Mesenchymal Stem Cells (MSCs) for Perianal Fistula

• Register: ClinicalTrials.gov
• Last refreshed on: 9 January 2023
• Main ID: NCT05407766
• Date of registration: 09/05/2022
• Prospective Registration: Yes
• Primary sponsor: Ossium Health, Inc.
• Public title: Mesenchymal Stem Cells (MSCs) for Perianal Fistula
• Scientific title: A Phase I Safety Study of Allogeneic Bone Marrow Derived MSCs for Refractory Perianal Fistulizing Crohn's Disease
• Date of first enrolment: August 2022
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT05407766
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Care Provider, Investigator).
• Phase: Phase 1

Contacts

• Name: Sagar Munjal, MD
• Affiliation: Ossium Health, Inc.

• Name: Amy Lightner, MD
• Affiliation: The Cleveland Clinic

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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.

Exclusion Criteria:

- 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

- 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



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Rectal Fistula

Intervention(s)

Other: Placebo

Biological: OSSM-001

Primary Outcome(s)

OSSM-001 (100M dose) related adverse events as assessed by CTCAE v5.0 [Time Frame: 12 weeks]

OSSM-001 (300M dose) related adverse events as assessed by CTCAE v5.0 [Time Frame: 12 weeks]

Secondary Outcome(s)

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 (100M dose) [Time Frame: 12 weeks]

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 (300M dose) [Time Frame: 12 weeks]

Secondary ID(s)

OSSM-001-001-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors