CompletedPhase 4

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

United States88 participantsStarted 2010-10-04

Plain-language 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.

Who can participate

Age range1 Year

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Number of Participants With at Least One Adverse Event

Timeframe: From the time of informed consent until 7 days after the last dose of study drug, up to 66 months

Trial details

NCT IDNCT01257737

SponsorAmgen

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2017-02-16

Results submitted2018-02-16