Study of ExoFlo for the Treatment of Perianal Fistulas

NCT ID: NCT05836883

Last Updated: 2025-02-11

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 5 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-08-28
• Study Completion Date: 2024-12-01

Brief Summary

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

Detailed Description

This is a phase IB/IIA, multicenter, single-blind, placebo-controlled, dose-escalation design, randomized controlled trial for the treatment of Perianal Fistulizing Crohn's Disease.

Subjects will be randomized 2:1 Investigational Medicinal Product (IMP) to normal saline (NS) in 3 cohorts of 12 subjects as follows:

Cohort 1: Local injection of 15 mL of IMP or NS on Day 0 (8 IMP, 4 NS) Cohort 2: Local injection of 30 mL of IMP or NS on Day 0 (8 IMP, 4 NS) Cohort 3: Local injection of 30 mL of IMP or NS on Day 0 and Month 3 (8 IMP, 4 NS)

Conditions

Perianal Fistula; Crohn's Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: SINGLE

Study Groups

Cohort 1: Placebo

Local injection 15 mL of normal saline on Day 0

Group Type: PLACEBO_COMPARATOR

Local injection of normal saline

Intervention Type: OTHER

Placebo

Cohort 2: Placebo

Local injection 30 mL of normal saline on Day 0

Group Type: PLACEBO_COMPARATOR

Local injection of normal saline

Intervention Type: OTHER

Placebo

Cohort 3: Placebo

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

Group Type: PLACEBO_COMPARATOR

Local injection of normal saline

Intervention Type: OTHER

Placebo

Cohort 1: Treatment

Local injection of 15 mL of ExoFlo on Day 0

Group Type: EXPERIMENTAL

ExoFlo

Intervention Type: BIOLOGICAL

Local injection of bone marrow mesenchymal stem cell derived extracellular vesicles

Cohort 2: Treatment

Local injection of 30 mL of ExoFlo on Day 0

Group Type: EXPERIMENTAL

ExoFlo

Intervention Type: BIOLOGICAL

Local injection of bone marrow mesenchymal stem cell derived extracellular vesicles

Cohort 3: Treatment

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

Group Type: EXPERIMENTAL

ExoFlo

Intervention Type: BIOLOGICAL

Local injection of bone marrow mesenchymal stem cell derived extracellular vesicles

Interventions

ExoFlo

Local injection of bone marrow mesenchymal stem cell derived extracellular vesicles

Intervention Type: BIOLOGICAL

Local injection of normal saline

Placebo

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

1. Men and Women 18-75 years of age with a diagnosis of Crohn's Disease (CD) for at least six months duration.

2. Single and/or Multi-tract Perianal fistula(s).

3. 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.

4. 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.

5. 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.

6. Ability to comply with protocol.

7. Competent and able to provide written informed consent.

Exclusion Criteria

1. Active perianal abscess or infection at the time of screening, enrollment, or at the time of investigational product administration

2. 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.

3. History of cancer including melanoma (with the exception of localized skin cancers) within one year of screening.

4. History of colorectal cancer within 2 years

5. Subjects who have a diagnosis of coagulation disorders and/or are currently on anti-coagulant therapy

6. Investigational drug within one month of treatment

7. Pregnant or breast feeding or trying to become pregnant.

8. Presence of a rectovaginal fistula

9. Presence of an ileal anal pouch and/or history of proctectomy

10. The presence of severe proctitis

11. 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.).

12. A participant who is unwilling to use medically acceptable contraception methods during participation in study

13. The following out of range laboratory results at screening (result may be repeated)

• WBC >11 x 109 /L
• Hemoglobin < 8 g/dl
• Platelet count <100,000/mL
• AST/ALT >2 times the upper limit of normal
• Creatinine >2 umol/L
• PT/INR: outside normal limits
• Hemoglobin A1c>6.5 mg/dL

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Direct Biologics, LLC

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Bill Arana

Role: STUDY_DIRECTOR

Direct Biologics, LLC

Locations

Direct Biologics Investigational Site

Los Angeles, California, United States

Countries

United States

Related Links

https://directbiologics.com/

Direct Biologics, LLC

Other Identifiers

DB-EF-PFCD-1a

Identifier Type: -

Identifier Source: org_study_id