Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet
Information source: BioMarin Pharmaceutical
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Phenylketonurias
Intervention: Sapropterin Dihydrochloride (Drug); Placebo (Drug)
Phase: Phase 3
Status: Completed
Sponsored by: BioMarin Pharmaceutical
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.
Clinical Details
Official title: A Phase 3, Double-blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet
Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double-Blind, Primary Purpose: Treatment
Primary outcome: Amount of Dietary Supplemented Phenylalanine (Phe)Tolerated in Children With Phenylketonuria
Secondary outcome: Change in Phenylalanine Levels From Baseline to Week 3
Eligibility
Minimum age: 4 Years.
Maximum age: 12 Years.
Gender(s): Both.
Criteria:
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)
Locations and Contacts
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
Additional Information
BioMarin Pharmaceutical Inc website NDA Review Package
Starting date: February 2006
Last updated: July 23, 2015
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