Safety, Tolerability, and Efficacy of MN-001 (Tipelukast) in Patients With Idiopathic Pulmonary Fibrosis

NCT02503657 | Completed Phase 2 Results

• 1 other identifier: MN-001-IPF-201
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Idiopathic Pulmonary Fibrosis; IPF

Keywords

tipelukast; IPF; fibrosis; Idiopathic Pulmonary Fibrosis

Outcome Measures

Primary Outcomes (5)

• 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

  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.

Secondary Outcomes (4)

• Number of Participants With Treatment-related Serious Adverse Events.

  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

  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

  Baseline, and Week 26 at the endpoint of the Double-blind treatment period

• Frequency of Worsening IPF

  Baseline, and Week 26 at the endpoint of the Double-blind treatment period

Study Arms (2)

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.

Drug: MN-001

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.

Drug: Placebo

Interventions

MN-001 DRUG

A novel, orally bioavailable small molecule compound that demonstrates anti-inflammatory and anti-fibrotic activity

Also known as: Tipelukast

MN-001 in Double-blind period for 26 weeks, then MN-001 in Open-Label period for 26 weeks

Placebo DRUG

Excipients of MN-001/tipelukast

Placebo during Double-blind period for 26 weeks, then MN-001 in Open-Label period for 26 weeks

Eligibility Criteria

• Age: 21 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female subjects ages 21 to 80, inclusive
• Presence of IPF confirmed per ATS criteria (2011)
• Presence of moderate to severe disease, stage II-III defined by GAP index (Gender, Age and Physiology)
• Subjects who are currently treated with OFEV™/Nintedanib should be on a stable dose for at least 3 months prior to initiation of the study drug.
• Females of child-bearing potential must have a negative serum ß-hCG (human chorionic gonadotropin) at screening and must be willing to use appropriate contraception (as defined by the investigator) for the duration of study treatment and 30 days after the last dose of study treatment.
• Males should practice contraception for the duration of study treatment and 30 days after the last dose of study treatment as follows: condom use and contraception by female partner.
• Subject is in stable condition on the basis of medical history, physical examination, and laboratory screening, as determined by the investigator.
• Subject is willing and able to comply with the protocol assessments and visits, in the opinion of the study nurse/coordinator and the Investigator.
• Written informed consent is obtained prior to participating the study.

You may not qualify if:

• Expected to receive a lung transplant within 1 year from the start of the Treatment Phase or on a lung transplant waiting list at the start of the Treatment Phase.
• Known explanation for interstitial lung disease
• Subjects on OFEV™/Nintedanib with a dose interruption due to significant adverse events within 6 weeks of screening visits.
• Ongoing IPF treatments with investigational therapy
• Ongoing IPF treatments with Esbriet® (Pirfenidone)
• Immunosuppressants (i.e., Mycophenolate, Imuran, Cyclophosphamide), and cytokine modulating agents within 1 month of Screening Visit and throughout the study
• Use of antibiotics and systemic steroids due to IPF exacerbation within 1 month of Screening Visit
• Clinically significant cardiovascular disease, including myocardial infarct within last 6 months, unstable ischemic heart disease, congestive heart failure or angina
• Resting pulse \< 50 bpm, SA (sinoatrial) or AV (atrioventricular) block, uncontrolled hypertension, or QTcF (QT interval corrected using the Fridericia formula) \> 450 ms
• Immune system disease
• Any significant laboratory abnormality which, in the opinion of the Investigator, may put the subject at risk
• History of malignancy \< 5 years prior to signing the informed consent, except for adequately treated basal cell or squamous cell skin cancer or in situ cervical cancer.
• History or evidence of drug or alcohol abuse
• History of HIV (human immunodeficiency virus) or other active infection.
• Currently has a clinically significant medical condition including the following: neurological, psychiatric, immunological, metabolic, hepatic, hematological, pulmonary (other than IPF) , cardiovascular (including uncontrolled hypertension), gastrointestinal, urological disorder, or central nervous system (CNS) infection that would pose a risk to the subject if they were to participate in the study or that might confound the results of the study.

Sponsors & Collaborators

• MediciNova: lead

Study Sites (1)

Penn State University College of Medicine, Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

MeSH Terms

Conditions

Idiopathic Pulmonary Fibrosis; Fibrosis

Interventions

4-(6-acetyl-3-(3-(4-acetyl-3-hydroxy-2-propylphenylthio)propoxy)-2-propylphenoxy)butyric acid

Condition Hierarchy (Ancestors)

Pulmonary Fibrosis; Lung Diseases, Interstitial; Lung Diseases; Respiratory Tract Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Director, Research and Development
• Organization: Medicinova Inc

makhay@medicinova.com

8582468680

Study Officials

• Rebecca Bascom, MD

  PSU Research, Department of Medicine

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: The double-blind period is masked by the participants, care provider, investigator, and outcomes assessor.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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).

Study Record Dates

• First Submitted: July 14, 2015
• First Posted: July 21, 2015
• Study Start: March 9, 2016
• Primary Completion: December 7, 2020
• Study Completion: March 15, 2022
• Last Updated: January 23, 2026
• Results First Posted: January 23, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)