NCT05176366

Study of ExoFlo for the Treatment of Medically Refractory Ulcerative Colitis

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 Ulcerative Colitis

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 4 (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 ulcerative colitis. * Short Title: ExoFlo for ulcerative colitis * 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 ulcerative colitis 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 ulcerative colitis 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 ulcerative colitis 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 ulcerative colitis 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 medically refractory ulcerative colitis and have no prior intestinal surgery for ulcerative colitis. Study Product, Dose, Route, Regimen: IV administration of 15 mL of 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

• Ulcerative Colitis
• Inflammatory Bowel Diseases

Interventions

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

Timeline

• Start date: 2022-12-19
• Primary completion: 2024-12-01
• Completion: 2024-12-01
• First posted: 2022-01-04
• Last updated: 2025-02-11

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study