAS score
timeFrame: 84 days
measure: Anal sphincter function：Wexner incontinence score
timeFrame: 84 days
measure: Anal sphincter function： rectal pressure
timeFrame: 84 days
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Shanghai
state: Shanghai
Study officials:
References: