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Trial record 27 of 52 for:    phenylbutyrate

Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle Disorders

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ClinicalTrials.gov Identifier: NCT00992459
Recruitment Status : Completed
First Posted : October 9, 2009
Results First Posted : August 12, 2013
Last Update Posted : January 16, 2017
Sponsor:
Information provided by (Responsible Party):
Horizon Pharma Ireland, Ltd., Dublin Ireland

Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Urea Cycle Disorders
Interventions: Drug: HPN-100
Drug: Buphenyl (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
Forty six subjects were screened and randomized at the participating sites between October 2009 to August 2010.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
There were no screen failures.

Reporting Groups
  Description
Arm A Subjects in Arm A were assigned to receive NaPBA + HPN 100 placebo for 2 weeks All patients in Arm A received HPN100 placebo (+ concomitant active NaPBA)
Arm B Subjects in Arm B were assigned to receive HPN-100 + NaPBA placebo for 2 weeks All patients in Arm B received NaPBA placebo (+ concomitant active HPN 100)

Participant Flow:   Overall Study
    Arm A   Arm B
STARTED   22 [1]   24 [2] 
COMPLETED   21 [3]   23 [4] 
NOT COMPLETED   1   1 
Withdrawal by Subject                0                1 
Adverse Event                1                0 
[1] All patients received HPN 100 placebo in Arm A (+ concomitant active NaPBA)
[2] All patients received NaPBA placebo in Arm B (+ concomitant active HPN 100)
[3] Totals enrolled and completed in Arm A NaPBA treatment + HPN 100 Placebo are not mutually exclusive.
[4] Totals enrolled and completed in Arm B HPN 100 treatment + NaPBA Placebo are not mutually exclusive.



  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
Treatment Arm A Patients randomized to receive NaPBA + HPN 100 placebo for 2 weeks (Treatment Period 1) followed by HPN-100 + NaPBA placebo for 2 weeks (Treatment Period 2)
Treatment Arm B Patients randomized to receive HPN-100 + NaPBA placebo for 2 weeks (Treatment Period 1) followed by NaPBA + HPN-100 placebo for 2 weeks (Treatment Period 2)
Total Total of all reporting groups

Baseline Measures
   Treatment Arm A   Treatment Arm B   Total 
Overall Participants Analyzed 
[Units: Participants]
 22   24   46 
Age 
[Units: Participants]
Count of Participants
     
<=18 years      0   0.0%      0   0.0%      0   0.0% 
Between 18 and 65 years      22 100.0%      24 100.0%      46 100.0% 
>=65 years      0   0.0%      0   0.0%      0   0.0% 
Gender 
[Units: Participants]
Count of Participants
     
Female      15  68.2%      17  70.8%      32  69.6% 
Male      7  31.8%      7  29.2%      14  30.4% 


  Outcome Measures

1.  Primary:   The Primary Endpoint Was the 24-hour Area Under the Curve for Blood Ammonia (NH324-hour AUC) on Days 14 and 28.   [ Time Frame: pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28 ]

2.  Secondary:   Correlation Between Urinary Phenylacetylglutamine (PAGN) Excretion Over 24 Hours (U-PAGN24-hour Excr) and Venous Ammonia - Area Under the Concentration-time Curve From Time 0 (Predose) to 24 Hours (AUC0-24)   [ Time Frame: 28 Days ]

3.  Secondary:   Maximum Ammonia Values Observed on NaPBA Versus HPN-100   [ Time Frame: pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28 ]

4.  Secondary:   Rate (Percentage) of Ammonia Values Above Upper Limit of Normal (ULN) on NaPBA Versus HPN-100   [ Time Frame: on Day 14 and Day 28 ]

5.  Secondary:   Number and Severity of Symptomatic Hyperammonemic Crises   [ Time Frame: 29 Days ]

6.  Secondary:   Rate of Adverse Events in Each Treatment Group   [ Time Frame: 29 Days ]

7.  Secondary:   Cmax for PAA of NaPBA and HPN-100 in Plasma   [ Time Frame: pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28 ]

8.  Secondary:   Cmax for PBA of NaPBA and HPN-100 in Plasma   [ Time Frame: pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28 ]

9.  Secondary:   Cmax PAGN of NaPBA and HPN-100 in Plasma   [ Time Frame: pre-dose, 2, 4, 8, 12, 16, 20 and 24 hours after first dose on days 14 and 28 ]

10.  Secondary:   U-PAGN24-hour Excr of NaPBA and HPN-100   [ Time Frame: 24 hours on Day 14 of each treatments ]


  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: Bruce Scharschmidt, MD, SVP, Chief MedicaL & Development Officer
Organization: Hyperion Therapeutics
phone: 650-745-7851
e-mail: bruce.scharschmidt@hyperiontx.com


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

Responsible Party: Horizon Pharma Ireland, Ltd., Dublin Ireland
ClinicalTrials.gov Identifier: NCT00992459     History of Changes
Other Study ID Numbers: HPN-100-006
First Submitted: October 8, 2009
First Posted: October 9, 2009
Results First Submitted: April 4, 2013
Results First Posted: August 12, 2013
Last Update Posted: January 16, 2017