Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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).
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| MN-001 in Double-blind period for 26 weeks, then MN-001 in Open-Label period for 26 weeks | Experimental | MN-001 750 mg twice a day during the double-blind period (26 weeks) and MN-001 750 mg twice a day during the open-label period for 26 weeks. The arm title is shortened to MN-001/MN-001 for all results sections. |
|
| Placebo during Double-blind period for 26 weeks, then MN-001 in Open-Label period for 26 weeks | Placebo Comparator | Placebo twice a day during the double-blind period for 26 weeks and MN-001 750 mg twice daily during the open-label period for 26 weeks. The arm title is shortened to Placebo/MN-001 for all results sections. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MN-001 | Drug | A novel, orally bioavailable small molecule compound that demonstrates anti-inflammatory and anti-fibrotic activity |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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). | 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]. | 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. | 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.). | Baseline and Week 26 at the end of Double-blind treatment period. |
| Semiannual Rate of Decline of Disease Activity Based on Forced Vital Capacity |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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 |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Note: Active medical conditions that are minor or well-controlled are not exclusionary if, in the judgment of the Investigator, they do not affect risk to the subject or the study results. In cases in which the impact of the condition upon risk to the subject or study results is unclear, the Medical Monitor should be consulted.
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Rebecca Bascom, MD | PSU Research, Department of Medicine | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Penn State University College of Medicine, Milton S. Hershey Medical Center | Hershey | Pennsylvania | 17033 | United States |
Not provided
Not provided
Not provided
Not provided
Not provided
In the double-blind period, participants were randomly assigned to receive MN-001 or placebo in a 2:1 ratio, meaning that 10 participants received MN-001 and 5 participants received placebo. In the Open-label period, all 15 participants who completed the double-blind period received MN-001.
The study was conducted at a single site.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | MN-001/MN-001 | MN-001 (tipelukast) 750 mg tablet by mouth twice daily for 26 weeks. MN-001: A novel, orally bioavailable small molecule compound that demonstrates anti-inflammatory and anti-fibrotic activity |
| FG001 | Placebo/MN-001 | Matching placebo twice a day for 26 weeks. Matching Placebo: Excipients of MN-001/tipelukast |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Double-blind Period |
| |||||||||||||
| Open-label Period |
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | MN-001/MN-001 | MN-001 (tipelukast) 750 mg tablet by mouth twice daily for 26 weeks. MN-001: A novel, orally bioavailable small molecule compound that demonstrates anti-inflammatory and anti-fibrotic activity |
| BG001 | Placebo/MN-001 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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). | Posted | Mean | Standard Deviation | Liters | Baseline and Week 26 at the end of Double-blind treatment period. |
|
26 weeks in the double-blind treatment period, 26 weeks in the open-label period
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Double-blind MN-001 | MN-001 (tipelukast) 750 mg tablet by mouth twice daily for 26 weeks (n=10) MN-001: A novel, orally bioavailable small molecule compound that demonstrates anti-inflammatory and anti-fibrotic activity |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Femur fracture | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Director, Research and Development | Medicinova Inc | 8582468680 | makhay@medicinova.com |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_ICF | Yes | No | Yes | Study Protocol and Informed Consent Form | Dec 21, 2015 | Jan 6, 2026 | Prot_ICF_002.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 7, 2021 | Aug 25, 2025 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D054990 | Idiopathic Pulmonary Fibrosis |
| D005355 | Fibrosis |
| ID | Term |
|---|---|
| D011658 | Pulmonary Fibrosis |
| D017563 | Lung Diseases, Interstitial |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C526359 | 4-(6-acetyl-3-(3-(4-acetyl-3-hydroxy-2-propylphenylthio)propoxy)-2-propylphenoxy)butyric acid |
Not provided
Not provided
Not provided
This study evaluated the efficacy, safety, and tolerability of MN-001 in male and female patients aged 21 to 80 years, inclusive, with moderate-to-severe idiopathic pulmonary fibrosis (IPF). Fifteen (15) participants received MN-001 750 mg or a matching placebo twice daily for 26 weeks in a 2:1 (MN-001:Placebo) ratio. The study consisted of a Screening period (up to 3 months before baseline), a 26-week double-blind treatment (DBT) period, a 26-week open-label (OL) treatment period, and a follow-up visit (within 4 weeks after the last dose). Efficacy results are reported only for the DBT period. Safety findings are reported from Baseline (Day 1 of MN-001 or placebo treatment) to the end of the Open-Label period (52 weeks total).
Not provided
Not provided
The double-blind period is masked by the participants, care provider, investigator, and outcomes assessor.
|
| Placebo | Drug | Excipients of MN-001/tipelukast |
|
The semiannual rates of change in FVC, measured in liters, were estimated using simple linear regression, with time measured in half-years. |
| Baseline and Week 26 at the end of Double-blind treatment period. |
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. |
| 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. | 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. | Baseline, and Week 26 at the endpoint of the Double-blind treatment period |
| NOT COMPLETED |
|
|
Matching placebo twice a day for 26 weeks.
Matching Placebo: Excipients of MN-001/tipelukast
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants | No |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
Matching placebo twice daily for 26 weeks.
Matching Placebo: Excipients of MN-001/tipelukast
|
|
| Primary | 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]. | Posted | Mean | Standard Deviation | percent of change | Baseline, and Week 26 at the endpoint of the Double-blind treatment period. |
|
|
|
| Primary | 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. | Posted | Mean | Standard Deviation | percent predicted normal volume | Baseline and Week 26 at the end of Double-blind treatment period. |
|
|
|
| Primary | 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.). | Posted | Mean | Standard Deviation | percent of change | Baseline and Week 26 at the end of Double-blind treatment period. |
|
|
|
| Primary | 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. | Posted | Mean | Standard Deviation | Liters per year | Baseline and Week 26 at the end of Double-blind treatment period. |
|
|
|
| Secondary | 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. | (TRSAEs) are reported by treatment period: 1) Double-blind MN-001 treatment, 2) Double-blind Placebo treatment, and 3) Open-Label MN-001 treatment for all participants, regardless of assignment in the Double-blind period. | Posted | Count of Participants | Participants | Baseline, and Week 26 at the endpoint of the Double-blind treatment period |
|
|
|
| Secondary | 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. | Posted | Mean | Standard Deviation | score on a scale | Baseline, and Week 26 at the endpoint of the Double-blind treatment period |
|
|
|
| Secondary | 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. | Posted | Mean | Standard Deviation | score on a scale | Baseline, and Week 26 at the endpoint of the Double-blind treatment period |
|
|
|
| Secondary | 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. | Posted | Number | number of participants | Baseline, and Week 26 at the endpoint of the Double-blind treatment period |
|
|
|
| 0 |
| 10 |
| 1 |
| 10 |
| 10 |
| 10 |
| EG001 | Double-blind Placebo | Matching placebo twice daily for 26 weeks (n=5) Matching Placebo: Excipients of MN-001/tipelukast | 0 | 5 | 3 | 5 | 5 | 5 |
| EG002 | Open Label MN-001/Former Placebo | MN-001 (tipelukast) 750 mg tablet by mouth twice daily for 26 weeks (n=15). MN-001: A novel, orally bioavailable small molecule compound that demonstrates anti-inflammatory and anti-fibrotic activity | 0 | 15 | 3 | 15 | 15 | 15 |
| Orthostatic hypotension | Vascular disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Acute myocardial infarction | Cardiac disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Pneumonia | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Abdominal discomfort | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Abdominal distention | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Haemorrhoidal haemorrhage | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Rectal fissure | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Toothache | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Bronchitis | Infections and infestations | MedDRA 21.0 | Non-systematic Assessment |
|
| Pharyngitis | Infections and infestations | MedDRA 21.0 | Non-systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA 21.0 | Non-systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA 21.0 | Non-systematic Assessment |
|
| Candida infection | Infections and infestations | MedDRA 21.0 | Non-systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA 21.0 | Non-systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA 21.0 | Non-systematic Assessment |
|
| Chest discomfort | General disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Chest pain | General disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Fatigue | General disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Gamma-glutamyltransferase increased | Investigations | MedDRA 21.0 | Non-systematic Assessment |
|
| Alanine aminotransferase increased | Investigations | MedDRA 21.0 | Non-systematic Assessment |
|
| Aspartate aminotransferase increased | Investigations | MedDRA 21.0 | Non-systematic Assessment |
|
| Heart rate increased | Investigations | MedDRA 21.0 | Non-systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Lower respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Eosinophelia | Blood and lymphatic system disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Femur fracture | Injury, poisoning and procedural complications | MedDRA 21.0 | Non-systematic Assessment |
|
| Laceration | Injury, poisoning and procedural complications | MedDRA 21.0 | Non-systematic Assessment |
|
| Angioedema | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Night sweats | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Hypertension | Vascular disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Orthostatic hypotension | Vascular disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Acute myocardial infarction | Cardiac disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Blepharitis | Eye disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Trigger finger | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Polllakiuria | Renal and urinary disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Skin lesion | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Skin ulcer | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Ear pain | Ear and labyrinth disorders | MedDRA 21.0 | Non-systematic Assessment |
|
| Ureterolithiasis | Renal and urinary disorders | MedDRA 21.0 | Non-systematic Assessment |
|
Not provided
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |