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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 - At least 6 months of blood Phe control (mean level of enrolling in the study

- Aged 4 to 12 years inclusive at screening

- A blood Phe level

- 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

Page last updated: August 23, 2015

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