Sjogren's SyndromePhase 1NCT03926286
Fecal Microbial Transplant (FMT) for Sjogrens Syndrome
A Phase 1 study testing FMP-30 in people with Sjogren's Syndrome, run by University of Miami at 1 site in United States.
Trial summary
This is an open label study to evaluate the effect of Fecal Microbiota Transplantation (FMT) on the gut microbiome and Systemic parameters. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sjogren's Syndrome
- What is being tested?
- FMP-30
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Apr 2019to 1 Jun 2020
- Phase
- Phase 1
- 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
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Positive diagnosis of Sjogrens syndrome, defined by meeting two or more of the following three criteria:
- Positive serum anti-SS-A/Ro and/or anti-SS-B/La (or positive rheumatoid factor and ANA ≥ 1:320)
- Labial salivary gland biopsy exhibiting focal lymphocytic sialadenitis with a focus score ≥ focus/4 mm2
- Keratoconjunctivitis sicca with ocular staining score ≥ 3 (assuming that individual is not currently using daily eye drops for glaucoma, and has not had corneal surgery or cosmetic eyelid surgery in the last 5 years)
- Or by both of the following:
- Positive antibodies to one of the early markers of Sjogrens Syndrome:
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- Anti-salivary gland protein 1 (SP1)
- Anti-carbonic anhydrase 6 (CA6)
- Parotid secretory protein (PSP) Ocular staining score ≥ 3
- Age ≥ 18 years at time of enrollment
- Able to provide signed and dated informed consent
- Women of child childbearing potential in sexual relationships with men must use an acceptable method of contraception§ from 30 days prior to enrollment until 4 weeks after completing study treatment.
- Males must agree to avoid impregnation of women during and for four weeks after completing study treatment through use of an acceptable method of contraception*.
- Includes, but is not limited to, barrier with additional spermicidal foam or jelly, intrauterine device, hormonal contraception (started at least 30 days prior to study enrollment), intercourse with men who underwent vasectomy.
- Includes, but is not limited to, barrier with additional spermicidal foam or jelly and vasectomy.
- Participant exclusion criteria
Who cannot join
- Inability to provide informed consent and/or unable or unwilling to comply with protocol requirements.
- Antibiotics for at least 2 weeks prior to FMT.
- Active infection for >15 days: bacteremia, urinary tract infection, pneumonia or abdominal collection.
- Known or suspected toxic megacolon and/or known small bowel ileus.
- Previous FMT
- Major gastrointestinal surgery (e.g. significant bowel resection) within 3 months before enrollment. This does not include appendectomy or cholecystectomy.
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- History of total colectomy or bariatric surgery.
- Antibiotics for the treatment of an active infection or anticipated antibiotic use during trial duration.
- Concurrent intensive induction chemotherapy, radiation therapy or biological treatment for active malignancy.
- Expected life expectancy < 6 months
- Patients with a history of severe anaphylactic or anaphylactoid food allergy.
- Solid organ transplant patients ≤ 90 days post-transplant or on active treatment for rejection.
- Neutropenia (<500 neutrophils/mL) or other severe immunosuppression. Patients on monoclonal antibodies to B and T cells, anti-tumor necrosis factor, glucocorticoids, antimetabolites, calcineurin inhibitors may be enrolled after consultation with their medical doctor.
- Renal failure (GFR <30 or dialysis)
- Human immunodeficiency virus+ controlled or not well controlled on antiretroviral therapy
- Regular probiotic supplement use within prior 2 weeks to enrollment
- Pregnancy or inability/unwillingness to use contraceptives.
- A condition that would jeopardize the safety or rights of the subject, would make it unlikely for the subject to complete the study, or would confound the results of the study.
- Exclusion on the discretion of the PI.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Fecal Microbial Transplant for Sjogrens Syndrome
This is an open label study to evaluate the effect of Fecal Microbiota Transplantation (FMT) on the gut microbiome and Systemic parameters.
- Conditions
- Sjogren's Syndrome
- Being tested
- FMP-30
- Keywords
- Dry Eye, Auto-Immune Disorder
- Medical subject headings
- Sjogren's Syndrome, Dry Eye Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FMP-30Drug
FMP-30 containing frozen human fecal microbiota administered as (3) units of FMP30 enema on Day 0 and Week1
| Study group | Role | What they receive |
|---|---|---|
| FMT for Sjogrens | Experimental | FMT- active ingredient coming from participant's screening stool Receives: Drug: FMP-30 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with reported adverse events (AEs) and serious adverse events (SAEs)
As an evaluation of the safety of FMT, the number of participants with reported AEs and SAEs will be collected. All occurrences of AEs and SAEs, regardless of relatedness to FMT, will be reported and assessed by the clinician using the NIH CTCAE.
Measured: 7 months
Number of participants with stable microbiome engraftment
Engraftment will be analyzed via the Jensen-Shannon divergence (JSD). The engraftment scores will be the ratio between the donors and recipients at the bacterial genus level. Participants who successfully engraft will more closely resemble the donor microbial profile on JSD analysis.
Measured: month 3
Secondary outcomes (11)
Change in dry eye symptoms
Dry eye symptoms will be measured by the Ocular Surface Disease Index (OSDI) Scale 0-100 Continuous with higher scores representing greater dry eye symptoms
Measured: baseline, 1 week, 1 month, 3 months
Change in dry eye symptoms
Dry eye symptoms will be measured by the Dry Eye Questionnaire 5 (DEQ5) Scale Range 0-22 Continuous with a higher number representing greater symptomatology of dry eye
Measured: baseline, 1 week, 1 month, 3 months
Change in diversity of bacterial communities
This will be captured via high-throughput 16S gene sequencing using DNA extracted from stool specimens in study participants. The Shannon diversity index will be used as our primary measure of diversity.
Measured: Pre FMT, 3 months post FMT
Change in system immune profiles as measured by T cell populations
System immune profiles will be evaluated by completing a comprehensive immuno-phenotypic profile from blood samples evaluating T cell populations including Th1, Th17, and T regulatory cells.
Measured: Pre-FMT, 1 Week, 1 Month, 3 months post FMT
Change in ocular and systemic symptoms as measured by the quality of life SF-12 Questionnaire
Ranges 0-100 with higher scores representing a better quality of life
Measured: Pre-FMT, 1 Week, 1 Month, 3 months post FMT
Change in self-reported ocular pain as assessed by the Numerical Rating Scale(NRS)
NRS Scoring Ranges from 0-10 with 0=no pain sensation and 10=the most intense eye pain imaginable
Measured: Pre-FMT, 1 Week, 1 Month, 3 months post FMT
Change in self-reported ocular pain as assessed by the Short-form McGill Pain Questionnaire(SFM-PQ)
SFM-PQ Scoring Ranges from 0-45 with zero to 45 with a higher score indicating more server eye pain
Measured: Pre-FMT, 1 Week, 1 Month, 3 months post FMT
Change in self-reported ocular pain as assessed by the Neuropathic Pain Symptom Inventory (NPSI)
NPSI Scoring Ranges from 0-100 with the higher score indicating the worse pain imaginable.
Measured: Pre-FMT, 1 Week, 1 Month, 3 months post FMT
Depression as assessed by the Patient Health Questionnaire-9 (PHQ-9)
PHQ-9 scoring Ranges from 0-27 with the higher score indicating a greater degree of depression
Measured: Pre-FMT, 1 Week, 1 Month, 3 months post FMT
Depression as assessed by the Symptom Checklist 90 for Depression (SCL-90 Depression)
SCL-90 Depression scoring ranges from 0-4 with the higher score indicating a greater degree of depression.
Measured: Pre-FMT, 1 Week, 1 Month, 3 months post FMT
Anxiety as assessed by the Symptom Checklist 90 for Anxiety (SCL-90 Anxiety)
SCL-90 Anxiety scoring ranges from 0-4 with the higher score indicating a greater degree of anxiety.
Measured: Pre-FMT, 1 Week, 1 Month, 3 months post FMT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Apr 2019
- Study start15 Apr 2019
- Primary completion1 Jun 2020
- Study completion1 Jun 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Sjogren's SyndromeStudies of the same condition, open and under way.
- More trials run by University of MiamiThe 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Miami
- Sponsor type
- University, hospital or other
- Collaborators
- Sjogrens Syndrome Foundation, Microbiome Health Research Institute
- Responsible party
- Principal investigator, Anat Galor, University of Miami
- Investigators
- Anat Galor, MD, MSPH, Principal investigator, University of Miami
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03926286
- Sponsor's protocol ID
- 20170733
- Status last verified
- Jun 2020
- First submitted
- 16 Apr 2019
- Last updated
- 5 Jun 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Zaheer M, Wang C, Bian F, Yu Z, Hernandez H, de Souza RG, Simmons KT, Schady D, Swennes AG, Pflugfelder SC, Britton RA, de Paiva CS. Protective role of commensal bacteria in Sjogren Syndrome. J Autoimmun. 2018 Sep;93:45-56. doi: 10.1016/j.jaut.2018.06.004. Epub 2018 Jun 20.
PubMed 29934134De Luca F, Shoenfeld Y. The microbiome in autoimmune diseases. Clin Exp Immunol. 2019 Jan;195(1):74-85. doi: 10.1111/cei.13158.
PubMed 29920643Wang C, Zaheer M, Bian F, Quach D, Swennes AG, Britton RA, Pflugfelder SC, de Paiva CS. Sjogren-Like Lacrimal Keratoconjunctivitis in Germ-Free Mice. Int J Mol Sci. 2018 Feb 13;19(2):565. doi: 10.3390/ijms19020565.
PubMed 29438346de Paiva CS, Jones DB, Stern ME, Bian F, Moore QL, Corbiere S, Streckfus CF, Hutchinson DS, Ajami NJ, Petrosino JF, Pflugfelder SC. Altered Mucosal Microbiome Diversity and Disease Severity in Sjogren Syndrome. Sci Rep. 2016 Apr 18;6:23561. doi: 10.1038/srep23561.
PubMed 27087247
Study at a glance
From the registry record, last updated by the sponsor on 5 Jun 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Fecal Microbial Transplant for Sjogrens Syndrome
- ClinicalTrials.gov ID
- NCT03926286
- Acronym / sponsor ID
- 20170733
- Conditions
- Sjogren's Syndrome
- Keywords
- Dry Eye, Auto-Immune Disorder
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- University of MiamiUniversity, hospital or other
- Collaborators
- Sjogrens Syndrome Foundation, Microbiome Health Research Institute
- Study start
- 15 Apr 2019 (actual)
- Primary completion
- 1 Jun 2020 (actual)
- Study completion
- 1 Jun 2020 (actual)
- First posted
- 24 Apr 2019 (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.
This is an open label study to evaluate the effect of Fecal Microbiota Transplantation (FMT) on the gut microbiome and Systemic parameters.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| FMT for Sjogrens | Experimental | Drug: FMP-30FMT- active ingredient coming from participant's screening stool |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- Positive diagnosis of Sjogrens syndrome, defined by meeting two or more of the following three criteria:
- Age ≥ 18 years at time of enrollment
- Able to provide signed and dated informed consent
- Women of child childbearing potential in sexual relationships with men must use an acceptable method of contraception§ from 30 days prior to enrollment until 4 weeks after completing study treatment.
- Males must agree to avoid impregnation of women during and for four weeks after completing study treatment through use of an acceptable method of contraception*.
Key exclusion criteria (19 listed)
- Inability to provide informed consent and/or unable or unwilling to comply with protocol requirements.
- Antibiotics for at least 2 weeks prior to FMT.
- Active infection for >15 days: bacteremia, urinary tract infection, pneumonia or abdominal collection.
- Known or suspected toxic megacolon and/or known small bowel ileus.
- Previous FMT
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 |
|---|---|---|
| FMP-30FMP-30 containing frozen human fecal microbiota administered as (3) units of FMP30 enema on Day 0 and Week1 | Drug | FMT for Sjogrens |
Primary outcome
Number of participants with reported adverse events (AEs) and serious adverse events (SAEs)
Time frame: 7 months
As an evaluation of the safety of FMT, the number of participants with reported AEs and SAEs will be collected. All occurrences of AEs and SAEs, regardless of relatedness to FMT, will be reported and assessed by the clinician using the NIH CTCAE.
Number of participants with stable microbiome engraftment
Time frame: month 3
Engraftment will be analyzed via the Jensen-Shannon divergence (JSD). The engraftment scores will be the ratio between the donors and recipients at the bacterial genus level. Participants who successfully engraft will more closely resemble the donor microbial profile on JSD analysis.
11 secondary outcomes
Change in dry eye symptoms
Time frame: baseline, 1 week, 1 month, 3 months
Change in dry eye symptoms
Time frame: baseline, 1 week, 1 month, 3 months
Change in diversity of bacterial communities
Time frame: Pre FMT, 3 months post FMT
Change in system immune profiles as measured by T cell populations
Time frame: Pre-FMT, 1 Week, 1 Month, 3 months post FMT
Change in ocular and systemic symptoms as measured by the quality of life SF-12 Questionnaire
Time frame: Pre-FMT, 1 Week, 1 Month, 3 months post FMT
Change in self-reported ocular pain as assessed by the Numerical Rating Scale(NRS)
Time frame: Pre-FMT, 1 Week, 1 Month, 3 months post FMT
Change in self-reported ocular pain as assessed by the Short-form McGill Pain Questionnaire(SFM-PQ)
Time frame: Pre-FMT, 1 Week, 1 Month, 3 months post FMT
Change in self-reported ocular pain as assessed by the Neuropathic Pain Symptom Inventory (NPSI)
Time frame: Pre-FMT, 1 Week, 1 Month, 3 months post FMT
Depression as assessed by the Patient Health Questionnaire-9 (PHQ-9)
Time frame: Pre-FMT, 1 Week, 1 Month, 3 months post FMT
Depression as assessed by the Symptom Checklist 90 for Depression (SCL-90 Depression)
Time frame: Pre-FMT, 1 Week, 1 Month, 3 months post FMT
Anxiety as assessed by the Symptom Checklist 90 for Anxiety (SCL-90 Anxiety)
Time frame: Pre-FMT, 1 Week, 1 Month, 3 months post FMT
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 5 Jun 2020.
- Background: PubMed 29934134Zaheer M, Wang C, Bian F, Yu Z, Hernandez H, de Souza RG, Simmons KT, Schady D, Swennes AG, Pflugfelder SC, Britton RA, de Paiva CS. Protective role of commensal bacteria in Sjogren Syndrome. J Autoimmun. 2018 Sep;93:45-56. doi: 10.1016/j.jaut.2018.06.004. Epub 2018 Jun 20.
- Background: PubMed 29920643De Luca F, Shoenfeld Y. The microbiome in autoimmune diseases. Clin Exp Immunol. 2019 Jan;195(1):74-85. doi: 10.1111/cei.13158.
- Background: PubMed 29438346Wang C, Zaheer M, Bian F, Quach D, Swennes AG, Britton RA, Pflugfelder SC, de Paiva CS. Sjogren-Like Lacrimal Keratoconjunctivitis in Germ-Free Mice. Int J Mol Sci. 2018 Feb 13;19(2):565. doi: 10.3390/ijms19020565.
- Background: PubMed 27087247de Paiva CS, Jones DB, Stern ME, Bian F, Moore QL, Corbiere S, Streckfus CF, Hutchinson DS, Ajami NJ, Petrosino JF, Pflugfelder SC. Altered Mucosal Microbiome Diversity and Disease Severity in Sjogren Syndrome. Sci Rep. 2016 Apr 18;6:23561. doi: 10.1038/srep23561.
- Individual participant dataSharing planned: No
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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.
Anat Galor, MD, MSPH principal investigator
University of Miami
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing FMP-30 in people with Sjogren's Syndrome, run by University of Miami at 1 site in United States.
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 16 inclusion and 19 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 5 Jun 2020. 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 United States. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this 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.
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?
Number of participants with reported adverse events (AEs) and serious adverse events (SAEs) (time frame: 7 months). The study lists 2 primary outcomes in all. 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 NCT03926286, which also lists 4 linked publications.
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 5 Jun 2020.