Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
This is a 24-month, multi-center, prospective, open-label, safety evaluation in PAH subjects following transition from a stable dose of inhaled iloprost (Ventavis).
Subjects are to be evaluated for safety throughout the course of the study while secondary assessments will be conducted at Baseline, Week 6, Week 12, and Months 6, 12, 18 and 24 following initiation of treprostinil sodium.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Inhaled treprostinil | Experimental | Solution for oral inhalation treprostinil (0.6 mg/mL). Inhaled via an ultrasonic nebulizer which provides a dose of 6mcg of treprostinil per breath. Doses are titrated up to 12 breaths four times daily. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Inhaled treprostinil | Drug |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Adverse Events | Overall safety of transitioning from inhaled iloprost to inhaled treprostinil was assessed by type and frequency of adverse events. | up to 24 months |
| Measure | Description | Time Frame |
|---|---|---|
| Six-minute Walk Distance (6MWD) | Change in 6MWD from Baseline to Week 12. The 6-minute walk test (6MWT) was conducted at Baseline (10-30 minutes following the last dose of inhaled iloprost) and at Week 12 (10-60 minutes following the dose of inhaled treprostinil). The change in distance (meters) between Baseline and Week 12 is reported below. | Baseline and 12 weeks |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama Birmingham | Birmingham | Alabama | 35294 | United States | ||
| UCSD Medical Center |
Not applicable given this was an open-label study.
Recruitment began 09-Dec-2008 and ended 09-Mar-2010. All recruitment took place in medical clinic settings.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Inhaled Treprostinil | Inhaled treprostinil was given four times daily at doses titrated up to 12 breaths. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Quality of Life (QoL) Assessment: Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) | Change in CAMPHOR Scores from Baseline to Week 12. The CAMPHOR is a health related quality of life instrument validated for pulmonary hypertension that assesses impairment (symptoms), disability (activities) and quality of life. The questionnaire is divided into three sections; Symptoms (Scores 0-25; high scores indicate more symptoms), Activity (Score 0-30; low score indicates good functioning)and Quality of Life (0-25; high scores indicate poor QoL). The sum of these scores equates to the Total score (0-80). In the CAMPHOR scores, lower scores indicate improvements. | Baseline and 12 weeks |
| Treatment Satisfaction Questionnaire of Medication (TSQM) | Change in TSQM score from Baseline to Week 12. The TSQM is a validated instrument (Health and Quality of Life Outcomes 2004, 2:12) that measures major dimensions of patient satisfaction with medications. The questionnaire is comprised of 15 questions which fall into one of four categories; Effectiveness, Side-Effects, Convenience, and Global Satisfaction. Responses are scaled on a seven point bipolar scale from 'Extremely Satisfied' to 'Extremely Dissatisfied' where higher scores indicate improvements (total scores from 0-100). The questionnaire was completed at Baseline and Week 12. The Baseline questionnaire focused on the subject's satisfaction with inhaled iloprost treatment, while the questionnaire completed at Week 12 focused on the subject's satisfaction with inhaled treprostinil. | Baseline and 12 weeks |
| Patient Impression of Change | The patient impression of change (PIC) was three single therapy-related questions related to the subjects overall impression of the transition from inhaled iloprost to inhaled treprostinil. Subjects were surveyed related to their overall impression of the transition from inhaled iloprost to inhaled treprostinil at Week 12. | Baseline and 12 weeks |
| N-terminal Prohormone of Brain Natriuretic Peptide (NT-proBNP) | Change in NTpro-BNP from Baseline to Week 12. Blood samples were collected for plasma NTpro-BNP analysis during the study. | Baseline and Week 12 |
| World Health Organization (WHO) Functional Class | Change in WHO Functional Class (FC) from Baseline to Week 12. Data presented as percent of subjects who either improved FC, worsened FC, or had no change in FC from Baseline to Week 12. | Baseline and 12 Weeks |
| Drug Administration Activities Questionnaire | Change in tasks from Baseline to Week 12. At Baseline and Week 12, subjects provided information related to the daily administration and time requirements of inhaled iloprost (Baseline) and inhaled treprostinil (Week 12). | Baseline and 12 weeks |
| La Jolla |
| California |
| 82037 |
| United States |
| Ronald Reagan UCLA Medical Center | Los Angeles | California | 90095 | United States |
| Kansas University Medical Center | Kansas City | Kansas | 66160 | United States |
| Long Island Jewish Medical Center - North Shore | New Hyde Park | New York | 11040 | United States |
| Beth Israel Medical Center | New York | New York | 10003-3314 | United States |
| Cornell University Medical Center | New York | New York | 10021 | United States |
| Columbia Presbyterian Medical Center | New York | New York | 10032 | United States |
| Mary M. Parkes Center for Asthma, Allergy and Pulmonary Care | Rochester | New York | 14623 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| Ohio State University Medical Center | Columbus | Ohio | 43210 | United States |
| Allegheny General Hospital | Pittsburgh | Pennsylvania | 15212 | United States |
| University of Pittsburg Medical Center | Pittsburgh | Pennsylvania | 15213 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | United States |
| Baylor College of Medicine | Houston | Texas | 77030 | United States |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Inhaled Treprostinil | Inhaled treprostinil was titrated up to 12 breaths four times daily. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| |||||||||||||||||||||||
| Age Continuous | Mean | Standard Deviation | years |
| ||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||||||||||||||
| Region of Enrollment | Number | participants |
| |||||||||||||||||||||||
| Pulmonary Arterial Hypertension (PAH) etiology | Number | participants |
| |||||||||||||||||||||||
| Background PAH therapy | Number | participants |
| |||||||||||||||||||||||
| Six-minute walk distance (6MWD) | Median | Full Range | meters |
| ||||||||||||||||||||||
| N-terminal prohormone brain natriuretic peptide (NT-proBNP) | Median | Full Range | pg/mL |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Adverse Events | Overall safety of transitioning from inhaled iloprost to inhaled treprostinil was assessed by type and frequency of adverse events. | All subjects who received at least one dose of inhaled treprostinil were included in the safety analysis population. | Posted | Number | number of events | up to 24 months |
|
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Six-minute Walk Distance (6MWD) | Change in 6MWD from Baseline to Week 12. The 6-minute walk test (6MWT) was conducted at Baseline (10-30 minutes following the last dose of inhaled iloprost) and at Week 12 (10-60 minutes following the dose of inhaled treprostinil). The change in distance (meters) between Baseline and Week 12 is reported below. | Subjects enrolled at Week 12 visit. | Posted | Median | Full Range | meters | Baseline and 12 weeks |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Quality of Life (QoL) Assessment: Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) | Change in CAMPHOR Scores from Baseline to Week 12. The CAMPHOR is a health related quality of life instrument validated for pulmonary hypertension that assesses impairment (symptoms), disability (activities) and quality of life. The questionnaire is divided into three sections; Symptoms (Scores 0-25; high scores indicate more symptoms), Activity (Score 0-30; low score indicates good functioning)and Quality of Life (0-25; high scores indicate poor QoL). The sum of these scores equates to the Total score (0-80). In the CAMPHOR scores, lower scores indicate improvements. | Subjects who were still enrolled and completed the questionnaire at Baseline and Week 12. Total analysis population was less for the activity score and total score (N = 67). | Posted | Mean | Full Range | units on a scale | Baseline and 12 weeks |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Treatment Satisfaction Questionnaire of Medication (TSQM) | Change in TSQM score from Baseline to Week 12. The TSQM is a validated instrument (Health and Quality of Life Outcomes 2004, 2:12) that measures major dimensions of patient satisfaction with medications. The questionnaire is comprised of 15 questions which fall into one of four categories; Effectiveness, Side-Effects, Convenience, and Global Satisfaction. Responses are scaled on a seven point bipolar scale from 'Extremely Satisfied' to 'Extremely Dissatisfied' where higher scores indicate improvements (total scores from 0-100). The questionnaire was completed at Baseline and Week 12. The Baseline questionnaire focused on the subject's satisfaction with inhaled iloprost treatment, while the questionnaire completed at Week 12 focused on the subject's satisfaction with inhaled treprostinil. | Subjects who completed the TSQM at Baseline and Week 12. Total analysis population was less for the Convenience Score (N=67) and Global Satisfaction Score (N=66). | Posted | Mean | Full Range | units on a scale | Baseline and 12 weeks |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Patient Impression of Change | The patient impression of change (PIC) was three single therapy-related questions related to the subjects overall impression of the transition from inhaled iloprost to inhaled treprostinil. Subjects were surveyed related to their overall impression of the transition from inhaled iloprost to inhaled treprostinil at Week 12. | Subjects who completed questionnaire at Baseline and Week 12 | Posted | Number | percentage of patients | Baseline and 12 weeks |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | N-terminal Prohormone of Brain Natriuretic Peptide (NT-proBNP) | Change in NTpro-BNP from Baseline to Week 12. Blood samples were collected for plasma NTpro-BNP analysis during the study. | Subjects with a NTproBNP sample drawn at Baseline and Week 12 | Posted | Median | Full Range | pg/mL | Baseline and Week 12 |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | World Health Organization (WHO) Functional Class | Change in WHO Functional Class (FC) from Baseline to Week 12. Data presented as percent of subjects who either improved FC, worsened FC, or had no change in FC from Baseline to Week 12. | Subjects still enrolled at Week 12 with a WHO Functional Class at Baseline and Week 12. | Posted | Number | percentage of subjects | Baseline and 12 Weeks |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Drug Administration Activities Questionnaire | Change in tasks from Baseline to Week 12. At Baseline and Week 12, subjects provided information related to the daily administration and time requirements of inhaled iloprost (Baseline) and inhaled treprostinil (Week 12). | Subjects who completed the questionnaire at Baseline and Week 12. | Posted | Mean | Standard Deviation | minutes | Baseline and 12 weeks |
|
|
up to 56 weeks
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Inhaled Treprostinil | Inhaled treprostinil was titrated up to 12 breaths four times daily. | 10 | 73 | 71 | 73 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pneumonia | Infections and infestations | MedDRA (12.0) |
| ||
| Pulmonary hypertension | Cardiac disorders | MedDRA (12.0) |
| ||
| Cor pulmonale | Cardiac disorders | MedDRA (12.0) |
| ||
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) |
| ||
| Atrial fibrillation | Cardiac disorders | MedDRA (12.0) |
| ||
| Dehydration | Metabolism and nutrition disorders | MedDRA (12.0) |
| ||
| gastrointestinal hemorrhage | Gastrointestinal disorders | MedDRA (12.0) |
| ||
| Influenza | Infections and infestations | MedDRA (12.0) |
| ||
| Myocardial infarction | Cardiac disorders | MedDRA (12.0) |
| ||
| Sick sinus syndrome | Cardiac disorders | MedDRA (12.0) |
| ||
| Syncope | Nervous system disorders | MedDRA (12.0) |
| ||
| psychotic disorder | Psychiatric disorders | MedDRA (12.0) |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) |
| ||
| Headache | Nervous system disorders | MedDRA (12.0) |
| ||
| Nausea | Gastrointestinal disorders | MedDRA (12.0) |
| ||
| Chest discomfort | General disorders | MedDRA (12.0) |
| ||
| upper respiratory tract infection | Infections and infestations | MedDRA (12.0) |
| ||
| nasopharyngitis | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) |
| ||
| flushing | Vascular disorders | MedDRA (12.0) |
| ||
| dizziness | Nervous system disorders | MedDRA (12.0) |
| ||
| palpitations | Cardiac disorders | MedDRA (12.0) |
| ||
| throat irritation | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) |
| ||
| fatigue | General disorders | MedDRA (12.0) |
| ||
| productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) |
| ||
| oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) |
| ||
| diarrhea | Gastrointestinal disorders | MedDRA (12.0) |
| ||
| bronchitis | Infections and infestations | MedDRA (12.0) |
| ||
| chest pain | General disorders | MedDRA (12.0) |
| ||
| dyspnea | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) |
| ||
| pain in jaw | Musculoskeletal and connective tissue disorders | MedDRA (12.0) |
| ||
| rash | Skin and subcutaneous tissue disorders | MedDRA (12.0) |
| ||
| somnolence | Nervous system disorders | MedDRA (12.0) |
| ||
| epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) |
| ||
| back pain | Musculoskeletal and connective tissue disorders | MedDRA (12.0) |
| ||
| fluid retention | Metabolism and nutrition disorders | MedDRA (12.0) |
| ||
| pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (12.0) |
| ||
| sinusitis | Infections and infestations | MedDRA (12.0) |
|
Small, open-label study without comparison arm.
Any publication of the result of this trial must be consistent with the United Therapeutics publication policy. The rights of the investigator and of the sponsor with regard to publication of the results of this trial are described in the investigator contract.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Inhaled Treprostinil Program Head | United Therpaeutics Corporation | 919-485-8350 | clinicalrecordsmanagment@unither.com |
| ID | Term |
|---|---|
| D000081029 | Pulmonary Arterial Hypertension |
| D053120 | Respiratory Aspiration |
| ID | Term |
|---|---|
| D006976 | Hypertension, Pulmonary |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D012120 | Respiration Disorders |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| C427248 | treprostinil |
Not provided
Not provided
Not provided
| Congenital heart disease (CHD)-repaired shunt |
|
| CHD-unrepaired shunt |
|
| HIV |
|
| Phosphodiesterase-5 inhibitor (PDE-5i) |
|
| ERA and PDE-5i |
|
| Title | Measurements |
|---|---|
|
| Serious events |
|
|
|
|
|
|
|
|
| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
|
|
| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
| Improvement |
| |||||
| No Change |
| |||||
| Worsened |
|
| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
| Week 12 Total Daily Dosing Time |
| |||||
| Change in Time Total Daily Dosing Time |
|