A Phase I Safety Study of Allogeneic Bone Marrow Derived MSCs for Refractory Perianal Fistulizing Crohn's Disease

Ossium Health, Inc.0 sitesAugust 2022

ConditionsRectal Fistula

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Rectal Fistula
Sponsor: Ossium Health, Inc.
Primary Endpoint: OSSM-001 (300M dose) related adverse events as assessed by CTCAE v5.0
Status: Withdrawn
Last Updated: 3 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief 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. 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.

Registry

clinicaltrials.gov

Start Date

August 2022

End Date

August 2024

Last Updated

3 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Ossium Health, Inc.

Responsible Party

Sponsor

Eligibility 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.

Outcomes

Primary Outcomes

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.

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.

Secondary Outcomes

Fistula healing assessed by radiographic (MRI) assessments for OSSM-001 (300M dose)(12 weeks)
Fistula healing assessed by clinical assessments for OSSM-001 (300M dose)(12 weeks)
Fistula healing assessed by radiographic (MRI) assessments for OSSM-001 (100M dose)(12 weeks)
Fistula healing assessed by clinical assessments for OSSM-001 (100M dose)(12 weeks)