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Trial record 1 of 1 for:    A Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Safety and Efficacy of Inhaled Treprostinil
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Safety and Efficacy of Inhaled Treprostinil in Adult PH With ILD Including CPFE

This study is currently recruiting participants.
Verified November 2017 by United Therapeutics
Sponsor:
ClinicalTrials.gov Identifier:
NCT02630316
First Posted: December 15, 2015
Last Update Posted: November 17, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
United Therapeutics
December 11, 2015
December 15, 2015
November 17, 2017
May 2016
December 2018   (Final data collection date for primary outcome measure)
Change in 6-minute Walk Distance (6MWD) Measured at Peak Exposure from Baseline to Week 16 [ Time Frame: Baseline and Week 16 ]
The intent of the 6MWD test is to evaluate exercise capacity associated with carrying out activities of daily living. Change in 6MWD from Baseline to Week 16, correlates with the current clinical standard for assessing patient functional status in the treatment of PH and is considered an objective measure of patient functional status. Subjects will be instructed to walk down a corridor at a comfortable speed as far as they can manage for six minutes. Distance <500 meters suggests considerable exercise limitation; Distance 500-800 meters suggests moderate limitation; Distance >800 meters (with no rests) suggests mild or no limitation. Peak exposure 6MWD will occur by conducting 6-minute walk test (6MWT) within 10 to 60 minutes after the most recent dose of study drug dose.
Same as current
Complete list of historical versions of study NCT02630316 on ClinicalTrials.gov Archive Site
  • Change in Peak 6-minute Walk Distance (6MWD) from Baseline to Week 12 [ Time Frame: Baseline and Week 12 ]
    The intent of the 6MWD test is to evaluate exercise capacity associated with carrying out activities of daily living. Change in 6MWD from Baseline to Week 12, correlates with the current clinical standard for assessing patient functional status in the treatment of PH and is considered an objective measure of patient functional status. Subjects will be instructed to walk down a corridor at a comfortable speed as far as they can manage for six minutes. Distance <500 meters suggests considerable exercise limitation; Distance 500-800 meters suggests moderate limitation; Distance >800 meters (with no rests) suggests mild or no limitation. Peak exposure 6MWD will occur by conducting 6-minute walk test (6MWT) within 10 to 60 minutes after the most recent dose of study drug dose.
  • Change in Trough 6-minute Walk Distance (6MWD) from Baseline to Week 15 [ Time Frame: Baseline and Week 15 ]
    The intent of the 6MWD test is to evaluate exercise capacity associated with carrying out activities of daily living. Change in 6MWD from Baseline to Week 15, correlates with the current clinical standard for assessing patient functional status in the treatment of PH and is considered an objective measure of patient functional status. Subjects will be instructed to walk down a corridor at a comfortable speed as far as they can manage for six minutes. Distance <500 meters suggests considerable exercise limitation; Distance 500-800 meters suggests moderate limitation; Distance >800 meters (with no rests) suggests mild or no limitation. Trough exposure 6MWD will occur by conducting 6-minute walk test (6MWT) at least four hours after the most recent study drug dose.
  • Change in plasma concentration of N-terminal pro-Brain Natriuretic Peptide (NT-proBNP) from Baseline to Week 16 [ Time Frame: Baseline and Week 16 ]
    The N-terminal pro-BNP (NT-proBNP) serum concentration is a useful biomarker associated with changes in right heart morphology and function. NT-proBNP serum concentration will be assessed to compare the severity of heart failure at Baseline and Week 16. Blood for NT-proBNP assessment must be drawn prior to conducting the 6-minute walk test (6MWT).
  • Change in Peak 6-minute Walk Distance (6MWD) from Baseline to Week 12 [ Time Frame: Baseline and Week 12 ]
    The intent of the 6MWD test is to evaluate exercise capacity associated with carrying out activities of daily living. Change in 6MWD from Baseline to Week 12, correlates with the current clinical standard for assessing patient functional status in the treatment of PH and is considered an objective measure of patient functional status. Subjects will be instructed to walk down a corridor at a comfortable speed as far as they can manage for six minutes. Distance <500 meters suggests considerable exercise limitation; Distance 500-800 meters suggests moderate limitation; Distance >800 meters (with no rests) suggests mild or no limitation. Peak exposure 6MWD will occur by conducting 6-minute walk test (6MWT) within 10 to 60 minutes after the most recent dose of study drug dose.
  • Change in Trough 6-minute Walk Distance (6MWD) from Baseline to Week 15 [ Time Frame: Baseline and Week 15 ]
    The intent of the 6MWD test is to evaluate exercise capacity associated with carrying out activities of daily living. Change in 6MWD from Baseline to Week 15, correlates with the current clinical standard for assessing patient functional status in the treatment of PH and is considered an objective measure of patient functional status. Subjects will be instructed to walk down a corridor at a comfortable speed as far as they can manage for six minutes. Distance <500 meters suggests considerable exercise limitation; Distance 500-800 meters suggests moderate limitation; Distance >800 meters (with no rests) suggests mild or no limitation. Trough exposure 6MWD will occur by conducting 6-minute walk test (6MWT) at least four hours after the most recent study drug dose.
  • Change in plasma concentration of N-terminal pro-Brain Natriuretic Peptide (NT-proBNP) from Baseline to Week 16 [ Time Frame: Baseline and Week 16 ]
    The N-terminal pro-BNP (NT-proBNP) serum concentration is a useful biomarker associated with changes in right heart morphology and function. NT-proBNP serum concentration will be assessed to compare the severity of heart failure at Baseline and Week 16. Blood for NT-proBNP assessment must be drawn prior to conducting the 6-minute walk test (6MWT).
  • Change in Forced Expiratory Volume (FEV1) in One Second from Baseline to Week 16 [ Time Frame: Baseline and Week 16 ]
    Change in pulmonary function following inhaled treprostinil therapy will be measured by Forced Expiratory Volume in One Second (FEV1), the maximal amount of air forcefully exhaled in 1 second, calculated from a Pulmonary Function Test (PFT) performed at Baseline and Week 16.
  • Change in Forced Vital Capacity (FVC) from Baseline to Week 16 [ Time Frame: Baseline and Week 16 ]
    Change in pulmonary function following inhaled treprostinil therapy will be measured by Forced Vital Capacity (FVC), calculated from a Pulmonary Function Test (PFT) performed at Baseline and Week 16.
  • Change in Total Lung Capacity (TLC) from Baseline to Week 16 [ Time Frame: Baseline and Week 16 ]
    Change in pulmonary function following inhaled treprostinil therapy will be measured by Total Lung Capacity (TLC), calculated from a Pulmonary Function Test (PFT) performed at Baseline and Week 16.
  • Change in Lung Diffusion Capacity (DLCO) from Baseline to Week 16 [ Time Frame: Baseline and Week 16 ]
    Change in pulmonary function following inhaled treprostinil therapy will be measured by Lung Diffusion Capacity (DLCO), calculated from a Pulmonary Function Test (PFT) performed at Baseline and Week 16.
  • Incidence of Adverse Events Among Participants through 16 Weeks [ Time Frame: 16 Weeks ]
    The incidence of adverse events among participants throughout the 16 week study will be measured by the number of participants analyzed and the percentage of those participants who experienced an adverse event.
Not Provided
Not Provided
 
Safety and Efficacy of Inhaled Treprostinil in Adult PH With ILD Including CPFE
A Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Safety and Efficacy of Inhaled Treprostinil in Subjects With Pulmonary Hypertension Due to Parenchymal Lung Disease
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).
Not Provided
Interventional
Phase 2
Phase 3
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
  • Pulmonary Hypertension
  • Interstitial Lung Disease
  • Combined Pulmonary Fibrosis and Emphysema
  • Drug: Inhaled Treprostinil
    Inhaled treprostinil (6 mcg/breath) administered four times daily
    Other Name: Tyvaso
  • Drug: Placebo
    Placebo administered four times daily
  • Placebo Comparator: Placebo
    Matching placebo inhaled using an ultrasonic nebulizer four times daily
    Intervention: Drug: Placebo
  • Active Comparator: Active Inhaled Treprostinil
    Active Treprostinil for inhalation solution (0.6 mg/mL) delivered via an ultrasonic nebulizer which emits a dose of approximately 6 mcg per breath. Inhaled four times daily and titrated up to a maximum of 12 breaths four times daily
    Intervention: Drug: Inhaled Treprostinil
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
314
December 2018
December 2018   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: All
18 Years and older   (Adult, Senior)
No
Contact: Kristan Rollins krollins@unither.com
Contact: Millicent Clark mclark@unither.com
United States
 
 
NCT02630316
RIN-PH-201
Yes
Not Provided
Not Provided
United Therapeutics
United Therapeutics
Not Provided
Not Provided
United Therapeutics
November 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP