Phenylketonurias
Conditions
Keywords
Phenylalanine Hydroxylase
Brief summary
The primary objective of this trial is to evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU.
Interventions
Sponsors
BioMarin Pharmaceutical
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
8 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary objective: | — |
| - To evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU. | — |
Secondary
| Measure | Time frame |
|---|---|
| To determine the effect of various doses of Phenoptin on blood phenylalanine (Phe) levels. | — |
| To evaluate the population pharmacokinetics of Phenoptin. | — |
| Secondary objectives: | — |
| To evaluate the persistence of benefit of Phenoptin treatment in the subject population as evidenced by long-term control of blood Phe levels. | — |
| To evaluate the ability of Phenoptin to reduce phenylalanine (Phe) levels over a 24-hour period. | — |
| To compare the safety and tolerability of three different doses of Phenoptin treatment in subjects with PKU. | — |
Countries
United States
Outcome results
None listed