Pulmonary Arterial HypertensionPhase 3NCT02999906
Study to Compare Triple Therapy (Oral Treprostinil, Ambrisentan, and Tadalafil) With Dual Therapy (Ambrisentan, Tadalafil, and Placebo) in Subjects With Pulmonary Arterial Hypertension
A Phase 3 study testing Oral Treprostinil in people with Pulmonary Arterial Hypertension, run by United Therapeutics.
Trial summary
This is a multicenter, randomized (2:1 oral treprostinil: placebo), double-blind, placebo-controlled study in subjects with Pulmonary Arterial Hypertension (PAH) who are currently receiving background dual therapy (ambrisentan, tadalafil) for at least 30 days at randomization for their PAH. Read more about this study
- Who is it for?
- Ages 18 to 79 years
- Conditions the study is for
- Pulmonary Arterial Hypertension
- What is being tested?
- Oral Treprostinil
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Oct 2017to Jun 2022
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 79 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Between 18 and 79 years of age, inclusive
- PAH that is idiopathic / heritable, PAH associated with connective tissue disease, HIV infection, repaired congenital systemic-to-pulmonary shunts (repaired > 1 year), or appetite suppressant / toxin use
- Receiving dual therapy (ambrisentan and tadalafil)
- Previous testing (e.g. right heart catheterization, echocardiography) consistent with diagnosis of PAH
Who cannot join
- Nursing or pregnant
- PAH due to conditions other than noted in the above inclusion criteria
- Received PAH-specific drug therapy for >2 years
- History of uncontrolled sleep apnea, severe liver disease, severe renal impairment, left sided heart disease, uncontrolled systemic hypertension
- Participated in an investigational drug or device study within 90 days prior to signing consent.
About this study
Official title: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled, Study to Compare the Efficacy and Safety of Triple Therapy (Oral Treprostinil, Ambrisentan, and Tadalafil) With Dual Therapy (Ambrisentan, Tadalafil, and Placebo) in Subjects With Pulmonary Arterial Hypertension
This is a multicenter, randomized (2:1 oral treprostinil: placebo), double-blind, placebo-controlled study in subjects with Pulmonary Arterial Hypertension (PAH) who are currently receiving background dual therapy (ambrisentan, tadalafil) for at least 30 days at randomization for their PAH. Once randomized, subjects will return for 5 study visits up to Week 28 during the blinded period. After the Week 28 Visit, eligible subjects will transition to the open-label period of the study for up to 20 weeks.
- Conditions
- Pulmonary Arterial Hypertension
- Being tested
- Oral Treprostinil, Placebo
- Keywords
- PAH, Oral Treprostinil, dual therapy, 6 Minute Walk Test
- Medical subject headings
- Pulmonary Arterial Hypertension
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Oral TreprostinilDrug
Also called treprostinil diethanolamine
Sustained release oral tablets for three times daily administration
PlaceboDrug
Also called matching placebo (sugar pill)
Placebo (sugar pill) for three times daily oral administration
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Matching placebo (sugar pill) Receives: Drug: Placebo |
| Active | Active comparator | Oral treprostinil sustained release tablet Receives: Drug: Oral Treprostinil |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in 6-Minute Walk Distance (6MWD)
Measured: Baseline to Week 28
Secondary outcomes (4)
Compare the effects of triple therapy versus dual therapy on the time to clinical worsening
clinical worsening is defined by 1 of the following:
- Death (all causes)
- Hospitalization due to worsening PAH
- Initiation of long-term parenteral therapy
- Disease progression
- Unsatisfactory long-term clinical response
Measured: Baseline to Week 28
Change in N-terminal pro-B-type natriuretic peptide (NT-proBNP) concentrations From Baseline to Week 28
The level of this biomarker of the (NT-proBNP) serum concentration will be assessed to compare the severity of heart failure at Baseline and Week 28.
Measured: Baseline to week 28
Number of Participants with a Change From Baseline World Health Organization (WHO) Functional Classification at Week 28.
The WHO Functional Class of pulmonary hypertension is a physical activity rating scale as follows: Class I: No limitation of physical activity. Class II: Slight limitation of physical activity. Class III: Marked limitation of physical activity. Class IV: Inability to carry out any physical activity without symptoms.
Measured: Baseline to week 28
Change in Quality of Life (QoL) Assessment: Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) From Baseline to Week 28.
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 025; high scores indicate more symptoms), Activity (Score 030; low score indicates good functioning) and Quality of Life (025; high scores indicate poor QoL). The sum of these scores equates to the Total score (080). In the CAMPHOR scores, lower scores indicate improvements.
Measured: Baseline to week 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Dec 2016 (estimated)
- Study startOct 2017 (estimated)
- Primary completionDec 2021 (estimated)
- Study completionJun 2022 (estimated)
Why it stopped: For business reasons.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 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
- NCT02999906
- Sponsor's protocol ID
- TDE-PH-313
- Status last verified
- Oct 2017
- First submitted
- 19 Dec 2016
- Last updated
- 1 Nov 2017
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Nov 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled, Study to Compare the Efficacy and Safety of Triple Therapy (Oral Treprostinil, Ambrisentan, and Tadalafil) With Dual Therapy (Ambrisentan, Tadalafil, and Placebo) in Subjects With Pulmonary Arterial Hypertension
- ClinicalTrials.gov ID
- NCT02999906
- Acronym / sponsor ID
- TDE-PH-313
- Conditions
- Pulmonary Arterial Hypertension
- Keywords
- PAH, Oral Treprostinil, dual therapy, 6 Minute Walk Test
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: For business reasons.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- United TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Oct 2017 (estimated)
- Primary completion
- Dec 2021 (estimated)
- Study completion
- Jun 2022 (estimated)
- First posted
- 21 Dec 2016 (estimated)
- Results
- Not posted in the source record
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 multicenter, randomized (2:1 oral treprostinil: placebo), double-blind, placebo-controlled study in subjects with Pulmonary Arterial Hypertension (PAH) who are currently receiving background dual therapy (ambrisentan, tadalafil) for at least 30 days at randomization for their PAH. Once randomized, subjects will return for 5 study visits up to Week 28 during the blinded period. After the Week 28 Visit, eligible subjects will transition to the open-label period of the study for up to 20 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboMatching placebo (sugar pill) |
| Active | Active comparator | Drug: Oral TreprostinilOral treprostinil sustained release tablet |
Who may qualify
- Ages
- 18 to 79 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Between 18 and 79 years of age, inclusive
- PAH that is idiopathic / heritable, PAH associated with connective tissue disease, HIV infection, repaired congenital systemic-to-pulmonary shunts (repaired > 1 year), or appetite suppressant / toxin use
- Receiving dual therapy (ambrisentan and tadalafil)
- Previous testing (e.g. right heart catheterization, echocardiography) consistent with diagnosis of PAH
Key exclusion criteria (5 listed)
- Nursing or pregnant
- PAH due to conditions other than noted in the above inclusion criteria
- Received PAH-specific drug therapy for >2 years
- History of uncontrolled sleep apnea, severe liver disease, severe renal impairment, left sided heart disease, uncontrolled systemic hypertension
- Participated in an investigational drug or device study within 90 days prior to signing consent.
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 |
|---|---|---|
| Oral TreprostinilAlso: treprostinil diethanolamineSustained release oral tablets for three times daily administration | Drug | Active |
| PlaceboAlso: matching placebo (sugar pill)Placebo (sugar pill) for three times daily oral administration | Drug | Placebo |
Primary outcome
Change in 6-Minute Walk Distance (6MWD)
Time frame: Baseline to Week 28
4 secondary outcomes
Compare the effects of triple therapy versus dual therapy on the time to clinical worsening
Time frame: Baseline to Week 28
Change in N-terminal pro-B-type natriuretic peptide (NT-proBNP) concentrations From Baseline to Week 28
Time frame: Baseline to week 28
Number of Participants with a Change From Baseline World Health Organization (WHO) Functional Classification at Week 28.
Time frame: Baseline to week 28
Change in Quality of Life (QoL) Assessment: Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) From Baseline to Week 28.
Time frame: Baseline to week 28
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Nov 2017.
- Individual participant dataSharing planned: No
Related trials for Pulmonary Arterial Hypertension
Not yet recruitingPhase 2/3
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A Pilot Study of Microbiota Transplant With Antibiotic Preconditioning and Fiber Supplementation in Pulmonary Arterial Hypertension (GUT RESTORE-PAH)
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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.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Oral Treprostinil in people with Pulmonary Arterial Hypertension, run by United Therapeutics.
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 4 inclusion and 5 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: withdrawn, as of the sponsor's last update on 1 Nov 2017. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
Change in 6-Minute Walk Distance (6MWD) (time frame: Baseline to Week 28). 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 1 Nov 2017.