To Assess the Efficacy of the Investigational Products Compared to Placebo in Participants With IPF
- Conditions
- Idiopathic Pulmonary Fibrosis
- Interventions
- Registration Number
- NCT05497284
- Lead Sponsor
- Novartis Pharmaceuticals
- Brief Summary
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
- Detailed Description
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.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 46
- 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
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description LTP001 LTP001 LTP001 orally once daily in the morning for approximately 26 weeks. LTP001 Standard of Care (SoC) LTP001 orally once daily in the morning for approximately 26 weeks. Placebo Placebo Placebo orally once daily in the morning for approximately 26 weeks. Placebo Standard of Care (SoC) Placebo orally once daily in the morning for approximately 26 weeks.
- Primary Outcome Measures
Name Time Method Change From Baseline to End of Treatment Epoch in Forced Vital Capacity (FVC) Expressed in Percent Predicted Baseline, up to approximately 26 weeks 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.
- Secondary Outcome Measures
Name Time Method Change From Baseline to End of Treatment Epoch in FVC Baseline, up to approximately 26 weeks 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. A positive change from baseline is considered a favorable outcome.
Progression-free Survival (PFS) Baseline, up to approximately 26 weeks 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.
Number of Participants With Absolute Decline of ≥10% Predicted in FVC Baseline, up to approximately 26 weeks Binary output of absolute decline of ≥ 10% predicted in FVC (Yes/No) at the end of treatment epoch.
Change From Baseline to the End of Treatment Epoch in Diffusion Capacity of Lung for Carbon Monoxide (DLCO) Baseline, up to approximately 26 weeks 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.
Change From Baseline to the End of Treatment Epoch in 6-minute Walk Distance (6MWD) Baseline, up to approximately 26 weeks 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.
Change From Baseline to the End of Treatment Epoch in Total Score From the K-BILD Questionnaire Baseline, up to approximately 26 weeks 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.
Change From Baseline to the End of Treatment Epoch in Scores From Leicester Cough Questionnaire Baseline, up to approximately 26 weeks 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.
Change From Baseline to the End of Treatment Epoch in Scores From the the R-Scale for IPF Questionnaire Baseline, up to approximately 26 weeks 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.
Change From Baseline to the End of Treatment Epoch in Total Score From the Living With IPF Questionnaire Baseline, up to approximately 26 weeks 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.
Trial Locations
- Locations (3)
University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
University of Kansas Hospital
🇺🇸Kansas City, Kansas, United States
Novartis Investigative Site
🇵🇱Bialystok, Poland
University of Alabama at Birmingham🇺🇸Birmingham, Alabama, United States