Overview

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

Status:
Completed
Trial end date:
2006-06-01
Target enrollment:
0
Participant gender:
All
Summary
The primary objective of this trial is to evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU.
Phase:
Phase 3
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
BioMarin Pharmaceutical
Treatments:
Verapamil
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