Back to Search Results
Study Complete
Sponsor ID
UP1204-003 (HPN-100-003)

Dose-Escalation Safety Study of HPN-100 to Treat Urea Cycle Disorders

Sponsor ID
UP1204-003 (HPN-100-003)
Clinicaltrials.gov ID
NCT00551200
EUCTIS ID
N/A
EudraCT ID
N/A
Contact Us

Study Details

The purpose of this study is to determine whether HPN-100 is safe and tolerable in subjects with Urea Cycle Disorders.

Additional Study Details

Phase
Phase 2
Product
N/A
Type
Interventional
Masking
None (Open Label)
Enrollment
14
Additional Study Details

Protocol Summary

Primary Outcome Measures

Venous Ammonia Levels at the Peak and Mean TNUAC Time-normalized Area Under the Curve)

Timeframe: At steady state (1 week) on each medication (Buphenyl® alone, HPN-100 alone), and at steady state (1 week) after each dose escalation

Number of Subjects Experienced Adverse Events

Timeframe: during the period on 100% Buphenyl (up to 4 weeks) or HPN-100 (up to 10 weeks)

Number of Subjects Experienced Serious Adverse Events

Timeframe: during the period subjects on 100% Buphenyl (up to 4 weeks) or HPN-100 (up to 10 weeks)

Secondary Outcome Measures

Pharmacokinetics (Plasma and Urine PK Parameters of Study Drugs and Their Metabolites)

Timeframe: At steady state (1 week) on each medication (Buphenyl® alone, HPN-100 alone)

Drug Preference for HPN-100 or Buphenyl® (as Assessed by Global Preference Question)

Timeframe: End of Study

Locations

Location
Status
Contact Us
Location
Baylor College of Medicine
Houston, TX, United States, 77030
Status
Completed
Location
University of Minnesota
Minneapolis, MN, United States, 55455
Status
Completed
Study Info
Age
18+ years
Condition
  • Urea Cycle Disorder
    • Sex
    All
    Trial Dates
    Oct 2007 - Jul 2008
    Inclusion and Exclusion Criteria
    Inclusion Criteria:
    - Male and female patients at least 18 years old - Signed written informed consent by patient or patient’s representative - Diagnosis of urea cycle enzyme deficiency confirmed via enzymatic or genetic testing - Currently treated with Buphenyl® TID for a minimum of 2 weeks prior to Visit 1 - Able to perform study activities (including the ability to collect all urine in the clinic, i.e., no patients in diapers) - Negative pregnancy test for all females of childbearing potential. All females of childbearing potential must agree to use an acceptable method of contraception throughout the study
    Exclusion Criteria:
    - Use of any investigational drug within 30 days of Buphenyl® Visit 1 - Active infection (viral or bacterial) or any other condition that may increase ammonia levels - Laboratory values outside the normal range that are determined to be clinically significant by the investigator - Any clinical or laboratory abnormality of Grade 3 or greater severity according to the Common Terminology Criteria for Adverse Events v3.0 (CTCAE) (or for conditions not covered by the CTCAE, a severe or life-threatening toxicity); except that Grade 3 elevations in liver enzymes are allowed in an otherwise clinically stable patient - Use of any medication known to significantly affect renal clearance (e.g., probenecid) or to increase protein catabolism (e.g., corticosteroids), or other medication (e.g., valproate) known to increase ammonia levels, within the 24 hours prior to Visit 1 - Preexisting QTc interval prolongation (> 450 msec for males or > 460 msec for females) - Other severe chronic medical conditions - Known hypersensitivity to PAA, PBA, or benzoate - Creatinine levels equal to or greater than 1.5 × ULN - Liver transplant