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: Not yet recruiting
• Trial end date: 2024-03-01

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: 70 weeks Single or Multi-Site: Single site Primary Objectives: • To evaluate the feasibility of intravenous ExoFlo in subjects with moderately to severely active Crohn's disease who have failed or are intolerant to one or more monoclonal antibodies.

• To evaluate the safety of intravenous ExoFlo in subjects with moderately to severely active Crohn's disease who have failed or are intolerant to one or more monoclonal antibodies.

Secondary Objectives: • To evaluate the efficacy of intravenous ExoFlo in inducing clinical remission in subjects with moderately to severely active Crohn's disease who have failed or are intolerant to one or more monoclonal antibodies.

• To evaluate the efficacy of intravenous ExoFlo in inducing clinical response in subjects with moderately to severely active Crohn's disease who have failed or are intolerant 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), fecal calprotectin, fecal lactoferrin, and other pharmacodynamic biomarkers.

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 TNF antagonist use.

Study Product, Dose, Route, Regimen: Arm 1: IV administration of study agent at Day 0, Day 2, Day 4, Week 2, Week 6 and every 8 weeks thereafter to week 46 (n=5), (total # doses = 10).

Arm 2: IV administration of study agent at Day 0, Day 2, Day 4, Week 2, Week 6 and every 4 weeks thereafter to week 46 (n=5), (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.

• Phase: Phase 1

Details

• Lead Sponsor: Direct Biologics, LLC