Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet
Condition: Phenylketonurias
MeSH terms: Phenylketonurias
Intervention: Sapropterin Dihydrochloride; Placebo
Intervention description: Phenoptin, provided in tablets containing 100 mg of sapropterin dihydrochloride each, was administered orally once daily in the morning as the number of tablets equivalent to a 20mg/kg/day dose dissolved in 4-8 oz (120-240 mL) of water or apple juice for 6 weeks. A follow-up call or visit was made 4 weeks later during this double-blind, placebo-controlled study.; Placebo, provided as tablets similar to Phenoptin tablets, was administered orally once daily in the morning as the number of tablets equivalent to a 20mg/kg/day dose dissolved in 4 8 oz (120-240 mL) of water or apple juice. for 6 weeks. A follow-up call or visit was made 4 weeks later during this double-blind, placebo-controlled study.
Intervention MeSH terms: Verapamil
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double
Enrollment: 45.0
Sex: All
Minimum age: 4 Years
Maximum age: 12 Years
Healthy volunteers: No

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.
    

Eligibility 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 </=1000 mg/day

          -  At least 6 months of blood Phe control (mean level of </=480 μmol/L) prior to
             enrolling in the study

          -  Aged 4 to 12 years inclusive at screening

          -  A blood Phe level </=480 μmol/L at screening

          -  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)