Human Autologous Lung Stem Cell Transplant for Idiopathic Pulmonary Fibrosis

NCT ID: NCT04262167

Last Updated: 2026-01-29

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-10-22
• Study Completion Date: 2027-12-31

Brief 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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Low Dose LSCs (cohort 1) n = 4 planned

4-8 weeks following transbronchial biopsy, participants in this arm will receive 100 million Lung Spheroid Stem Cell (LSC) infusion.

Group Type: EXPERIMENTAL

Lung Spheroid Stem Cells 100 million

Intervention Type: BIOLOGICAL

LSCs grown from autologous trans-bronchial biopsy specimens

Usual Care (Cohort 1) n = 2 planned

Patients will receive standard of care with no biopsy and no infusion. Placebo will not be used.

Group Type: NO_INTERVENTION

No interventions assigned to this group

High Dose LSCs (Cohort 2) n = 12 planned

4-8 weeks following transbronchial biopsy, participants in this arm will receive 200 million LSC infusion.

Group Type: EXPERIMENTAL

Lung Spheroid Stem Cells 200 million

Intervention Type: BIOLOGICAL

LSCs grown from autologous trans-bronchial biopsy specimens

Usual Care (Cohort 2) n = 6 planned

Patients will receive standard of care with no biopsy and no infusion. Placebo will not be used.

Group Type: NO_INTERVENTION

No interventions assigned to this group

Interventions

Lung Spheroid Stem Cells 100 million

LSCs grown from autologous trans-bronchial biopsy specimens

Intervention Type: BIOLOGICAL

Lung Spheroid Stem Cells 200 million

LSCs grown from autologous trans-bronchial biopsy specimens

Intervention Type: BIOLOGICAL

Other Intervention Names

LSCs; LSCs

Eligibility Criteria

Inclusion Criteria

• Male or female between the ages of 40 to 80.
• Diagnosis of a Progressive Fibrotic Interstitial Lung Disease
• Diagnosis of IPF based on the following criteria in accordance with American Thoracic Society (ATS) guidelines for diagnosing IPF:

1. Definite usual interstitial pneumonia (UIP) confirmed on surgical lung biopsy (SLB) with all other etiologies for UIP excluded OR High resolution CT scan (HRCT) showing definite UIP with all other etiologies for UIP excluded.

2. Probable UIP on both imaging and surgical lung biopsy with all other etiologies for UIP excluded.

• Forced vital capacity (FVC) greater than 50% of predicted with a ratio of forced expiratory volume in 1 second to FVC (FEV1/FVC) greater than 0.75 (Pulmonary function tests must be completed no more than 90 days before screening).
• Diffusing capacity for carbon monoxide (DLCO) greater than 25% of predicted capacity.
• Ability to perform a 6-Minute Walk Test (6MWT) at screening.
• Competency to understand the information given in the Human Research and Ethics Committee (HREC) approved Informed Consent Form and must sign the form prior to the initiation of any study procedures

Exclusion Criteria

• Diagnosis of an interstitial lung disease (ILD) or restrictive lung disease other than IPF or Progressive Fibrotic Interstitial Lung Disease.
• Obstructive lung disease as determined by evidence of airflow obstruction on HRCT or physiologic criteria including: FEV1/FVC ratio less than 0.75, Residual volume (RV) greater than 120% by plethysmography or significant (verified by radiologist) emphysema on HRCT or evidence of reactive airway disease by change in FEV1 of greater than 12% following bronchodilator challenge.
• Evidence of sustained improvement lung function defined as improvement from pre-therapy pulmonary function tests (PFTs) observed with two or more successive post-therapy PFTs over the year prior to randomization.
• Active or recent (less than 60 days prior to enrollment) significant respiratory tract infections, or a history of frequent (greater than 2 per year for the last 2 years) infective exacerbations of IPF.
• Hospitalization within 60 days of screening for an acute exacerbation of IPF (AE-IPF).

Chronic heart failure (NYHA class III/IV) or known left ventricular ejection fraction less than 45%.

• Acute or chronic impairment (other than dyspnea) which limits the ability to comply with study requirements and procedures including the 6MWT.
• Subject requires hemodialysis, peritoneal dialysis or hemofiltration.
• Infection with HIV
• Viral Hepatitis
• Resting oxygen requirements or >4 L of nasal canula oxygen needed with exertion

• Minimum Eligible Age: 40 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

North Carolina State University

OTHER

Sponsor Role: collaborator

University of North Carolina, Chapel Hill

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Leonard Lobo, MD

Role: PRINCIPAL_INVESTIGATOR

University of North Carolina, Chapel Hill

Locations

University of North Carolina as Chapel Hill

Chapel Hill, North Carolina, United States

Countries

United States

Other Identifiers

18-1430

Identifier Type: -

Identifier Source: org_study_id