Safety, Tolerability, and Pharmacokinetic Study of TRK-250 for Patients With Idiopathic Pulmonary Fibrosis

Study Overview
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Brief Summary
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TRK-250 is a nucleic acid medicine that inhibits the progression of pulmonary fibrosis by selectively suppressing the expression of transforming growth factor-beta 1 (TGF-β1) protein, at the gene expression level. This study is a double-blind, randomized, placebo-controlled Phase I study. The primary objective of the study is to assess the safety and tolerability of single and multiple inhaled doses of TRK-250 in subjects with idiopathic pulmonary fibrosis (IPF).

Official Title
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TRK-250 - A Phase I, Double-Blind, Placebo-Controlled, Single and Multiple Inhaled Dose, Safety, Tolerability, and Pharmacokinetic Study of TRK-250 in Subjects With Idiopathic Pulmonary Fibrosis

Conditions
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Idiopathic Pulmonary Fibrosis

Intervention / Treatment
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* Drug: TRK-250
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Clinical, radiographic, and histologic features consistent with the diagnosis of IPF SpO2 ≥90% at rest by pulse oximetry while breathing ambient air. FVC ≥50% of predicted. FEV1 ≥50% of predicted. Ratio of FEV1 to FVC ≥0.7. DLCO corrected for hemoglobin 30% to 79% of predicted, inclusive. Exclusion Criteria: History of acute exacerbation of IPF or respiratory tract infection within 3 months prior to Screening. Planned surgery during the study. History of malignant tumor within 5 years prior to Screening. History of emphysema or clinically significant respiratory diseases (other than IPF). Other known causes of interstitial lung disease (eg, drug toxicities, environmental exposures, connective tissue diseases). End-stage fibrotic disease expected to require organ transplantation within 6 months. Taking a systemic corticosteroid, cytotoxic therapy, vasodilator therapy for pulmonary hypertension, or unapproved treatment for IPF within 4 weeks prior to Screening. (Treatment with pirfenidone or nintedanib, though not both concurrently, is permitted, provided that the subject has been on a stable dose for at least 4 weeks prior to Screening and it is anticipated the dose will remain unchanged throughout enrollment.)

Ages Eligible for Study
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Minimum Age: 40 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: TRK-250<br> | Drug: TRK-250<br>* single and multiple doses (4 weeks)<br>|
| Placebo Comparator: Placebo<br> | Drug: Placebo<br>* single and multiple doses (4 weeks)<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence and Severity of Adverse Events | Number of patients reporting Adverse Events Number of patients by severity reporting Adverse Events | Up to 14 days after last dose | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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TRK-250, Idiopathic Pulmonary Fibrosis