Efficacy and Safety of Allogenic Cultured Adipose-derived Mesenchymal Stromal Cell Injections on MoUth Fibrosis and Handicap in Patients With Systemic sclEroderma
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 50
- 试验地点
- 5
- 主要终点
- Change in the Mouth Handicap in Systemic Sclerosis scale (MHISS)
研究概览
简要总结
Orofacial manifestations and microstomia are a frequent complication in systemic sclerosis (SSc) with a major impact on oral hygiene, dental care and quality of life. Peri-oral injection of allogeneic cultured adipose-derived stromal cells constitutes a promising approach to treat scleroderma-induced mouth fibrosis where no alternative therapy is validated. The aim of this phase 2 study is to compare efficacy and safety of perioral injection of allogeneic cultured adipose-derived stromal cells (AdMSC) versus placebo to improve oro-facial fibrosis in patients with systemic sclerosis.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •Male or female patient ≥18 years of age,
- •Patient with systemic scleroderma according to the 2013 ACR/EULAR classification criteria,
- •Mouth Handicap in Systemic Sclerosis Scale (MHISS) score more than or equal to 20 (0-48),
- •Rodnan skin score on the face more than or equal to 1,
- •Maximal mouth opening of less than 40 mm (distance between the dental arches)
- •Patient must have provided written informed consent prior to enrolment,
- •Patient must be able to understand their requirements of participating in the protocol.
- •Patient affiliated to a social security system.
排除标准
- •Patient participating in a clinical trial or having participated in a clinical trial within the previous 3 months,
- •Injection of botulinum toxin within 4 weeks prior to "inclusion visit",
- •Patient who underwent autologous hematopoietic stem cell transplantation (HSCT) within less than 1 year,
- •Local active labial herpes virus within 1 week prior to "inclusion visit",
- •Patients with an indication for intensification by autologous HSCT (according to EBMT guidelines and national MATHEC-SFGMTC guidelines),
- •History of cancer in the last five years, except for successfully excised basal cell/squamous cell carcinoma, or successfully excised early melanoma of the skin. Subjects, who had a successful tumor resection or radiation or chemotherapy more than 5 years prior to inclusion and no recurrence, may be enrolled in the study,
- •Radio- or chemotherapy in progress,
- •Females who are pregnant or breastfeeding or plan to be or do so during the course of this study,
- •Women of childbearing potential (WOCBP) who are sexually active and unwilling to use an adequate birth control method,
- •Vulnerable patient (persons deprived of their liberty by judicial or administrative decision, persons undergoing psychiatric treatment, persons admitted to a health or social establishment for purposes other than research) according to article L1121-6 of the Public Health Code
研究组 & 干预措施
AdMSC
Patients will be standard of care (physiotherapy and daily self-administered exercises) and receive allogeneic AdMSC injection in the perioral (lips) region.
干预措施: AdMSC (Drug)
Placebo
Patients will be standard of care (physiotherapy and daily self-administered exercises) and receive placebo injections in the perioral (lips) region
干预措施: Placebo (Drug)
结局指标
主要结局
Change in the Mouth Handicap in Systemic Sclerosis scale (MHISS)
时间窗: Day 0, 12 weeks after injection
the change in the Mouth Handicap in Systemic Sclerosis scale (MHISS) between baseline and week 12. An improvement of at least 5 points will be considered clinically significant
次要结局
- Safety of treatment(Day 0, 4, 12 and 24 weeks after injection)
- Efficacy on oral function by facial scanners(Day 0, 4, 12 and 24 weeks after injection)
- Efficacy on orofacial handicap(Day 0, 4, 12 and 24 weeks after injection)
- Patient satisfaction(Day 0, 4, 12 and 24 weeks after injection)
- Oral habits and hygiene(Day 0 and 24 weeks after injection)
- oral microbiota(Day 0, 12 and 24 weeks after injection)
- Plaque index(Day 0, 12 and 24 weeks after injection)
- Change in decayed missing filled teeth(Day 0 and 24 weeks after injection)
- Change in mandibular tracking(Day 0, 4, 12 and 24 weeks after injection)
- Posture by stabilometry(Day 0, 12 and 24 weeks after injection)
- psycho-social aspects and oro-facial pains(Day 0, 4, 12 and 24 weeks after injection)
- Rodnan skin score(Day 0, 12 and 24 weekds after injection)
- Dry mouth syndrome(Day 0, 4, 12 and 24 weeks after injection)
- Efficacy on aesthetics(Day 0, 4 , 12 and 24 weeks after injection)
- Immunomonitoring of vascular and antifibrotic biomarkers(Day 0 and 12 weekds after injection)