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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05943535




Registration number
NCT05943535
Ethics application status
Date submitted
5/07/2023
Date registered
13/07/2023
Date last updated
16/12/2024

Titles & IDs
Public title
Study of the Efficacy and Safety of Inhaled Treprostinil in Subjects With Progressive Pulmonary Fibrosis (TETON-PPF)
Scientific title
A Randomized, Double-blind, Placebo-controlled, Multinational, Phase 3 Study of the Efficacy and Safety of Inhaled Treprostinil in Subjects With Progressive Pulmonary Fibrosis (TETON-PPF)
Secondary ID [1] 0 0
RIN-PF-305
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Progressive Pulmonary Fibrosis 0 0
Interstitial Lung Disease 0 0
Condition category
Condition code
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Placebo
Treatment: Drugs - Inhaled Treprostinil
Treatment: Devices - Treprostinil Ultrasonic Nebulizer

Placebo comparator: Placebo - Matching placebo inhaled using an ultrasonic nebulizer QID

Experimental: Inhaled Treprostinil - Treprostinil for inhalation solution (0.6 mg/mL) delivered via an ultrasonic nebulizer which emits a dose of approximately 6 mcg per breath. Inhaled QID and titrated up to a target of 12 breaths QID or until the subject reaches their maximum clinically tolerated dose.


Treatment: Drugs: Placebo
Placebo administered QID

Treatment: Drugs: Inhaled Treprostinil
Inhaled treprostinil (6 mcg/breath) administered QID

Treatment: Devices: Treprostinil Ultrasonic Nebulizer
Treprostinil ultrasonic nebulizer which emits a dose of approximately 6 mcg per breath.
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Treatment: Devices
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in Absolute FVC from Baseline to Week 52
Timepoint [1] 0 0
Baseline to Week 52
Secondary outcome [1] 0 0
Time to First Clinical Worsening
Timepoint [1] 0 0
Baseline to Week 52
Secondary outcome [2] 0 0
Time to First Acute Exacerbation of ILD
Timepoint [2] 0 0
Baseline to Week 52
Secondary outcome [3] 0 0
Overall Survival at Week 52
Timepoint [3] 0 0
Week 52
Secondary outcome [4] 0 0
Change in % Predicted FVC from Baseline to Week 52
Timepoint [4] 0 0
Baseline to Week 52
Secondary outcome [5] 0 0
Change in K-BILD Questionnaire Score from Baseline to Week 52
Timepoint [5] 0 0
Baseline to Week 52
Secondary outcome [6] 0 0
Change in DLCO from Baseline to Week 52
Timepoint [6] 0 0
Baseline to Week 52

Eligibility
Key inclusion criteria
1. Subject gives voluntary informed consent to participate in the study.
2. Subject is =18 years of age, inclusive, at the time of signing informed consent.
3. Subject has radiological evidence of pulmonary fibrosis of >10% extent on an HRCT scan in the previous 12 months (confirmed by central review).
4. Subject has a diagnosis of PPF (other than IPF) that fulfills at least 1 of the following criteria for progression within 24 months of screening despite standard treatment of ILD, as assessed by the Investigator:

1. Clinically significant decline in % predicted FVC based on =10% relative decline
2. Marginal decline in % predicted FVC based on =5% to <10% relative decline combined with worsening of respiratory symptoms
3. Marginal decline in % predicted FVC based on =5% to <10% relative decline combined with increasing extent of fibrotic changes on chest imaging
4. Worsening of respiratory symptoms as well as increasing extent of fibrotic changes on chest imaging
5. FVC =45% predicted at Screening (confirmed by central review).
6. Subjects must be on 1 of the following:

1. On nintedanib or pirfenidone for =90 days prior to Baseline and in the Investigator's opinion, are planning to continue treatment through the study
2. Not on treatment with nintedanib or pirfenidone for =90 days prior to Baseline and in the Investigator's opinion, not planning to initiate either treatment during the study.

Concomitant use of both nintedanib and pirfenidone is not permitted.
7. Subjects treated with immunosuppressive agents (eg, mycophenolate, methotrexate, azathioprine, oral corticosteroids, rituximab) need to be on treatment for at least 120 days prior to Baseline and, in the Investigator's clinical opinion, must be refractory to treatment.
8. Women of childbearing potential must be non-pregnant (as confirmed by a urine pregnancy test at Screening and Baseline) and non-lactating, and will agree to do 1 of the following:

1. Abstain from intercourse (when it is in line with their preferred and usual lifestyle)
2. Use 2 medically acceptable, highly effective forms of contraception for the duration of the study, and at least 30 days after discontinuing study drug.

i. Medically acceptable, highly effective forms of contraception can include approved hormonal contraceptives (oral, injectable, and implantable) and barrier methods (such as a condom or diaphragm) when used with a spermicide.

Women who are successfully sterilized (including hysterectomy, bilateral salpingectomy, or bilateral oophorectomy) or postmenopausal (defined as amenorrhea for at least 12 consecutive months) are not considered to be of reproductive potential.
9. Males with a partner of childbearing potential must agree to use a condom for the duration of treatment and for at least 48 hours after discontinuing study drug.
10. In the opinion of the Investigator, the subject is able to communicate effectively with study personnel, and is considered reliable, willing, and likely to be cooperative with protocol requirements, including attending all study visits.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Subject is pregnant or lactating.
2. Subject has primary obstructive airway physiology (forced expiratory volume in 1 second/FVC <0.70 at Screening) or greater extent of emphysema than fibrosis on HRCT (confirmed by central review).
3. Subject has a diagnosis of IPF.
4. Subject has shown intolerance or significant lack of efficacy to a prostacyclin or prostacyclin analogue that resulted in discontinuation or inability to effectively titrate that therapy.
5. Subject has received any PAH-approved therapy, including prostacyclin therapy (epoprostenol, treprostinil, iloprost, or beraprost; except for acute vasoreactivity testing), IP receptor agonists (selexipag), endothelin receptor antagonists, phosphodiesterase type 5 inhibitors (PDE5-Is), or soluble guanylate cyclase stimulators within 60 days prior to Baseline. As needed use of a PDE5-I for erectile dysfunction is permitted, provided no doses are taken within 48 hours prior to any study-related efficacy assessments.
6. Subject is receiving >10 L/min of oxygen supplementation by any mode of delivery at rest at Baseline.
7. Exacerbation of ILD or active pulmonary or upper respiratory infection within 30 days prior to Baseline. Subjects must have completed any antibiotic or steroid regimens for treatment of the infection or acute exacerbation more than 30 days prior to Baseline to be eligible. If hospitalized for an acute exacerbation of ILD or a pulmonary or upper respiratory infection, subjects must have been discharged more than 90 days prior to Baseline to be eligible.
8. Subject has uncontrolled cardiac disease, defined as myocardial infarction within 6 months prior to Baseline or unstable angina within 30 days prior to Baseline.
9. Use of any other investigational drug/device or participation in any investigational study in which the subject received a medical intervention (ie, procedure, device, medication/supplement) within 30 days prior to Screening. Subjects participating in non-interventional, observational, or registry studies are eligible.
10. Acute pulmonary embolism within 90 days prior to Baseline.
11. In the opinion of the Investigator, the subject has any condition that would interfere with the interpretation of study assessments or would impair study participation or cooperation.
12. In the opinion of the Investigator, life expectancy <12 months due to ILD or a concomitant illness.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
30/10/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/11/2027
Actual
Sample size
Target
698
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
West AustraliaWA
Recruitment hospital [1] 0 0
Institute for Respiratory Health - Midland - Midland
Recruitment hospital [2] 0 0
Institute for Respiratory Health - Nedlands
Recruitment postcode(s) [1] 0 0
6056 - Midland
Recruitment postcode(s) [2] 0 0
6009 - Nedlands
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Arizona
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
District of Columbia
Country [5] 0 0
United States of America
State/province [5] 0 0
Florida
Country [6] 0 0
United States of America
State/province [6] 0 0
Georgia
Country [7] 0 0
United States of America
State/province [7] 0 0
Illinois
Country [8] 0 0
United States of America
State/province [8] 0 0
Kansas
Country [9] 0 0
United States of America
State/province [9] 0 0
Kentucky
Country [10] 0 0
United States of America
State/province [10] 0 0
Louisiana
Country [11] 0 0
United States of America
State/province [11] 0 0
Maryland
Country [12] 0 0
United States of America
State/province [12] 0 0
Massachusetts
Country [13] 0 0
United States of America
State/province [13] 0 0
Michigan
Country [14] 0 0
United States of America
State/province [14] 0 0
Minnesota
Country [15] 0 0
United States of America
State/province [15] 0 0
Missouri
Country [16] 0 0
United States of America
State/province [16] 0 0
New Mexico
Country [17] 0 0
United States of America
State/province [17] 0 0
New York
Country [18] 0 0
United States of America
State/province [18] 0 0
North Carolina
Country [19] 0 0
United States of America
State/province [19] 0 0
Ohio
Country [20] 0 0
United States of America
State/province [20] 0 0
Pennsylvania
Country [21] 0 0
United States of America
State/province [21] 0 0
South Carolina
Country [22] 0 0
United States of America
State/province [22] 0 0
Tennessee
Country [23] 0 0
United States of America
State/province [23] 0 0
Texas
Country [24] 0 0
United States of America
State/province [24] 0 0
Utah
Country [25] 0 0
United States of America
State/province [25] 0 0
Virginia
Country [26] 0 0
United States of America
State/province [26] 0 0
Wisconsin
Country [27] 0 0
Canada
State/province [27] 0 0
British Columbia
Country [28] 0 0
Canada
State/province [28] 0 0
Ontario
Country [29] 0 0
Canada
State/province [29] 0 0
Quebec
Country [30] 0 0
Israel
State/province [30] 0 0
Hadera
Country [31] 0 0
Israel
State/province [31] 0 0
Jerusalem
Country [32] 0 0
New Zealand
State/province [32] 0 0
Canterbury
Country [33] 0 0
Taiwan
State/province [33] 0 0
Kaohsiung City
Country [34] 0 0
Taiwan
State/province [34] 0 0
Kaohsiung
Country [35] 0 0
Taiwan
State/province [35] 0 0
Taichung

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
United Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
United Therapeutics Global Medical Information
Address 0 0
Country 0 0
Phone 0 0
919-485-8350
Fax 0 0
Email 0 0
clinicaltrials@unither.com
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT05943535