NCT04262167

Human Autologous Lung Stem Cell Transplant for Idiopathic Pulmonary Fibrosis

A Phase I, Randomized Study of the Safety and Efficacy of Intravenous Delivery of Lung Spheroid Stem Cells (LSCs) in Patients With Idiopathic Pulmonary Fibrosis

• Status: Active Not Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 24 (estimated)
• Sponsor: University of North Carolina, Chapel Hill · Academic / Other
• Sex: All
• Age: 40 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

Purpose: To demonstrate the safety and efficacy of autologous Lung Spheroid Stem Cells (LSCs) administered by intravenous infusion in patients with idiopathic pulmonary fibrosis Progressive Fibrotic Interstitial Lung Disease. Participants: Patients with Idiopathic Pulmonary Fibrosis (IPF) and Progressive Fibrotic Interstitial Lung Disease Procedures (methods): 24 patients previously diagnosed with idiopathic pulmonary fibrosis or Progressive Fibrotic Interstitial Lung Disease meeting all inclusion/exclusion criteria will be evaluated at baseline. LSCs will be grown from autologous trans-bronchial pulmonary biopsy specimens. The first group, consisting of 6 patients will be randomized after completion of the screening procedures to either a treatment group of 100 million LSCs administered via intravenous infusion or to a control group (standard care) in a 2:1 LSC to control group ratio. The second group of 18 patients will be randomized after completion of the screening procedures to either a treatment group of 200 million LSCs administered via intravenous infusion or to a control group (standard care) in a 2:1 LSC to control group ratio. Patients will be randomized using permuted blocks in a 2:1 LSC to control group ratio, providing a distribution of 8:4:12 patients among the control, low dose, and high dose groups, respectively. If the patient is randomized and 100 million LSCs are not achieved, then the patient will be analyzed separately and another patient enrolled. Intravenous infusion of LSCs will take place 4-8 weeks after the pulmonary biopsies are obtained. All patients will be followed up at months 0.5, 1, 3, 6, 9, 12, 18, and 24 after infusion to complete the safety and efficacy assessments listed herein. All patients will receive standard of care for their IPF.

Conditions

• Idiopathic Pulmonary Fibrosis and Progressive Fibrotic Interstitial Lung Disease

Interventions

Type	Name	Description
BIOLOGICAL	Lung Spheroid Stem Cells 100 million	LSCs grown from autologous trans-bronchial biopsy specimens
BIOLOGICAL	Lung Spheroid Stem Cells 200 million	LSCs grown from autologous trans-bronchial biopsy specimens

Timeline

• Start date: 2020-10-22
• Primary completion: 2027-12-01
• Completion: 2027-12-01
• First posted: 2020-02-10
• Last updated: 2026-01-29

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study