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

Phenylketonurias Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00225615
• Other study ID #: PKU-004
• Status: Completed
• Phase: Phase 3
• First received: September 22, 2005
• Last updated: August 18, 2009
• Start date: November 2005
• Est. completion date: June 2006

Study information

• 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

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 100
• Est. completion date: June 2006
• 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

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Phenylketonurias

Intervention

Drug:
• sapropterin dihydrochloride

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Country where clinical trial is conducted

United States, 

Outcome

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.

External Links

•   BioMarin Pharmaceutical Inc website