Idiopathic Pulmonary Fibrosis
Conditions
Keywords
TRK-250, Idiopathic Pulmonary Fibrosis
Brief summary
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).
Interventions
DRUGPlacebo
single and multiple doses (4 weeks)
DRUGTRK-250
single and multiple doses (4 weeks)
Sponsors
Toray Industries, Inc
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 80 Years
Healthy volunteers
No
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.)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence and Severity of Adverse Events | Up to 14 days after last dose | Number of patients reporting Adverse Events Number of patients by severity reporting Adverse Events |
Countries
United States
Participant flow
Recruitment details
A total of 34 subjects were randomized to treatment, with 16 subjects in Part A and 18 subjects in Part B.
Participants by arm
| Arm | Count |
|---|---|
| PartA:TRK-250 2mg single dose of TRK-250 | 3 |
| PartA:TRK-250 10mg single dose of TRK-250 | 3 |
| PartA:TRK-250 30mg single dose of TRK-250 | 3 |
| PartA:TRK-250 60mg single dose of TRK-250 | 3 |
| PartA:Pooled Placebo single dose of Placebo | 4 |
| PartB:TRK-250 10mg multiple doses (4 weeks) of TRK-250 | 4 |
| PartB:TRK-250 30mg multiple doses (4 weeks) of TRK-250 | 4 |
| PartB:TRK-250 60mg multiple doses (4 weeks) of TRK-250 | 4 |
| PartB:Pooled Placebo multiple doses (4 weeks) of Placebo | 6 |
| Total | 34 |
Baseline characteristics
| Characteristic | PartA:TRK-250 2mg | PartA:TRK-250 10mg | PartA:TRK-250 30mg | PartA:TRK-250 60mg | PartA:Pooled Placebo | PartB:TRK-250 10mg | PartB:TRK-250 30mg | PartB:TRK-250 60mg | PartB:Pooled Placebo | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| Age, Customized Age | 74 years | 70 years | 76 years | 69 years | 75 years | 64 years | 70 years | 74 years | 75 years | 72 years |
| Race/Ethnicity, Customized Race Black or African Black or African American | 0 Participants | 0 Participants | 1 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 1 Participants |
| Race/Ethnicity, Customized Race White | 3 Participants | 3 Participants | 2 Participants | 3 Participants | 4 Participants | 4 Participants | 4 Participants | 4 Participants | 6 Participants | 33 Participants |
| Sex: Female, Male Female | 2 Participants | 1 Participants | 1 Participants | 1 Participants | 0 Participants | 0 Participants | 0 Participants | 2 Participants | 0 Participants | 7 Participants |
| Sex: Female, Male Male | 1 Participants | 2 Participants | 2 Participants | 2 Participants | 4 Participants | 4 Participants | 4 Participants | 2 Participants | 6 Participants | 27 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk | EG005 affected / at risk | EG006 affected / at risk | EG007 affected / at risk | EG008 affected / at risk |
|---|---|---|---|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 3 | 0 / 3 | 0 / 3 | 0 / 3 | 0 / 4 | 0 / 4 | 0 / 4 | 0 / 4 | 0 / 6 |
| other Total, other adverse events | 1 / 3 | 0 / 3 | 3 / 3 | 2 / 3 | 1 / 4 | 3 / 4 | 2 / 4 | 3 / 4 | 3 / 6 |
| serious Total, serious adverse events | 0 / 3 | 0 / 3 | 0 / 3 | 0 / 3 | 0 / 4 | 0 / 4 | 0 / 4 | 1 / 4 | 0 / 6 |
Outcome results
Primary
Incidence and Severity of Adverse Events
Number of patients reporting Adverse Events Number of patients by severity reporting Adverse Events
Time frame: Up to 14 days after last dose
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| PartA:TRK-250 2mg | Incidence and Severity of Adverse Events | Severe(Severity) | 0 Participants |
| PartA:TRK-250 2mg | Incidence and Severity of Adverse Events | Mild(Severity) | 0 Participants |
| PartA:TRK-250 2mg | Incidence and Severity of Adverse Events | Moderate(Severity) | 1 Participants |
| PartA:TRK-250 2mg | Incidence and Severity of Adverse Events | Subjects with AEs | 1 Participants |
| PartA:TRK-250 10mg | Incidence and Severity of Adverse Events | Severe(Severity) | 0 Participants |
| PartA:TRK-250 10mg | Incidence and Severity of Adverse Events | Subjects with AEs | 0 Participants |
| PartA:TRK-250 10mg | Incidence and Severity of Adverse Events | Mild(Severity) | 0 Participants |
| PartA:TRK-250 10mg | Incidence and Severity of Adverse Events | Moderate(Severity) | 0 Participants |
| PartA:TRK-250 30mg | Incidence and Severity of Adverse Events | Severe(Severity) | 0 Participants |
| PartA:TRK-250 30mg | Incidence and Severity of Adverse Events | Subjects with AEs | 3 Participants |
| PartA:TRK-250 30mg | Incidence and Severity of Adverse Events | Mild(Severity) | 2 Participants |
| PartA:TRK-250 30mg | Incidence and Severity of Adverse Events | Moderate(Severity) | 1 Participants |
| PartA:TRK-250 60mg | Incidence and Severity of Adverse Events | Severe(Severity) | 0 Participants |
| PartA:TRK-250 60mg | Incidence and Severity of Adverse Events | Moderate(Severity) | 1 Participants |
| PartA:TRK-250 60mg | Incidence and Severity of Adverse Events | Mild(Severity) | 1 Participants |
| PartA:TRK-250 60mg | Incidence and Severity of Adverse Events | Subjects with AEs | 2 Participants |
| PartA:Pooled Placebo | Incidence and Severity of Adverse Events | Subjects with AEs | 1 Participants |
| PartA:Pooled Placebo | Incidence and Severity of Adverse Events | Severe(Severity) | 0 Participants |
| PartA:Pooled Placebo | Incidence and Severity of Adverse Events | Mild(Severity) | 1 Participants |
| PartA:Pooled Placebo | Incidence and Severity of Adverse Events | Moderate(Severity) | 0 Participants |
| PartB:TRK-250 10mg | Incidence and Severity of Adverse Events | Subjects with AEs | 3 Participants |
| PartB:TRK-250 10mg | Incidence and Severity of Adverse Events | Severe(Severity) | 0 Participants |
| PartB:TRK-250 10mg | Incidence and Severity of Adverse Events | Mild(Severity) | 2 Participants |
| PartB:TRK-250 10mg | Incidence and Severity of Adverse Events | Moderate(Severity) | 1 Participants |
| PartB:TRK-250 30mg | Incidence and Severity of Adverse Events | Moderate(Severity) | 1 Participants |
| PartB:TRK-250 30mg | Incidence and Severity of Adverse Events | Mild(Severity) | 1 Participants |
| PartB:TRK-250 30mg | Incidence and Severity of Adverse Events | Severe(Severity) | 0 Participants |
| PartB:TRK-250 30mg | Incidence and Severity of Adverse Events | Subjects with AEs | 2 Participants |
| PartB:TRK-250 60mg | Incidence and Severity of Adverse Events | Mild(Severity) | 0 Participants |
| PartB:TRK-250 60mg | Incidence and Severity of Adverse Events | Subjects with AEs | 3 Participants |
| PartB:TRK-250 60mg | Incidence and Severity of Adverse Events | Moderate(Severity) | 2 Participants |
| PartB:TRK-250 60mg | Incidence and Severity of Adverse Events | Severe(Severity) | 1 Participants |
| PartB:Pooled Placebo | Incidence and Severity of Adverse Events | Subjects with AEs | 3 Participants |
| PartB:Pooled Placebo | Incidence and Severity of Adverse Events | Mild(Severity) | 3 Participants |
| PartB:Pooled Placebo | Incidence and Severity of Adverse Events | Severe(Severity) | 0 Participants |
| PartB:Pooled Placebo | Incidence and Severity of Adverse Events | Moderate(Severity) | 0 Participants |