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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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2 August 2023 |
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Main ID: |
NCT03335488 |
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Date of registration:
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18/10/2017 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)
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Scientific title:
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A Randomised, Controlled, Open-Label Parallel Arm Study of Safety, PK and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate) Oral Liquid and Sodium Phenylbutyrate in Phenylbutyrate Treatment Naïve Patients With Urea Cycle Disorders |
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Date of first enrolment:
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February 20, 2018 |
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Target sample size:
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16 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/ct2/show/NCT03335488 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 4
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Countries of recruitment
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Belgium
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Italy
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Spain
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Switzerland
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United States
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Contacts
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Name:
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Denise Coughlan |
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Address:
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Telephone:
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Email:
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Affiliation:
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Horizon Therapeutics, LLC |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Signed informed consent given by the subject or the subject's parent/legal guardian
for those under 18 years of age or the age of consent by local regulation.
- Male and female subjects with a suspected or confirmed UCD diagnosis of any subtype,
except n-acetylglutamate synthetase (NAGS) deficiency.
- Suspected diagnosis is defined as having experienced a hyperammonemic crisis (HAC) or
a documented high ammonia of >=100 µmol/L
- Confirmed diagnosis is determined via enzymatic, biochemical, or genetic testing.
- Requires nitrogen-binding agents according to the judgment of the Investigator
- Birth and older.
- All females of childbearing potential and all sexually active males must agree to
use an acceptable method of contraception from signing the informed consent
throughout the study and for 30 days after the last dose of study drug.
Acceptable forms of contraception are (oral, injected, implanted or transdermal),
tubal ligation, intrauterine device, hysterectomy, vasectomy, or double barrier
methods. Abstinence is an acceptable form of birth control, though appropriate
contraception must be used if the subject becomes sexually active.
Exclusion Criteria:
- Subject has received chronic treatment with an oral phenylbutyrate (RAVICTI, NaPBA,
Pheburane, or other) longer than 14 consecutive days within one year prior to
enrollment.
- Temporary use of NaPBA for acute management of a hyperammonemic crisis in the past is
acceptable.
- Any concomitant illness (e.g., malabsorption or clinically significant liver or
bowel disease) which would preclude the subject's safe participation, as judged
by the Investigator.
- Has undergone liver transplantation, including hepatocellular transplant.
- Subjects on sodium benzoate (NaBz) at Baseline will be excluded if they are
viewed by the Investigator as being unable to undergo NaBz transition to a PAA
prodrug during the Initial Treatment Period.
- Known hypersensitivity to phenylbutyric acid (PBA) or any excipients of the
NaPBA/PBA formulations.
- Pregnant or breast-feeding patients. Women of childbearing potential must have a
pregnancy test performed at the Baseline Visit prior to the start of study drug.
Age minimum:
N/A
Age maximum:
99 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Urea Cycle Disorder
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Intervention(s)
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Drug: NaPBA
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Drug: RAVICTI
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Primary Outcome(s)
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Rate of Treatment Success (Percentage of Participants Defined as Treatment Success at Week 4) During the Initial Treatment Period
[Time Frame: Week 4]
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Secondary Outcome(s)
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Change From Baseline in Fasting Plasma Ammonia Levels During the Initial Treatment Period
[Time Frame: Baseline, Initial Treatment Period Week 1, Week 2, Week 3, Week 4 (0, 4, 8 hours post dose)]
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Peak Plasma Concentration (Cmax) of Ammonia at the End of the Initial Treatment Period
[Time Frame: Week 4: hour 0 (predose), and hours 4 and 8 postdose]
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Plasma Ammonia Area Under the Curve (AUC) 0 to 8h at the End of the Initial Treatment Period
[Time Frame: Week 4: hour 0 (predose), and hours 4 and 8 postdose]
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Rate of Drug Discontinuations (Percentage of Participants Who Discontinued Study Drug) Due to Any Reason in the Initial Treatment Period
[Time Frame: Baseline through Week 4]
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Secondary ID(s)
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2015-000075-27
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HPN-100-021
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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