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Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet

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Trial Status Completed

Trial Identifier

NCT00272792

Condition

Phenylketonuria, Phenylketonurias

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial Summary

The primary objective of this trial is to evaluate the ability of Phenoptin to increase phenylalanine (phe) tolerance in children with phenylketonuria who are following a phe-restricted diet.

Eligibility Criteria

Birth Sex

All

Age

4 Years to 12 Years

Healthy Volunteers

No

Drug/Treatment:

Sapropterin Dihydrochloride, Placebo

Phase:

Phase 3

Study Type:

Interventional

Number of Participants:

45

Study Started:

2006-02

Study Updated:

2015-08-13

Trial Locations

  • Los Angeles, California, United States

  • Oakland, California, United States

  • Sacramento, California, United States

  • San Jose, California, United States

  • Chicago, Illinois, United States

  • Boston, Massachusetts, United States

  • Minneapolis, Minnesota, United States

  • St. Louis, Missouri, United States

  • Salt Lake City, Utah, United States

Inclusion Criteria

  • Clinical diagnosis of PKU with hyperphenylalaninemia (HPA) documented by at least one blood Phe measurement >/=360 umol/L (6 mg/dL)
  • Under dietary control with a Phe-restricted diet as evidenced by:· Estimated daily Phe tolerance </=1000 mg/day
  • At least 6 months of blood Phe control (mean level of </=480 μmol/L) prior to enrolling in the study
  • Aged 4 to 12 years inclusive at screening
  • A blood Phe level </=480 μmol/L at screening
  • Female subjects of childbearing potential (as determined by the principal investigator) must have a negative blood or urine pregnancy test at entry (prior to the first dose). Note: All female subjects of childbearing potential and sexually mature male subjects must be advised to use a medically accepted method of contraception throughout the study. Female subjects of childbearing potential must be willing to undergo periodic pregnancy tests during the course of the study
  • Willing and able to comply with all study procedures
  • Willing to provide written assent (if applicable) and written informed consent by a parent or legal guardian after the nature of the study has been explained and prior to any research-related procedures

Exclusion Criteria

  • Any condition that, in the view of the PI, renders the subject at high risk from treatment compliance and/or completing the study
  • Prior history of organ transplantation
  • Perceived to be unreliable or unavailable for study participation or have parents or legal guardians who are perceived to be unreliable or unavailable
  • Use of any investigational agent within 30 days prior to screening, or requirement for any investigational agent or vaccine prior to completion of all scheduled study assessments
  • ALT > 2 times the upper limit of normal (i.e., Grade 1 or higher based on World Health Organization Toxicity Criteria) at screening
  • 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

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