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

Amgen

Status and phase

Completed

Phase 4

Conditions

Urea Cycle Disorders

Treatments

Drug: HPN-100

Study type

Interventional

Funder types

Industry

Identifiers

NCT01257737

HPN-100-011

Details and patient eligibility

About

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.

Full description

The duration of treatment in this study was open-ended. Participants were to return for clinic visits as prescribed by the investigator, and were to be seen at a minimum of every 6 months. At each clinic visit, participants were queried about any adverse events (AEs) or hyperammonemic crises (HACs) that occurred since the last visit. Physical and neurological examinations were performed, and blood samples were collected for the analysis of ammonia, amino acid panels, and routine clinical laboratory safety tests. Participants underwent neuropsychological testing at baseline, every 12 months thereafter, and at the final study visit.

Study acquired from Horizon in 2024.

Enrollment

88 patients

Sex

All

Ages

1+ year old

Volunteers

No Healthy Volunteers

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