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Clinical Trials/NCT00225615
NCT00225615CompletedPhase 3

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

BioMarin Pharmaceutical14 sites in 1 country100 target enrollmentStarted: November 1, 2005Last updated:
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Conditions
Drugs

Trial Snapshot

Phase
Phase 3
Status
Completed
Enrollment
100
Locations
14
Primary Endpoint
Primary objective:
Study OverviewStudy DesignEligibility CriteriaOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Study Overview

Brief Summary

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

Study Design

Study Type
Interventional
Allocation
Non Randomized
Intervention Model
Single Group
Primary Purpose
Treatment
Masking
None

Eligibility Criteria

Ages
8 Years to — (Child, Adult, Older Adult)
Sex
All
Accepts Healthy Volunteers
No

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

Outcomes

Primary Outcomes

Primary objective:

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

Secondary Outcomes

  • Secondary objectives:
  • To compare the safety and tolerability of three different doses of Phenoptin treatment in subjects with PKU.
  • To determine the effect of various doses of Phenoptin on blood phenylalanine (Phe) levels.
  • To evaluate the population pharmacokinetics of Phenoptin.
  • To evaluate the ability of Phenoptin to reduce phenylalanine (Phe) levels over a 24-hour period.
  • To evaluate the persistence of benefit of Phenoptin treatment in the subject population as evidenced by long-term control of blood Phe levels.

Investigators

Sponsor Class
Industry

Study Sites (14)

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