To Assess the Efficacy of the Investigational Products Compared to Placebo in Participants With IPF

Phase 2TerminatedNCT05497284

Novartis Pharmaceuticals46 enrolled

Overview

A participant- and investigator-blinded, randomized, placebo-controlled, multicenter, platform study to investigate efficacy, safety, and tolerability of various single treatments in participants with idiopathic pulmonary fibrosis

This was a randomized, placebo-controlled, participant- and investigator-blinded platform study in participants with idiopathic pulmonary fibrosis. Participants underwent a screening period of 42 days, a treatment period of 26 weeks and a post-treatment safety follow-up period of 30 days. This study was designed to safely allow rapid and efficient screening of potentially efficacious investigational products in participants with IPF. The study was terminated for strategic reasons and no additional cohorts were created.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Idiopathic Pulmonary Fibrosis

Interventions

LTP001DRUG

LTP001 administered once daily in the morning

PlaceboDRUG

Placebo to LTP001 administered once daily in the morning

Standard of Care (SoC)DRUG

nintedanib, pirfenidone, or neither

Eligibility

Sex: ALLMin age: 40 YearsMax age: 100 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Male and female participants at least 40 years of age * IPF diagnosed based on ATS/ERS/JRS/ALAT IPF 2018 modified guidelines * FVC ≥45% predicted * DLCO, corrected for hemoglobin, ≥25% predicted (inclusive) * Unlikely to undergo lung transplantation during this trial in the opinion of the investigator * If a participant is taking nintedanib or pirfenidone, they must be on a stable regimen for at least 8 weeks prior to randomization Exclusion Criteria: * Airway obstruction (i.e. prebronchodilator FEV1/ FVC \< 0.7) or evidence of a bronchodilator response at screening * Emphysema \>20% on screening HRCT * Fibrosis \<10% on screening HRCT * Clinical diagnosis of any connective tissue disease * Clinically diagnosed acute exacerbation of IPF (AE-IPF) or other significant clinical worsening within 3 months of randomization Additional protocol-defined inclusion / exclusion criteria may apply.

Locations (15)

University of Alabama at Birmingham

Birmingham, Alabama, United States

University of Kansas Hospital

Kansas City, Kansas, United States

Novartis Investigative Site

Outcomes

Primary Outcomes

Change From Baseline to End of Treatment Epoch in Forced Vital Capacity (FVC) Expressed in Percent Predicted

Forced Vital Capacity (FVC) is the total amount of air exhaled during the Forced expiratory volume (FEV) test measured through spirometry testing. FEV measures how much air a person can exhale during a forced breath. It is expressed as percent predicted, defined as FVC of the participant divided by the average FVC in the population for any person of similar age, sex, and body composition multiplied by 100. A positive change from baseline is considered a favorable outcome.

Time frame: Baseline, up to approximately 26 weeks

Secondary Outcomes

Change From Baseline to End of Treatment Epoch in FVC

Time frame: Baseline, up to approximately 26 weeks

Progression-free Survival (PFS)

PFS is defined as the time from the date of randomization to the date of the any of the following events: Absolute reduction from baseline of ≥10% predicted in FVC, Nonelective hospitalization for respiratory events, Lung Transplant, Death. PFS was analyzed based on Kaplan-Meier estimates.

Time frame: Baseline, up to approximately 26 weeks

Number of Participants With Absolute Decline of ≥10% Predicted in FVC

Binary output of absolute decline of ≥ 10% predicted in FVC (Yes/No) at the end of treatment epoch.

Time frame: Baseline, up to approximately 26 weeks

Change From Baseline to the End of Treatment Epoch in Diffusion Capacity of Lung for Carbon Monoxide (DLCO)

Diffusing capacity of the lungs for carbon monoxide (DLCO) is a measurement to assess the ability of the lungs to transfer gas from inspired air to the bloodstream. Inhaled carbon monoxide (CO) is used for this test due to its high affinity for hemoglobin. During a ten-second breath-hold, DLCO measures uptake of CO per time per CO pressure.

Data from ClinicalTrials.gov

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CABA, Buenos Aires, Argentina

Novartis Investigative Site

Ranelagh Partido de Berazate, Buenos Aires, Argentina

Novartis Investigative Site

Paraná, Argentina

Novartis Investigative Site

Camperdown, New South Wales, Australia

Novartis Investigative Site

Chermside, Queensland, Australia

Novartis Investigative Site

Spearwood, Western Australia, Australia

Novartis Investigative Site

Prague, Czechia

Novartis Investigative Site

Munich, Bavaria, Germany

...and 5 more locations

Time frame: Baseline, up to approximately 26 weeks

Change From Baseline to the End of Treatment Epoch in 6-minute Walk Distance (6MWD)

The 6MWD test is self-paced, with standardized instructions and encouragement being given as participants walk as far as possible over 6 minutes through a flat corridor. The final distance is recorded in meters. A positive change from baseline in 6MWD is considered a favourable outcome.

Time frame: Baseline, up to approximately 26 weeks

Change From Baseline to the End of Treatment Epoch in Total Score From the K-BILD Questionnaire

The King's Brief Interstitial Lung Disease (K-BILD) is a 15-item HRQOL questionnaire that range from 0 to 100. Higher scores indicate better health-related quality of life, with 100 representing the best possible health status. A positive change from baseline in K-BILD questionnaire is considered a favourable outcome.

Time frame: Baseline, up to approximately 26 weeks

Change From Baseline to the End of Treatment Epoch in Scores From Leicester Cough Questionnaire

The Leicester Cough Questionnaire (LCQ) is a 19-item validated patient-report questionnaire that measures the impact of cough on quality of life. It takes about 5 minutes to complete and results in three domain scores (Social, Psychological, and Physical), and one Total score. Domain scores are averages of the questions that make up each domain. Minimum and maximum domain scores are 1 and 7, respectively. The domain scores are summed to determine the Total score, which can range from 3 to 21. A higher score means less impact on quality of life. A positive LCQ change score indicates an improvement.

Time frame: Baseline, up to approximately 26 weeks

Change From Baseline to the End of Treatment Epoch in Scores From the the R-Scale for IPF Questionnaire

The Raghu-Scale for Pulmonary Fibrosis (R-Scale for PF) is a questionnaire designed to assess the impact of lung disease on quality of life. The R-Scale-PF uses a numerical rating scale to assess the severity of five symptoms: cough, shortness of breath, fatigue, depressed mood, and overall sense of wellbeing, over the past two weeks. Each item is scored from 0 to 10, with lower scores indicating better health-related quality of life. The total R-Scale-PF score ranges from 0 to 50. A decrease from baseline in R-Scale for PF is considered a favourable outcome.

Time frame: Baseline, up to approximately 26 weeks

Change From Baseline to the End of Treatment Epoch in Total Score From the Living With IPF Questionnaire

Living with idiopathic pulmonary fibrosis (L-IPF) is a tool that assesses symptoms and impacts of IPF. The Symptoms Module consists of 20 questions to assess symptoms experienced from IPF over the last 24 hours. The Impacts Module consists of 20 questions on how IPF affects quality of life over the last 7 days. The total score ranging from 0 to 100, with higher scores indicating greater symptom severity. A positive change from baseline in L-IPF is considered a favourable outcome.

Time frame: Baseline, up to approximately 26 weeks