Phenylketonurias Clinical Trial
Official title:
A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
Verified date | August 2009 |
Source | BioMarin Pharmaceutical |
Contact | n/a |
Is FDA regulated | No |
Health authority | United States: Food and Drug Administration |
Study type | Interventional |
The primary objective of this trial is to evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU.
Status | Completed |
Enrollment | 100 |
Est. completion date | June 2006 |
Est. primary completion date | |
Accepts healthy volunteers | No |
Gender | Both |
Age group | 8 Years and older |
Eligibility |
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 |
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
n/a |
Lead Sponsor | Collaborator |
---|---|
BioMarin Pharmaceutical |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Primary objective: | |||
Primary | - To evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU. | |||
Secondary | Secondary objectives: | |||
Secondary | To compare the safety and tolerability of three different doses of Phenoptin treatment in subjects with PKU. | |||
Secondary | To determine the effect of various doses of Phenoptin on blood phenylalanine (Phe) levels. | |||
Secondary | To evaluate the population pharmacokinetics of Phenoptin. | |||
Secondary | To evaluate the ability of Phenoptin to reduce phenylalanine (Phe) levels over a 24-hour period. | |||
Secondary | To evaluate the persistence of benefit of Phenoptin treatment in the subject population as evidenced by long-term control of blood Phe levels. |
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