Study of ExoFlo for the Treatment of Perianal Fistulas

Crohn's Disease Clinical Trial

Official title:

A Phase IB/IIA Study of ExoFlo, an Ex Vivo Culture-expanded Adult Allogeneic Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicle Isolate Product, for the Treatment of Perianal Fistulizing Crohn's Disease

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05836883
• Other study ID #: DB-EF-PFCD-1a
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: August 28, 2023
• Est. completion date: December 2024

Study information

• Verified date: February 2024
• Source: Direct Biologics, LLC
• Contact: Bill Arana
• Phone: 1-800-791-1021
• Email: clinicalaffairs[@]directbiologics.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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)

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 36
• Est. completion date: December 2024
• Est. primary completion date: December 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

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

Related Conditions & MeSH terms

• Crohn Disease
• Crohn's Disease
• Fistula
• Perianal Fistula
• Rectal Fistula

Intervention

Biological:
• ExoFlo
Local injection of bone marrow mesenchymal stem cell derived extracellular vesicles

Other:
• Local injection of normal saline
Placebo

Locations

Country	Name	City	State
United States	Direct Biologics Investigational Site	Los Angeles	California

Sponsors (1)

Lead Sponsor	Collaborator
Direct Biologics, LLC

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Healing	To evaluate the efficacy 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. Subjects will be evaluated for healing progress: complete healing, partial healing, lack of response, or treatment failure.	12 Months

External Links

•   Direct Biologics, LLC