A Dose Response Study of UT-15C SR in Patients With Exercise-Induced Pulmonary Hypertension

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
United Therapeutics
ClinicalTrials.gov Identifier:
NCT01104870
First received: January 4, 2010
Last updated: May 25, 2016
Last verified: May 2016
Results First Received: November 24, 2015  
Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Single Blind (Outcomes Assessor);   Primary Purpose: Treatment
Condition: Pulmonary Hypertension
Intervention: Drug: UT-15C

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
The recruitment period for this study was May 2010 to October 2012. Sites were located in the US only.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
The 50 subjects who received a dose of study drug are presented here.

Reporting Groups
  Description
Dose Group 1

0.25 mg twice daily

UT-15C: oral

Dose Group 2

1.25 mg twice daily

UT-15C: oral

Dose Group 3

individual Maximum Tolerated Dose

UT-15C: oral


Participant Flow:   Overall Study
    Dose Group 1     Dose Group 2     Dose Group 3  
STARTED     11     19     20  
COMPLETED     10     18     18  
NOT COMPLETED     1     1     2  
Adverse Event                 1                 1                 0  
Death                 0                 0                 1  
Withdrawal by Subject                 0                 0                 1  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Dose Group 1

0.25 mg twice daily

UT-15C: oral

Dose Group 2

1.25 mg twice daily

UT-15C: oral

Dose Group 3

individual Maximum Tolerated Dose

UT-15C: oral

Total Total of all reporting groups

Baseline Measures
    Dose Group 1     Dose Group 2     Dose Group 3     Total  
Number of Participants  
[units: participants]
  11     19     20     50  
Age  
[units: years]
Mean (Full Range)
  53.1   (22 to 74)     58.9   (43 to 75)     57.6   (32 to 72)     57.1   (22 to 75)  
Gender  
[units: participants]
       
Female     11     15     15     41  
Male     0     4     5     9  
Ethnicity (NIH/OMB)  
[units: participants]
       
Hispanic or Latino     0     3     3     6  
Not Hispanic or Latino     11     16     17     44  
Unknown or Not Reported     0     0     0     0  
Race (NIH/OMB)  
[units: participants]
       
American Indian or Alaska Native     0     0     0     0  
Asian     0     0     0     0  
Native Hawaiian or Other Pacific Islander     0     0     0     0  
Black or African American     1     1     1     3  
White     10     18     19     47  
More than one race     0     0     0     0  
Unknown or Not Reported     0     0     0     0  
Region of Enrollment  
[units: participants]
       
United States     11     19     20     50  
PAH History  
[units: participants]
       
Idiopathic, heritable or drug or toxin-induced PAH     5     6     6     17  
PAH associated with connective tissue diseases     5     9     10     24  
PAH associated with HIV     0     0     0     0  
PH associated with interstitial lung disease (ILD)     0     4     3     7  
PH associated with sarcoidosis     1     0     1     2  
Background PAH Therapy  
[units: participants]
       
Both PDE-5i and ERA Dual Therapy     0     0     1     1  
ERA     4     4     1     9  
PDE-5i     3     6     7     16  
No Background Therapy     4     9     11     24  
Baseline Six-Minute Walk Distance (6MWD)  
[units: meters]
Mean (Standard Deviation)
  366.0  (79.6)     338.1  (55.7)     311.4  (67.4)     333.6  (68.1)  
World Health Organization (WHO) Functional Class [1]
[units: participants]
       
Class I     0     0     0     0  
Class II     2     5     4     11  
Class III     9     14     16     39  
Class IV     0     0     0     0  
[1] 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.



  Outcome Measures
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1.  Primary:   Change in Peak Total Pulmonary Resistance Index (TPRI) During Exercise From Baseline to Week 12   [ Time Frame: Baseline and Week 12 ]

2.  Secondary:   Change in Mean Pulmonary Artery Pressure (PAPm) From Baseline to Week 12   [ Time Frame: Baseline and Week 12 ]

3.  Secondary:   Change in Cardiac Index (CI) From Baseline to Week 12   [ Time Frame: Baseline and Week 12 ]

4.  Secondary:   Change in 6-minute Walk Distance (6MWD) From Baseline to Week 12   [ Time Frame: Baseline and Week 12 ]

5.  Secondary:   Change in Borg Dyspnea Score (Following 6MWT) From Baseline to Week 12   [ Time Frame: Baseline and Week 12 ]

6.  Secondary:   Change in PH Symptoms From Baseline to Week 12   [ Time Frame: Change from Baseline at 12 Weeks ]

7.  Secondary:   Number of Participants With a Change From Baseline World Health Organization (WHO) Functional Classification at Week 12   [ Time Frame: Change from Baseline at Week 12 ]

8.  Secondary:   Change in N-terminal Pro-B-type Natriuretic Peptide (NT-proBNP) Concentrations From Baseline to Week 12   [ Time Frame: Baseline and Week 12 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Kevin Laliberte, PharmD
Organization: United Therapeutics, Corp.
phone: 919-425-8176
e-mail: KLaliberte@unither.com



Responsible Party: United Therapeutics
ClinicalTrials.gov Identifier: NCT01104870     History of Changes
Other Study ID Numbers: TDE-PH-202
Study First Received: January 4, 2010
Results First Received: November 24, 2015
Last Updated: May 25, 2016
Health Authority: United States: Food and Drug Administration