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15 studies found for:    Open Studies | "Phenylketonurias"

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1 Recruiting Liver Cell Transplant for Phenylketonuria
Condition: Phenylketonuria
Interventions: Radiation: Preparative Radiation Therapy;   Procedure: Hepatocyte Transplant;   Drug: Immunosuppression;   Other: Liver Evaluation
2 Unknown † Response to Phenylketonuria to Tetrahydrobiopterin (BH4)
Condition: Phenylketonuria
Intervention: Drug: tetrahydrobiopterin (BH4)
3 Recruiting Phenylketonuria, Oxidative Stress, and BH4
Condition: Phenylketonuria
Interventions: Drug: Kuvan;   Other: Meal Challenge
4 Recruiting PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
Conditions: Phenylketonuria;   Hyperphenylalaninaemia
Intervention: Drug: Sapropterin Dihydrochloride
5 Recruiting Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Patients With Phenylketonuria
Condition: Phenylketonuria
Intervention: Drug: Kuvan®
6 Recruiting Kuvan® in Phenylketonuria (PKU) Patients Less Than 4 Years Old
Condition: Phenylketonuria (PKU)
Interventions: Drug: Kuvan®;   Other: Phe-restricted diet
7 Unknown † Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response
Condition: Phenylketonuria
Intervention: Drug: Kuvan
8 Not yet recruiting An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165
Condition: Phenylketonuria
Intervention: Drug: BMN 165
9 Not yet recruiting The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical PKU
Condition: Classical Phenylketonuria(PKU)
Intervention:
10 Recruiting Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency
Condition: Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency
Intervention:
11 Unknown † Bone Mineral Density in Adults With Hyperphenylalaninemia
Condition: Phenylketonurias
Intervention:
12 Unknown † Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression Kuvan Nonresponders
Conditions: Phenylketonuria;   Behavior and Behavior Mechanisms;   PAH Gene Expression
Intervention: Drug: sapropterin dihydrochloride
13 Recruiting Study of a National Cohort of Adult Patients With Phenylketonuria
Conditions: PKU;   Hyperphenylalaninemia;   BMD;   Quality of Life
Intervention:
14 Not yet recruiting A Pilot Study on Diurnal Variation
Condition: Phenylketonuria (PKU)
Intervention: Drug: Kuvan
15 Recruiting Neuroimaging and Neurocognitive Assessment and Response to Sapropterin Dihydrochloride Treatment in Phenylketonuria
Condition: PKU
Interventions: Dietary Supplement: diet;   Drug: sapropterin dihydrochloride
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† Indicates status has not been verified in more than two years