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

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

Status

Terminated

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT05497284

Enrollment

Registered

2022-08-11

Start date

2022-11-10

Completion date

2024-09-26

Last updated

2026-01-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Idiopathic Pulmonary Fibrosis

Keywords

Idiopathic pulmonary fibrosis, FVC, DLCO, 6-minute walk test, patient reported outcome, HRCT

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.

Interventions

DRUGLTP001

LTP001 administered once daily in the morning

DRUGPlacebo

Placebo to LTP001 administered once daily in the morning

DRUGStandard of Care (SoC)

nintedanib, pirfenidone, or neither

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender

ALL

Age

40 Years to 100 Years

Healthy volunteers

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 /

Design outcomes

Primary

Measure	Time frame	Description
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

Measure	Time frame	Description
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 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.
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.
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.

Countries

Argentina, Australia, Czechia, Germany, Netherlands, Poland, United States

Participant flow

Recruitment details

Participants took part at 15 investigative sites in 7 countries.

Pre-assignment details

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.

Participants by arm

Arm	Count
LTP001 6mg LTP001 6mg orally once daily in the morning for approximately 26 weeks.	23
Placebo Placebo orally once daily in the morning for approximately 26 weeks.	23
Total	46

Withdrawals & dropouts

Period	Reason	FG000	FG001
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

Characteristic	LTP001 6mg	Placebo	Total
Age, Continuous	70.3 Years STANDARD_DEVIATION 7.07	69.5 Years STANDARD_DEVIATION 5.34	69.9 Years STANDARD_DEVIATION 6.21
Race/Ethnicity, Customized White	23 Participants	23 Participants	46 Participants
Sex: Female, Male Female	2 Participants	11 Participants	13 Participants
Sex: Female, Male Male	21 Participants	12 Participants	33 Participants

Adverse events

Event type	EG000 affected / at risk	EG001 affected / at risk	EG002 affected / at risk
deaths Total, all-cause mortality	1 / 23	0 / 23	1 / 46
other Total, other adverse events	14 / 23	7 / 23	21 / 46
serious Total, serious adverse events	2 / 23	1 / 23	3 / 46

Outcome results

Primary

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.