Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00947297
Recruitment Status : Completed
First Posted : July 28, 2009
Results First Posted : August 15, 2013
Last Update Posted : January 16, 2017
Information provided by (Responsible Party):
Horizon Pharma Ireland, Ltd., Dublin Ireland

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Urea Cycle Disorders
Intervention: Drug: HPN-100

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
HPN-100 Patients who were treated with HPN-100

Participant Flow:   Overall Study

  Baseline Characteristics

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.
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Reporting Groups
HPN-100 Patients who were treated with HPN-100

Baseline Measures
Overall Participants Analyzed 
[Units: Participants]
[Units: Participants]
Count of Participants
<=18 years      9  15.0% 
Between 18 and 65 years      51  85.0% 
>=65 years      0   0.0% 
[Units: Years]
Mean (Standard Deviation)
 28.83  (13.933) 
[Units: Participants]
Count of Participants
Female      41  68.3% 
Male      19  31.7% 
Region of Enrollment 
[Units: Participants]
United States   54 
Canada   6 

  Outcome Measures

1.  Primary:   Rate of Adverse Events (Number of Participants Who Experienced Any AE Considered Related to Study Drug)   [ Time Frame: 1 year ]

2.  Secondary:   Number and Causes of Hyperammonemic Events   [ Time Frame: 1 year ]

3.  Secondary:   Blood Ammonia Levels   [ Time Frame: 1 Year ]

4.  Secondary:   Patient Satisfaction With HPN-100   [ Time Frame: Month 1 post dose ]

  Serious Adverse Events

  Other Adverse Events

  Limitations and Caveats

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

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 Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed.

Results Point of Contact:  
Name/Title: Craig James
Organization: Hyperion Therapeutics
phone: 650-745 7840

Publications automatically indexed to this study by Identifier (NCT Number):

Responsible Party: Horizon Pharma Ireland, Ltd., Dublin Ireland Identifier: NCT00947297     History of Changes
Other Study ID Numbers: HPN-100-007
First Submitted: July 24, 2009
First Posted: July 28, 2009
Results First Submitted: April 30, 2013
Results First Posted: August 15, 2013
Last Update Posted: January 16, 2017