Trial Outcomes & Findings for To Assess the Efficacy of the Investigational Products Compared to Placebo in Participants With IPF (NCT NCT05497284)

NCT ID: NCT05497284

Last Updated: 2026-01-13

Results Overview

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.

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 46 participants
• Primary outcome timeframe: Baseline, up to approximately 26 weeks
• Results posted on: 2026-01-13

Participant Flow

Participants took part at 15 investigative sites in 7 countries.

The screening period took place over 42 days to assess participants eligibility to take part. Written informed consent was obtained from each participant before any study specific proceedure(s) were performed. The study was explained to each participant or designee, who answered and questions, and written information was also provided.

Participant milestones

Measure	LTP001 6mg LTP001 6mg orally once daily in the morning for approximately 26 weeks.	Placebo Placebo orally once daily in the morning for approximately 26 weeks.
Overall Study STARTED	23	23
Overall Study COMPLETED	15	17
Overall Study NOT COMPLETED	8	6

Reasons for withdrawal

Measure	LTP001 6mg LTP001 6mg orally once daily in the morning for approximately 26 weeks.	Placebo Placebo orally once daily in the morning for approximately 26 weeks.
Overall Study Adverse Event	1	0
Overall Study Physician Decision	0	1
Overall Study Study terminated by sponsor	7	4
Overall Study Subject Decision	0	1

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	LTP001 6mg n=23 Participants LTP001 6mg orally once daily in the morning for approximately 26 weeks.	Placebo n=23 Participants Placebo orally once daily in the morning for approximately 26 weeks.	Total n=46 Participants Total of all reporting groups
Age, Continuous	70.3 Years STANDARD_DEVIATION 7.07 • n=210 Participants	69.5 Years STANDARD_DEVIATION 5.34 • n=19 Participants	69.9 Years STANDARD_DEVIATION 6.21 • n=123 Participants
Sex: Female, Male Female	2 Participants n=210 Participants	11 Participants n=19 Participants	13 Participants n=123 Participants
Sex: Female, Male Male	21 Participants n=210 Participants	12 Participants n=19 Participants	33 Participants n=123 Participants
Race/Ethnicity, Customized White	23 Participants n=210 Participants	23 Participants n=19 Participants	46 Participants n=123 Participants

PRIMARY outcome

Timeframe: Baseline, up to approximately 26 weeks

Population: The Pharmacodynamic (PD) analysis set included all participants with available PD data and no protocol deviations with relevant impact on PD data. Only participants with evaluable measurements are included.

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.

Outcome measures

Measure	LTP001 6mg n=17 Participants LTP001 6mg orally once daily in the morning for approximately 26 weeks.	Placebo n=22 Participants Placebo orally once daily in the morning for approximately 26 weeks.
Change From Baseline to End of Treatment Epoch in Forced Vital Capacity (FVC) Expressed in Percent Predicted	-3.1 Percentage of predicted FVC Standard Deviation 4.80	-1.1 Percentage of predicted FVC Standard Deviation 5.31

SECONDARY outcome

Timeframe: Baseline, up to approximately 26 weeks

Population: The Pharmacodynamic (PD) analysis set included all participants with available PD data and no protocol deviations with relevant impact on PD data. Only participants with evaluable measurements are included.

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.

Outcome measures

Measure	LTP001 6mg n=17 Participants LTP001 6mg orally once daily in the morning for approximately 26 weeks.	Placebo n=22 Participants Placebo orally once daily in the morning for approximately 26 weeks.
Change From Baseline to End of Treatment Epoch in FVC	-120.0 mL Standard Deviation 181.56	-45.5 mL Standard Deviation 176.25

SECONDARY outcome

Timeframe: Baseline, up to approximately 26 weeks

Population: The Pharmacodynamic (PD) analysis set included all participants with available PD data and no protocol deviations with relevant impact on PD data.

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.

Outcome measures

Measure	LTP001 6mg n=19 Participants LTP001 6mg orally once daily in the morning for approximately 26 weeks.	Placebo n=23 Participants Placebo orally once daily in the morning for approximately 26 weeks.
Progression-free Survival (PFS)	185.0 Days Interval 180.0 to Not estimable due to insufficient number of participants with events.	185.0 Days Interval 183.0 to Not estimable due to insufficient number of participants with events.

SECONDARY outcome

Timeframe: Baseline, up to approximately 26 weeks

Population: The Pharmacodynamic (PD) analysis set included all participants with available PD data and no protocol deviations with relevant impact on PD data.

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

Outcome measures

Measure	LTP001 6mg n=19 Participants LTP001 6mg orally once daily in the morning for approximately 26 weeks.	Placebo n=23 Participants Placebo orally once daily in the morning for approximately 26 weeks.
Number of Participants With Absolute Decline of ≥10% Predicted in FVC	6 Participants	5 Participants

SECONDARY outcome

Timeframe: Baseline, up to approximately 26 weeks

Population: The Pharmacodynamic (PD) analysis set included all participants with available PD data and no protocol deviations with relevant impact on PD data. Only participants with evaluable measurements are included.

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.

Outcome measures

Measure	LTP001 6mg n=16 Participants LTP001 6mg orally once daily in the morning for approximately 26 weeks.	Placebo n=17 Participants Placebo orally once daily in the morning for approximately 26 weeks.
Change From Baseline to the End of Treatment Epoch in Diffusion Capacity of Lung for Carbon Monoxide (DLCO)	-0.167 mL/min/mmHg Standard Deviation 1.5568	-0.334 mL/min/mmHg Standard Deviation 2.3838

SECONDARY outcome

Timeframe: Baseline, up to approximately 26 weeks

Population: The Pharmacodynamic (PD) analysis set included all participants with available PD data and no protocol deviations with relevant impact on PD data. Only participants with evaluable measurements are included.

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.

Outcome measures

Measure	LTP001 6mg n=14 Participants LTP001 6mg orally once daily in the morning for approximately 26 weeks.	Placebo n=20 Participants Placebo orally once daily in the morning for approximately 26 weeks.
Change From Baseline to the End of Treatment Epoch in 6-minute Walk Distance (6MWD)	9.2 meters Standard Deviation 63.06	4.7 meters Standard Deviation 56.66

SECONDARY outcome

Timeframe: Baseline, up to approximately 26 weeks

Population: The Pharmacodynamic (PD) analysis set included all participants with available PD data and no protocol deviations with relevant impact on PD data. Only participants with evaluable measurements are included.

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.

Outcome measures

Measure	LTP001 6mg n=17 Participants LTP001 6mg orally once daily in the morning for approximately 26 weeks.	Placebo n=22 Participants Placebo orally once daily in the morning for approximately 26 weeks.
Change From Baseline to the End of Treatment Epoch in Total Score From the K-BILD Questionnaire	17.17 score on scale Standard Deviation 174.009	7.94 score on scale Standard Deviation 136.768

SECONDARY outcome

Timeframe: Baseline, up to approximately 26 weeks

Population: The Pharmacodynamic (PD) analysis set included all participants with available PD data and no protocol deviations with relevant impact on PD data. Only participants with evaluable measurements are included.

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.

Outcome measures

Measure	LTP001 6mg n=17 Participants LTP001 6mg orally once daily in the morning for approximately 26 weeks.	Placebo n=22 Participants Placebo orally once daily in the morning for approximately 26 weeks.
Change From Baseline to the End of Treatment Epoch in Scores From Leicester Cough Questionnaire	-3.9 score on scale Standard Deviation 14.23	2.7 score on scale Standard Deviation 18.35

SECONDARY outcome

Timeframe: Baseline, up to approximately 26 weeks

Population: The Pharmacodynamic (PD) analysis set included all participants with available PD data and no protocol deviations with relevant impact on PD data. Only participants with evaluable measurements are included.

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.

Outcome measures

Measure	LTP001 6mg n=17 Participants LTP001 6mg orally once daily in the morning for approximately 26 weeks.	Placebo n=22 Participants Placebo orally once daily in the morning for approximately 26 weeks.
Change From Baseline to the End of Treatment Epoch in Scores From the the R-Scale for IPF Questionnaire	1.53 score on scale Standard Deviation 5.784	-0.93 score on scale Standard Deviation 6.020

SECONDARY outcome

Timeframe: Baseline, up to approximately 26 weeks

Population: The Pharmacodynamic (PD) analysis set included all participants with available PD data and no protocol deviations with relevant impact on PD data. Only participants with evaluable measurements are included.

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.

Outcome measures

Measure	LTP001 6mg n=17 Participants LTP001 6mg orally once daily in the morning for approximately 26 weeks.	Placebo n=22 Participants Placebo orally once daily in the morning for approximately 26 weeks.
Change From Baseline to the End of Treatment Epoch in Total Score From the Living With IPF Questionnaire	7.15 score on scale Standard Deviation 14.392	-4.48 score on scale Standard Deviation 11.067

Adverse Events

Placebo

Serious events: 1 serious events

Other events: 7 other events

Deaths: 0 deaths

Total

Serious events: 3 serious events

Other events: 21 other events

Deaths: 1 deaths

LTP001 6 mg

Serious events: 2 serious events

Other events: 14 other events

Deaths: 1 deaths

Serious adverse events

Measure	Placebo n=23 participants at risk Placebo orally once daily in the morning for approximately 26 weeks.	Total n=46 participants at risk Total	LTP001 6 mg n=23 participants at risk LTP001 6mg orally once daily in the morning for approximately 26 weeks.
Infections and infestations Herpes zoster	0.00% 0/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	2.2% 1/46 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	4.3% 1/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.
Infections and infestations Infection	0.00% 0/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	2.2% 1/46 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	4.3% 1/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.
Renal and urinary disorders Bladder disorder	0.00% 0/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	2.2% 1/46 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	4.3% 1/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.
Respiratory, thoracic and mediastinal disorders Idiopathic pulmonary fibrosis	4.3% 1/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	4.3% 2/46 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	4.3% 1/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.

Other adverse events

Measure	Placebo n=23 participants at risk Placebo orally once daily in the morning for approximately 26 weeks.	Total n=46 participants at risk Total	LTP001 6 mg n=23 participants at risk LTP001 6mg orally once daily in the morning for approximately 26 weeks.
Gastrointestinal disorders Diarrhoea	4.3% 1/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	13.0% 6/46 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	21.7% 5/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.
Infections and infestations Bronchitis	0.00% 0/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	4.3% 2/46 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	8.7% 2/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.
Infections and infestations Pneumonia	8.7% 2/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	4.3% 2/46 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	0.00% 0/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.
Infections and infestations Urinary tract infection	8.7% 2/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	6.5% 3/46 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	4.3% 1/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.
Investigations Amylase increased	0.00% 0/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	4.3% 2/46 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	8.7% 2/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.
Investigations Lipase increased	0.00% 0/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	4.3% 2/46 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	8.7% 2/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.
Musculoskeletal and connective tissue disorders Arthralgia	4.3% 1/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	8.7% 4/46 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	13.0% 3/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.
Nervous system disorders Headache	8.7% 2/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	6.5% 3/46 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	4.3% 1/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.
Respiratory, thoracic and mediastinal disorders Cough	4.3% 1/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	6.5% 3/46 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	8.7% 2/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.
Vascular disorders Hypertension	0.00% 0/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	4.3% 2/46 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.	8.7% 2/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus follow up period, up to a maximum duration of approximately 30 weeks.

Additional Information

Study Director

Novartis Pharmaceuticals

Phone: + 1 862 778 8300

Email: novartis.email@novartis.com

Results disclosure agreements

• Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.
• Publication restrictions are in place

Restriction type: OTHER