Pulmonary Arterial HypertensionPhase 4NCT00741819
Safety Evaluation of Inhaled Treprostinil Administration Following Transition From Inhaled Ventavis in Pulmonary Arterial Hypertension (PAH) Subjects
A Phase 4 study testing Inhaled treprostinil in people with Pulmonary Arterial Hypertension, run by United Therapeutics at 15 sites in United States.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Pulmonary Arterial Hypertension
- What is being tested?
- Inhaled treprostinil
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 73 participants
- Where
- 15 sitesUnited States
- Study dates
- Sep 2008to Dec 2010
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Between 18 and 75 years of age
- PAH diagnosis defined by following WHO Group I categories: idiopathic/familial, associated with unrepaired or repaired congenital systemic-to-pulmonary shunts (repaired >/= 5 years), associated with collagen vascular disease, associated with HIV, associated with appetite suppressant/toxin use
- Baseline six-minute walk distance (6MWD) >/= 250 meters
- Currently receiving Ventavis and be stable at current dose for 30 days prior to Baseline
- If currently receiving other approved background therapy (i.e. endothelin receptor antagonist or phosphodiesterase-5-inhibitor or both) must be on stable dose for 30 days prior to Baseline
- Previous testing (e.g. right heart catheterization, echocardiography) consistent with diagnosis of PAH
Who cannot join
- Nursing or pregnant
- Has acute concomitant disease (e.g. portal hypertension, chronic thromboembolic disease, pulmonary veno-occlusive disease, etc) other than those accepted as part of the inclusion criteria or has had atrial septostomy
- History of uncontrolled sleep apnea, left-sided heart disease, or parenchymal lung disease
- Use of investigational drug within 30 days of Baseline
Trial locations and contacts
15 sites in 1 country.
15 sites in 1 country
United States15 sites
University of Alabama Birmingham
Birmingham, Alabama, 35294
Ohio State University Medical Center
Columbus, Ohio, 43210
Duke University Medical Center
Durham, North Carolina, 27710
Baylor College of Medicine
Houston, Texas, 77030
Kansas University Medical Center
Kansas City, Kansas, 66160
UCSD Medical Center
La Jolla, California, 82037
Ronald Reagan UCLA Medical Center
Los Angeles, California, 90095
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Long Island Jewish Medical Center - North Shore
New Hyde Park, New York, 11040
Beth Israel Medical Center
New York, New York, 10003-3314
Cornell University Medical Center
New York, New York, 10021
Columbia Presbyterian Medical Center
New York, New York, 10032
Allegheny General Hospital
Pittsburgh, Pennsylvania, 15212
University of Pittsburg Medical Center
Pittsburgh, Pennsylvania, 15213
Mary M. Parkes Center for Asthma, Allergy and Pulmonary Care
Rochester, New York, 14623
About this study
Official title: An Open Label, Multi-center Study Evaluating the Safety of Long-term Inhaled Treprostinil Administration Following Transition From Inhaled Ventavis® (Iloprost) in Subjects With Pulmonary Arterial Hypertension.
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.
- Conditions
- Pulmonary Arterial Hypertension
- Being tested
- Inhaled treprostinil
- Keywords
- pulmonary arterial hypertension, PAH, treprostinil sodium, inhalation, ventavis, iloprost
- Medical subject headings
- Pulmonary Arterial Hypertension, Respiratory Aspiration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Inhaled treprostinilDrug
Also called Tyvaso
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Inhaled treprostinil |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Adverse Events
Overall safety of transitioning from inhaled iloprost to inhaled treprostinil was assessed by type and frequency of adverse events.
Measured: up to 24 months
Secondary outcomes (7)
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.
Measured: Baseline and 12 weeks
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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).
Measured: Baseline and 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Aug 2008 (estimated)
- Study startSep 2008
- Primary completionMar 2010
- Study completionDec 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Pulmonary Arterial HypertensionStudies of the same condition, open and under way.
- More trials run by United TherapeuticsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 73 participants (actual)
Sponsor and investigators
- Lead sponsor
- United Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00741819
- Sponsor's protocol ID
- RIN-PH-401
- Status last verified
- Jan 2013
- First submitted
- 23 Aug 2008
- Last updated
- 20 Feb 2013
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Feb 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label, Multi-center Study Evaluating the Safety of Long-term Inhaled Treprostinil Administration Following Transition From Inhaled Ventavis® (Iloprost) in Subjects With Pulmonary Arterial Hypertension.
- ClinicalTrials.gov ID
- NCT00741819
- Acronym / sponsor ID
- RIN-PH-401
- Conditions
- Pulmonary Arterial Hypertension
- Keywords
- pulmonary arterial hypertension, PAH, treprostinil sodium, inhalation, ventavis, iloprost
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 73 participants (actual)
- Lead sponsor
- United TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Sep 2008
- Primary completion
- Mar 2010 (actual)
- Study completion
- Dec 2010 (actual)
- First posted
- 26 Aug 2008 (estimated)
- Results
- Posted 20 Feb 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Inhaled treprostinil | Experimental | Drug: Inhaled treprostinilSolution 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. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Between 18 and 75 years of age
- PAH diagnosis defined by following WHO Group I categories: idiopathic/familial, associated with unrepaired or repaired congenital systemic-to-pulmonary shunts (repaired >/= 5 years), associated with collagen vascular disease, associated with HIV, associated with appetite suppressant/toxin use
- Baseline six-minute walk distance (6MWD) >/= 250 meters
- Currently receiving Ventavis and be stable at current dose for 30 days prior to Baseline
- If currently receiving other approved background therapy (i.e. endothelin receptor antagonist or phosphodiesterase-5-inhibitor or both) must be on stable dose for 30 days prior to Baseline
Key exclusion criteria (4 listed)
- Nursing or pregnant
- Has acute concomitant disease (e.g. portal hypertension, chronic thromboembolic disease, pulmonary veno-occlusive disease, etc) other than those accepted as part of the inclusion criteria or has had atrial septostomy
- History of uncontrolled sleep apnea, left-sided heart disease, or parenchymal lung disease
- Use of investigational drug within 30 days of Baseline
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Inhaled treprostinilAlso: Tyvaso | Drug | Inhaled treprostinil |
Primary outcome
Number of Adverse Events
Time frame: up to 24 months
Overall safety of transitioning from inhaled iloprost to inhaled treprostinil was assessed by type and frequency of adverse events.
7 secondary outcomes
Six-minute Walk Distance (6MWD)
Time frame: Baseline and 12 weeks
Quality of Life (QoL) Assessment: Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR)
Time frame: Baseline and 12 weeks
Treatment Satisfaction Questionnaire of Medication (TSQM)
Time frame: Baseline and 12 weeks
Patient Impression of Change
Time frame: Baseline and 12 weeks
N-terminal Prohormone of Brain Natriuretic Peptide (NT-proBNP)
Time frame: Baseline and Week 12
World Health Organization (WHO) Functional Class
Time frame: Baseline and 12 Weeks
Drug Administration Activities Questionnaire
Time frame: Baseline and 12 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Feb 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
15 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Inhaled treprostinil in people with Pulmonary Arterial Hypertension, run by United Therapeutics at 15 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Feb 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 20 Feb 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Adverse Events (time frame: up to 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Feb 2013.