Safety and Efficacy of Inhaled Treprostinil in Adult PH With ILD Including CPFE

Overview

  • Study type

    Interventional
  • Study phase

    II/III
  • Study IDs

  • Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

  • Site IRB
    • Jacksonville, Florida: 16-002642
    • Rochester, Minnesota: 16-002642
    NCT ID: NCT02630316
    Sponsor Protocol Number: RIN-PH-201

About this study

This is a multicenter, randomized (1:1 inhaled treprostinil: placebo), double-blinded, placebo-controlled trial to evaluate the safety and efficacy of inhaled treprostinil in subjects with pre-capillary pulmonary hypertension (PH) associated with interstitial lung disease (ILD) including combined pulmonary fibrosis and emphysema (CPFE). The study will include about 314 patients at approximately 120 clinical trial centers. The treatment phase of the study will last approximately 16 weeks. Patients who complete all required assessments will also be eligible to enter an open-label, extension study (RIN-PH-202).

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. If you need assistance understanding the eligibility criteria, please contact the study team.

Inclusion Criteria:

  1. Subject voluntarily gives informed consent to participate in the study.
  2. Males and females aged 18 years or older at the time of informed consent. a. Females of reproductive potential must be non-pregnant (as confirmed by a urine pregnancy test at screening) and non-lactating, and will: i. Either abstain from intercourse (when it is in line with their preferred and usual lifestyle), or ii. Use two medically acceptable, highly-effective forms of contraception for the duration of study, and at least 30 days after discontinuing study drug. b. Males with a partner of childbearing potential must use a condom for the duration of treatment and for at least 48 hours after discontinuing study drug.
  3. The subject has a confirmed diagnosis of WHO Group 3 PH based on CT imaging, which demonstrates evidence of diffuse parenchymal lung disease performed within 6 months prior to randomization. Subjects may have any form of ILD or CPFE.
  4. Subjects are required to have a right heart catheterization (RHC) within one year prior to randomization with the following documented parameters:
    1. Pulmonary vascular resistance (PVR) > 3 Wood Units (WU) and
    2. A pulmonary capillary wedge pressure (PCWP) of < 15 mmHg and and
    3. A mean pulmonary arterial pressure (mPAP) of ≥ 25 mmHg
  5. Baseline 6MWD ≥ 100 meters
  6. Subjects on a chronic medication for underlying lung disease (ie, pirfenidone, nintedanib, etc) must be on a stable and optimized dose for ≥ 30 days prior to randomization. Subjects receiving pirfenidone or nintedanib must have been receiving treatment for at least 90 days and on a stable dose for at least 30 days prior to randomization.
  7. 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.
  8. Subjects with connective tissue disease (CTD) must have a Baseline FVC of < 70%.

Exclusion Criteria:

  1. The subject has a diagnosis of pulmonary arterial hypertension (PAH) or PH for reasons other than WHO Group 3 PH-ILD as outlined in inclusion criterion 3.
  2. The 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.
  3. The subject has received any PAH approved therapy including: prostacyclin therapy (i.e., epoprostenol, treprostinil, iloprost, or beraprost; except for acute vasoreactivity testing), IP receptor agonist (selexipag), endothelin receptor antagonist (ERA), phosphodiesterase type 5 inhibitor (PDE-5I), or soluble guanylate cyclase (sGC) within 60 days of randomization.
  4. The subject has evidence of clinically significant left-sided heart disease as defined by:
    1. PCWP > 15 mmHg
    2. Left ventricular ejection fraction < 40%.
    Note: Subjects with abnormal left ventricular function attributable entirely to impaired left ventricular filling due to the effects of right ventricular overload (i.e., right ventricular hypertrophy and/or dilatation) will not be excluded
  5. The subject is receiving > 10 L/min of oxygen supplementation by any mode of delivery at rest at Baseline.
  6. Current use of any inhaled tobacco/marijuana products or significant history of drug abuse at the time of informed consent.
  7. Exacerbation of underlying lung disease or active pulmonary or upper respiratory infection within 30 days of randomization.
  8. Initiation of pulmonary rehabilitation within 12 weeks prior to the randomization.
  9. In the opinion of the Investigator, the subject has any condition that would interfere with the interpretation of study assessments or has any disease or condition (ie, peripheral vascular disease, musculoskeletal disorder, morbid obesity) that would likely be the primary limit to ambulation (as opposed to PH).
  10. Use of any investigational drug/device, or participation in any investigational study with therapeutic intent within 30 days prior to randomization.
  11. Severe concomitant illness limiting life expectancy (< 6 months).
  12. Acute pulmonary embolism within 90 days of randomization.

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status Contact

Jacksonville, Fla.

Mayo Clinic principal investigator

Charles Burger, M.D.

Closed for enrollment

Contact information:

Melissa Stratoberdha

7832111

Stratoberdha.Melissa@mayo.edu

Rochester, Minn.

Mayo Clinic principal investigator

Hilary DuBrock, M.D.

Closed for enrollment

Contact information:

Pulmonary Clinical Research Unit

(800) 753-1606

PCRUE18@mayo.edu

More information

Publications

Publications are currently not available

Study Results Summary

Not yet available

Supplemental Study Information

Not yet available

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CLS-20423500

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