Clinical trial NCT00225615: A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels
Official title: A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Phenylketonurias
Keywords: Phenylalanine Hydroxylase
Lead sponsor: BioMarin Pharmaceutical
Sponsor class: INDUSTRY
First posted: 2005-09-26
Enrollment: 100
Sex: ALL
Minimum age (years): 8
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The primary objective of this trial is to evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU.


Eligibility criteria: Inclusion Criteria:

* 8 years of age and older
* Prior successful participation in Study PKU-003
* Willing and able to provide written informed consent or assent and written informed consent (if required) by a parent or legal guardian
* For females of child-bearing potential only: Negative urine pregnancy test within 24 hours prior to enrollment. Women using acceptable birth control measures must agree to continue to use those measures while participating in the study
* Willing and able to comply with study procedures
* Willing to continue current diet unchanged while participating in the study

Exclusion Criteria:

* Perceived to be unreliable or unavailable for study participation or, if under the age of 18, have parents or legal guardians who are perceived to be unreliable or unavailable
* Withdrew from, or otherwise did not successfully complete, study PKU-003, except for subjects who were removed from the study because their blood Phe exceeded the alert level
* Expected to require any investigational agent or vaccine prior to completion of all scheduled study assessments
* Pregnant or breastfeeding, or planning pregnancy
* Concurrent disease or condition that would interfere with study participation or safety (e.g., seizure disorder, oral steroid-dependent asthma or other condition requiring oral or parenteral corticosteroid administration, or insulin-dependent diabetes)
* Requirement for concomitant treatment with any drug known to inhibit folate synthesis (e.g., methotrexate)
* Concurrent use of levodopa

Primary outcomes:
measure: Primary objective:
measure: - To evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU.
Secondary outcomes:
measure: Secondary objectives:
measure: To compare the safety and tolerability of three different doses of Phenoptin treatment in subjects with PKU.
measure: To determine the effect of various doses of Phenoptin on blood phenylalanine (Phe) levels.
measure: To evaluate the population pharmacokinetics of Phenoptin.
measure: To evaluate the ability of Phenoptin to reduce phenylalanine (Phe) levels over a 24-hour period.
measure: To evaluate the persistence of benefit of Phenoptin treatment in the subject population as evidenced by long-term control of blood Phe levels.
Other outcomes:
Locations:
city: Los Angeles
country: United States
state: California
city: Oakland
country: United States
state: California
city: Sacramento
country: United States
state: California
city: San Jose
country: United States
state: California
city: New Haven
country: United States
state: Connecticut
city: Atlanta
country: United States
state: Georgia
city: Chicago
country: United States
state: Illinois
city: Minneapolis
country: United States
state: Minnesota
city: St. Louis
country: United States
state: Missouri
city: New York
country: United States
state: New York
city: Portland
country: United States
state: Oregon
city: Dallas
country: United States
state: Texas
city: Salt Lake City
country: United States
state: Utah
city: Madison
country: United States
state: Wisconsin
Study officials:
affiliation: BioMarin Pharmaceutical
name: Alex Dorenbaum, MD
role: STUDY_CHAIR
References: