Status | Study |
Recruiting |
Study Name: Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia Condition: Metabolic Disease Date: 2016-09-03 |
Recruiting |
Study Name: GMP Drink for PKU Study Condition: Phenylketonuria Date: 2016-08-08 Interventions: Dietary Supplement: Glycomacropeptide-based protein substitute |
Completed |
Study Name: COMPARATIVE EFFECTIVENESS OF MCI and DEMENTIA TREATMENTS IN A COMMUNITY-BASED DEMENTIA PRACTICE Condition: Mild Cognitive Impairment Dementia Hypoxia Date: 2016-07-29 |
Recruiting |
Study Name: A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients Condition: AADC Deficiency Date: 2016-07-20 Interventions: Drug: AAV2-hAADC Subjects will be enrolled sequentially in |
Enrolling by invitation |
Study Name: Feasibility Study of the Intensive Systolic Blood Pressure Control Condition: Hypertension Hyperhomocysteinemia Date: 2016-06-18 Interventions: Drug: Standard BP control Enal |
Not yet recruiting |
Study Name: Review of French Cases of Glutathione Synthetase Deficiency Condition: The Glutathione Synthetase Deficiency Date: 2016-06-17 |
Recruiting |
Study Name: Neuroimaging and Neuropsychological Outcomes in Urea Cycle Disorders Condition: Urea Cycle Disorders Date: 2016-05-12 Interventions: Procedure: MRI MRI, fMRI, 1H M |
Completed |
Study Name: Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1) Condition: Hereditary Tyrosinemia, Type I Date: 2016-04-21 Interventions: Drug: Nitisinone A single oral |
Completed |
Study Name: Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1) Condition: Hereditary Tyrosinemia, Type I Date: 2016-04-21 Interventions: Drug: Nitisinone A single oral dose of Nitisinone 10 mg Tablet will be administered. |
Completed |
Study Name: Bioequivalence Study of Two Nitisinone Formulations Compared to Orfadin Condition: Hereditary Tyrosinemia, Type I Date: 2016-04-20 Interventions: Drug: Nitisinone A single oral |