This is a randomized, placebo-controlled, double-blind, 6-month study followed by a 6-month open-label extension phase to evaluate the efficacy, safety, and tolerability of MN-001 in patients diagnosed with moderate to severe idiopathic pulmonary fibrosis (IPF). Participants were randomly assigned to receive MN-001 or matching placebo twice daily over a 26-week period. A total of 15 participants were enrolled.
This study was a single-center, randomized (2:1), placebo-controlled, double-blind, 6-month study followed by a 6-month open-label extension (OLE) phase in patients with moderate to severe IPF. Major inclusion criteria: physician diagnosed IPF (ATS Guidelines, 2011), males and females aged 21 to 80 years, GAP Stage II-III; on no anti-fibrotic treatment. Patients on stable dose of nintedanib for at least 3 months prior to the study were allowed. The study consisted of a Screening Phase (up to 3 months prior to Day1), a 26- week Double-Blind Treatment (DBT) period, a 26-week Open-Label Extension (OLE) period, and a Follow-up / End of Study Visit (within 4 weeks of the last dose taken). A total of 15 patients were enrolled in the study. During the DBT period, participants were randomly assigned to receive MN-001 750 mg twice daily or a matching placebo in a 2:1 ratio (MN-001: placebo) for 26 weeks. During the OLE period, all participants received MN-001 750 mg twice daily for 26 weeks. Taken together, participants (n=15) received either MN-001 50 mg twice daily for 12 months (MN-001/MN-001) or matching placebo for 6 months and MN-001 750 mg twice daily for 6 months (Placebo/MN-001).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
15
Penn State University College of Medicine, Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States
Absolute Change From Baseline in Forced Vital Capacity for 26 Weeks
Predicted forced vital capacity (FVC) is a reference value for lung function based on your age, height, sex, and ethnicity measured by a spirometer and is an established method of pulmonary function. FVC is the volume of air that can be forcibly blown out after full inspiration, measured in liters (L).
Time frame: Baseline and Week 26 at the end of Double-blind treatment period.
Percent Change in Forced Vital Capacity From Baseline to Week 26
FVC is the volume of air that can be forcibly blown out after full inspiration, measured in liters (L). The mean relative change was calculated as 100\*\[FVC (L) at Week 26 - FVC (L) at baseline\].
Time frame: Baseline, and Week 26 at the endpoint of the Double-blind treatment period.
Absolute Change From Baseline in Forced Vital Capacity (% Predicted)
FVC(%pred.) refers to the expected FVC for a healthy individual with the same age, sex, height, and weight. The actual FVC result is then expressed as a percentage of this predicted value; values of 80% or higher are generally considered normal and indicate no significant impairment.
Time frame: Baseline and Week 26 at the end of Double-blind treatment period.
Relative Change From Baseline in Percent Predicted Forced Vital Capacity From Baseline to Week 26
FVC (%pred/) refers to the expected FVC for a healthy individual with the same age, sex, height, and weight. The actual FVC result is then expressed as a percentage of this predicted value; values of 80% or higher are generally considered normal and indicate no significant impairment. Relative change is measured as percent (%) change from FVC (%pred.).
Time frame: Baseline and Week 26 at the end of Double-blind treatment period.
Semiannual Rate of Decline of Disease Activity Based on Forced Vital Capacity
The semiannual rates of change in FVC, measured in liters, were estimated using simple linear regression, with time measured in half-years.
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Time frame: Baseline and Week 26 at the end of Double-blind treatment period.
Number of Participants With Treatment-related Serious Adverse Events.
Treatment-related serious adverse events (TRSAEs) are defined as possibly related, probably related, or related to MN-001 treatment.
Time frame: Baseline, and Week 26 at the endpoint of the Double-blind treatment period
Change From Baseline on Disease Activity Based on Modified Medical Research Council Dyspnea Score
The Modified Medical Research Council Dyspnea Score is a simple grading system widely used in the assessment of dyspnea (difficulty breathing) in chronic respiratory diseases, such as IPF. The breathlessness scale comprises five statements that cover respiratory disability from 0 (zero) to 4, in which 0 = not troubled by breathlessness, except on strenuous exercise, 1= shortness of breath when hurrying on the level or walking up a slight hill, 2 = walks slower than people of the same age or has to stop for breath when walking at own pace on level ground, 3 = stops for breath after walking just 100 meters (100 yards) or after a few minutes, and 4 = too breathless to leave the house or breathless when dressing or undressing. A higher score corresponds to greater difficult in breathing.
Time frame: Baseline, and Week 26 at the endpoint of the Double-blind treatment period
Change From Baseline on Quality of Life (QOL) Measured by A Tool to Assess Quality of Life in Idiopathic Pulmonary Fibrosis
The A Tool to Assess Quality of Life in Idiopathic Pulmonary Fibrosis score is a quality-of-life questionnaire that has 13 domains (cough, dyspnea, forethought, sleep, mortality, exhaustion, emotional well-being, social participation, finances, independence, sexual health, relationships, therapy). The domain scores and the Total score from these domain scores are calculated by summation. Higher scores correspond to greater impairment. The Total score ranges from 74 to 370.
Time frame: Baseline, and Week 26 at the endpoint of the Double-blind treatment period
Frequency of Worsening IPF
The number of participants who experienced worsening in IPF. Worsening of IPF is defined as acute IPF exacerbation, hospitalization due to respiratory symptoms, IPF-related fatal events, and/or lung transplantation.
Time frame: Baseline, and Week 26 at the endpoint of the Double-blind treatment period