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Clinical Trials for
Patients with Urea Cycle Disorders
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About the Studies
Enrolling New Patients:
Study in Pediatric Patients (HPN-100-012)
This study will
enroll approximately 20 pediatric subjects, ages 29 days to less than 6 years
of age with UCD (all subtypes except NAGS deficiency) currently treated with
BUPHENYL® for at least 5 days prior to enrollment. The study includes both a 10 day
‘switch-over’ phase from BUPHENYL to HPN-100, involving two
overnight stays, and a one-year safety follow-up phase. Approximately 12
patients will be enrolled into the ‘switch over’ phase of the
study before entering the one-year safety phase. The sponsor may decide to enroll an
additional 8 subjects directly into the one-year safety study following
complete recruitment of the switch-over phase. Patients who enroll in the one-year safety
study (only) will receive HPN-100 and return for a study visit one week
later. Visits during the one-year
safety phase for all patients will occur monthly for 3 months and then
quarterly thereafter. The following is a brief summary of eligibility criteria.
Your physician will make the final decision whether or not you are medically
eligible based on your age, UCD type, and medical history. Who is
eligible for the pediatric study (HPN-100-012)?
Participants in the pediatric trial must: ·
Be at least 29 days but less
than 6 years of age ·
Suspected or confirmed UCD
diagnosis of any subtype, except N-acetyl glutamate synthetase
deficiency ·
Sign an informed consent by
subject’s legally acceptable representative ·
Be treated with BUPHENYL®
for at least 5 days prior to the Day 1 visit. ·
Not receive sodium benzoate
for at least 5 days prior to the Day 1 visit. ·
Be able to complete required
study visits and activities, including blood draws Participating Centers in Pediatric study (HPN-100-012) No Longer Enrolling
New Patients/Completed: Study in Pediatric Patients (HPN-100-005)-Enrollment
Closed
This
study enrolled approximately 20 children ages 6-17 with UCDs
(all subtypes) treated with BUPHENYL® and included both a
two-week ‘switch-over’ phase from BUPHENYL to HPN-100, involving
two overnight stays, and a one-year safety follow-up study. The first 11
patients enrolled have already completed the ‘switch over’ phase
and enrolled in the one-year safety. Newly enrolled patients directly entered
the one-year safety study, in which they received HPN-100 and returned for a
study visit to be seen by study personnel one week after starting HPN-100 and
then monthly thereafter. Study in Adult Patients (HPN-100-006)-Study Complete
This
study enrolled approximately 44 UCD adults (OTC, CPS and ASS deficiencies) on
BUPHENYL®. The study lasted 4 weeks. For the first two weeks,
participants took either BUPHENYL plus a HPN-100
placebo or HPN-100 plus a BUPHENYL placebo; for the second 2 weeks,
participants received whatever they did not receive during the first two
weeks. Blood ammonia was measured
to determine whether HPN-100 works as well as BUPHENYL. Weekly visits to the study site and
two overnight stays in the hospital were required, one at the end of the
first 2 weeks and a second at the end of the study. Long Term Study (HPN-100-007)-Enrollment Closed
This
study enrolled patients completing protocol HPN-100-006 as well as
approximately 20 pediatric (ages 6-17) and/or adult UCD patients (all UCD
subtypes) who were not eligible for the HPN-100-006 study or are not
currently being treated with BUPHENYL® but who had the
potential to benefit from treatment with HPN-100 (e.g., history of hyperammonemia). Study participants received HPN-100 for
one-year and were required to go to the study site for monthly appointments. Long Term Use of HPN-100 (HPN-100-011)-Enrollment Open
Only to Patients Who Complete HPN-100-005 & HPN-100-007 Studies
This
study will enroll approximately 70 subjects, pediatric (ages 6-17) and/or
adult UCD patients (all UCD subtypes), completing protocol HPN-100-005 or
HPN-100-007. Study participants
will continue to receive HPN-100 until the subjects can be transitioned to
commercially available HPN-100 or until the Sponsor decides not to pursue
submitting a NDA (new drug application) to the FDA (Food and Drug
Administration). Subjects will be
required to go to the study site every six months for appointments. |
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BUPHENYL® is a registered
trademark of Ucyclyd Pharma,
Inc. |
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