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Clinical Trials for Patients with Urea Cycle Disorders
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About the
Studies
Study in Pediatric
Patients (HPN-100-005)
This study will enroll approximately 10 children ages
6-17 with UCDs (all subtypes) currently treated with BUPHENYL®.
Like the phase 2 adult UCD study completed in 2008, patients will ‘switch
over’ from BUPHENYL to HPN-100. The first portion of the study will
last 2-3 weeks. Patients will take BUPHENYL for one or two weeks and then
receive HPN-100 for one week. Unless there are concerns about safety,
participants will be eligible to receive HPN-100 at no cost for one year as
part of a safety study. More
information. Study in Adult Patients
(HPN-100-006)
This study will enroll approximately 44 UCD adults (OTC,
CPS and ASS deficiencies) currently on BUPHENYL®. The study
will last 4 weeks, and for the first two weeks participants will take either
BUPHENYL plus a HPN-100 placebo or HPN-100 plus a BUPHENYL placebo; for the
second 2 weeks participants will receive whatever they did not receive during
the first two weeks. Blood
ammonia will be measured to determine whether HPN-100 works as well as
BUPHENYL. Weekly visits to the
study site and two overnight stays in the hospital will be required, one at
the end of the first 2 weeks and a second at the end of the study. More
information. Long Term Study
(HPN-100-007)
This study will enroll adults completing protocol
HPN-100-006 as well as approximately 20 adult UCD patients (all UCD subtypes)
not currently being treated with BUPHENYL® who have the
potential to benefit from treatment (e.g., history of hyperammonemia). Study
participants will receive HPN-100 at no charge for one-year and will be
required to go to the study site for monthly appointments. More
information. |
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BUPHENYL® is a
registered trademark of Ucyclyd Pharma, Inc. |
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