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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
24
LSCs grown from autologous trans-bronchial biopsy specimens
LSCs grown from autologous trans-bronchial biopsy specimens
University of North Carolina as Chapel Hill
Chapel Hill, North Carolina, United States
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study treatment, whether or not considered related to the study treatment. An SAE is defined as any untoward medical occurrence that, at any dose: results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent disability/incapacity; is a congenital anomaly/birth defect; other important medical events that may jeopardize the participant or may require medical or surgical intervention to prevent one of the other outcomes listed before.
Time frame: Through study completion, 24 months
Number of Participants With Hematological Parameters of Potential Clinical Importance
Blood samples will be collected for the assessment of hematology parameters. The clinical concern range for the parameters will be: hematocrit (high: \>0.54 proportion of red blood cells in blood); hemoglobin (high: \>180 grams per liter \[g/L\]), lymphocytes (low: \<0.8x10\^9 cells per liter \[cells/L\]); neutrophil count (low: \<1.5x10\^9 cells/L); platelet count (low: \<100x10\^9 cells/L and high: \>550x10\^9 cells/L); white blood cells count (low: \<3x10\^9 cells/L and high: \>20x10\^9 cells/L).
Time frame: Through study completion, 24 months
Number of Participants With Clinical Chemistry Parameters of Potential Clinical Importance
Blood samples will be collected for the assessment of clinical chemistry parameters. The clinical concern range for the parameters are: albumin (low: \<30 millimoles per liter \[mmol/L\]); alanine aminotransferase (ALT) (high: \>=2xupper limit of normal \[ULN\]); aspartate aminotransferase (AST) (high: \>=2xULN); alkaline phosphatase (ALP) (high: \>=2xULN); total bilirubin (high: \>=1.5xULN); calcium (low: \<2 mmol/L and high: \>2.75 mmol/L); glucose (low: \<3 mmol/L and high: \>9 mmol/L); potassium (low: \<3 mmol/L and high: \>5.5 mmol/L) and sodium (low: \<130 mmol/L and high: \>150 mmol/L).
Time frame: Through study completion, 24 months
Change from Baseline in High Resolution CT scan Fibrosis Score (0-50)
Each lobe of the lung will be numerically scored on findings seen with fibrosis utilizing High resolution CT Chest (HRCT). A numerical value is assigned to a specific finding related to fibrosis, with higher values assigned to more specific findings of fibrosis. A total composite overall numerical score will be obtained for the lungs, with a maximum possible pulmonary fibrosis score of 50 (Right upper lobe, right middle lobe, right lower lobe, left upper lobe, and left lower lobe). An azygous lobe if present will be considered as part of the right upper lobe.
Time frame: screening visit prior to infusion and 12 months after infusion
Change from baseline in Forced Vital Capacity (FVC)
Efficacy of therapy as measured by the annual rate of decline in FVC expressed in mL
Time frame: screening visit prior to infusion and 360days post infusion
Change from baseline in 6 minute walk test distance (meters)
A 6 minute walk test will be done at the indicated time points to determine change in exercise capacity with the intervention and meters walked in 6 minutes on a flat surface will be recorded
Time frame: screening visit prior to infusion and 360days post infusion
Change from baseline in Diffusion capacity of the lung for carbon monoxide (DLCO)
Change from baseline in DLCO, corrected for Hemaglobin (mmol/min/kPa)
Time frame: screening visit prior to infusion and 360days post infusion
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