Study of ExoFlo for the Treatment of Medically Refractory Crohn's Disease

Crohn Disease Clinical Trial

Official title: A Phase I Study of ExoFlo, an ex Vivo Culture-expanded Adult Allogeneic Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicle Isolate Product, for the Treatment of Medically Refractory Crohn's Disease

Status Recruiting

Clinical Trial Summary

Protocol Summary

• Title: A Phase I study of ExoFlo, an ex vivo culture-expanded adult allogeneic bone marrow mesenchymal stem cell derived extracellular vesicle isolate product, for the treatment of medically refractory Crohn's disease.

• Short Title: ExoFlo for Crohn's Disease

• Phase: 1

• Methodology: Open label

• Study Duration: 24 months

• Subject Participation: 58 weeks

• Single or Multi-Site: Multi-Site

Clinical Trial Description

Primary Objectives:

• To evaluate the feasibility of intravenous ExoFlo in subjects with medically refractory Crohn's disease who have failed, or are intolerant, or have a contraindication to one or more monoclonal antibodies.

• To evaluate the safety of intravenous ExoFlo in subjects with medically refractory Crohn's disease who have failed, or are intolerant, or have a contraindication to one or more monoclonal antibodies.

Secondary Objectives:

• To evaluate the efficacy of intravenous ExoFlo in inducing clinical remission in subjects with medically refractory Crohn's disease who have failed, or are intolerant, or have a contraindication to one or more monoclonal antibodies.

• To evaluate the efficacy of intravenous ExoFlo in inducing clinical response in subjects with medically refractory Crohn's disease who have failed, or are intolerant, or have a contraindication to one or more monoclonal antibodies.

• To evaluate the efficacy of intravenous ExoFlo in improving disease-specific health-related quality of life.

• To evaluate the pharmacokinetics and pharmacodynamics of ExoFlo therapy, including changes in C-reactive protein (CRP) and fecal calprotectin.

Number of Subjects: 10

Diagnosis and Main Inclusion Criteria: Subjects must have colitis, ileitis, or ileocolitis previously confirmed at any time in the past by radiography, histology, and/or endoscopy, and must allow a ≥ 8-week washout for prior monoclonal antibody therapy.

Study Product, Dose, Route, Regimen:

IV administration of 15 mL study agent at Day 0, Day 2, Day 4 and 30 mL at Week 2, Week 6 and every 4 weeks thereafter to week 46 (n=10), (total # doses =15)

Statistical Methodology: This is a safety study with exploratory assessment of efficacy. The study has insufficient power to confirm efficacy. All assessments of efficacy will be exploratory for the purpose of hypothesis-generation in larger sample sizes. ;

Related Conditions & MeSH terms

• Crohn Disease
• Inflammatory Bowel Diseases
• Intestinal Diseases

• NCT number: NCT05130983
• Study type: Interventional
• Source: Direct Biologics, LLC
• Contact: Bill Arana
• Phone: 1-800-791-1021
• Email: clinicalaffairs@directbiologics.com
• Status: Recruiting
• Phase: Phase 1
• Start date: January 23, 2023
• Completion date: September 1, 2024