Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
1 other identifier
interventional
89
1 country
13
Brief Summary
The primary objective of this study is to evaluate the efficacy of Phenoptin™ (sapropterin dihydrochloride) in reducing blood phenylalanine (Phe) levels in subjects with phenylketonuria.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Mar 2005
Shorter than P25 for phase_3
13 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 24, 2005
CompletedFirst Posted
Study publicly available on registry
February 25, 2005
CompletedStudy Start
First participant enrolled
March 1, 2005
CompletedStudy Completion
Last participant's last visit for all outcomes
February 1, 2006
CompletedResults Posted
Study results publicly available
April 16, 2009
CompletedJuly 23, 2014
July 1, 2014
February 24, 2005
January 29, 2009
July 15, 2014
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change in Blood Phenylalanine Levels From Baseline to Week 6.
baseline to week 6
Interventions
Eligibility Criteria
You may qualify if:
- years of age and older
- Received at least 7 out of 8 scheduled doses in Study PKU 001
- Responsive to Phenoptin™ in Study PKU-001, defined as a reduction in blood Phenylalanine level of \>/=30% compared with baseline
- Blood Phenylalanine level \>/=450 μmol/L at screening
- Willing and able to provide written informed consent or, in the case of subjects under the age of 18, provide written assent (if required) and written informed consent by a parent or legal guardian, after the nature of the study has been explained
- Negative urine pregnancy test at screening (females of child-bearing potential)
- Male and Female subjects of childbearing potential (if sexually active) must be using acceptable birth control measures, as determined by the investigator, and willing to continue to use acceptable birth control 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
You may not qualify if:
- 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
- Use of any investigational agent other than Phenoptin™ within 30 days prior to screening, or requirement for any investigational agent or investigational vaccine prior to completion of all scheduled study assessments
- Pregnant or breastfeeding, or considering pregnancy
- ALT \>5 times the upper limit of normal (i.e., Grade 3 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, or organ transplantation recipient)
- Serious neuropsychiatric illness (e.g., major depression) not currently under medical management
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (13)
Unknown Facility
Los Angeles, California, United States
Unknown Facility
Oakland, California, United States
Unknown Facility
New Haven, Connecticut, United States
Unknown Facility
Chicago, Illinois, United States
Unknown Facility
Boston, Massachusetts, United States
Unknown Facility
Minneapolis, Minnesota, United States
Unknown Facility
St Louis, Missouri, United States
Unknown Facility
New York, New York, United States
Unknown Facility
Portland, Oregon, United States
Unknown Facility
Pittsburgh, Pennsylvania, United States
Unknown Facility
Dallas, Texas, United States
Unknown Facility
Salt Lake City, Utah, United States
Unknown Facility
Madison, Wisconsin, United States
Related Publications (1)
Levy HL, Milanowski A, Chakrapani A, Cleary M, Lee P, Trefz FK, Whitley CB, Feillet F, Feigenbaum AS, Bebchuk JD, Christ-Schmidt H, Dorenbaum A; Sapropterin Research Group. Efficacy of sapropterin dihydrochloride (tetrahydrobiopterin, 6R-BH4) for reduction of phenylalanine concentration in patients with phenylketonuria: a phase III randomised placebo-controlled study. Lancet. 2007 Aug 11;370(9586):504-10. doi: 10.1016/S0140-6736(07)61234-3.
PMID: 17693179DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Information Services
- Organization
- BioMarin Pharmaceutical Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
Study Record Dates
First Submitted
February 24, 2005
First Posted
February 25, 2005
Study Start
March 1, 2005
Study Completion
February 1, 2006
Last Updated
July 23, 2014
Results First Posted
April 16, 2009
Record last verified: 2014-07