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Trial record 6 of 52 for:    phenylbutyrate

Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders

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ClinicalTrials.gov Identifier: NCT00947544
Recruitment Status : Completed
First Posted : July 28, 2009
Results First Posted : December 25, 2013
Last Update Posted : January 16, 2017
Sponsor:
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
Interventions: Drug: HPN-100
Drug: NaPBA

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Swich Over and Safety Extension NaPBA was dosed three times daily (TID) with during the first week and the same PBA mole-equivalent dose of HPN-100 during the second week. If there were safety concerns regarding a single-step transition from NaPBA to HPN-100, at the investigator's discretion, the transition could occur in 2 steps such that in the second week, subjects might receive 50% of the PBA equivalent dose as NaPBA and 50% as HPN-100 before receiving 100% of Serial blood samples were collected for PK and blood ammonia assessments after each drug reached steady state, which was achieved approximately 4 days after initiation of 100% NaPBA or HPN100 treatment. After the switch over, participants entered the safety extension part of the study and continued receiving open-label HPN-100 for up to 12 months.
Safety Extension Only Participants entered the safety extension part of the study only, and received open-label HPN-100 for up to 12 months.

Participant Flow for 2 periods

Period 1:   Switch-Over Period (2 Weeks)
    Swich Over and Safety Extension   Safety Extension Only
STARTED   11   0 
COMPLETED   11   0 
NOT COMPLETED   0   0 

Period 2:   Safety-Extension Period (12 Months)
    Swich Over and Safety Extension   Safety Extension Only
STARTED   11   6 [1] 
COMPLETED   10   6 
NOT COMPLETED   1   0 
Withdrawal by Subject                1                0 
[1] New participants were enrolled due to DSMB approval.



  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.
No text entered.

Reporting Groups
  Description
Participants in SO and SE Patients who completed switch over study and enrolled safety extension study

Baseline Measures
   Participants in SO and SE 
Overall Participants Analyzed 
[Units: Participants]
 17 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      17 100.0% 
Between 18 and 65 years      0   0.0% 
>=65 years      0   0.0% 
Age 
[Units: Years]
Mean (Standard Deviation)
 10  (3.482) 
Gender 
[Units: Participants]
Count of Participants
 
Female      14  82.4% 
Male      3  17.6% 
Region of Enrollment 
[Units: Participants]
 
United States   16 
Canada   1 


  Outcome Measures

1.  Primary:   Rate of Adverse Events During the Switchover Part of the Study Rate of Adverse Events (Number of Participants Showing Adverse Events)   [ Time Frame: 1 week on each treatment for a total of 2 week. ]

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

3.  Secondary:   Blood Ammonia Control   [ Time Frame: Day 7 (NaPBA) and Day 14 (HPN-100) ]

4.  Secondary:   NH3 Cmax on NaPBA vs. HPN-100 on the Last Day of Treatment With Each Drug   [ Time Frame: Day 7 (NaPBA) and Day 14 (HPN-100) ]

5.  Secondary:   Average Ammonia Values on NaPBA vs. HPN-100 on the Last Day of Treatment With Each Drug (Switch Over)   [ Time Frame: Day 7 (NaPBA) and Day 14 (HPN-100) ]

6.  Secondary:   Rate (Percentage) of Ammonia Values Above Upper Limit of Normal (ULN) on NaPBA vs. HPN-100   [ Time Frame: Day 7 (NaPBA) and Day 14 (HPN-100) ]

7.  Secondary:   Urinary PAGN 24-hour Excretion Values on NaPBA vs. HPN-100 (Switch Over)   [ Time Frame: Day 7 (NaPBA) and Day 14 (HPN-100) ]

8.  Secondary:   Plasma PAA (Phenylacetate) AUC0-24 Values on NaPBA vs. HPN-100 on on the Last Day of Treatment With Each Drug   [ Time Frame: Day 7 (NaPBA) and Day 14 (HPN-100) ]

9.  Secondary:   Plasma PBA (Phenylbutyrate) AUC0-24 Values on NaPBA vs. HPN-100 on on the Last Day of Treatment With Each Drug   [ Time Frame: Day 7 (NaPBA) and Day 14 (HPN-100) ]

10.  Secondary:   Plasma PAGN AUC0-24 Values on NaPBA vs. HPN-100 on on the Last Day of Treatment With Each Drug   [ Time Frame: Day 7 (NaPBA) and Day 14 (HPN-100) ]

11.  Secondary:   Quality of Life Assessed by the SF-15 Questionnaire   [ Time Frame: 1 year ]


  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
No text entered.


  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-Associate Director, Clinical Operations
Organization: Hyperion Therapeutics
phone: 650-745-7840
e-mail: craig.james@hyperiontx.com


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

Responsible Party: Horizon Pharma Ireland, Ltd., Dublin Ireland
ClinicalTrials.gov Identifier: NCT00947544     History of Changes
Other Study ID Numbers: HPN-100-005
First Submitted: July 24, 2009
First Posted: July 28, 2009
Results First Submitted: April 30, 2013
Results First Posted: December 25, 2013
Last Update Posted: January 16, 2017