Phase 3 Study of Human TH-SC01 Cell Injection for Treating Perianal Fistulas in Patients With Crohn's Disease

Официальное название

A Randomized, Double-blind, Placebo-controlled, Multi-center Phase 3 Clinical Trial to Evaluate the Efficacy and Safety of Human TH-SC01 Cell Injection in the Treatment of Anal Fistulas in Patients With Non-active/Mildly Active Crohn's Disease

Обзор

The purpose of this study is to evaluate the efficacy of human TH-SC01 cell injection for the treatment of perianal fistulas in Crohn's Disease over 24 weeks.

Вмешательства

• Другое Human TH-SC01 cell injection
  Human TH-SC01 cell injection is a cell suspension in aseptic buffered solution containing human expanded umbilical cord-derived stem cells of allogeneic origin in disposable vials with no preservative agents. The cells will be given at a dose of 120 million cells (5 million cells / mL) for intralesional injection.
• Другое Saline solution
  Placebo (saline solution) will be given also for intralesional injection at the same quantity (volume, 24 mL) and following the same schedule.

Первичные конечные точки

• The proportion of subjects achieved combined remission [Срок оценки: 24 weeks]

Вторичные конечные точки (11)

• The proportion of subjects achieved clinical remission [Срок оценки: 24 weeks ，52 weeks and104 weeks]
• The proportion of subjects achieved a clinical response [Срок оценки: 24 weeks，52 weeks and104 weeks]
• The proportion of subjects recurrence [Срок оценки: 24 weeks，52 weeks and104 weeks]
• Time to achieve clinical remission, clinical response and recurrence [Срок оценки: 24 weeks，52 weeks and104 weeks]
• The proportion of subjects achieved combined remission [Срок оценки: 52 weeks and104 weeks]
• VAS score [Срок оценки: 24 weeks，52 weeks and104 weeks]
• Perianal Disease Activity Index (PDAI) [Срок оценки: 24 weeks，52 weeks and104 weeks]
• Crohn's Disease Activity Index (CDAI) score [Срок оценки: 24 weeks，52 weeks and104 weeks]
• Inflammatory Bowel Disease Questionnaire（IBDQ）score [Срок оценки: 24 weeks，52 weeks and104 weeks]
• Anal sphincter function：Wexner incontinence score [Срок оценки: 24 weeks，52 weeks and104 weeks]
• peripheral blood cytokines [Срок оценки: 2 weeks，4 weeks ，8 weeks ，12weeks and 24 weeks]

Критерии участия

Критерии включения

• Signed informed consent
• 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)
• Subjects with active perianal fistula and non active luminal CD defined by a CDAI ≤ 200.
• Presence of perianal fistulas with a maximum of 2 fistulas (internal openings) and a maximum of 3 external openings, assessed by clinical assessment and MRI.
• Eastern Cooperative Oncology Group (ECOG) performance status score of 0 to 1.
• American Society of Anesthesiologists (ASA) physical status classification of grade I to grade II.
• 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.

Критерии исключения

• Subjects allergic to human serum albumin, human platelet lysate, gentamicin sulfate, anesthetic drugs or MRI contrast agents.
• Subjects with active infection evaluated by the investigator
• Subjects with active Crohn's disease before drug administration or have not completed the induction-phase treatment.
• Subjects with abscess or collections >2 cm.
• Rectal and/or anal stenosis , if this means a limitation for any surgical procedure
• Subjects with concurrent active proctitis.
• Subjects with other fistulas such as rectum/anal canal-vaginal fistulas or rectum-bladder fistulas.
• Patients have undergone a diverting stoma operation in the past, except for patients have completed stoma closure at least one month before drug administration.
• Patients plan to undergo surgery in the perianal area during the trial period due to other diseases other than anal fistula.
• 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).
• Serum virology test (HBeAg, HCV antibody, HIV antibody, Treponema pallidum antibody) positive.
• Subjects with malignant tumors or a history of malignant tumors.
• Subjects with congenital or acquired immunodeficiency.
• Contraindication to MRI scan
• Current or recent history of abnormal, severe, progressive, uncontrolled hepatic, haematological, gastrointestinal (except CD), endocrine, pulmonary, cardiac, neurological, psychiatric, or cerebral disease.
• Subjects received stem cell treatment within two years prior to screening.
• Patient with ongoing steroid treatment or treated with steroids in the last 4 weeks.
• Patient undergone major surgical operations (such as orthopedic surgery, abdominal surgery) within one month before drug administration, or Patient plan to receive surgeries during the study period (except for the surgeries required by the procedures of this trial).
• Patients with a history of drug abuse or alcoholism (Criteria for alcoholism: having a long-term drinking history of more than 5 years, with the amount of ethanol equivalent to ≥40g/d for men and ≥20g/d for women, or having a history of heavy drinking within 2 weeks, with the amount of ethanol equivalent to >80g/d. The conversion formula for the amount of ethanol (g) = the amount of alcohol consumed (ml) × the ethanol content (%) × 0.8).
• Pregnant or lactating women.
• Patients with a history of diverting ostomy, except those who have undergone stoma reversal at least 1 month prior to drug administration.
• Other diseases or conditions that the investigator deems inappropriate for participating in this clinical trial, such as poor subject compliance and the expectation of being unable to complete follow-up and evaluation.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Тройное слепое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• Jiangsu Topcel-KH Pharmaceutical Co., Ltd. — Нанкин

Идентификаторы

NCT: NCT06925594 · TH-SC01-CD-Ⅲ-01

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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