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Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels

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.
 
ClinicalTrials.gov Identifier: NCT00104247
Recruitment Status : Completed
First Posted : February 25, 2005
Results First Posted : April 16, 2009
Last Update Posted : July 23, 2014
Sponsor:
Information provided by:
BioMarin Pharmaceutical

Tracking Information
First Submitted Date  ICMJE February 24, 2005
First Posted Date  ICMJE February 25, 2005
Results First Submitted Date  ICMJE January 29, 2009
Results First Posted Date  ICMJE April 16, 2009
Last Update Posted Date July 23, 2014
Study Start Date  ICMJE March 2005
Primary Completion Date Not Provided
Current Primary Outcome Measures  ICMJE
 (submitted: March 12, 2009)
Change in Blood Phenylalanine Levels From Baseline to Week 6. [ Time Frame: baseline to week 6 ]
Original Primary Outcome Measures  ICMJE
 (submitted: June 23, 2005)
The primary objective is to evaluate the efficacy of Phenoptin™ in reducing blood Phe levels in subjects with phenylketonuria
Change History Complete list of historical versions of study NCT00104247 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE
 (submitted: June 23, 2005)
  • A secondary objective of this study is to evaluate the safety of Phenoptin™ versus placebo in this subject population.
  • A secondary objective is to evaluate the efficacy of Phenoptin™ versus placebo in this subject population with respect to: The mean change in weekly blood Phe levels during the 6 weeks of treatment
  • The proportion of subjects who have blood Phe levels </=600 μmol/L at Week 6.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
Official Title  ICMJE 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
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double
Primary Purpose: Treatment
Condition  ICMJE Phenylketonurias
Intervention  ICMJE Drug: sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin
Study Arms  ICMJE Not Provided
Publications * 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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: March 12, 2009)
89
Original Enrollment  ICMJE
 (submitted: June 23, 2005)
100
Actual Study Completion Date  ICMJE February 2006
Primary Completion Date Not Provided
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • 8 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

Exclusion Criteria:

  • 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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 8 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00104247
Other Study ID Numbers  ICMJE PKU-003
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Not Provided
Study Sponsor  ICMJE BioMarin Pharmaceutical
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account BioMarin Pharmaceutical
Verification Date July 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP