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

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BioMarin Pharmaceutical

Status and phase

Completed
Phase 3

Conditions

Phenylketonurias

Treatments

Drug: sapropterin dihydrochloride

Study type

Interventional

Funder types

Industry

Identifiers

NCT00225615
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

Trial design

Primary purpose

Treatment

Allocation

Non-Randomized

Interventional model

Single Group Assignment

Masking

None (Open label)

Trial contacts and locations

14

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Data sourced from clinicaltrials.gov

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