ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 42 of 52 for:    phenylbutyrate

To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)

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.
ClinicalTrials.gov Identifier: NCT01257737
Recruitment Status : Completed
First Posted : December 10, 2010
Results First Posted : May 4, 2018
Last Update Posted : May 4, 2018
Sponsor:
Information provided by (Responsible Party):
Horizon Pharma Ireland, Ltd., Dublin Ireland ( Horizon Therapeutics, LLC )

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
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
HPN-100- Pediatric Pediatric participants (age 1-17) continued HPN-100 treatment after completion of HPN-100-005SE, HPN-100-007, or HPN-100-012SE.
HPN-100 - Adult Adult participants (age 19-61) continued HPN-100 treatment after completion of HPN-100-005SE, HPN-100-007, or HPN-100-012SE.

Participant Flow:   Overall Study
    HPN-100- Pediatric   HPN-100 - Adult
STARTED   45   43 
COMPLETED   43   39 
NOT COMPLETED   2   4 
Lost to Follow-up                1                2 
Liver transplant                1                0 
Relocation out of the country                0                1 
Withdrew from study                0                1 



  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.
Safety Population: All participants who received any amount of study medication

Reporting Groups
  Description
HPN-100- Pediatric Pediatric participants (age 1-17) continued HPN-100 treatment after completion of HPN-100-005SE, HPN-100-007, or HPN-100-012SE.
HPN-100 - Adult Adult participants (age 19-61) continued HPN-100 treatment after completion of HPN-100-005SE, HPN-100-007, or HPN-100-012SE.
Total Total of all reporting groups

Baseline Measures
   HPN-100- Pediatric   HPN-100 - Adult   Total 
Overall Participants Analyzed 
[Units: Participants]
 45   43   88 
Age 
[Units: Years]
Mean (Standard Deviation)
 7.3  (4.33)   33.3  (11.90)   20.0  (15.76) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Female      32  71.1%      26  60.5%      58  65.9% 
Male      13  28.9%      17  39.5%      30  34.1% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
     
American Indian or Alaska Native      1   2.2%      1   2.3%      2   2.3% 
Asian      2   4.4%      2   4.7%      4   4.5% 
Native Hawaiian or Other Pacific Islander      0   0.0%      0   0.0%      0   0.0% 
Black or African American      3   6.7%      2   4.7%      5   5.7% 
White      38  84.4%      36  83.7%      74  84.1% 
More than one race      0   0.0%      0   0.0%      0   0.0% 
Unknown or Not Reported      1   2.2%      2   4.7%      3   3.4% 
Urea Cycle Disorder Diagnosis 
[Units: Participants]
Count of Participants
     
Argininosuccinate synthetase (ASS) deficiency      7  15.6%      2   4.7%      9  10.2% 
Ornithine transcarbamylase (OTC) deficiency      26  57.8%      35  81.4%      61  69.3% 
Arginase (ARG) deficiency      1   2.2%      1   2.3%      2   2.3% 
Argininosuccinate lyase (ASL) deficiency      11  24.4%      2   4.7%      13  14.8% 
Hyperornithinemia-hyperammonemia-homocitrullinuria      0   0.0%      3   7.0%      3   3.4% 


  Outcome Measures

1.  Primary:   Number of Participants With at Least One Adverse Event   [ Time Frame: From the time of informed consent until 7 days after the last dose of study drug, up to 66 months ]

2.  Secondary:   Mean Normalized Blood Ammonia Levels   [ Time Frame: From baseline through the end of the study, up to 66 months ]

3.  Secondary:   Number of Hyperammonemic Crises   [ Time Frame: From the time of informed consent until 7 days after the last dose of study drug, up to 66 months ]

4.  Secondary:   Causes of Hyperammonemic Crises   [ Time Frame: From the time of informed consent until 7 days after the last dose of study drug, up to 66 months ]

5.  Secondary:   Mean Wechsler Abbreviated Scale of Intelligence (WASI) Scores   [ Time Frame: Baseline, Month 12, Month 24, Month 36, Month 48, and study exit visit (up to 66 months) ]

6.  Secondary:   Mean Child Behavior Checklist (CBCL) Problems Scores   [ Time Frame: Baseline, Month 12, Month 24, and study exit visit (up to 66 months) ]

7.  Secondary:   Mean Behavior Rating Inventory of Executive Function (BRIEF) Scores   [ Time Frame: Baseline, Month 12, Month 24, and study exit visit (up to 66 months) ]

8.  Secondary:   Mean California Verbal Learning Test Scores: List A Total 1-5 T-Scores   [ Time Frame: Baseline, Month 12, Month 24, Month 36, Month 48, and study exit visit (up to 66 months) ]

9.  Secondary:   Mean California Verbal Learning Test Scores: Short and Long Delay Free Recall, Short and Long Delay Cued Recall, CVLT-II-Learning Slope, and Total Word Recognition Discrimination   [ Time Frame: Baseline, Month 12, Month 24, Month 36, Month 48, and study exit visit (up to 66 months) ]


  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 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: Colleen Canavan, Director
Organization: Horizon Therapeutics, LLC
phone: 1-224-383-3000
e-mail: clinicaltrials@horizonpharma.com



Responsible Party: Horizon Pharma Ireland, Ltd., Dublin Ireland ( Horizon Therapeutics, LLC )
ClinicalTrials.gov Identifier: NCT01257737     History of Changes
Other Study ID Numbers: HPN-100-011
First Submitted: December 2, 2010
First Posted: December 10, 2010
Results First Submitted: February 16, 2018
Results First Posted: May 4, 2018
Last Update Posted: May 4, 2018