Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Horizon Pharma Ireland, Ltd., Dublin Ireland
ClinicalTrials.gov Identifier:
NCT01347073
First received: April 29, 2011
Last updated: June 8, 2015
Last verified: June 2015
Results First Received: April 3, 2015  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Urea Cycle Disorders
Intervention: Drug: HPN-100

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations

Study Locations: Houston, TX; Minneapolis, MN; Washington, DC; New York, NY; Cleveland, OH; Portland, ME; Portland, OR

Study Initiation Date: September 9, 2011 Study Completion Date: April 4, 2013


Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
This is an open-label, fixed-sequence NaPBA to HPN-100 switchover study with no control group.

Reporting Groups
  Description
HPN-100 The first part of this open-label study consisted of a switch-over period during which patients were switched from the current medication (NaPBA) to HPN-100. The second part of this open-label study consisted of a 12-month long-term treatment phase with HPN-100. All participants in the switch-over were enrolled into the long-term treatment phase.

Participant Flow:   Overall Study
    HPN-100  
STARTED     23  
COMPLETED     21  
NOT COMPLETED     2  
Adverse Event                 1  
Liver Transplant                 1  



  Baseline Characteristics
  Hide 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.
Analyses were based on the safety population, defined as all patients who received any amount of study medication.

Reporting Groups
  Description
HPN-100 The first part of this open-label study consisted of a switch-over period during which patients were switched from the current medication (NaPBA) to HPN-100. The second part of this open-label study consisted of a 12-month long-term treatment phase with HPN-100. All participants in the switch-over were enrolled into the long-term treatment phase.

Baseline Measures
    HPN-100  
Number of Participants  
[units: participants]
  23  
Age  
[units: participants]
 
<=18 years     23  
Between 18 and 65 years     0  
>=65 years     0  
Gender  
[units: participants]
 
Female     12  
Male     11  
Region of Enrollment  
[units: participants]
 
United States     23  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Adverse Events   [ Time Frame: 2 weeks ]

2.  Primary:   Adverse Events   [ Time Frame: 12 months ]

3.  Secondary:   Blood Ammonia   [ Time Frame: 2 weeks ]

4.  Secondary:   Frequency of Ammonia Levels Greater Than the Upper Limit of Normal (ULN) on HPN-100 Compared With NaPBA   [ Time Frame: 2 weeks ]

5.  Secondary:   Hyperammonemic Crisis   [ Time Frame: 1 year ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide 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
The protocol was designed to capture information important for evaluating safety, pharmacokinetics, and efficacy while recognizing sampling limitations in young children and current standard of care.


  More Information
  Hide More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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.


Results Point of Contact:  
Name/Title: Craig James R.N. Associate Dir. UCD Clinical Operations
Organization: Hyperion Therapeutics, Inc.
phone: (650) 745- 7480
e-mail: craig.james@hyperiontx.com


No publications provided by Horizon Pharma Ireland, Ltd., Dublin Ireland

Publications automatically indexed to this study:

Responsible Party: Horizon Pharma Ireland, Ltd., Dublin Ireland
ClinicalTrials.gov Identifier: NCT01347073     History of Changes
Other Study ID Numbers: HPN-100-012
Study First Received: April 29, 2011
Results First Received: April 3, 2015
Last Updated: June 8, 2015
Health Authority: United States: Food and Drug Administration