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52 studies found for:    Phenylketonuria

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Rank Status Study
21 Completed Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria
Condition: Phenylketonurias
Intervention: Drug: sapropterin dihydrochloride
22 Completed
Has Results 		Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
Condition: Phenylketonurias
Intervention: Drug: sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin
23 Recruiting Study of a National Cohort of Adult Patients With Phenylketonuria
Conditions: PKU;   Hyperphenylalaninemia;   BMD;   Quality of Life
Intervention:
24 Active, not recruiting Follow-up of Adult Phenylketonuria (PKU) Patients
Conditions: Nutritional Deficiencies;   Phenylketonuria
Intervention:
25 Unknown † Nutritional and Neurotransmitter Changes in PKU Subjects on BH4
Condition: Phenylketonuria
Intervention:
26 Completed Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria
Condition: Phenylketonuria
Intervention: Drug: rAvPAL-PEG
27 Active, not recruiting Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)
Condition: Phenylketonuria
Intervention: Drug: rAvPAL-PEG
28 Unknown † Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response
Condition: Phenylketonuria
Intervention: Drug: Kuvan
29 Enrolling by invitation Phase 2 Study of Glycomacropeptide Versus Amino Acid Diet for Management of Phenylketonuria
Condition: Phenylketonuria
Interventions: Dietary Supplement: Amino Acid (AA) Diet Given First;   Dietary Supplement: Glycomacropeptide (GMP) diet given first
30 Active, not recruiting Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU
Condition: Phenylketonuria
Intervention: Drug: sapropterin dihydrochloride
31 Terminated Long-Term Tetrahydrobiopterin Treatment in PKU Patients of 0-18 Years - Study on Phenylalanine Tolerance and Safety
Condition: Phenylalanine Hydroxylase Deficiencies
Intervention: Drug: tetrahydrobiopterin (BH4)
32 Recruiting Neuroimaging and Neurocognitive Assessment and Response to Sapropterin Dihydrochloride Treatment in Phenylketonuria
Condition: PKU
Interventions: Dietary Supplement: diet;   Drug: sapropterin dihydrochloride
33 Approved for marketing Sapropterin Expanded Access Program
Condition: Phenylketonuria
Intervention: Drug: Sapropterin dihydrochloride
34 Enrolling by invitation Effects of Kuvan on Melatonin Secretion
Condition: Phenylketonuria (PKU)
Interventions: Drug: Kuvan;   Dietary Supplement: Large Neutral Amino Acid Therapy
35 Completed
Has Results 		Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet
Condition: Phenylketonurias
Interventions: Drug: Sapropterin Dihydrochloride;   Drug: Placebo
36 Active, not recruiting A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks
Condition: Phenylketonuria
Intervention: Biological: rAvPAL-PEG
37 Enrolling by invitation Long-Term Extension of a Dose Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With Phenylketonuria (PKU) (PAL-003)
Condition: Phenylketonuria
Intervention: Drug: rAvPAL-PEG
38 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
39 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:
40 Completed A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels
Condition: Phenylketonurias
Intervention: Drug: sapropterin dihydrochloride
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† Indicates status has not been verified in more than two years