Pulmonary Arterial HypertensionPhase 2NCT01477333

Addition of UT-15C SR to Pulmonary Arterial Hypertension Patients Currently Receiving Tyvaso®

A Phase 2 study testing UT-15C SR and Tyvaso Inhalation Solution in people with Pulmonary Arterial Hypertension, run by United Therapeutics at 6 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 18 to 75 years
Conditions the study is for: Pulmonary Arterial Hypertension
What is being tested?: UT-15C SR and Tyvaso Inhalation Solution
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 18 participants
Where: 6 sitesUnited States
Study dates: Oct 2011to Nov 2013
Phase: Phase 2
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

Significant inclusion criteria included:

Subjects were between 18 to 75 years of age
Diagnosis of PAH: Idiopathic; Heritable; Associated with: Collagen vascular disease, Human immunodeficiency virus (HIV) infection, appetite suppressant or toxin use, or repaired congenital systemic-to-pulmonary shunts (repaired ≥5 years)
Had been receiving Tyvaso for at least 4 weeks (≥9 breaths, 4 times a day [QID]) and required additional therapy
In addition to Tyvaso, subjects may have been receiving other approved PAH specific oral therapies (ERAs and/or PDE-5 inhibitors, if at a stable dose)

Significant exclusion criteria included:

The subject was pregnant or lactating
The subject had previously received UT-15C SR
The subject had added, discontinued or changed the dosing regimen (i.e., prescription) of conventional PAH therapies (e.g., oral vasodilators, oxygen, digoxin) within 21 days of Baseline
The subject was receiving a FDA approved PAH therapy (e.g., ERA and/or PDE-5 inhibitor) for less than 30 days prior to Baseline, or the dose had been modified within 30 days of Baseline
The subject had any disease associated with PAH other than collagen vascular disease, HIV infection, repaired congenital systemic-to-pulmonary shunts (for at least 5 years), or appetite suppressant/toxin use (e.g., portal hypertension, chronic thromboembolic disease, pulmonary veno-occlusive disease, etc.), or had an atrial septostomy
The subject had ischemic heart disease prior to Screening, or left ventricular dysfunction as evidenced by a mean PCWP (or a left ventricular end diastolic pressure) greater than 15 mmHg or left ventricular ejection fraction (LVEF) less than 40% as assessed by either multigated angiogram (MUGA), angiography or echocardiography. Patients with abnormal left ventricular function attributable entirely to impaired left ventricular filling due to the effects of right ventricular overload were not excluded.
The subject had uncontrolled systemic hypertension as evidenced by systolic blood pressure greater than 160 mmHg or diastolic blood pressure greater than 100 mmHg at Baseline.

Trial locations and contacts

6 sites in 1 country.

City, postcode or hospital

6 sites in 1 country

United States6 sites

The University of Alabama at Birmingham
Birmingham, Alabama, 35294
UT Southwestern Medical Center
Dallas, Texas, 75390
Duke University Medical Center
Durham, North Carolina, 27710
Allegheny General Hospital
Pittsburgh, Pennsylvania, 15212
Mayo Clinic
Rochester, Minnesota, 55905
Washington University School of Medicine
St Louis, Missouri, 63110

Other trials in United States

About this study

Official title: An Evaluation of the Safety and Efficacy of the Addition of UT-15C SR to Pulmonary Arterial Hypertension Patients Currently Receiving Tyvaso®

The purpose of this multi-center, open-label, safety and tolerability study was to assess the addition of oral treprostinil (UT-15C sustained release [SR] tablets) to subjects currently receiving Tyvaso (treprostinil) inhalation solution. During the 24-week evaluation period, the study evaluated the changes in the following assessments: hemodynamics, 6-minute walk test (6MWT), Borg dyspnea score, N-Terminal pro-brain natriuretic peptide (NT-proBNP), World Health Organization (WHO) Functional Class, and safety assessments.

Eligible subjects had a diagnosis of pulmonary arterial hypertension (PAH), currently were receiving Tyvaso, and may have been receiving other approved PAH specific oral therapies (endothelin receptor antagonists [ERAs] and/or phosphodiesterase type 5 inhibitor [PDE5-I], if at a stable dose for ≥30 days). At Baseline, subjects received the first dose of 0.125 mg UT-15C SR.

Read the detailed description

This was a multi-center, open-label, safety and tolerability study in WHO Group 1 PAH subjects adding UT-15C SR to Tyvaso and PAH approved background therapy. This study had a 24-week evaluation period followed by a long term safety period. Six study visits occurred in the first 24 weeks of study; Screening, Baseline, Week 4, Week 8, Week 12, and Week 24 visits. Right heart catheterization occurred between 2-4 hours following the last Tyvaso dose at Baseline (prior to the administration of UT-15C SR) and Week 24.

Conditions: Pulmonary Arterial Hypertension
Being tested: UT-15C SR, Tyvaso Inhalation Solution
Keywords: Tyvaso, UT-15C SR, PAH
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.

UT-15C SRDrug
Also called treprostinil diethanolamine, sustained release
Initiated at 0.125 mg BID, titrated as clinically indicated.
Tyvaso Inhalation SolutionDrug
Also called Treprostinil
Administered as at least 9 breaths 4 times daily for at least 4 weeks prior to Baseline

Study groups in NCT01477333
Study group	Role	What they receive
UT-15C SR BID	Experimental	Initiated at 0.125 mg twice daily (BID), titrated as clinically indicated. Receives: Drug: UT-15C SR; Drug: Tyvaso Inhalation Solution

What is being measured

The primary outcome is the main question the study is designed to answer.

Change in Hemodynamic Parameters From Baseline to Week 24.
Hemodynamics (via right heart catheterization [RHC]) were assessed at Baseline and Week 24 or at the time of premature termination of study drug if prior to the Week 24 visit.
Cardiopulmonary hemodynamic measurements included the following: mean pulmonary arterial pressure (PAPm), mean systemic arterial pressure (SAPm), mean right atrial pressure (RAPm), and mean pulmonary capillary wedge pressure (PCWPm).
Measured: Baseline and Week 24
Change in Hemodynamic Parameter (Heart Rate) From Baseline to Week 24.
Heart rate was assessed at Baseline and Week 24 or at the time of premature termination of study drug if prior to the Week 24 visit.
Measured: Baseline and Week 24
Change in Hemodynamic Parameters (Arterial and Venous Oxygen Saturation) From Baseline to Week 24.
Hemodynamics (via right heart catheterization [RHC]) were assessed at Baseline and Week 24 or at the time of premature termination of study drug if prior to the Week 24 visit.
Cardiopulmonary hemodynamic measurements included arterial oxygen saturation (SaO2) and mixed venous oxygen saturation (SvO2).
Measured: Baseline and Week 24
Change in Hemodynamic Parameter (Cardiac Output) From Baseline to Week 24.
Hemodynamics (via right heart catheterization [RHC]) were assessed at Baseline and Week 24 or at the time of premature termination of study drug if prior to the Week 24 visit.
Measured: Baseline and Week 24
Change in Hemodynamic Parameter (Cardiac Index) From Baseline to Week 24.
Hemodynamics (via right heart catheterization [RHC]) were assessed at Baseline and Week 24 or at the time of premature termination of study drug if prior to the Week 24 visit.
Cardiopulmonary hemodynamic measurements included cardiac index (CI).
Measured: Baseline and Week 24
Change in Hemodynamic Parameter (Pulmonary Vascular Resistance Index) From Baseline to Week 24.
Hemodynamics (via right heart catheterization [RHC]) were assessed at Baseline and Week 24 or at the time of premature termination of study drug if prior to the Week 24 visit.
Cardiopulmonary hemodynamic measurements included the following: pulmonary vascular resistance index (PVRI).
Measured: Baseline and Week 24

Secondary outcomes (4)

Change in Exercise Capacity as Measured by the 6-minute Walk Test (6MWT) Over the 24-week Treatment Period.
The 6MWT was conducted at Baseline, 2 to 4 hours after the last Tyvaso dose, and prior to first dose of UT-15C SR. The 6MWT was also performed at Weeks 4, 12, and 24, or at the time of premature termination of study drug if prior to the Week 24 visit.
Measured: Baseline and Weeks 4, 12, and 24
Time to Clinical Worsening Over the Treatment Period.
Clinical worsening was assessed continuously from Baseline through each subject's last study visit. Clinical worsening was defined as the occurrence of any one or more of the following: death (all causes), hospitalization as a result of worsening pulmonary arterial hypertension (PAH), and initiation of a parenteral prostacyclin.
Measured: Clinical worsening was assessed continuously from Baseline through each subject's last study visit
Shift From Baseline in World Health Organization (WHO) Functional Class Over the 24-week Treatment Period.
The WHO functional classification for pulmonary hypertension was assessed at Baseline prior to starting UT-15C SR, Weeks 4, 8, 12, and 24, or at the time of premature termination if prior to Week 24. The WHO functional classification ranges from I (patient's disease does not affect daily activities) to IV (patient's disease causes severe impairment).
Measured: Baseline and Weeks 4, 8, 12, and 24
N-terminal Pro-Brain Natriuretic Peptide (NT-proBNP) Over the 24-week Treatment Period.
NT-proBNP was assessed at Baseline prior to starting UT-15C SR, Weeks 12 and 24, or at the time of premature termination if prior to Week 24. Blood for NT-proBNP assessment was collected before the 6MWT was conducted.
Measured: Baseline and Weeks 12 and 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Oct 2011Nov 2013

First posted22 Nov 2011 (estimated)
Study startOct 2011
Primary completionNov 2013
Study completionNov 2013

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 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 18 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01477333
Sponsor's protocol ID: TDE-PH-203
Status last verified: Jan 2017
First submitted: 14 Nov 2011
Last updated: 27 Dec 2023
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Pulmonary Arterial Hypertension trials

Study at a glance

From the registry record, last updated by the sponsor on 27 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Evaluation of the Safety and Efficacy of the Addition of UT-15C SR to Pulmonary Arterial Hypertension Patients Currently Receiving Tyvaso®
ClinicalTrials.gov ID: NCT01477333
Acronym / sponsor ID: TDE-PH-203
Conditions: Pulmonary Arterial Hypertension
Keywords: Tyvaso, UT-15C SR, PAH
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 18 participants (actual)
Lead sponsor: United TherapeuticsIndustry (pharma, biotech, devices)
Study start: Oct 2011
Primary completion: Nov 2013 (actual)
Study completion: Nov 2013 (actual)
First posted: 22 Nov 2011 (estimated)
Results: Posted 6 Mar 2017

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
UT-15C SR BID	Experimental	Drug: UT-15C SR, Drug: Tyvaso Inhalation SolutionInitiated at 0.125 mg twice daily (BID), titrated as clinically indicated.

Who may qualify

Ages: 18 to 75 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

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
UT-15C SRAlso: treprostinil diethanolamine, sustained releaseInitiated at 0.125 mg BID, titrated as clinically indicated.	Drug	UT-15C SR BID
Tyvaso Inhalation SolutionAlso: TreprostinilAdministered as at least 9 breaths 4 times daily for at least 4 weeks prior to Baseline	Drug	UT-15C SR BID

Primary outcome

Change in Hemodynamic Parameters From Baseline to Week 24.
Time frame: Baseline and Week 24
Hemodynamics (via right heart catheterization [RHC]) were assessed at Baseline and Week 24 or at the time of premature termination of study drug if prior to the Week 24 visit. Cardiopulmonary hemodynamic measurements included the following: mean pulmonary arterial pressure (PAPm), mean systemic arterial pressure (SAPm), mean right atrial pressure (RAPm), and mean pulmonary capillary wedge pressure (PCWPm).
Change in Hemodynamic Parameter (Heart Rate) From Baseline to Week 24.
Time frame: Baseline and Week 24
Heart rate was assessed at Baseline and Week 24 or at the time of premature termination of study drug if prior to the Week 24 visit.
Change in Hemodynamic Parameters (Arterial and Venous Oxygen Saturation) From Baseline to Week 24.
Time frame: Baseline and Week 24
Hemodynamics (via right heart catheterization [RHC]) were assessed at Baseline and Week 24 or at the time of premature termination of study drug if prior to the Week 24 visit. Cardiopulmonary hemodynamic measurements included arterial oxygen saturation (SaO2) and mixed venous oxygen saturation (SvO2).
Change in Hemodynamic Parameter (Cardiac Output) From Baseline to Week 24.
Time frame: Baseline and Week 24
Hemodynamics (via right heart catheterization [RHC]) were assessed at Baseline and Week 24 or at the time of premature termination of study drug if prior to the Week 24 visit.
Change in Hemodynamic Parameter (Cardiac Index) From Baseline to Week 24.
Time frame: Baseline and Week 24
Hemodynamics (via right heart catheterization [RHC]) were assessed at Baseline and Week 24 or at the time of premature termination of study drug if prior to the Week 24 visit. Cardiopulmonary hemodynamic measurements included cardiac index (CI).
Change in Hemodynamic Parameter (Pulmonary Vascular Resistance Index) From Baseline to Week 24.
Time frame: Baseline and Week 24
Hemodynamics (via right heart catheterization [RHC]) were assessed at Baseline and Week 24 or at the time of premature termination of study drug if prior to the Week 24 visit. Cardiopulmonary hemodynamic measurements included the following: pulmonary vascular resistance index (PVRI).

4 secondary outcomes

Change in Exercise Capacity as Measured by the 6-minute Walk Test (6MWT) Over the 24-week Treatment Period.
Time frame: Baseline and Weeks 4, 12, and 24
Time to Clinical Worsening Over the Treatment Period.
Time frame: Clinical worsening was assessed continuously from Baseline through each subject's last study visit
Shift From Baseline in World Health Organization (WHO) Functional Class Over the 24-week Treatment Period.
Time frame: Baseline and Weeks 4, 8, 12, and 24
N-terminal Pro-Brain Natriuretic Peptide (NT-proBNP) Over the 24-week Treatment Period.
Time frame: Baseline and Weeks 12 and 24

Results and safety

Results posted to the registry on 6 Mar 2017, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
UT-15C SR BID	18	15

Primary outcome: Change in Hemodynamic Parameters From Baseline to Week 24.

Time frame: Baseline and Week 24. Median (Full Range). Unit: mmHg

Measure	UT-15C SR BID n = 15
Systolic systemic arterial pressure (mmHg)	-2.0 (-22 to 21)
Diastolic systemic arterial pressure (mmHg)	0 (-20 to 18)
Mean systemic arterial pressure (mmHg)	-1.0 (-29 to 18)
Systolic pulmonary arterial pressure (mmHg)	3.0 (-51 to 12)
Diastolic pulmonary arterial pressure (mmHg)	0 (-16 to 10)
Mean pulmonary arterial pressure (mmHg)	0 (-26 to 10)
Mean right atrial pressure (mmHg)	0 (-9 to 6)
Mean pulmonary capillary wedge pressure (mmHg)	-1.0 (-15 to 8)

Population analysed: Intent-to-treat population with data available at Baseline and Week 24.

Primary outcome: Change in Hemodynamic Parameter (Heart Rate) From Baseline to Week 24.

Time frame: Baseline and Week 24. Median (Full Range). Unit: beats/min

Measure	UT-15C SR BID n = 15
Value	4.00 (-29.0 to 25.0)

Population analysed: Intent-to-treat population with data available at Baseline and Week 24.

Primary outcome: Change in Hemodynamic Parameters (Arterial and Venous Oxygen Saturation) From Baseline to Week 24.

Time frame: Baseline and Week 24. Median (Full Range). Unit: percentage bound to hemoglobin

Measure	UT-15C SR BID n = 15
Arterial oxygen saturation (%)	1.0 (-5 to 5)
Mixed venous oxygen saturation (%)	0 (-9 to 4)

Population analysed: Intent-to-treat population with data available at Baseline and Week 24.

Primary outcome: Change in Hemodynamic Parameter (Cardiac Output) From Baseline to Week 24.

Time frame: Baseline and Week 24. Median (Full Range). Unit: L/min

Measure	UT-15C SR BID n = 15
Value	0 (-1 to 4)

Population analysed: Intent-to-treat population with data available at Baseline and Week 24.

4 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse events were recorded throughout the 24-week study, including the screening phase, as well as during the long-term follow-up period.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
UT-15C SR BID	2 of 18 (11.1%)	18 of 18 (100%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 27 Dec 2023.
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.

Cynthia Madden, MD, MPH study chair

Associate Director, Medical Development

6 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing UT-15C SR and Tyvaso Inhalation Solution in people with Pulmonary Arterial Hypertension, run by United Therapeutics at 6 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?

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 27 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 6 Mar 2017). 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?

Change in Hemodynamic Parameters From Baseline to Week 24. (time frame: Baseline and Week 24). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01477333.

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 27 Dec 2023.