Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00332189
Recruitment Status : Completed
First Posted : June 1, 2006
Results First Posted : November 16, 2012
Last Update Posted : November 16, 2012
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Brief Summary:
The objective of this study is to evaluate the safety of long-term treatment with Phenoptin in subjects with phenylketonuria (PKU) who participated in Phase 3 clinical studies with Phenoptin.

Condition or disease Intervention/treatment Phase
Phenylketonuria Drug: sapropterin dihydrochloride Phase 3

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 111 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006
Study Start Date : July 2006
Actual Primary Completion Date : August 2009
Actual Study Completion Date : August 2009

Intervention Details:
  • Drug: sapropterin dihydrochloride
    5-20mg/kg/day orally, dose may be adjusted up or down as needed at the discretion of the investigator in increments of 5mg/kg/day.
    Other Name: Kuvan

Primary Outcome Measures :
  1. Tabulation of the Incidence and Frequency of All AEs and SAEs That Occur Throughout the Study. [ Time Frame: Baseline through Final Visit (a Maximum of 30 months) with AEs collected at month 3 and 6 then at 6 month intervals ]
    Safety was assessed with regards to the rate and type of AEs, clinically significant changes to vital signs and/or physical examination findings, and clinically significant changes in laboratory test results.

Secondary Outcome Measures :
  1. Following Blood Phe Levels. [ Time Frame: Baseline through Final Visit (a Maximum of 30 months) with blood phe samples taken at months 3 and 6 then at 6 month intervals ]
    There were no pre-specified efficacy analysis, blood Phe samples were taken at each visit. Blood Phe concentrations remained within levels consistent with local clinical site recommendations for blood Phe control.

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Ages Eligible for Study:   4 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Participation in study PKU-004 or PKU-006
  • Willing and able to provide written, signed informed consent or, in the case of subjects under the age of 18 years, provide written assent (if required) 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
  • Negative urine pregnancy test at screening (females of child-bearing potential)
  • Willing and able to comply with all study procedures

Exclusion Criteria:

  • Non-responsive to prior treatment with Phenoptin based on participation in PKU-004 or PKU-006
  • Perceived to be unreliable or unavailable for study participation or, if under the age of 18 years, have parents or legal guardians who are perceived to be unreliable or unavailable
  • Terminated early from PKU-004 or PKU-006, except for subjects in PKU-004 that rolled into PKU-008 at Week 22, subjects in PKU-006 that rolled into PKU-008 at Week 10, or subjects in PKU-006 that terminated due to elevated Phe levels following dietary Phe increases
  • Use of any investigational product other than Phenoptin within 30 days prior to screening, or anticipated requirement for any investigational agent prior to completion of all scheduled study assessments
  • Positive urine pregnancy test at screening (non-sterile females of child-bearing potential only), already known to be pregnant or breastfeeding or planning a pregnancy in self or partner during the study
  • Female subjects of childbearing potential must be using an effective method of birth control, as determined by the PI, and willing to continue to use acceptable birth control measures
  • 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)
  • Any condition that, in the view of the PI, renders the subject at high risk from treatment compliance and/or completing the study
  • ALT > 2 times the upper limit of normal (i.e., Grade 1 or higher based on World Health Organization Toxicity Criteria) at screening (see Appendix 2)
  • Serious neuropsychiatric illness (e.g., major depression) not currently under medical control
  • Prior history of organ transplantation
  • Requirement for concomitant treatment with any drug known to inhibit folate synthesis (e.g., methotrexate)
  • Concurrent use of levodopa
  • Clinical diagnosis of primary BH4 deficiency

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00332189

United States, California
Los Angeles, California, United States
Sacramento, California, United States
San Jose, California, United States
United States, Connecticut
New Haven, Connecticut, United States
United States, Georgia
Atlanta, Georgia, United States
United States, Illinois
Chicago, Illinois, United States
United States, Minnesota
Minneapolis, Minnesota, United States
United States, Missouri
St. Louis, Missouri, United States
United States, New York
New York, New York, United States
United States, Oregon
Portland, Oregon, United States
United States, Texas
Dallas, Texas, United States
United States, Utah
Salt Lake City, Utah, United States
United States, Washington
Seattle, Washington, United States
United States, Wisconsin
Madison, Wisconsin, United States
Sponsors and Collaborators
BioMarin Pharmaceutical

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: BioMarin Pharmaceutical Identifier: NCT00332189     History of Changes
Other Study ID Numbers: PKU-008
First Posted: June 1, 2006    Key Record Dates
Results First Posted: November 16, 2012
Last Update Posted: November 16, 2012
Last Verified: October 2012

Keywords provided by BioMarin Pharmaceutical:

Additional relevant MeSH terms:
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Anti-Arrhythmia Agents
Calcium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Vasodilator Agents