240. フェニルケトン尿症 Phenylketonuria Clinical trials / Disease details
臨床試験数 : 143 / 薬物数 : 90 - (DrugBank : 10) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 5
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT05356377 (ClinicalTrials.gov) | May 25, 2022 | 27/4/2022 | Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU | A Preliminary Study of the Neurological and Neuropsychological Effects of Palynziq-Related Changes in Phenylalanine in Individuals With Phenylketonuria (PKU) | Phenylketonurias | Drug: Pegvaliase-Pqpz | University of Missouri-Columbia | BioMarin Pharmaceutical | Enrolling by invitation | 18 Years | 55 Years | All | 13 | United States | |
| 2 | NCT04375592 (ClinicalTrials.gov) | February 1, 2021 | 21/4/2020 | Acceptability and Tolerance of a Ready-to-use Protein Substitute in Tablet Form for the Dietary Management of Phenylketonuria | XPhe Minis - Acceptability and Tolerance Market Research | Phenylketonurias;Hyperphenylalaninaemia;Tetrahydrobiopterin Deficiency | Dietary Supplement: Phenylalanine-free protein substitute in tablet form (XPhe minis) | metaX Institut fuer Diatetik GmbH | Birmingham Children's Hospital | Recruiting | 7 Years | 18 Years | All | 10 | United Kingdom | |
| 3 | NCT04480567 (ClinicalTrials.gov) | September 24, 2020 | 8/7/2020 | AAV Gene Therapy Study for Subjects With PKU | A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects With Phenylketonuria | Phenylketonuria (PKU) | Drug: BMN 307 | BioMarin Pharmaceutical | NULL | Active, not recruiting | 15 Years | N/A | All | 100 | Phase 1/Phase 2 | United States;United Kingdom |
| 4 | NCT04302194 (ClinicalTrials.gov) | May 1, 2020 | 7/3/2020 | Early Dietary Treated Patients With Phenylketonuria Can Achieve Normal Growth and Mental Development. | Early Dietary Treated Patients With Phenylketonuria Can Achieve Normal Growth and Mental Development. | Phenylketonurias | Dietary Supplement: phenylalanine restricted diet | Assiut University | NULL | Not yet recruiting | 1 Month | 3 Years | All | 50 | NULL | |
| 5 | EUCTR2019-001878-28-GB (EUCTR) | 17/12/2019 | 21/11/2019 | A Phase 1/2 study to evaluate the safety and efficacy of BMN 307 gene therapy in patients with phenylketonuria | A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects with Phenylketonuria and Plasma Phe Levels > 600 µmol/L - A Phase 1/2 study to evaluate the safety and efficacy of BMN 307 | Phenylketonuria MedDRA version: 20.0;Level: PT;Classification code 10034872;Term: Phenylketonuria;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Code: BMN 307 | BioMarin Pharmaceutical Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 1;Phase 2 | United States;Taiwan;Spain;Turkey;Australia;Germany;Italy;United Kingdom | ||
| 6 | NCT03788343 (ClinicalTrials.gov) | August 19, 2019 | 17/12/2018 | Phenylalanine and Its Impact on Cognition | PICO: Phenylalanine and Its Impact on Cognition - Impact of Phenylalanine on Cognitive, Cerebral and Neurometabolic Parameters in Adult Patients With Phenylketonuria | Phenylketonuria | Dietary Supplement: Phenylalanine;Drug: Placebo | University Hospital Inselspital, Berne | University of Zurich | Completed | 18 Years | N/A | All | 90 | Phase 4 | Switzerland |
| 7 | NCT03333720 (ClinicalTrials.gov) | July 15, 2019 | 18/9/2017 | Compliance, Tolerance and Acceptability of a Tablet Protein Substitute for the Dietary Management of Phenylketonuria | Compliance, Tolerance and Acceptability of a Tablet Protein Substitute for the Dietary Management of Phenylketonuria | Phenylketonurias | Dietary Supplement: Phenylalanine-free protein substitute tablets | Nutricia UK Ltd | NULL | Withdrawn | 8 Years | 100 Years | All | 0 | N/A | NULL |
| 8 | NCT03168399 (ClinicalTrials.gov) | June 8, 2017 | 24/5/2017 | Evaluation of PKU Explore | A Study to Evaluate the Acceptability of PKU Explore, a Renovated Phenylalanine Free Second Stage Protein Substitute for Use in the Dietary Management of Phenylketonuria in Infants From 6 Months to 5 Years of Age With Regard to Product Tolerance and Adherence. | Phenylketonuria;Inborn Errors of Metabolism | Dietary Supplement: PKU Explore | Vitaflo International, Ltd | Birmingham Women's and Children's NHS Foundation Trust | Completed | 6 Months | 5 Years | All | 22 | N/A | United Kingdom |
| 9 | NCT03058848 (ClinicalTrials.gov) | March 6, 2017 | 16/2/2017 | Evaluation of PKU Start | A Study to Evaluate the Acceptability of a New Phenylalanine Free Infant Formula for Use in the Dietary Management of Phenylketonuria in Infants From Birth to 2 Year of Age With Regard to Product Tolerance and Adherence. | Phenylketonuria;Inborn Errors of Metabolism | Dietary Supplement: PKU Start | Vitaflo International, Ltd | NULL | Completed | N/A | 2 Years | All | 10 | N/A | United Kingdom |
| 10 | EUCTR2010-021343-41-NL (EUCTR) | 21/03/2014 | 30/11/2011 | Variations of blood phenylalanine and tyrosine in children with phenylketonuria under sapropterin | Effect of sapropterin on variations of blood phenylalanine and tyrosine over 24 hours and from day to day in children with phenylketonuria | Phenylketonuria (PKU) is an autosomal recessive metabolic genetic disorder by a mutation in the gene for the enzyme phenylalanine hydroxylase (PAH), rendering it nonfunctional. Left untreated, the disease will result in high concentrations of phenylalanine (Phe) in blood and tissues, likely resulting in severe mental retardation and behavioural problems. Treatment focusus on the restriction of dietary phenylalanine intake with supplementation of a synthetic phenylalaninefree amino acid mixture.;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Kuvan | University Medical Center Groningen | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Phase 4 | Netherlands | |||
| 11 | NCT01732471 (ClinicalTrials.gov) | November 2012 | 19/11/2012 | Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria | A Phase III Non-comparative Open-label Clinical Study to Evaluate the Response to and Safety of Kuvan (Sapropterin Dihydrochloride) After 6 Weeks of Treatment in Patients of 4 to 18 Years of Age With Phenylketonuria Who Have Elevated Blood Phenylalanine Levels | Phenylketonuria | Drug: Kuvan® | Merck KGaA | NULL | Completed | 4 Years | 18 Years | All | 90 | Phase 3 | Germany |
| 12 | NCT01376908 (ClinicalTrials.gov) | June 2011 | 17/6/2011 | Kuvan® in Phenylketonuria Patients Less Than 4 Years Old | A Phase IIIb, Multicentre, Open-Label, Randomized, Controlled Study of the Efficacy, Safety, and Population Pharmacokinetics of Sapropterin Dihydrochloride (Kuvan®) in Phenylketonuria (PKU) Patients <4 Years Old. | Phenylketonuria | Drug: Kuvan®;Other: Phenylalanine (Phe)-restricted diet | BioMarin Pharmaceutical | NULL | Completed | N/A | 4 Years | All | 56 | Phase 3 | Austria;Belgium;Czechia;Germany;Italy;Netherlands;Slovakia;Turkey;United Kingdom;Czech Republic;Portugal |
| 13 | NCT00838435 (ClinicalTrials.gov) | February 2009 | 5/2/2009 | Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU | A 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 | Phenylketonuria | Drug: sapropterin dihydrochloride | BioMarin Pharmaceutical | NULL | Completed | N/A | 6 Years | All | 95 | Phase 3 | United States;Canada |
| 14 | NCT00841100 (ClinicalTrials.gov) | December 2008 | 10/2/2009 | Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response | Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response | Phenylketonuria | Drug: Kuvan;Other: Diet | University of Miami | NULL | Completed | 4 Years | N/A | All | 21 | Phase 2 | United States |
| 15 | NCT00432822 (ClinicalTrials.gov) | February 2007 | 7/2/2007 | Long-Term Tetrahydrobiopterin Treatment in PKU Patients of 0-18 Years - Study on Phenylalanine Tolerance and Safety | Double-Blind, Placebo Controlled, Multicentre Study With an Open Label Extension to Evaluate the Efficacy and Safety of Tetrahydrobiopterin (BH4) in Children and Adolescents With Hyperphenylalaninemia Caused by Phenylalanine Hydroxylase Deficiency | Phenylalanine Hydroxylase Deficiencies | Drug: tetrahydrobiopterin (BH4) | Orphanetics Pharma Entwicklungs GmbH | NULL | Terminated | N/A | 18 Years | Both | 50 | Phase 2/Phase 3 | NULL |
| 16 | EUCTR2005-003777-24-GB (EUCTR) | 17/08/2006 | 20/04/2006 | A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of Phenoptin 20 mg/kg/day to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet - NA | A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of Phenoptin 20 mg/kg/day to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet - NA | Phenylketonuria (PKU) MedDRA version: 6.1;Classification code 10034872 | Product Name: Phenoptin Product Code: T1401 INN or Proposed INN: Sapropterin Other descriptive name: sapropterin dihydrochloride | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 300 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Spain;Germany;United Kingdom | ||
| 17 | EUCTR2006-000648-15-AT (EUCTR) | 08/06/2006 | 05/04/2006 | A double-blind, placebo-controlled, multicentre study with an open-label extension to evaluate the efficacy and safety of tetrahydrobiopterin (BH4) in children and adolescents with hyperphenylalaninemia caused by phenylalanine hydroxylase deficiency | A double-blind, placebo-controlled, multicentre study with an open-label extension to evaluate the efficacy and safety of tetrahydrobiopterin (BH4) in children and adolescents with hyperphenylalaninemia caused by phenylalanine hydroxylase deficiency | Hyperphenylalaninemia due to phenylalanine hydroxylase deficiency. Phenotypes: classic phenylketonuria (PKU), mild PKU (MPK) or mild hyperphenylalaninemia (HPA). MedDRA version: 81;Level: LLT;Classification code 10034873 | Product Name: tetrahydrobiopterin Product Code: BH4 INN or Proposed INN: Sapropterin Other descriptive name: n.a. | ORPHANETICS Pharma Entwicklungs- GmbH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 75 | Austria | |||
| 18 | EUCTR2005-003777-24-DE (EUCTR) | 24/03/2006 | 14/12/2005 | A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of Phenoptin 20 mg/kg/day to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet - NA | A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of Phenoptin 20 mg/kg/day to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet - NA | Phenylketonuria (PKU) MedDRA version: 6.1;Classification code 10034872 | Product Name: Phenoptin Product Code: T1401 INN or Proposed INN: Sapropterin Other descriptive name: sapropterin dihydrochloride | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 300 | Phase 3 | Germany;Spain | ||
| 19 | EUCTR2005-003777-24-ES (EUCTR) | 01/03/2006 | 19/01/2006 | Estudio fase 3, multicéntrico, aleatorizado, doble ciego y controlado con placebo para evaluar la seguridad y la eficacia de PhenoptinTM en dosis de 20 mg/kg/dia para aumentar la tolerancia a la fenilalanina en niños fenilcetonúricos que siguen dietas restrictivas en fenilalanina. | Estudio fase 3, multicéntrico, aleatorizado, doble ciego y controlado con placebo para evaluar la seguridad y la eficacia de PhenoptinTM en dosis de 20 mg/kg/dia para aumentar la tolerancia a la fenilalanina en niños fenilcetonúricos que siguen dietas restrictivas en fenilalanina. | Fenilcetonuria (Phenylketonuria-PKU) MedDRA version: 6.1;Classification code 10034872 | Product Name: Phenoptin Product Code: T1401 INN or Proposed INN: Sapropterin Other descriptive name: Sapropterin dihydrochloride | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 300 | Phase 3 | Spain;Germany;United Kingdom | ||
| 20 | NCT00225615 (ClinicalTrials.gov) | November 2005 | 22/9/2005 | A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels | A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels | Phenylketonurias | Drug: sapropterin dihydrochloride | BioMarin Pharmaceutical | NULL | Completed | 8 Years | N/A | Both | 100 | Phase 3 | United States |
| 21 | EUCTR2004-004513-41-DE (EUCTR) | 27/10/2005 | 12/05/2006 | A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels | A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels | Phenylketonuria (PKU) MedDRA version: 6.1;Classification code 10034872 | Product Name: Phenoptin Product Code: T1401 INN or Proposed INN: Sapropterin, sapropterin dihydrochloride | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | Ireland;Germany;Italy;United Kingdom | ||
| 22 | EUCTR2004-004513-41-GB (EUCTR) | 25/10/2005 | 02/06/2005 | A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels | A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels | Phenylketonuria (PKU) MedDRA version: 6.1;Classification code 10034872 | Product Name: Phenoptin Product Code: T1401 INN or Proposed INN: Sapropterin, sapropterin dihydrochloride | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Human pharmacology (Phase 1): Therapeutic exploratory (Phase 2): Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): | Ireland;Germany;Italy;United Kingdom | ||
| 23 | EUCTR2004-004513-41-IT (EUCTR) | 23/09/2005 | 11/11/2005 | A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels | A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels | Treatment of phenylketonuria MedDRA version: 6.1;Level: SOC;Classification code 10027433 | Product Name: Phenoptin | BIOMARIN | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | Germany;United Kingdom;Ireland;Italy | ||
| 24 | EUCTR2004-004513-41-IE (EUCTR) | 19/08/2005 | 10/06/2005 | A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels | A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels | Phenylketonuria (PKU) MedDRA version: 6.1;Classification code 10034872 | Product Name: Phenoptin Product Code: T1401 INN or Proposed INN: Sapropterin, sapropterin hydrochloride | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Human pharmacology (Phase 1): Therapeutic exploratory (Phase 2): Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): | Ireland;Germany;Italy;United Kingdom | ||
| 25 | EUCTR2004-004512-23-GB (EUCTR) | 15/08/2005 | 23/02/2005 | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA | Phenylketonuria (PKU) MedDRA version: 6.1;Classification code 10034872 | Product Name: Phenoptin Product Code: T1401 INN or Proposed INN: Sapropterin, sapropterin hydrochloride | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Human pharmacology (Phase 1): Therapeutic exploratory (Phase 2): Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): | Ireland;Germany;Italy;United Kingdom | ||
| 26 | EUCTR2004-004512-23-IT (EUCTR) | 09/06/2005 | 22/06/2005 | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels | Treatment of phenylketonuria MedDRA version: 6.1;Level: SOC;Classification code 10027433 | Product Name: Phenoptin | BIOMARIN | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | Germany;United Kingdom;Ireland;Italy | ||
| 27 | EUCTR2004-004512-23-IE (EUCTR) | 22/05/2005 | 15/02/2005 | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA | Phenylketonuria (PKU) MedDRA version: 6.1;Classification code 10034872 | Product Name: Phenoptin Product Code: T1401 INN or Proposed INN: Sapropterin, sapropterin hydrochloride | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Human pharmacology (Phase 1): Therapeutic exploratory (Phase 2): Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): | Ireland;Germany;Italy;United Kingdom | ||
| 28 | NCT00260000 (ClinicalTrials.gov) | April 2005 | 30/11/2005 | Study of BH4, a New and Simple Treatment of Mild PKU | Study of the Response of Tetrahydrobiopterin on S-Phenylalanine in Patients With PKU Housing the Y414C Mutation | Phenylketonuria | Drug: 5,6,7,8-tetrahydrobiopterin | The Kennedy Institute-National Eye Clinic | Sygekassernes Helsefond | Completed | 8 Years | N/A | Both | 15 | Phase 2 | Denmark |
| 29 | EUCTR2004-002071-16-GB (EUCTR) | 18/03/2005 | 22/02/2005 | A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA | A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA | Phenylketonuria (PKU) MedDRA version: 6.1;Classification code 10034872 | Product Name: Phenoptin Product Code: T1401 INN or Proposed INN: Sapropterin, sapropterin hydrochloride | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 700 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | Ireland;Germany;Italy;United Kingdom | ||
| 30 | EUCTR2004-002071-16-IT (EUCTR) | 17/03/2005 | 27/04/2005 | A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels | A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels | Treatment of Phenylketonuria MedDRA version: 6.1;Level: SOC;Classification code 10027433 | Product Name: Phenoptin Product Code: NA INN or Proposed INN: sapropterin hydrochloride Other descriptive name: NA | BIOMARIN | NULL | Not Recruiting | Female: yes Male: yes | Phase 2 | United Kingdom;Germany;Italy | |||
| 31 | NCT00104247 (ClinicalTrials.gov) | March 2005 | 24/2/2005 | Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels | Phenylketonurias | Drug: sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin | BioMarin Pharmaceutical | NULL | Completed | 8 Years | N/A | All | 89 | Phase 3 | United States |
| 32 | EUCTR2004-002071-16-DE (EUCTR) | 28/01/2005 | 14/10/2004 | A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA | A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA | Phenylketonuria (PKU) MedDRA version: 6.1;Classification code 10034872 | Product Name: Phenoptin Product Code: T1401 INN or Proposed INN: Sapropterin, sapropterin hydrochloride | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 700 | Phase 2 | United Kingdom;Germany;Italy | ||
| 33 | NCT00104260 (ClinicalTrials.gov) | December 2004 | 24/2/2005 | Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria | A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels | Phenylketonurias | Drug: sapropterin dihydrochloride | BioMarin Pharmaceutical | NULL | Completed | 8 Years | N/A | Both | 700 | Phase 2 | United States |
| 34 | EUCTR2004-002365-21-DK (EUCTR) | 03/11/2004 | 09/07/2008 | Trial with BH4, a new and simple treatment of phenylketonuria, PKU - BH4 and PKU | Trial with BH4, a new and simple treatment of phenylketonuria, PKU - BH4 and PKU | PKU, phenylketonuria, is a rare, inherited metabolic disease that results in mental retardation if not a very strict low-protein diet is started within the first weeks of life. The conversion of phenylalanine to tyrosine is defect, phe accumulates and leads to brain damage. There are different degrees of severity, reflecting the spectrum of mutant genes. BH4, tetrahydrobiopterin, is co-enzym for the conversion of phe to tyrosine. BH4 can lower phe in some patients with milder forms of PKU. | Product Name: tetrahydrobiopterin | John F. Kennedy Institute | NULL | Not Recruiting | Female: yes Male: yes | Denmark | ||||
| 35 | EUCTR2015-001650-15-Outside-EU/EEA (EUCTR) | 26/06/2015 | Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria | A Phase III Non-comparative Open-label Clinical Study to Evaluate the Response to and Safety of Kuvan (Sapropterin Dihydrochloride) After 6 Weeks of Treatment in Patients of 4 to 18 Years of Age With Phenylketonuria Who Have Elevated Blood Phenylalanine Levels | Phenylketonuria;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Kuvan 100mg soluble tablet Product Name: Sapropterin Dihydrocholoride | Merck Serono Middle East FZ-LLC | NULL | NA | Female: yes Male: yes | 90 | Phase 3 | Ukraine;Russian Federation | |||
| 36 | EUCTR2009-012978-12-NO (EUCTR) | 02/06/2009 | A Phase IV, prospective, open-label, uncontrolled, single-centre cohort trial to be conducted in Norway to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 7 days. - Response to Kuvan® in subjects with PKU from a single centre in Norway | A Phase IV, prospective, open-label, uncontrolled, single-centre cohort trial to be conducted in Norway to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 7 days. - Response to Kuvan® in subjects with PKU from a single centre in Norway | Hyperphenylalaninemia (HPA) in adults and children >=16 years of age with phenylketonuri (PKU) (classic or mild PKU, or mild hyperphenylalaninemia (HPA)). MedDRA version: 9.1;Level: LLT;Classification code 10034872;Term: Phenylketonuria MedDRA version: 9.1;Classification code 10034873;Term: Phenylketonuria (PKU) MedDRA version: 9.1;Level: PT;Classification code 10034871;Term: Phenylalaninemia | Trade Name: Kuvan® (sapropterin dihydrochloride) INN or Proposed INN: sapropterin | Merck Serono, an affiliate of E. Merck AB | NULL | Not Recruiting | Female: yes Male: yes | Phase 4 | Norway | ||||
| 37 | EUCTR2004-004512-23-DE (EUCTR) | 17/03/2005 | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA | Phenylketonuria (PKU) MedDRA version: 6.1;Classification code 10034872 | Product Name: Phenoptin Product Code: T1401 INN or Proposed INN: Sapropterin, sapropterin hydrochloride | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | United Kingdom;Germany;Ireland;Italy | |||
| 38 | EUCTR2004-002071-16-IE (EUCTR) | 13/10/2004 | A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA | A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA | Phenylketonuria (PKU) MedDRA version: 6.1;Classification code 10034872 | Product Name: Phenoptin Product Code: T1401 INN or Proposed INN: Sapropterin, sapropterin hydrochloride | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 400 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | Ireland;Germany;Italy;United Kingdom |