Study of the Efficacy and Safety of Inhaled Treprostinil in Subjects With Progressive Pulmonary Fibrosis (TETON-PPF)

Overview

About this study

The purpose of this study is to evaluate the safety and effectiveness of inhaled treprostinil in subjects with progressive pulmonary fibrosis (PPF) over a 52-week period.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

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.

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.

Note: Other protocol defined Inclusion/Exclusion Criteria may apply.

Eligibility last updated 9/12/23. Questions regarding updates should be directed to the study team contact

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Teng Moua, M.D.

Open for enrollment

Contact information:

Samuel Nascak

(507) 422-3403

Nascak.Samuel@mayo.edu

Jacksonville, Fla.

Mayo Clinic principal investigator

Augustine Lee, M.D.

Contact us for the latest status

Contact information:

Caroline Chang

Chang.Caroline@mayo.edu

More information

Publications

Publications are currently not available
.
CLS-20564514

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