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

BioMarin Pharmaceutical

Status and phase

Completed

Phase 3

Conditions

Phenylketonurias

Treatments

Drug: sapropterin dihydrochloride

Study type

Interventional

Funder types

Industry

Identifiers

PKU-004

Details and patient eligibility

About

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

Sex

All

Ages

8+ years old

Volunteers

No Healthy Volunteers

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