An Open Label Extension Study to Evaluate Inhaled Treprostinil in Adult PH With ILD Including CPFE

Sponsor
United Therapeutics (Industry)
Overall Status
Completed
CT.gov ID
NCT02633293
Collaborator
(none)
243
93
1
58.9
2.6
0

Study Details

Study Description

Brief Summary

This is a multicenter, open-label 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 266 patients who completed all required assessments in the RIN-PH-201 study at approximately 100 clinical trial centers. The study will continue Your participation in this study is voluntary and will last until you discontinue from the study or the study ends. The study will continue until each subject reaches the Week 108 visit or until inhaled treprostinil become commercially available for patients with PH associated with ILD including CPFE (whichever is sooner).

Condition or Disease Intervention/Treatment Phase
  • Drug: Inhaled Treprostinil
Phase 2/Phase 3

Study Design

Study Type:
Interventional
Actual Enrollment :
243 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
An Open-Label Extension Study of Inhaled Treprostinil in Subjects With Pulmonary Hypertension Due to Parenchymal Lung Disease
Actual Study Start Date :
Sep 1, 2016
Actual Primary Completion Date :
Jul 30, 2021
Actual Study Completion Date :
Jul 30, 2021

Arms and Interventions

Arm Intervention/Treatment
Experimental: Inhaled Treprostinil

Open-label access

Drug: Inhaled Treprostinil
Inhaled treprostinil (6 mcg/breath) administered four times daily
Other Names:
  • Tyvaso
  • Outcome Measures

    Primary Outcome Measures

    1. Change in Peak 6-Minute Walk Distance (6MWD) from Baseline to the Week 48 Visit [Baseline and Week 48]

      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 48, 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 6MWT within 10 to 60 minutes after the most recent dose of study drug dose.

    Secondary Outcome Measures

    1. Change in plasma concentration of N-terminal pro-Brain Natriuretic Peptide (NT-proBNP) from Baseline to the Week 48 Visit [Baseline and Week 48]

      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 48. Blood for NT-proBNP assessment must be drawn prior to conducting the 6-minute walk test (6MWT).

    2. Change in Forced Expiratory Volume (FEV1) in One Second from Baseline to Week 48 [Baseline and Week 48]

      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 48.

    3. Change in Forced Vital Capacity (FVC) from Baseline to Week 48 [Baseline and Week 48]

      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 48.

    4. Change in Total Lung Capacity (TLC) from Baseline to Week 48 [Baseline and Week 48]

      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 48.

    5. Change in Lung Diffusion Capacity (DLCO) from Baseline to Week 48 [Baseline and Week 48]

      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 48.

    6. Incidence of Adverse Events Among Participants through Week 108 [Week 108]

      Adverse events among participants will be recorded throughout participation in the study. The incidence of adverse events among participants through Week 108 will be described by the number of participants analyzed and the percentage of those participants who experienced an adverse event until Week 108.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:
    1. Subject voluntarily gives informed consent to participate in the study.

    2. The subject participated in study RIN-PH-201, remained on study drug, was compliant with RIN-PH-201 study procedures or was enrolled in study RIN-PH-201 at the time that the study was discontinued by the sponsor.

    3. Females of reproductive potential must be non-pregnant (as confirmed by a urine pregnancy test at Baseline) and non-lactating, and will:

    • Either abstain from intercourse (when it is in line with their preferred and usual lifestyle), or

    • Use two medically acceptable, highly-effective forms of contraception for the duration of study, and at least 30 days after discontinuing study drug.

    1. Males must use a condom for the duration of treatment and for at least 48 hours after discontinuing study drug.
    Exclusion Criteria:
    1. The subject is pregnant or lactating.

    2. The subject was prematurely discontinued from study RIN-PH-201.

    3. The subject developed a concurrent illness or condition during the conduct of RIN-PH-201 which, in the opinion of the Investigator, would represent a risk to overall health if they enrolled in this study.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 University of Alabama at Birmingham Birmingham Alabama United States 35294
    2 IMC-Diagnostic & Medical Clinic Mobile Alabama United States 36604
    3 Arizona Pulmonary Specialists, Ltd. Phoenix Arizona United States 85012
    4 University of Arizona Tucson Arizona United States 85724
    5 Cedars-Sinai Medical Center Beverly Hills California United States 90211
    6 University of California, San Francisco-Fresno Fresno California United States 93701
    7 University of California San Diego La Jolla California United States 92093
    8 VA Long Beach Healthcare System Long Beach California United States 90822
    9 University of Southern California Los Angeles California United States 90033
    10 Department of Veterans Affairs Greater Los Angeles Healthcare System Los Angeles California United States 90073
    11 Pacific Pulmonary Medical Group Riverside California United States 92505
    12 Kaiser Permanente - Roseville Roseville California United States 95825
    13 University of California Davis Medical Center Sacramento California United States 95817
    14 Kaiser Permanente San Francisco California United States 94115
    15 University of Colorado Hospital - Cardiac and Vascular Center Aurora Colorado United States 80045
    16 National Jewish Health Denver Colorado United States 80206
    17 Georgetown University Hospital Washington District of Columbia United States 20007
    18 MedStar Heart & Vascular Institute Washington District of Columbia United States 20010
    19 Florida Lung, Asthma & Sleep Specialists, P.A. Celebration Florida United States 34747
    20 St. Francis Sleep, Allergy and Lung Institute Clearwater Florida United States 33765
    21 University of Florida Clinical Research Center Gainesville Florida United States 32610
    22 University of Florida College of Medicine, Jacksonville Jacksonville Florida United States 32209
    23 Mayo Clinic Jacksonville Jacksonville Florida United States 32224
    24 St. Vincent's Lung, Sleep, and Critical Care Specialists Jacksonville Florida United States 33204
    25 University of Miami Miami Florida United States 33136
    26 Florida Hospital Orlando Florida United States 32804
    27 University of South Florida Tampa Florida United States 33606
    28 Cleveland Clinic Florida Weston Florida United States 33331
    29 The Emory Clinic Atlanta Georgia United States 30322
    30 Piedmont - Georgia Lung Associates Austell Georgia United States 30106
    31 Wellstar Medical Group Marietta Georgia United States 30060
    32 Northwestern University Chicago Illinois United States 60611
    33 Rush University Medical Center Chicago Illinois United States 60612
    34 University of Illinois at Chicago Hospital Chicago Illinois United States 60612
    35 University of Chicago Medical Center Chicago Illinois United States 60637
    36 Loyola University Medical Center Maywood Illinois United States 60153
    37 Indiana University Hospital Indianapolis Indiana United States 46202
    38 Community Heart and Vascular Hospital Indianapolis Indiana United States 46250
    39 St. Vincent Medical Group, Inc. Indianapolis Indiana United States 46260
    40 University of Iowa Hospitals & Clinics Iowa City Iowa United States 52242
    41 University of Kansas Medical Center Kansas City Kansas United States 66160
    42 University of Louisville Physicians Outpatient Center Louisville Kentucky United States 40202
    43 Louisiana State University Health Sciences Center New Orleans New Orleans Louisiana United States 70112
    44 Maine Medical Center South Portland Maine United States 04106
    45 University of Maryland Medical Center Baltimore Maryland United States 21201
    46 Johns Hopkins University Baltimore Maryland United States 21205
    47 Tufts Medical Center Boston Massachusetts United States 02111
    48 Massachusetts General Hospital Boston Massachusetts United States 02114
    49 Brigham and Women's Hospital Boston Massachusetts United States 02115
    50 Spectrum Health Medical Center Grand Rapids Michigan United States 49503
    51 University of Minnesota Minneapolis Minnesota United States 55455
    52 Mayo Clinic Rochester Minnesota United States 55905
    53 University of Mississippi Medical Center Jackson Mississippi United States 39216
    54 Saint Luke's Hospital of Kansas City Kansas City Missouri United States 64111
    55 Washington University Hospital Saint Louis Missouri United States 63110
    56 The University of New Mexico Albuquerque New Mexico United States 87131
    57 Albany Medical College Albany New York United States 12208
    58 New York Methodist Hospital Brooklyn New York United States 11215
    59 Northwell Health New Hyde Park New York United States 11040
    60 NYU Langone Medical Center New York New York United States 10016
    61 The Mount Sinai Hospital New York New York United States 10029
    62 Weill Cornell Medical Center New York New York United States 10065
    63 University of North Carolina at Chapel Hill Chapel Hill North Carolina United States 27599
    64 Duke University Medical Center Durham North Carolina United States 27710
    65 Pinehurst Medical Clinic, Inc. Pinehurst North Carolina United States 28374
    66 The Carl and Edyth Lindner Research Center at The Christ Hospital Cincinnati Ohio United States 45219
    67 University of Cincinnati Health Cincinnati Ohio United States 45267
    68 University Hospitals Cleveland Medical Center Cleveland Ohio United States 44106
    69 Cleveland Clinic Cleveland Ohio United States 44195
    70 The Ohio State University Medical Center Columbus Ohio United States 43221
    71 Integris Baptist Medical Center Oklahoma City Oklahoma United States 73112
    72 Hospital of University of Pennsylvania Philadelphia Pennsylvania United States 19104
    73 Allegheny General Hospital Pittsburgh Pennsylvania United States 15212
    74 University of Pittsburgh Medical Center Pittsburgh Pennsylvania United States 15213
    75 AnMed Health Pulmonary and Sleep Medicine Anderson South Carolina United States 29621
    76 Medical University of South Carolina Charleston South Carolina United States 29425
    77 Statcare Pulmonary Consultants Knoxville Tennessee United States 37919
    78 Baylor University Medical Center at Dallas Dallas Texas United States 75246
    79 UT Southwestern Medical Center Dallas Texas United States 75390
    80 Texas Tech University Health Sciences Center El Paso Texas United States 79905
    81 Houston Methodist Houston Texas United States 77030
    82 Michael E. DeBakey VA Medical Center Houston Texas United States 77030
    83 The University of Texas Health Science Center at Houston Houston Texas United States 77030
    84 The University of Texas Health Science Center at San Antonio San Antonio Texas United States 78229
    85 University of Vermont, Vermont Lung Center Colchester Vermont United States 05446
    86 Inova Fairfax Hospital Fairfax Virginia United States 22042
    87 Sentara Cardiovascular Research Institute Norfolk Virginia United States 23507
    88 Pulmonary Associates of Richmond Richmond Virginia United States 23229
    89 University of Washington Medical Center Seattle Washington United States 98195
    90 University of Wisconsin School of Medicine and Public Health Madison Wisconsin United States 53792
    91 Aurora St. Luke's Medical Center Milwaukee Wisconsin United States 53215
    92 Medical College of Wisconsin/Froedtert Hospital Milwaukee Wisconsin United States 53226
    93 Alliance Cardio Pulmonary Research Group, Inc. Guaynabo Puerto Rico 00968

    Sponsors and Collaborators

    • United Therapeutics

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    United Therapeutics
    ClinicalTrials.gov Identifier:
    NCT02633293
    Other Study ID Numbers:
    • RIN-PH-202
    First Posted:
    Dec 17, 2015
    Last Update Posted:
    Nov 5, 2021
    Last Verified:
    Nov 1, 2021

    Study Results

    No Results Posted as of Nov 5, 2021