Dose Escalation of Allogeneic Adipose Derived Stroma/Stem Cells for the Treatment of Crohn's Fistula:a Phase I/II Clinical Study
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 9
- 试验地点
- 1
- 主要终点
- adverse events of grade ≥ 2 at 6 months
研究概览
简要总结
Perianal fistulas are in the forefront (42 to 72, 4%) of morbid complication of Crohn's disease, affecting nearly one- third of patients and complicating abscesses in 35-48% of cases. The current treatment is based on the combination of drainage (proctologic and surgical), and biologics techniques, but the failure rate varies from 30 to 80%. Actually, innovative cell therapy procedures are validated by Cell-Easy with the use of allogenic mesenchymal stem cells for the immunomodulatory, anti-inflammatory, angiogenic and trophic properties (CellReady®) and represent a promising option in the treatment of perianal fistulas associated with Crohn's disease. This phase I/II study is designed to evaluate the treatment of complex perianal fistulas associated with Crohn's disease, after failure of conventional treatment by injection of allogeneic cultured adipose-derived stromal cell (AdMSC) into the fistula.
详细描述
The injection of adipose stromal cells is currently evaluated in clinical studies for repair-damaged tissues in various diseases (limb ischemia, osteoarthritis, systemic slerosis...). Immunoregulatory and anti-inflammatory properties of AdMSC's are responsible for accelerating healing and represents an innovative approach to treat perianal fistulas associated with Crohn's disease.
This phase I/II study is designed to evaluate the treatment of complex perianal fistulas associated with Crohn's disease, after failure of conventional treatment by injection of AdMSC (CellReady®) into the fistula.
Different doses of AdMSC will be tested for a dose escalation (5.10*7 and 10.10*7 cells) and injected in the in the wall of the fistula.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients over 18 years old,
- •Patients who signed the informed consent,
- •Patient affiliated to a social security system,
- •Controlled luminal Crohn's disease characterized by an Harvey-Bradshaw score less or equal than 8 and diagnosed on clinical, endoscopic, histological and/or radiological criteria for more than 3 months,
- •Colonoscopy less than a year old without ulcer in the rectum,
- •Presence of complex chronic perianal fistula with a maximum of two internal ports and three external ports,
- •Patient treated with a combined treatment (drainage on setons + anti-TNFα) and who failed conventional treatment after 6 months and whose intraluminal disease (intestinal damage) is controlled
排除标准
- •Refusal of the patient to participate in the study,
- •Positive QuantiFERON test,
- •Patient with transplanted organ,
- •History of cancer in the last five years or lympho-proliferative disease,
- •Persistent bacterial or viral infection,
- •Patient with a contraindication to MRI,
- •Known allergy to Gadolinium,
- •Known allergy to Albumine,
- •End-stage organ failure,
- •Pregnant or breastfeeding women,
- •Women of childbearing age without effective contraception throughout the duration of the study,
- •Patient under judicial protection, under guardianship or curatorship.
- •Patient previously treated with ALOFISEL®
研究组 & 干预措施
AdMSC (CellReady®)
Different doses of AdMSC will be tested for a dose escalation (5.10*7 and 10.10*7 cells) and injected in the in the wall of the fistula
干预措施: AdMSC (CellReady®) (Drug)
结局指标
主要结局
adverse events of grade ≥ 2 at 6 months
时间窗: 6 months after injection
Number of adverse events of grade ≥ 2 related to the experimental treatment (CellReady®) or related to surgical/medical procedures,
Efficacy of AdMSC by clinical evaluation at 6 months
时间窗: 6 months after injection
This evaluation will be measured by the presence or absence of flow through the internal or external orifice see in anoscopy and through the external orifice
Efficacy of AdMSC by radiological evaluation at 6 months
时间窗: 6 months after injection
This evaluation will be measured by disappearance of fistula tract or fistula tract present but inactive.
次要结局
- Safety at 1 and 3 and 6 months(1, 3 and 6 months after injection)
- Efficacy of AdMSC by clinical evaluation 1 and 3 months(1 and 3 months after injection)
- Efficacy of AdMSC by biological evaluation(1, 3 and 6 months after injection)
- Change from Baseline in quality of life(1, 3 and 6 months after injection)
- Change from Baseline in disease activity(1, 3 and 6 months after injection)
- Change from Baseline in perianal disease activity(1, 3 and 6 months after injection)
- Change from Baseline in lesions(1, 3 and 6 months after injection)
- Change from Baseline in anal incontinence(1, 3 and 6 months after injection)
- Change from Baseline in patient symptoms(1, 3 and 6 months after injection)
- tryptophan metabolists(3 and 6 months after injection)
- faecal microbiota(3 and 6 months after injection)