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

NCT ID: NCT03727802

Last Updated: 2023-10-23

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

34 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-11-28

Study Completion Date

2022-04-01

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).

Detailed Description

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Conditions

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Idiopathic Pulmonary Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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TRK-250

Group Type EXPERIMENTAL

TRK-250

Intervention Type DRUG

single and multiple doses (4 weeks)

Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

single and multiple doses (4 weeks)

Interventions

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TRK-250

single and multiple doses (4 weeks)

Intervention Type DRUG

Placebo

single and multiple doses (4 weeks)

Intervention Type DRUG

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.)
Minimum Eligible Age

40 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Toray Industries, Inc

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Pulmonary Associates

Phoenix, Arizona, United States

Site Status

Cedars-Sinai Medical Center (CSMC) - Women's Guild Lung Institute

Los Angeles, California, United States

Site Status

UC Davis Medical Center

Sacramento, California, United States

Site Status

Advanced Pulmonary & Sleep Research Institute of Florida

Daytona Beach, Florida, United States

Site Status

Mayo Clinic

Jacksonville, Florida, United States

Site Status

Pulmonary Disease Specialists, PA, d/b/a PDS Research. Kissimmee, FL

Kissimmee, Florida, United States

Site Status

Weill Cornell Medicine

New York, New York, United States

Site Status

PulmonIx LLC

Greensboro, North Carolina, United States

Site Status

Cleveland Clinic

Cleveland, Ohio, United States

Site Status

Medical University of South Carolina - Pulmonary & Critical Care Clinical Research Program

Charleston, South Carolina, United States

Site Status

The University of Texas Southwestern Medical Center

Dallas, Texas, United States

Site Status

Countries

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United States

References

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Doi H, Atsumi J, Baratz D, Miyamoto Y. A Phase I Study of TRK-250, a Novel siRNA-Based Oligonucleotide, in Patients with Idiopathic Pulmonary Fibrosis. J Aerosol Med Pulm Drug Deliv. 2023 Dec;36(6):300-308. doi: 10.1089/jamp.2023.0014. Epub 2023 Sep 22.

Reference Type DERIVED
PMID: 37738329 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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250IPF01

Identifier Type: -

Identifier Source: org_study_id

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