A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00225615
• Date of registration: 22/09/2005
• Prospective Registration: Yes
• Primary sponsor: BioMarin Pharmaceutical
• Public title: A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels
• Scientific title: A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
• Date of first enrolment: November 2005
• Target sample size: 100
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00225615
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 3

Countries of recruitment

United States

Contacts

• Name: Alex Dorenbaum, MD
• Affiliation: BioMarin Pharmaceutical

Key inclusion & exclusion 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



Age minimum: 8 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Phenylketonurias

Intervention(s)

Drug: sapropterin dihydrochloride

Primary Outcome(s)

- To evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU.

Primary objective:

Secondary Outcome(s)

To determine the effect of various doses of Phenoptin on blood phenylalanine (Phe) levels.

Secondary objectives:

To compare the safety and tolerability of three different doses of Phenoptin treatment in subjects with PKU.

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 evaluate the population pharmacokinetics of Phenoptin.

Secondary ID(s)

PKU-004

Source(s) of Monetary Support

Please refer to primary and secondary sponsors