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 | EUCTR2009-015768-33-DE (EUCTR) | 20/05/2011 | 04/11/2010 | Safety Paediatric efficAcy phaRmacokinetic with Kuvan® | 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 - SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan®) | Phenylketonuria MedDRA version: 19.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Kuvan Product Name: Sapropterin Dihydrocholoride Product Code: NAP INN or Proposed INN: SAPROPTERIN Other descriptive name: tetrahydrobiopterin | BioMarin International Ltd | NULL | Not Recruiting | Female: yes Male: yes | 56 | Phase 3 | Portugal;Czech Republic;Slovakia;Belgium;Turkey;Austria;Netherlands;Germany;Italy;United Kingdom | ||
| 2 | EUCTR2009-015768-33-PT (EUCTR) | 13/05/2011 | 16/12/2010 | Safety Paediatric efficAcy phaRmacokinetic with Kuvan® | 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 - SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan®) | Phenylketonuria MedDRA version: 14.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Kuvan Product Name: Sapropterin Dihydrocholoride Product Code: NAP INN or Proposed INN: SAPROPTERIN Other descriptive name: tetrahydrobiopterin | Merck Serono S.A. - Geneva | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | Portugal;Czech Republic;Slovakia;Belgium;Turkey;Austria;Netherlands;Germany;Italy;United Kingdom | ||
| 3 | EUCTR2009-015768-33-BE (EUCTR) | 16/02/2011 | 19/01/2011 | Safety Paediatric efficAcy phaRmacokinetic with Kuvan® | 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 - SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan®) | Phenylketonuria MedDRA version: 18.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Kuvan Product Name: Sapropterin Dihydrocholoride Product Code: NAP INN or Proposed INN: SAPROPTERIN Other descriptive name: tetrahydrobiopterin | BioMarin International Ltd | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | Portugal;Czech Republic;Slovakia;Belgium;Turkey;Austria;Netherlands;Germany;Italy;United Kingdom | ||
| 4 | EUCTR2009-015768-33-SK (EUCTR) | 10/02/2011 | 03/11/2010 | Safety Paediatric efficAcy phaRmacokinetic with Kuvan® | 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 - SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan®) | Phenylketonuria MedDRA version: 18.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Kuvan Product Name: Sapropterin Dihydrocholoride Product Code: NAP INN or Proposed INN: SAPROPTERIN Other descriptive name: tetrahydrobiopterin | BioMarin International Ltd | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | Portugal;Czech Republic;Slovakia;Belgium;Turkey;Austria;Netherlands;Germany;Italy;United Kingdom | ||
| 5 | EUCTR2009-015768-33-AT (EUCTR) | 05/01/2011 | 25/11/2010 | Safety Paediatric efficAcy phaRmacokinetic with Kuvan® | 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 - SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan®) | Phenylketonuria MedDRA version: 18.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Kuvan Product Name: Sapropterin Dihydrocholoride Product Code: NAP INN or Proposed INN: SAPROPTERIN Other descriptive name: tetrahydrobiopterin | BioMarin International Ltd | NULL | Not Recruiting | Female: yes Male: yes | 54 | Phase 3 | Portugal;Czech Republic;Slovakia;Belgium;Turkey;Austria;Netherlands;Germany;Italy;United Kingdom | ||
| 6 | EUCTR2009-015768-33-GB (EUCTR) | 04/01/2011 | 11/10/2010 | Safety Paediatric efficAcy phaRmacokinetic with Kuvan® | 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 - SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan®) | Phenylketonuria MedDRA version: 19.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Kuvan Product Name: Sapropterin Dihydrocholoride Product Code: NAP INN or Proposed INN: SAPROPTERIN Other descriptive name: tetrahydrobiopterin | BioMarin International Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 56 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Portugal;Czech Republic;Slovakia;Belgium;Turkey;Austria;Netherlands;Germany;Italy;United Kingdom | ||
| 7 | EUCTR2009-015768-33-CZ (EUCTR) | 15/12/2010 | 10/12/2010 | Safety Paediatric efficAcy phaRmacokinetic with Kuvan® | 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 - SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan®) | Phenylketonuria MedDRA version: 19.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850 | Trade Name: Kuvan Product Name: Sapropterin Dihydrocholoride Product Code: NAP INN or Proposed INN: SAPROPTERIN Other descriptive name: tetrahydrobiopterin | BioMarin International Ltd | NULL | Not Recruiting | Female: yes Male: yes | 56 | Phase 3 | Portugal;Czech Republic;Slovakia;Belgium;Turkey;Austria;Netherlands;Germany;Italy;United Kingdom | ||
| 8 | 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 |