Mesenchymal Stem Cells (MSCs) for Perianal Fistula

NCT05407766 | Withdrawn Phase 1 DMC FDA Drug

• 1 other identifier: OSSM-001-001-01
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Rectal Fistula

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  12 weeks

Secondary Outcomes (4)

• 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

• 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

Study Arms (3)

100M of OSSM-001

EXPERIMENTAL

Single injection of mesenchymal stem cells at dose of 100M

Biological: OSSM-001

300M of OSSM-001

EXPERIMENTAL

Single injection of mesenchymal stem cells at dose of 300M

Biological: OSSM-001

Placebo

PLACEBO COMPARATOR

Single injection of normal saline

Other: Placebo

Interventions

OSSM-001 BIOLOGICAL

Single injection of OSSM-001 at dose of 100M or 300M cells

100M of OSSM-001; 300M of OSSM-001

Placebo OTHER

Single injection of placebo (saline)

Placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• 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).
• Any contraindications to MRI or surgical or anesthetic procedure(s)
• Any major GI surgery or major perianal local surgery within 6 months of randomization

Sponsors & Collaborators

• Ossium Health, Inc.: lead

MeSH Terms

Conditions

Rectal Fistula

Condition Hierarchy (Ancestors)

Intestinal Fistula; Digestive System Fistula; Digestive System Diseases; Intestinal Diseases; Gastrointestinal Diseases; Rectal Diseases; Fistula; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms

Study Officials

• Sagar Munjal, MD

  Ossium Health, Inc.

  STUDY DIRECTOR

• Amy Lightner, MD

  The Cleveland Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 9, 2022
• First Posted: June 7, 2022
• Study Start: August 1, 2022
• Primary Completion: April 1, 2024
• Study Completion: August 1, 2024
• Last Updated: December 23, 2022

Record last verified: 2022-12