Scleroderma, SystemicPhase 1/2ReSScueNCT03444220
Gut Microbiota Transplantation in Systemic Sclerosis
A Phase 1/2 study testing Anaerobically Cultivated Human Intestinal Microbiota and Anaerobically Cultivated medium in people with Scleroderma, Systemic, run by Oslo University Hospital at 1 site in Norway.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older, women only
- Conditions the study is for
- Scleroderma, Systemic
- What is being tested?
- Anaerobically Cultivated Human Intestinal Microbiota and Anaerobically Cultivated medium
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 10 participants
- Where
- 1 siteNorway
- Study dates
- 18 Jan 2018to 15 Oct 2018
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Women only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Fulfillment of the 2013 SSc classification criteria
- Objective GI involvement and provide informed consent.
Who cannot join
- Severe organ dysfunction (and risk of procedure related complications
- Recent exposure to antibiotics
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Reduce Disease Activity in Systemic Sclerosis by Transplantation of an Anaerobically Cultivated Human Intestinal Microbiota, a Controlled Interventional Pilot Trial
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.
Read the 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.
- Conditions
- Scleroderma, Systemic
- Being tested
- Anaerobically Cultivated Human Intestinal Microbiota, Anaerobically Cultivated medium
- Medical subject headings
- Scleroderma, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Anaerobically Cultivated Human Intestinal MicrobiotaBiological
30ml ACHIM will be administrated at week 0 and 2 with 14 days apart
Anaerobically Cultivated mediumBiological
30ml medium will be administrated at week 0 and 2 with 14 days apart
| Study group | Role | What they receive |
|---|---|---|
| ACHIM | Active comparator | Anaerobically Cultivated Human Intestinal Microbiota Receives: Biological: Anaerobically Cultivated Human Intestinal Microbiota |
| Placebo | Placebo comparator | Anaerobically Cultivated medium Receives: Biological: Anaerobically Cultivated medium |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical SSc-related GI parameters
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.
Measured: The change in the UCLA GIT score from baseline to week 16 will be evaluated.
Secondary outcomes (5)
Clinical SSc activity
Explore the effect of GMT on known clinical outcome measures. The investigators base these assessments on the validated SSc disease activity index which includes a set value for active disease and a definition of change.
Measured: The investigators will assess all the SSc disease activity index measures at study start and week 16 .
Gut microbiome composition changes
It is unknown how the gut microbiome composition changes after GMT and how long these changes persist. We will investigate these aspects, as well as the mechanisms by which ACHIM exert its effects. Specifically, the investigators will address how the intestinal IgA binding to gut commensals and other bacteria is regulated.
Measured: The investigators will assess the gut microbiome weekly from study start to week 16.
Incidence of Treatment-Emergent Adverse Events (Safety)
We define an adverse event as any untoward medical occurrence that may arise during the study period. The study investigator will fill out a safety form at each study visit (week, 0, 2, 4, 8, 12 and 16). The following questions will be asked: Hospital admission or contact with primary physician since last visit and reason why. Diarrhea, constipation, stomach pain, nausea, vomiting or fever since last study visit. Use of antibiotics since last visit.
Measured: Explore safety of GMT intervention at each study visit (week 0, 2, 4, 8,12 and 16)
Tissue architecture changes
Explore effects of GMT on tissue architecture in gut and skin, and on immune cell distribution in blood.
Measured: 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.
Clinical SSc-related GI parameters
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.
Measured: The change in the UCLA GIT score from baseline will be evaluated at each study visit (week 0,2,4,8 and 12).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Feb 2018
- Study start18 Jan 2018
- Primary completion18 May 2018
- Study completion15 Oct 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Scleroderma, SystemicStudies of the same condition, open and under way.
- More trials run by Oslo University HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Oslo University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Anna-Maria Hoffmann-Vold, Oslo University Hospital
- Investigators
- Anna-Maria Hoffmann-Vold, PhD, Principal investigator, Oslo University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03444220
- Sponsor's protocol ID
- 2016/1529
- Acronym
- ReSScue
- Status last verified
- Mar 2019
- First submitted
- 13 Feb 2018
- Last updated
- 13 Mar 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Strahm N, Didriksen H, Fretheim H, Molberg O, Midtvedt O, Farstad IN, Midtvedt T, Lundin KEA, Aabakken L, Blyszczuk P, Distler O, Kania G, Hoffmann-Vold AM. Effects of faecal microbiota transplantation on the small intestinal mucosa in systemic sclerosis. Rheumatology (Oxford). 2023 Aug 1;62(8):2918-2929. doi: 10.1093/rheumatology/kead014.
PubMed 36688692
Study at a glance
From the registry record, last updated by the sponsor on 13 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Reduce Disease Activity in Systemic Sclerosis by Transplantation of an Anaerobically Cultivated Human Intestinal Microbiota, a Controlled Interventional Pilot Trial
- ClinicalTrials.gov ID
- NCT03444220
- Acronym / sponsor ID
- ReSScue / 2016/1529
- Conditions
- Scleroderma, Systemic
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Oslo University HospitalUniversity, hospital or other
- Study start
- 18 Jan 2018 (actual)
- Primary completion
- 18 May 2018 (actual)
- Study completion
- 15 Oct 2018 (actual)
- First posted
- 23 Feb 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ACHIM | Active comparator | Biological: Anaerobically Cultivated Human Intestinal MicrobiotaAnaerobically Cultivated Human Intestinal Microbiota |
| Placebo | Placebo comparator | Biological: Anaerobically Cultivated mediumAnaerobically Cultivated medium |
Who may qualify
- Ages
- 18 years and older
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Fulfillment of the 2013 SSc classification criteria
- Objective GI involvement and provide informed consent.
Key exclusion criteria (2 listed)
- Severe organ dysfunction (and risk of procedure related complications
- Recent exposure to antibiotics
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Anaerobically Cultivated Human Intestinal Microbiota30ml ACHIM will be administrated at week 0 and 2 with 14 days apart | Biological | ACHIM |
| Anaerobically Cultivated medium30ml medium will be administrated at week 0 and 2 with 14 days apart | Biological | Placebo |
Primary outcome
Clinical SSc-related GI parameters
Time frame: 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.
5 secondary outcomes
Clinical SSc activity
Time frame: The investigators will assess all the SSc disease activity index measures at study start and week 16 .
Gut microbiome composition changes
Time frame: The investigators will assess the gut microbiome weekly from study start to week 16.
Incidence of Treatment-Emergent Adverse Events (Safety)
Time frame: Explore safety of GMT intervention at each study visit (week 0, 2, 4, 8,12 and 16)
Tissue architecture changes
Time frame: 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.
Clinical SSc-related GI parameters
Time frame: The change in the UCLA GIT score from baseline will be evaluated at each study visit (week 0,2,4,8 and 12).
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Mar 2019.
- Derived from this study: PubMed 36688692Strahm N, Didriksen H, Fretheim H, Molberg O, Midtvedt O, Farstad IN, Midtvedt T, Lundin KEA, Aabakken L, Blyszczuk P, Distler O, Kania G, Hoffmann-Vold AM. Effects of faecal microbiota transplantation on the small intestinal mucosa in systemic sclerosis. Rheumatology (Oxford). 2023 Aug 1;62(8):2918-2929. doi: 10.1093/rheumatology/kead014.
Related trials for Scleroderma, Systemic
Active, not recruiting
Patient Reported Outcomes With UVA-1 Therapy for Treatment of Sclerosing Skin Diseases
Scleroderma, Systemic, Morphea
RecruitingPhase 1
A Safety and Efficacy Study Evaluating CTX112 in Adult Subjects With Refractory Autoimmune Disease
SLE (Systemic Lupus), Lupus Erythematosus, Systemic
RecruitingObservational study
Collection of Airway, Blood and/or Urine Specimens From Subjects for Research Studies
Chronic Obstructive Pulmonary Disease (COPD), Smoking
Recruiting
An Clinical Study of YTS109 Cell Injection in Subjects With Recurrent/Refractory Autoimmune Disease
Systemic Lupus Erythematosus, Systemic Sclerosis
Not yet recruitingObservational study
A Clinical Study on Systemic Sclerosis and the Validation of French-language Questionnaires Assessing Disability, Quality of Life, and Disease Status.
Scleroderma
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Anna-Maria Hoffmann-Vold, PhD principal investigator
Oslo University Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Anaerobically Cultivated Human Intestinal Microbiota and Anaerobically Cultivated medium in people with Scleroderma, Systemic, run by Oslo University Hospital at 1 site in Norway.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Norway. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Clinical SSc-related GI parameters (time frame: The change in the UCLA GIT score from baseline to week 16 will be evaluated.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03444220, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Mar 2019.