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

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ClinicalTrials.gov Identifier: NCT00947297
Recruitment Status : Completed
First Posted : July 28, 2009
Results First Posted : August 15, 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
Intervention Drug: HPN-100
Enrollment 60

Recruitment Details  
Pre-assignment Details  
Arm/Group Title HPN-100
Hide Arm/Group Description Patients who were treated with HPN-100
Period Title: Overall Study
Started 60
Completed 53
Not Completed 7
Arm/Group Title HPN-100
Hide Arm/Group Description Patients who were treated with HPN-100
Overall Number of Baseline Participants 60
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 60 participants
<=18 years
9
  15.0%
Between 18 and 65 years
51
  85.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 60 participants
28.83  (13.933)
Gender  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 60 participants
Female
41
  68.3%
Male
19
  31.7%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 60 participants
United States 54
Canada 6
1.Primary Outcome
Title Rate of Adverse Events (Number of Participants Who Experienced Any AE Considered Related to Study Drug)
Hide Description [Not Specified]
Time Frame 1 year
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title HPN-100
Hide Arm/Group Description:
Patients who were treated with HPN-100
Overall Number of Participants Analyzed 60
Measure Type: Number
Unit of Measure: participants
33
2.Secondary Outcome
Title Number and Causes of Hyperammonemic Events
Hide Description Number of hyperammonemic crises per patient
Time Frame 1 year
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title HPN-100
Hide Arm/Group Description:
Patients who were treated with HPN-100
Overall Number of Participants Analyzed 60
Mean (Standard Deviation)
Unit of Measure: hyperammonemic events
0.20  (0.514)
3.Secondary Outcome
Title Blood Ammonia Levels
Hide Description Venous Ammonia levels over time
Time Frame 1 Year
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title HPN-100
Hide Arm/Group Description:

Patients who were treated with HPN-100

HPN-100: HPN-100 is a triglyceride that has a similar mechanism of action as NaPBA. It is a liquid with minimal taste and odor. Three teaspoons of HPN-100 (~17.4 mL) delivers equivalent of PBA that 40 tablets of NaPBA do.

Overall Number of Participants Analyzed 60
Mean (Standard Deviation)
Unit of Measure: Umol/L
Baseline 27.623  (15.8875)
Month 12 24.202  (20.6124)
4.Secondary Outcome
Title Patient Satisfaction With HPN-100
Hide Description Drug preference will be noted at week 3
Time Frame Month 1 post dose
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
all available questionnaires
Arm/Group Title HPN-100
Hide Arm/Group Description:

Patients who were treated with HPN-100

HPN-100: HPN-100 is a triglyceride that has a similar mechanism of action as NaPBA. It is a liquid with minimal taste and odor. Three teaspoons of HPN-100 (~17.4 mL) delivers equivalent of PBA that 40 tablets of NaPBA do.

Overall Number of Participants Analyzed 50
Measure Type: Number
Unit of Measure: % preferred HPN-100
90
Time Frame 1 year
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title HPN-100
Hide Arm/Group Description Patients who were treated with HPN-100
All-Cause Mortality
HPN-100
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
HPN-100
Affected / at Risk (%)
Total   12/60 (20.00%) 
Gastrointestinal disorders   
Abdominal pain  1  1/60 (1.67%) 
Infections and infestations   
Gastroenteritis  1  1/60 (1.67%) 
Lobar pneumonia  1  1/60 (1.67%) 
Metabolism and nutrition disorders   
Hyperammonaemia  1  9/60 (15.00%) 
Nervous system disorders   
Dizziness  1  1/60 (1.67%) 
Neuropathy peripheral  1  1/60 (1.67%) 
Psychiatric disorders   
Psychotic disorder  1  1/60 (1.67%) 
Reproductive system and breast disorders   
Pelvic pain  1  1/60 (1.67%) 
Respiratory, thoracic and mediastinal disorders   
Lung infiltration  1  1/60 (1.67%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (3.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
HPN-100
Affected / at Risk (%)
Total   59/60 (98.33%) 
Gastrointestinal disorders   
Abdominal discomfort  1  4/60 (6.67%) 
Abdominal distension  1  3/60 (5.00%) 
Diarrhoea  1  11/60 (18.33%) 
Dyspepsia  1  4/60 (6.67%) 
Nausea  1  13/60 (21.67%) 
Vomiting  1  19/60 (31.67%) 
General disorders   
Abdominal pain  1  6/60 (10.00%) 
Constipation  1  4/60 (6.67%) 
Fatigue  1  7/60 (11.67%) 
Pyrexia  1  4/60 (6.67%) 
Infections and infestations   
Bronchitis  1  4/60 (6.67%) 
Gastroenteritis  1  4/60 (6.67%) 
Gastroenteritis viral  1  6/60 (10.00%) 
Nasopharyngitis  1  11/60 (18.33%) 
Upper respiratory tract infection  1  19/60 (31.67%) 
Investigations   
Aspartate aminotransferase increased  1  3/60 (5.00%) 
Vitamin D decreased  1  4/60 (6.67%) 
Metabolism and nutrition disorders   
Decreased appetite  1  8/60 (13.33%) 
Hyperammonaemia  1  9/60 (15.00%) 
Increased appetite  1  3/60 (5.00%) 
Musculoskeletal and connective tissue disorders   
Back pain  1  4/60 (6.67%) 
Pain in extremity  1  4/60 (6.67%) 
Nervous system disorders   
Convulsion  1  3/60 (5.00%) 
Dizziness  1  9/60 (15.00%) 
Headache  1  10/60 (16.67%) 
Tremor  1  3/60 (5.00%) 
Reproductive system and breast disorders   
Metrorrhagia  1  3/60 (5.00%) 
Respiratory, thoracic and mediastinal disorders   
Cough  1  8/60 (13.33%) 
Nasal congestion  1  4/60 (6.67%) 
Oropharyngeal pain  1  7/60 (11.67%) 
Rhinorrhoea  1  4/60 (6.67%) 
Skin and subcutaneous tissue disorders   
Acne  1  3/60 (5.00%) 
Rash  1  6/60 (10.00%) 
Skin odour abnormal  1  4/60 (6.67%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (3.0)
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.
Results Point of Contact
Name/Title: Craig James
Organization: Hyperion Therapeutics
Phone: 650-745 7840
Responsible Party: Horizon Pharma Ireland, Ltd., Dublin Ireland
ClinicalTrials.gov 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