Study of ExoFlo for the Treatment of Perianal Fistulas

NCT05836883 | Terminated Phase 1 FDA Drug

• 1 other identifier: DB-EF-PFCD-1a
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief Summary

To evaluate the safety and feasibility of ExoFlo as a treatment for Perianal Fistulizing Crohn's Disease.

Conditions

Perianal Fistula; Crohn's Disease

Keywords

Perianal Fistula; Perianal Fistulizing Crohn's Disease

Outcome Measures

Primary Outcomes (1)

• Safety and Feasibility

  Safety will be defined as the rate of Adverse Events (AEs) or Severe Adverse Events (SAEs) related to IMP and administration of IMP in this patient population. Safety and feasibility will be evaluated for one dose of 15 mL or 30 mL ExoFlo or two doses of 30 mL ExoFlo in subjects with perianal fistula(s) in the setting of Crohn's Disease.

  12 Months

Secondary Outcomes (1)

• Healing

  12 Months

Study Arms (6)

Cohort 1: Placebo

PLACEBO COMPARATOR

Local injection 15 mL of normal saline on Day 0

Other: Local injection of normal saline

Cohort 2: Placebo

PLACEBO COMPARATOR

Local injection 30 mL of normal saline on Day 0

Other: Local injection of normal saline

Cohort 3: Placebo

PLACEBO COMPARATOR

Local injection 30 mL of normal saline on Day 0 and Month 3

Other: Local injection of normal saline

Cohort 1: Treatment

EXPERIMENTAL

Local injection of 15 mL of ExoFlo on Day 0

Biological: ExoFlo

Cohort 2: Treatment

EXPERIMENTAL

Local injection of 30 mL of ExoFlo on Day 0

Biological: ExoFlo

Cohort 3: Treatment

EXPERIMENTAL

Local injection of 30 mL of ExoFlo on Day 0 and Month 3

Biological: ExoFlo

Interventions

ExoFlo BIOLOGICAL

Local injection of bone marrow mesenchymal stem cell derived extracellular vesicles

Cohort 1: Treatment; Cohort 2: Treatment; Cohort 3: Treatment

Local injection of normal saline OTHER

Placebo

Cohort 1: Placebo; Cohort 2: Placebo; Cohort 3: 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 Crohn's Disease (CD) for at least six months duration.
• Single and/or Multi-tract Perianal fistula(s).
• Failed at least one medical therapy within the last year including, but not limited to, antibiotics, immunomodulators (6-MP, methotrexate, azathioprine), monoclonal antibodies (infliximab, adalimumab, certolizumab, ustekinumab, golimumab, vedolizumab), or small molecule inhibitors.
• Previous failed surgical intervention, including seton placement at least two weeks prior, or are not candidates for surgical intervention or are not willing to undergo surgical intervention for the management of their fistula.
• Medical therapy for CD stable for at least 2 months prior to administration of IMP. Changes in dosing or dosing intervals related to serum drug levels are permitted.
• Ability to comply with protocol.
• Competent and able to provide written informed consent.

You may not qualify if:

• Active perianal abscess or infection at the time of screening, enrollment, or at the time of investigational product administration
• Clinically significant medical conditions within the six months before administration of IMP that would, in the opinion of the investigators, compromise the safety of the subject.
• History of cancer including melanoma (with the exception of localized skin cancers) within one year of screening.
• History of colorectal cancer within 2 years
• Subjects who have a diagnosis of coagulation disorders and/or are currently on anti-coagulant therapy
• Investigational drug within one month of treatment
• Pregnant or breast feeding or trying to become pregnant.
• Presence of a rectovaginal fistula
• Presence of an ileal anal pouch and/or history of proctectomy
• The presence of severe proctitis
• Any condition which, in the opinion of the investigator, would make it unsafe or unsuitable for patients to undergo Magnetic Resonance (MR) evaluations (i.e., presence of implantable or external MR unsafe device that cannot be removed, body weight exceeding limitations, claustrophobia etc.).
• A participant who is unwilling to use medically acceptable contraception methods during participation in study
• The following out of range laboratory results at screening (result may be repeated)
• WBC \>11 x 109 /L
• Hemoglobin \< 8 g/dl

Sponsors & Collaborators

• Direct Biologics, LLC: lead

Study Sites (1)

Direct Biologics Investigational Site

Los Angeles, California, 90048, United States

Location

Related Links

• Direct Biologics, LLC

MeSH Terms

Conditions

Crohn Disease

Condition Hierarchy (Ancestors)

Inflammatory Bowel Diseases; Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Intestinal Diseases

Study Officials

• Bill Arana

  Direct Biologics, LLC

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Masking Details: Single-blind
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Multicenter, Placebo-controlled, Dose-escalation Design, Randomized Controlled Trial.

Study Record Dates

• First Submitted: April 19, 2023
• First Posted: May 1, 2023
• Study Start: August 28, 2023
• Primary Completion: December 1, 2024
• Study Completion: December 1, 2024
• Last Updated: February 11, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)