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Active, Not Recruiting

NCT Number: NCT04262167

Human Autologous Lung Stem Cell Transplant for Idiopathic Pulmonary Fibrosis

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Idiopathic Pulmonary Fibrosis and Progressive Fibrotic Interstitial Lung Disease Idiopathic Pulmonary Fibrosis Lung Diseases Lung Diseases, Interstitial Pulmonary Fibrosis Respiratory Tract Diseases

Age range

40 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

University of North Carolina as Chapel Hill

Chapel Hill, North Carolina, 27514, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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:
  • 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.
  • 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

Treatment and study plan

Lung Spheroid Stem Cells 100 million

Biological

LSCs grown from autologous trans-bronchial biopsy specimens

Other names: LSCs

Lung Spheroid Stem Cells 200 million

Biological

LSCs grown from autologous trans-bronchial biopsy specimens

Other names: LSCs

Primary outcomes

  1. Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Time frame: Through study completion, 24 months

    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.

  2. Number of Participants With Hematological Parameters of Potential Clinical Importance

    Time frame: Through study completion, 24 months

    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).

  3. Number of Participants With Clinical Chemistry Parameters of Potential Clinical Importance

    Time frame: Through study completion, 24 months

    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).

Secondary outcomes

  1. Change from Baseline in High Resolution CT scan Fibrosis Score (0-50)

    Time frame: screening visit prior to infusion and 12 months after infusion

    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.

  2. Change from baseline in Forced Vital Capacity (FVC)

    Time frame: screening visit prior to infusion and 360days post infusion

    Efficacy of therapy as measured by the annual rate of decline in FVC expressed in mL

  3. Change from baseline in 6 minute walk test distance (meters)

    Time frame: screening visit prior to infusion and 360days post infusion

    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

  4. Change from baseline in Diffusion capacity of the lung for carbon monoxide (DLCO)

    Time frame: screening visit prior to infusion and 360days post infusion

    Change from baseline in DLCO, corrected for Hemaglobin (mmol/min/kPa)

Sponsors and collaborators

Lead sponsor

University of North Carolina, Chapel Hill

Other

Collaborators

  • North Carolina State University

Registry information

Official study title

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

Acronym: HALT-IPF

Important dates

Study start
2020
Primary completion
2027
Study completion
2027
First posted
Feb 10, 2020
Registry last updated
Jan 29, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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