Evaluate the Distribution and Dynamic Behavior of TH-SC01 Cells in Vivo in Patients With Perianal Fistula

A Phase I Clinical Study Evaluating the Distribution and Dynamic Behavior of Nuclide Labeled TH-SC01 Cells in Vivo in Patients With Perianal Fistula

The purpose of this study is to evaluate the distribution and dynamic behavior of Nuclide labeled TH-SC01 cells in vivo in patients with perianal fistula

Study Overview

• Status: Recruiting
• Conditions: Crohn's Disease; Anal Fistula; Complex Perianal Fistulas
• Intervention / Treatment: Biological: Mesenchymal Stem Cells (MSCs)

Detailed Description

A Phase I clinical study evaluating the distribution and dynamic behavior of Nuclide labeled TH-SC01 cells in vivo in patients with perianal fistula

• Study Type: Interventional
• Enrollment (Estimated): 8
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Miu Li Yan The First Affiliated Hospital of Soochow University; Phone Number: 86+0512-67972858; Email: E-mail:sdfyec@163.com

Study Locations

• China
  • Jiangsu
    • Suzhou, Jiangsu, China, 215006
      • Recruiting
      • The First Affiliated Hospital of Soochow University
      • Contact: Yan Wang, MD; Phone Number: 0512-67972858; Email: sdfyy8040@126.com

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 or complex perianal fistula diagnosed at least 6 months earlier according to the Chinese Consensus Opinion on the Diagnosis and Treatment of Inflammatory Bowel Diseases (Beijing, 2018).Subjects with active perianal fistula and non active luminal CD defined by a CDAI ≤ 200.
3. For patients with perianal fistula, 1≤ the number of internal openings ≤2, and 1≤ the number of external openings ≤3, the fistula of the patient needs to be drained smoothly
4. 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.
5. ECOG score 0~1, ASA grade I~II
6. 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 Human serum albumin, human platelet lysate, gentamicin sulfate, anesthetic drug
11. Subjects who has received stem cells therapy.
12. Subjects who has major surgery or severe trauma within 6 months prior to the screening period.
13. Subjects who has received any investigational drug within 3 months prior to the screening.
14. Subjects deemed inappropriate by the investigator to participate in this clinical trial.
15. The female participant who is pregnant, or is lactating.
16. Not suitable for PET/CT examination.
17. Participants considered inappropriate to participate in this clinical trial

Study Plan

How is the study designed?

Design Details

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

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: MSCs treatment group; Intralesional injection of expanded human umbilical cord-derived mesenchymal stem cells suspension.	Biological: Mesenchymal Stem Cells (MSCs); single dose injection (120 million cells)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Standardized uptake value	Standardized values of organs or tissues after PET/CT imaging after local injection in patients with perianal fistula	8-12 hour、32-36 hour、56-60 hour、104-108 hour、152-156 hour，296-300 hour

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Effectiveness endpoint:Proportion of subjects with clinically significant effect at week 24 after local injection	Change from baseline in pain score (VAS score).Total score ranges from 0 to 10. Higher score means more pain.	Week 1，Week 4，Week 24
Safety endpoint: Treatment-related adverse events/adverse reactions, serious adverse events/serious adverse events	All subjects were observed for any adverse events/reactions or serious adverse events/reactions that occurred during the clinical trial, including but not limited to clinically significant abnormal changes in clinical symptoms, physical examination, vital signs examination, laboratory examination, 12-lead electrocardiogram examination, etc. The clinical features, severity, occurrence time, end time, treatment and outcome of the disease should be recorded, and the correlation between the disease and the investigational drug should be determined.	Week 1，Week 4，Week 24，Month 24
Radiation exposure: Uptake rate (%ID), absorbed dose, and systemic effective dose in vital organs or tissues after local injection of nuclide labeled TH-SC01 cells.	Based on the image data obtained by PET/CT scan, OLINDA's sphere model was used to delineate the injection site and important organs or tissues as areas of interest (ROI), obtain standardized uptake values (SUV) of each ROI, and obtain time-radioactive activity curves (TACs) of each tissue and organ. The absorbed dose of vital organs or tissues, the equivalent dose of the whole body, and the distribution and change of transplanted cells in different tissues or organs at different time points are described	Week 1

Collaborators and Investigators

• Sponsor: Jiangsu Topcel-KH Pharmaceutical Co., Ltd.
• Collaborators: The First Affiliated Hospital of Soochow University

Study record dates

Study Major Dates

• Study Start (Actual): April 8, 2024
• Primary Completion (Estimated): November 1, 2024
• Study Completion (Estimated): December 1, 2025

Study Registration Dates

• First Submitted: May 9, 2024
• First Submitted That Met QC Criteria: May 20, 2024
• First Posted (Actual): May 24, 2024

Study Record Updates

• Last Update Posted (Actual): June 12, 2024
• Last Update Submitted That Met QC Criteria: June 10, 2024
• Last Verified: May 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Gastrointestinal Diseases; Gastroenteritis; Intestinal Diseases; Pathological Conditions, Anatomical; Rectal Diseases; Inflammatory Bowel Diseases; Intestinal Fistula; Digestive System Fistula; Fistula; Crohn Disease; Rectal Fistula
• Other Study ID Numbers: 2023-TH-SC01-I-007

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

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