Reduce Disease Activity in Systemic Sclerosis by Transplantation of an Anaerobically Cultivated Human Intestinal Microbiota, a Controlled Interventional Pilot Trial
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 10
- 试验地点
- 2
- 主要终点
- Clinical SSc-related GI parameters
研究概览
简要总结
Systemic Sclerosis (SSc) is a progressive multi-organ disorder with high disease burden. Life expectancy in SSc is reduced by 25-40 years, mainly due to cardiopulmonary and gastro-intestinal (GI) disease involvement; and a very poor response to available treatment. Aiming to improve treatment for SSc, the ReSScue project will determine the therapeutic potential of standardized, cultivated gut microbiome transplantation (GMT), and assess the mechanisms by which this novel intervention strategy works. This approach is rationalized by studies indicating that skewed gut microbiomes could act as major, environmental risk factors in SSc; and thereby be rational targets for therapeutic manipulation. ReSScue is set up as a 4 months randomized double blind trial involving the University Hospital in Oslo. Trial participants will be recruited from the population-based, nationwide SSc cohort. Intervention will be by GMT or placebo. Primary end point is changes in SSc-related GI parameters , while secondary outcomes include safety, explorative clinical parameters, changes in oral, skin and gut microbiomes, and in immune cell phenotypes.
详细描述
This is a randomized, double blind clinical trial testing intervention by standardized GMT. The study cohort will include 10 SSc patients with GI symptoms. Evaluation of end points is at 16 weeks. The trial is set up with six study visits. Patient recruitment, eligibility screening and retrieval of informed consent will be performed at OUS before inclusion. At visit 1, and 6 patients will undergo clinical examination, PFTs, at all visits clinical examination and sampling of biological material.
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
- Female
- 接受健康志愿者
- 否
入选标准
- •Fulfillment of the 2013 SSc classification criteria
- •Objective GI involvement and provide informed consent.
排除标准
- •Severe organ dysfunction (and risk of procedure related complications
- •Recent exposure to antibiotics
研究组 & 干预措施
ACHIM
Anaerobically Cultivated Human Intestinal Microbiota
干预措施: Anaerobically Cultivated Human Intestinal Microbiota (Biological)
Placebo
Anaerobically Cultivated medium
干预措施: Anaerobically Cultivated medium (Biological)
结局指标
主要结局
Clinical SSc-related GI parameters
时间窗: The change in the UCLA GIT score from baseline to week 16 will be evaluated.
The investigators apply a patient-reported outcome measure; the validated UCLA Scleroderma Clinical Trial Consortium GIT 2.0 (UCLA GIT score 2.0) Instrument. The instrument includes seven multi-item scales: diarrhea, reflux, distention/bloating, fecal soilage, constipation, emotional well-being, and social functioning. Items are scored on a 0-3 range, and a lower value indicates a better health related quality of life. The Total GIT Score is a composite score that includes the total sum of all subgroups except constipation divided by 6. The Total GIT Score ranges from 0-3, and a lower value indicates a better health related quality of life.
次要结局
- Clinical SSc activity(The investigators will assess all the SSc disease activity index measures at study start and week 16 .)
- Clinical SSc-related GI parameters(The change in the UCLA GIT score from baseline will be evaluated at each study visit (week 0,2,4,8 and 12).)
- Gut microbiome composition changes(The investigators will assess the gut microbiome weekly from study start to week 16.)
- Tissue architecture changes(The investigators will analyse tissue architeure in the gut at week 0. 2 and 16. The skin tissue archtecture at week 0 and 16. The immune cell distribution in blood at week 0, 2, 4, 8, 12 and 16.)
- Incidence of Treatment-Emergent Adverse Events (Safety)(Explore safety of GMT intervention at each study visit (week 0, 2, 4, 8,12 and 16))
研究者
Anna-Maria Hoffmann-Vold
Principle Investigator
Oslo University Hospital