Phase 4
N=88
To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)
Urea Cycle Disorders
Bottom Line
View on ClinicalTrials.gov: NCT01257737 ↗Enrolled (actual)
88
Serious AEs
35.2%
Results posted
May 2018
Primary outcome: Primary: Number of Participants With at Least One Adverse Event — 34; 40 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- HPN-100 (Drug)
- Age
- Pediatric, Adult, Older Adult · 1+ yrs
- Sex
- All
- Sponsor
- Amgen
- Primary completion
- Feb 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With at Least One Adverse Event |
34; 40 | — |
| SECONDARY Mean Normalized Blood Ammonia Levels |
19.445; 26.005; 18.437; 24.235; 18.236; 27.843 | — |
| SECONDARY Number of Hyperammonemic Crises |
22; 22 | — |
| SECONDARY Causes of Hyperammonemic Crises |
0; 4; 1; 0; 6; 0 | — |
| SECONDARY Mean Wechsler Abbreviated Scale of Intelligence (WASI) Scores |
46.5; 45.2; 48.9; 44.2; 44.9; 45.2 | — |
| SECONDARY Mean Child Behavior Checklist (CBCL) Problems Scores |
53.4; 52.5; 55.5; 52.5; 53.5; 52.1 | — |
| SECONDARY Mean Behavior Rating Inventory of Executive Function (BRIEF) Scores |
54.0; 56.8; 57.3; 56.1; 57.7; 59.0 | — |
| SECONDARY Mean California Verbal Learning Test Scores: List A Total 1-5 T-Scores |
45.3; 45.4; 48.3; 30.0; 19.0; 46.3 | — |
| 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 |
-0.6; -0.4; -0.6; -2.0; -3.5; -0.4 | — |
Summary
This was an open-label, long-term safety study of HPN-100 (RAVICTI; glycerol phenylbutyrate) in participants with a urea cycle disorder (UCD) who completed the safety extensions of HPN-100-005 (NCT00947544; HPN-100-005SE), HPN-100-006 (NCT00947297; HPN-100-007), or HPN-100-012 (NCT01347073; HPN-100-012SE). The initial studies were 1- to 2-week crossover studies, and their associated safety extensions were 12-month, open-label studies. All participants who completed the initial studies were eligible to enroll in the associated safety extension studies, and new participants were also permitted to enroll directly into the safety extension studies.
Eligibility Criteria
Inclusion Criteria
- Male and female subjects who completed Studies HPN-100-005SE, HPN-100-007, or HPN-100-012SE
- Signed informed consent by participant and/or participant's legally authorized representative
- Negative pregnancy test for all females of childbearing potential
Exclusion Criteria
- Any clinical or laboratory abnormality or medical condition that, at the discretion of the investigator, may have put the participant at increased risk when participating
- Known hypersensitivity to PAA (phenylacetate) or PBA (phenylbutyrate).
- Liver transplant, including hepatocellular transplant
- Pregnant, breastfeeding or lactating females
Data sourced from ClinicalTrials.gov (NCT01257737). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.