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A Phase 3 Adaptive Study to Evaluate the Safety and Efficacy of Inhaled Treprostinil in Patients With PH Due to COPD

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ClinicalTrials.gov Identifier: NCT03496623
Recruitment Status : Recruiting
First Posted : April 12, 2018
Last Update Posted : April 22, 2019
Sponsor:
Information provided by (Responsible Party):
United Therapeutics

Brief Summary:
This is a multicenter, randomized, double-blind, placebo-controlled, 30-week, adaptive cross-over study, with a Treatment Period of approximately 26 weeks under the Original Design or, if applicable, a 17-week parallel study, with a Treatment Period of approximately 14 weeks under the Contingent Design.

Condition or disease Intervention/treatment Phase
Pulmonary Hypertension Chronic Obstructive Pulmonary Disease Drug: Inhaled treprostinil solution Drug: Placebo solution Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 314 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Placebo-controlled, Double-blind, Adaptive Study to Evaluate the Safety and Efficacy of Inhaled Treprostinil in Patients With Pulmonary Hypertension Due to Chronic Obstructive Pulmonary Disease (PH-COPD)
Actual Study Start Date : June 27, 2018
Estimated Primary Completion Date : September 2020
Estimated Study Completion Date : September 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Inhaled Treprostinil
Inhaled treprostinil delivered via an ultrasonic nebulizer with a target dosing regimen of 12 breaths (72 mcg) 4 times daily (QID)
Drug: Inhaled treprostinil solution
Treprostinil inhalation solution

Placebo Comparator: Placebo
Placebo delivered via an ultrasonic nebulizer for QID administration
Drug: Placebo solution
Placebo solution




Primary Outcome Measures :
  1. Change in 6-Minute Walk Distance (6MWD) from Baseline to Week 12 [ Time Frame: Baseline to Week 12 ]
    The intent of the 6-Minute Walk Test (6MWT) is a validated and reliable measure of exercise ability in patients with chronic respiratory diseases.


Secondary Outcome Measures :
  1. Change in Borg dyspnea score from Baseline to Week 12 [ Time Frame: Baseline to Week 12 ]
    The Borg dyspnea score is a 10 point scale rating the maximum level of dyspnea experienced during the 6MWT. Scores range from 0 (no dyspnea at all) to 10 (very, very severe dyspnea).

  2. Change in quality of life (QOL) measured by St. George's Respiratory Questionnaire (SGRQ) from Baseline to Week 12 [ Time Frame: Baseline to Week 12 ]
    The SGRQ is a designed to measure impact on overall health, daily life, and perceived well-being in patients with obstructive airways disease. Scores range from 0 to 100, with higher scores indicating more limitations.

  3. Change in QOL measured by the University of California San Diego Shortness of Breath Questionnaire (UCSD SOBQ) from Baseline to Week 12 [ Time Frame: Baseline to Week 12 ]
    The UCSD SOBQ is a self-administered rating of dyspnea associated with activities of daily living. The questionnaire uses a 6-point scale where 0 = "not at all" and 5 = "maximal or unable to do because of breathlessness"

  4. Change in plasma concentration of N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) levels from Baseline to Week 12 [ Time Frame: Baseline to Week 12 ]
    The NT-proBNP concentration is a biomarker associated with changes in right heart morphology and function.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

    1. Females of childbearing potential must not be pregnant or lactating, and will either abstain from intercourse, or use two medically acceptable, highly-effective forms of contraception for the duration of study.
    2. 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.
  3. The subject has a clinical diagnosis of PH-COPD (WHO Group 3) using the Global Initiative for Chronic Obstructive Lung Disease (GOLD) diagnostic criteria and post-bronchodilator spirometry.
  4. The subject has a resting peripheral capillary oxygen saturation (SpO2) ≥90%, with or without supplemental oxygen, not to exceed 10 L/min.
  5. The subject's baseline 6MWD must be at least 100 meters.
  6. Subjects are required to have a right heart catheterization (RHC) including a vasodilator test, with and without oxygen challenge, prior to randomization with the following documented parameters:

    1. Pulmonary vascular resistance (PVR) >4 Wood Units (WU) and
    2. A pulmonary artery wedge pressure (PAWP) or left ventricular end-diastolic pressure (LVEDP) of ≤15 mmHg
    3. A pulmonary arterial pressure mean (PAPm) of ≥30 mmHg
  7. Subjects on a chronic COPD medication therapy must be on a stable and optimized dose for ≥30 days prior to Screening Visit.
  8. Subjects on medications for conditions unrelated to COPD must be on a stable and optimized dose for ≥30 days prior to start of Screening Visit 1 and remain on stable doses throughout the Screening Period. Exceptions are anticoagulants and diuretics.
  9. 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. The subject has a diagnosis of pulmonary arterial hypertension (PAH) or PH for reasons other than COPD.
  2. The subject has a confirmed diagnosis of WHO Group 3 PH, other than COPD.
  3. The subject has received any FDA approved therapy including: prostacyclin therapy, prostacyclin receptor agonist, endothelin receptor antagonist (ERA), phosphodiesterase type 5 inhibitor (PDE5-I), or soluble guanylate cyclase (sGC) within 60 days of Screening.
  4. The subject has previously been diagnosed with alpha-1 antitrypsin deficiency.
  5. The subject has shown intolerance to prostanoid therapy.
  6. The subject is unable to tolerate low dose (3 breaths, 18 mcg) study drug and/or unable to follow dosing regimen during the Screening Period.
  7. The subject has evidence of clinically significant left-sided heart disease. Note: Subjects with abnormal left ventricular function attributable entirely to impaired left ventricular filling due to the effects of right ventricular overload will not be excluded, but requires the Sponsor's approval.
  8. The subject is receiving > 10 L/min of oxygen supplementation by any mode of delivery at rest between the Screening Visit and Baseline Visit.
  9. Current use of any inhaled tobacco/marijuana products.
  10. Significant history of substance abuse within 6 months prior to start of Screening Visit.
  11. Exacerbation of COPD for active pulmonary or upper respiratory infection within 60 days prior to Screening Visit.
  12. Initiation of pulmonary rehabilitation within 12 weeks prior to the Screening Visit 1.
  13. The subject has an uncontrolled medical condition deemed by an Investigator to pose an undue risk to the subject.
  14. The subject has any form of congenital heart disease (repaired or unrepaired; other than a patent foramen ovale).
  15. The subject has a Body Mass Index ≥45 kg/m2 at Screening Visit 1.
  16. The subject has any musculoskeletal disorder or has any other condition that would limit ambulation.
  17. Use of any investigational drug/device, or participation in any investigational study with therapeutic intent within 30 days prior to Screening Visit 1.
  18. Any other clinically significant illness or abnormal laboratory value(s) that might adversely affect the interpretation of the study data.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03496623


Contacts
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Contact: Prakash Sista, Ph.D. 240-821-1661 304perfectstudy@lungbiotechnology.com
Contact: Mary Lou Tomson, M.A. 240-821-1881 304perfectstudy@lungbiotechnology.com

  Show 24 Study Locations
Sponsors and Collaborators
United Therapeutics

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Responsible Party: United Therapeutics
ClinicalTrials.gov Identifier: NCT03496623     History of Changes
Other Study ID Numbers: RIN-PH-304
First Posted: April 12, 2018    Key Record Dates
Last Update Posted: April 22, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by United Therapeutics:
Pulmonary Hypertension
COPD
Inhaled Treprostinil
6-Minute Walk Test
Tyvaso

Additional relevant MeSH terms:
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Hypertension
Lung Diseases
Lung Diseases, Obstructive
Pulmonary Disease, Chronic Obstructive
Hypertension, Pulmonary
Vascular Diseases
Cardiovascular Diseases
Respiratory Tract Diseases
Pharmaceutical Solutions
Treprostinil
Antihypertensive Agents