NCT05130983

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

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: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 3 (actual)
• Sponsor: Direct Biologics, LLC · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

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

Detailed 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.

Conditions

• Crohn Disease
• Inflammatory Bowel Diseases

Interventions

Type	Name	Description
BIOLOGICAL	ExoFlo	Intravenous administration of bone marrow mesenchymal stem cell derived extracellular vesicles

Timeline

• Start date: 2023-01-23
• Primary completion: 2024-12-01
• Completion: 2024-12-01
• First posted: 2021-11-23
• Last updated: 2025-02-11

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study