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

This study has been completed.
Sponsor:
Information provided by:
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT00104247
First received: February 24, 2005
Last updated: July 15, 2014
Last verified: July 2014

February 24, 2005
July 15, 2014
March 2005
Not Provided
Change in Blood Phenylalanine Levels From Baseline to Week 6. [ Time Frame: baseline to week 6 ] [ Designated as safety issue: No ]
The primary objective is to evaluate the efficacy of Phenoptin™ in reducing blood Phe levels in subjects with phenylketonuria
Complete list of historical versions of study NCT00104247 on ClinicalTrials.gov Archive Site
Not Provided
  • 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.
Not Provided
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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

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.

Not Provided
Interventional
Phase 3
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double-Blind
Primary Purpose: Treatment
Phenylketonurias
Drug: sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin
Not Provided
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.
 
Completed
89
February 2006
Not Provided

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
Both
8 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00104247
PKU-003
Not Provided
Not Provided
BioMarin Pharmaceutical
Not Provided
Not Provided
BioMarin Pharmaceutical
July 2014

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