Completed Studies
Study KOGNITO – 162-502
KUVAN®‘s Effect on the Cognition of Children With Phenylketonuria
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Indication
Phenylketonuria (PKU)
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Investigational Therapeutic
KUVAN® (sapropterin dihydrochloride, Phenoptin™ or BMN 162)
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Study Type
Phase 4
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Goal
A Phase IV Open-Label, Single-Cohort Study of the Long-Term Neurocognitive Outcomes in 4 to 5-Year-Old Children with Phenylketonuria Treated with Sapropterin Dihydrochloride (KUVAN®) for 7 Years
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Status
This study has been completed
Study KAMPER – 162-501
Observational Study on the Long-Term Safety of KUVAN® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency (KAMPER)
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Indication
Phenylketonuria (PKU)
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Investigational Therapeutic
KUVAN® (sapropterin dihydrochloride, Phenoptin™ or BMN 162)
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Study Type
Phase IV, Observational
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Goal
Assess the long-term safety in subjects treated with KUVAN®
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Status
This study has been completed
Study PKUDOS
Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
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Indication
Phenylketonuria (PKU), Hyperphenylalaninaemia
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Investigational Therapeutic
KUVAN® (sapropterin dihydrochloride, Phenoptin™ or BMN 162)
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Study Type
Phase IV, Observational
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Goal
Evaluate the safety of long-term treatment with KUVAN
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Status
This study has been completed
Study SPARK – 162-503
KUVAN® in Phenylketonuria Patients Less Than 4 Years Old
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Indication
Phenylketonuria (PKU)
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Investigational Therapeutic
KUVAN® (sapropterin dihydrochloride, Phenoptin™ or BMN 162)
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Study Type
Phase 3b
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Goal
Evaluate the efficacy after 26 weeks of KUVAN® treatment + Phe-restricted diet therapy in increasing dietary Phe tolerance, as compared to dietary therapy alone in <4 year-old infants and children with phenylketonuria (PKU). Phe tolerance will be defined as the amount of dietary Phe (mg/kg/day) ingested while maintaining blood Phe levels within the range of 120–360 μmol/L (defined as >=120 to <360 μmol/L).
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Status
This study has been completed
Study 162-015
Phase 3b Open-Label Study to Evaluate the Effect of KUVAN® on Neurocognitive Function, Maintenance of Blood Phenylalanine Concentrations, Safety, and Population Pharmacokinetics in Young Children with Phenylketonuria
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Indication
Phenylketonuria (PKU)
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Investigational Therapeutic
KUVAN® (sapropterin dihydrochloride, Phenoptin™ or BMN 162)
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Study Type
Phase 4
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Goal
Primary Objective
– To evaluate the long-term efficacy of KUVAN in preserving neurocognitive function in children with phenylketonuria (PKU) when treatment is initiated at 0 to 6 years of age at date of screening.
Secondary Objectives
– To evaluate the long-term safety of KUVAN in the study population
– To evaluate the effect of KUVAN on growth parameters in the study population
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Status
This study has been completed
Study PKU-003
Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
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Indication
Phenylketonuria (PKU)
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Investigational Therapeutic
KUVAN® (sapropterin dihydrochloride, Phenoptin™ or BMN 162)
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Study Type
Phase 3
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Goal
Evaluate the efficacy of Phenoptin™ (sapropterin dihydrochloride) in reducing blood phenylalanine (Phe) levels in subjects with phenylketonuria
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Status
This study has been completed
Study PKU-016
A Double-blind, Placebo-controlled, Randomized Study to Evaluate the Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Subjects with Phenylketonuria
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Indication
Phenylketonuria (PKU)
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Investigational Therapeutic
KUVAN® (sapropterin dihydrochloride, Phenoptin™ or BMN 162)
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Study Type
Phase 3
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Goal
Evaluate the safety and therapeutic effects of sapropterin dihydrochloride on neuropsychiatric symptoms in subjects with PKU
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Status
This study has been completed