Amino Acid Metabolism, Inborn Errors - 53 Studies Found
| Active, not recruiting |
: Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003) : Phenylketonuria : 2009-06-18 : Drug: rAvPAL-PEG The doses are planned to be in the same range as those tested in PAL-002 or PAL-004 and |
| Active, not recruiting |
: Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU : Phenylketonuria : 2009-02-05 : Drug: sapropterin dihydrochloride A dose of 20 mg/kg will be administered dissolved in water or apple ju |
| Completed |
: A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects : Phenylketonuria : 2008-11-10 :
|
| Recruiting |
: PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry :
: 2008-10-21 : Drug: Kuvan
|
| Completed |
: Nutritional and Neurotransmitter Changes in PKU Subjects on BH4 : Phenylketonuria : 2008-05-29 : Drug: KuvanTM Therapy BH4 treatment was prescribed by each participant's medical provider, not an interv |
| Approved for marketing |
: Sapropterin Expanded Access Program : Phenylketonuria : 2007-06-08 : Drug: Sapropterin dihydrochloride 20mg/kg/day orally, physicians may adjust the dose within a range of 5 |
| Terminated |
: Long-Term Tetrahydrobiopterin Treatment in PKU Patients of 0-18 Years - Study on Phenylalanine Tolerance and Safety : Phenylalanine Hydroxylase Deficiencies : 2007-02-07 : Drug: tetrahydrobiopterin (BH4) |
| Completed |
: Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet : Phenylketonurias : 2006-01-05 :
|
| Completed |
: A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels : Phenylketonurias : 2005-09-22 : Drug: sapropterin dihydrochloride |
| Completed |
: Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria : Phenylketonurias : 2005-02-24 : Drug: sapropterin dihydrochloride |