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Current Studies


 

Study PKUDOS

Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry

  • Indication

    Phenylketonuria (PKU), Hyperphenylalaninaemia

  • Investigational Therapeutic

    Kuvan® (sapropterin dihydrochloride, Phenoptin™ or BMN 162)

  • Study Type

    Observational

  • Goal

    Evaluate the safety of long-term treatment with Kuvan

  • Status

    This study is ongoing, but not recruiting participants

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Study KOGNITO – 162-502

Kuvan®‘s Effect on the Cognition of Children With Phenylketonuria

  • Indication

    Phenylketonuria (PKU)

  • Investigational Therapeutic

    Kuvan® (sapropterin dihydrochloride, Phenoptin™ or BMN 162)

  • Study Type

    Phase 4

  • 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

  • Status

    This study is ongoing, but not recruiting participants

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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)

  • Indication

    PKU

  • Investigational Therapeutic

    Kuvan® (sapropterin dihydrochloride, Phenoptin™ or BMN 162)

  • Study Type

    Phase 4

  • Goal

    Assess the long-term safety in subjects treated with Kuvan®

  • Status

    This study is active, but not recruiting participants

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Completed Studies


 

Study SPARK – 162-503

Kuvan® in Phenylketonuria Patients Less Than 4 Years Old

  • Indication

    Phenylketonuria (PKU)

  • Investigational Therapeutic

    Kuvan® (sapropterin dihydrochloride, Phenoptin™ or BMN 162)

  • Study Type

    Phase 3b

  • 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).

  • Status

    This study has been completed

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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

  • Indication

    Phenylketonuria (PKU)

  • Investigational Therapeutic

    Kuvan® (sapropterin dihydrochloride, Phenoptin™ or BMN 162)

  • Study Type

    Phase 4

  • 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

  • Status

    This study has been completed

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Study PKU-003

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

  • Indication

    Phenylketonuria (PKU)

  • Investigational Therapeutic

    Kuvan® (sapropterin dihydrochloride, Phenoptin™ or BMN 162)

  • Study Type

    Phase 3

  • Goal

    Evaluate the efficacy of Phenoptin™ (sapropterin dihydrochloride) in reducing blood phenylalanine (Phe) levels in subjects with phenylketonuria

  • Status

    This study has been completed

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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

  • Indication

    Phenylketonuria (PKU)

  • Investigational Therapeutic

    Kuvan® (sapropterin dihydrochloride, Phenoptin™ or BMN 162)

  • Study Type

    Phase 3

  • Goal

    Evaluate the safety and therapeutic effects of sapropterin dihydrochloride on neuropsychiatric symptoms in subjects with PKU

  • Status

    This study has been completed

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