Clinical trial NCT03444220: Gut Microbiota Transplantation in Systemic Sclerosis
Official title: Reduce Disease Activity in Systemic Sclerosis by Transplantation of an Anaerobically Cultivated Human Intestinal Microbiota, a Controlled Interventional Pilot Trial
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Scleroderma, Systemic
Lead sponsor: Oslo University Hospital
Sponsor class: OTHER
First posted: 2018-02-23
Enrollment: 10
Sex: FEMALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* Fulfillment of the 2013 SSc classification criteria
* Objective GI involvement and provide informed consent.

Exclusion Criteria:

* Severe organ dysfunction (and risk of procedure related complications
* Recent exposure to antibiotics

Primary outcomes:
measure: Clinical SSc-related GI parameters
timeFrame: The change in the UCLA GIT score from baseline to week 16 will be evaluated.
Secondary outcomes:
measure: Clinical SSc activity
timeFrame: The investigators will assess all the SSc disease activity index measures at study start and week 16 .
measure: Gut microbiome composition changes
timeFrame: The investigators will assess the gut microbiome weekly from study start to week 16.
measure: Incidence of Treatment-Emergent Adverse Events (Safety)
timeFrame: Explore safety of GMT intervention at each study visit (week 0, 2, 4, 8,12 and 16)
measure: Tissue architecture changes
timeFrame: 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.
measure: Clinical SSc-related GI parameters
timeFrame: The change in the UCLA GIT score from baseline will be evaluated at each study visit (week 0,2,4,8 and 12).
Other outcomes:
Locations:
city: Oslo
country: Norway
facility: Oslo University Hospital
Study officials:
affiliation: Oslo University Hospital
name: Anna-Maria Hoffmann-Vold, PhD
role: PRINCIPAL_INVESTIGATOR
References: