Rectal FistulaPhase 1NCT05407766
Mesenchymal Stem Cells (MSCs) for Perianal Fistula
A Phase 1 study testing OSSM-001 in people with Rectal Fistula, run by Ossium Health, Inc..
Trial summary
OSSM-001 will be evaluated in a double blind, randomized phase I safety study of a single injection of 100 and 300 million bone marrow derived MSCs in 16 patients with refractory perianal fistulas in the setting of Crohn's disease. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Rectal Fistula
- What is being tested?
- OSSM-001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Aug 2022to Aug 2024
- 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 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Men and Women 18-75 years of age with a diagnosis of unhealing perianal fistulas in the setting of Crohn's disease and must be on maximal standard of care therapy (e.g., antibiotics, 5-ASA, and immunomodulator, anti-TNF, anti-integrin, and anti-interleukin therapies) for at least six months duration.
- Presence of perianal fistula with maximum of two internal fistula tracts and a maximum of three external openings based on clinical assessment and MRI. Fistula must have been draining for at least 6 weeks prior to the screening visit.
- Medically refractory perianal fistulizing Crohn's disease (i.e., standard of care such as antibiotics, immunosuppressives)
- Have no contraindications to MR evaluations: e.g., pacemaker or magnetically active metal fragments, claustrophobia
- Ability to comply with protocol
- Competent and able to provide written informed consent
- Concurrent Crohn's-related therapies with stable doses corticosteroids, 5- ASA drugs, immunomodulators, anti-TNF therapy, anti-integrin and anti-interleukin therapies are permitted.
- A female participant and of childbearing potential must have a negative serum or urine pregnancy test at time of screening, and must not plan to become pregnant during the study.
Who cannot join
- Inability to give informed consent.
- Clinically significant medical conditions within the six months before administration of MSCs: e.g. myocardial infarction, active angina, congestive heart failure or other conditions that would, in the opinion of the investigators, compromise the safety of the patient.
- Specific exclusions: Hepatitis B or C, HIV, Abnormal AST or ALT at screening
- History of cancer including melanoma (with the exception of localized skin cancers) in the past five years
- Received treatment with an investigational drug or device within 60 days of randomization or have not completed 5 half-life washout (whichever is later) for drugs or biologics. If the half-life of the product is not known, then subjects cannot be randomized within 6 months of investigation drug treatment.
- Participated in a cell therapy-based trial within 6 months before randomization
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- Participated in Crohn's/ulcerative colitis or any fistulizing Crohn's study within 6 months before randomization
- Pregnant or breast feeding or trying to become pregnant.
- Presence of a rectovaginal or perineal body fistula
- Change in Crohn's immunosuppressive regimen prior to enrollment. Subjects should be on stable, maximal medical therapy for 4 months prior to enrollment.
- Uncontrolled intestinal Crohn's disease which will require escalation for medical therapy or surgery within 3 months of enrollment
- Severe anal canal disease that is stenotic and requires dilation or severe proctitis making difficult to the surgery
- A female or male participant unwilling to agree to use acceptable contraception methods during participation in study. Acceptable contraception methods include: Implants, injectables, combined oral contraceptives, an intrauterine device, a bilateral tubal ligation, a vasectomy, a vasectomized partner for female participant, double-barrier methods, and physical barrier if male participant is not vasectomized
- Associated perianal abscess(es).
- Laboratory exclusions: Serum creatinine levels >1.5 times the upper limit of normal (ULN). Total bilirubin >1.5 × ULN. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >5.0 × ULN. Hemoglobin <10.0 g/dL for females or <11.0 g/dL for males. Platelets <75.0 × 109/L.
- Any contraindications to MRI or surgical or anesthetic procedure(s)
- Any major GI surgery or major perianal local surgery within 6 months of randomization
About this study
Official title: A Phase I Safety Study of Allogeneic Bone Marrow Derived MSCs for Refractory Perianal Fistulizing Crohn's Disease
OSSM-001 will be evaluated in a double blind, randomized phase I safety study of a single injection of 100 and 300 million bone marrow derived MSCs in 16 patients with refractory perianal fistulas in the setting of Crohn's disease. There will be 2 cohorts (100M and 300M MSCs); 8 subjects in each cohort; 6 subjects receiving the investigational product and 2 subjects receiving placebo (normal saline) in each cohort.
- Conditions
- Rectal Fistula
- Being tested
- OSSM-001, Placebo
- Medical subject headings
- Rectal Fistula
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OSSM-001Biological
Single injection of OSSM-001 at dose of 100M or 300M cells
PlaceboOther
Single injection of placebo (saline)
| Study group | Role | What they receive |
|---|---|---|
| 100M of OSSM-001 | Experimental | Single injection of mesenchymal stem cells at dose of 100M Receives: Biological: OSSM-001 |
| 300M of OSSM-001 | Experimental | Single injection of mesenchymal stem cells at dose of 300M Receives: Biological: OSSM-001 |
| Placebo | Placebo comparator | Single injection of normal saline Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
OSSM-001 (100M dose) related adverse events as assessed by CTCAE v5.0
To determine the safety of a single injection of 100M allogeneic bone marrow MSCs for the treatment of refractory perianal fistulizing Crohn's disease by assessing OSSM-001 related adverse events as assessed by CTCAE v5.0.
Measured: 12 weeks
OSSM-001 (300M dose) related adverse events as assessed by CTCAE v5.0
To determine the safety of a single injection of 300M allogeneic bone marrow MSCs for the treatment of refractory perianal fistulizing Crohn's disease by assessing OSSM-001 related adverse events as assessed by CTCAE v5.0.
Measured: 12 weeks
Secondary outcomes (4)
Fistula healing assessed by radiographic (MRI) assessments for OSSM-001 (100M dose)
To assess fistula healing induced by the delivery of a single injection of 100M allogeneic bone marrow derived MSCs on refractory perianal fistulizing Crohn's disease using radiographic assessment (MRI) assessments.
Complete healing defined as MRI with an absence of a fluid collection >2cm in 3 of 3 dimensions, lack of edema, inflammation, or sign of active inflammatory response. A remnant of scar of a fistula tract may remain.
Partial healing defined as MRI with an absence of a fluid collection >2cm in 2 of 3 dimensions, lack of edema, inflammation, or sign of active inflammatory response. A remnant of scar of a fistula tract may remain.
Lack of response defined as healing which does not meet the threshold for partial healing.
Worsening disease defined as MRI with fluid collection >2cm in 2 of 3 dimensions, edema, inflammation, or sign of active inflammatory response. An increased number of tracts may be seen, or increased branching from the primary tract.
Measured: 12 weeks
Fistula healing assessed by clinical assessments for OSSM-001 (100M dose)
To assess fistula healing induced by the delivery of a single injection of 100M allogeneic bone marrow derived MSCs on refractory perianal fistulizing Crohn's disease using clinical assessments.
Complete healing defined as 100% cessation of drainage on clinical exam and with gentle finger compression.
Partial healing defined as greater than or equal to 50% cessation of drainage on clinical exam with gentle finger compression.
Lack of response defined as healing which does not meet the threshold for partial healing.
Worsening disease defined as increased drainage on clinical exam.
Measured: 12 weeks
Fistula healing assessed by radiographic (MRI) assessments for OSSM-001 (300M dose)
To assess fistula healing induced by the delivery of a single injection of 300M allogeneic bone marrow derived MSCs on refractory perianal fistulizing Crohn's disease using radiographic assessment (MRI) assessments.
Complete healing defined as MRI with an absence of a fluid collection >2cm in 3 of 3 dimensions, lack of edema, inflammation, or sign of active inflammatory response. A remnant of scar of a fistula tract may remain.
Partial healing defined as MRI with an absence of a fluid collection >2cm in 2 of 3 dimensions, lack of edema, inflammation, or sign of active inflammatory response. A remnant of scar of a fistula tract may remain.
Lack of response defined as healing which does not meet the threshold for partial healing.
Worsening disease defined as MRI with fluid collection >2cm in 2 of 3 dimensions, edema, inflammation, or sign of active inflammatory response. An increased number of tracts may be seen, or increased branching from the primary tract.
Measured: 12 weeks
Fistula healing assessed by clinical assessments for OSSM-001 (300M dose)
To assess fistula healing induced by the delivery of a single injection of 300M allogeneic bone marrow derived MSCs on refractory perianal fistulizing Crohn's disease using clinical assessments.
Complete healing defined as 100% cessation of drainage on clinical exam and with gentle finger compression.
Partial healing defined as greater than or equal to 50% cessation of drainage on clinical exam with gentle finger compression.
Lack of response defined as healing which does not meet the threshold for partial healing.
Worsening disease defined as increased drainage on clinical exam.
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jun 2022
- Study startAug 2022 (estimated)
- Primary completionApr 2024 (estimated)
- Study completionAug 2024 (estimated)
Why it stopped: OSSM-001 will not be used in this clinical program.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rectal FistulaStudies of the same condition, open and under way.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ossium Health, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sagar Munjal, MD, Study director, Ossium Health, Inc.Amy Lightner, MD, Principal investigator, The Cleveland Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05407766
- Sponsor's protocol ID
- OSSM-001-001-01
- Status last verified
- Dec 2022
- First submitted
- 9 May 2022
- Last updated
- 23 Dec 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Safety Study of Allogeneic Bone Marrow Derived MSCs for Refractory Perianal Fistulizing Crohn's Disease
- ClinicalTrials.gov ID
- NCT05407766
- Acronym / sponsor ID
- OSSM-001-001-01
- Conditions
- Rectal Fistula
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: OSSM-001 will not be used in this clinical program.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Ossium Health, Inc.Industry (pharma, biotech, devices)
- Study start
- Aug 2022 (estimated)
- Primary completion
- Apr 2024 (estimated)
- Study completion
- Aug 2024 (estimated)
- First posted
- 7 Jun 2022 (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.
OSSM-001 will be evaluated in a double blind, randomized phase I safety study of a single injection of 100 and 300 million bone marrow derived MSCs in 16 patients with refractory perianal fistulas in the setting of Crohn's disease. There will be 2 cohorts (100M and 300M MSCs); 8 subjects in each cohort; 6 subjects receiving the investigational product and 2 subjects receiving placebo (normal saline) in each cohort.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 100M of OSSM-001 | Experimental | Biological: OSSM-001Single injection of mesenchymal stem cells at dose of 100M |
| 300M of OSSM-001 | Experimental | Biological: OSSM-001Single injection of mesenchymal stem cells at dose of 300M |
| Placebo | Placebo comparator | Other: PlaceboSingle injection of normal saline |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Men and Women 18-75 years of age with a diagnosis of unhealing perianal fistulas in the setting of Crohn's disease and must be on maximal standard of care therapy (e.g., antibiotics, 5-ASA, and immunomodulator, anti-TNF, anti-integrin, and anti-interleukin therapies) for at least six months duration.
- Presence of perianal fistula with maximum of two internal fistula tracts and a maximum of three external openings based on clinical assessment and MRI. Fistula must have been draining for at least 6 weeks prior to the screening visit.
- Medically refractory perianal fistulizing Crohn's disease (i.e., standard of care such as antibiotics, immunosuppressives)
- Have no contraindications to MR evaluations: e.g., pacemaker or magnetically active metal fragments, claustrophobia
- Ability to comply with protocol
Key exclusion criteria (17 listed)
- Inability to give informed consent.
- Clinically significant medical conditions within the six months before administration of MSCs: e.g. myocardial infarction, active angina, congestive heart failure or other conditions that would, in the opinion of the investigators, compromise the safety of the patient.
- Specific exclusions: Hepatitis B or C, HIV, Abnormal AST or ALT at screening
- History of cancer including melanoma (with the exception of localized skin cancers) in the past five years
- Received treatment with an investigational drug or device within 60 days of randomization or have not completed 5 half-life washout (whichever is later) for drugs or biologics. If the half-life of the product is not known, then subjects cannot be randomized within 6 months of investigation drug treatment.
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 |
|---|---|---|
| OSSM-001Single injection of OSSM-001 at dose of 100M or 300M cells | Biological | 100M of OSSM-001, 300M of OSSM-001 |
| PlaceboSingle injection of placebo (saline) | Other | Placebo |
Primary outcome
OSSM-001 (100M dose) related adverse events as assessed by CTCAE v5.0
Time frame: 12 weeks
To determine the safety of a single injection of 100M allogeneic bone marrow MSCs for the treatment of refractory perianal fistulizing Crohn's disease by assessing OSSM-001 related adverse events as assessed by CTCAE v5.0.
OSSM-001 (300M dose) related adverse events as assessed by CTCAE v5.0
Time frame: 12 weeks
To determine the safety of a single injection of 300M allogeneic bone marrow MSCs for the treatment of refractory perianal fistulizing Crohn's disease by assessing OSSM-001 related adverse events as assessed by CTCAE v5.0.
4 secondary outcomes
Fistula healing assessed by radiographic (MRI) assessments for OSSM-001 (100M dose)
Time frame: 12 weeks
Fistula healing assessed by clinical assessments for OSSM-001 (100M dose)
Time frame: 12 weeks
Fistula healing assessed by radiographic (MRI) assessments for OSSM-001 (300M dose)
Time frame: 12 weeks
Fistula healing assessed by clinical assessments for OSSM-001 (300M dose)
Time frame: 12 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Dec 2022.
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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.
Sagar Munjal, MD study director
Ossium Health, Inc.
Amy Lightner, MD principal investigator
The Cleveland Clinic
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing OSSM-001 in people with Rectal Fistula, run by Ossium Health, Inc..
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 8 inclusion and 17 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: withdrawn, as of the sponsor's last update on 23 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
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 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 was withdrawn before enrolling participants, so there are no results.
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?
OSSM-001 (100M dose) related adverse events as assessed by CTCAE v5.0 (time frame: 12 weeks). 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?
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 23 Dec 2022.