SAPROPTERIN DIHYDROCHLORIDE ( DrugBank: Sapropterin )
1 disease
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 240 | フェニルケトン尿症 | 45 |
240. フェニルケトン尿症
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 | EUCTR2010-024311-13-NL (EUCTR) | 13/05/2014 | 27/01/2014 | A clinical study, carried out in different centers and using a placebo, to assess the effect of a medicinal product, containing sapropterin as the active substance, on mental abilities in young adults suffering from phenylketonuria, a genetic disease in which an enzyme defect leads to accumulation of a waste product (phenylketone) which can be detected in urine. | A Multicenter, Double-Blind, Placebo Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing the Effect of Sapropterin on Cognitive Abilities in Young Adults with Phenylketonuria - SIGNAL (SapropterIn on coGnitive abilities in youNg Adults with phenyLketonuria) | Phenylketonuria MedDRA version: 16.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Kuvan® INN or Proposed INN: Sapropterin Other descriptive name: SAPROPTERIN DIHYDROCHLORIDE | Merck KGaA | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | France;Spain;Belgium;Germany;Netherlands;Italy;Switzerland | ||
| 2 | EUCTR2010-024311-13-DE (EUCTR) | 02/01/2014 | 23/07/2013 | A clinical study, carried out in different centers and using a placebo, to assess the effect of a medicinal product, containing sapropterin as the active substance, on mental abilities in young adults suffering from phenylketonuria, a genetic disease in which an enzyme defect leads to accumulation of a waste product (phenylketone) which can be detected in urine. | A Multicenter, Double-Blind, Placebo Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing the Effect of Sapropterin on Cognitive Abilities in Young Adults with Phenylketonuria - SIGNAL (SapropterIn on coGnitive abilities in youNg Adults with phenyLketonuria) | Phenylketonuria MedDRA version: 16.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Kuvan® INN or Proposed INN: Sapropterin Other descriptive name: SAPROPTERIN DIHYDROCHLORIDE | Merck KGaA | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | France;Belgium;Spain;Netherlands;Germany;Italy;Switzerland | ||
| 3 | EUCTR2010-024311-13-BE (EUCTR) | 23/12/2013 | 25/09/2013 | A clinical study, carried out in different centers and using a placebo, to assess the effect of a medicinal product, containing sapropterin as the active substance, on mental abilities in young adults suffering from phenylketonuria, a genetic disease in which an enzyme defect leads to accumulation of a waste product (phenylketone) which can be detected in urine. | A Multicenter, Double-Blind, Placebo Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing the Effect of Sapropterin on Cognitive Abilities in Young Adults with Phenylketonuria - SIGNAL (SapropterIn on coGnitive abilities in youNg Adults with phenyLketonuria) | Phenylketonuria MedDRA version: 16.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Kuvan® INN or Proposed INN: Sapropterin Other descriptive name: SAPROPTERIN DIHYDROCHLORIDE | Merck KGaA | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | France;Spain;Belgium;Netherlands;Germany;Italy;Switzerland | ||
| 4 | EUCTR2010-024311-13-ES (EUCTR) | 21/11/2013 | 01/10/2013 | A clinical study, carried out in different centers and using a placebo, to assess the effect of a medicinal product, containing sapropterin as the active substance, on mental abilities in young adults suffering from phenylketonuria, a genetic disease in which an enzyme defect leads to accumulation of a waste product (phenylketone) which can be detected in urine. | A Multicenter, Double-Blind, Placebo Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing the Effect of Sapropterin on Cognitive Abilities in Young Adults with Phenylketonuria - SIGNAL (SapropterIn on coGnitive abilities in youNg Adults with phenyLketonuria) | Phenylketonuria MedDRA version: 16.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Kuvan® INN or Proposed INN: Sapropterin Other descriptive name: SAPROPTERIN DIHYDROCHLORIDE | Merck KGaA | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | France;Belgium;Spain;Netherlands;Germany;Italy;Switzerland | ||
| 5 | EUCTR2009-015844-41-ES (EUCTR) | 31/10/2013 | 16/01/2014 | A Study of the Long-term Effects on Mental Abilities of 4 to 5 Year-old Children with a Disease Caused by an Enzyme Defect (Phenylketonuria) that Have Been Treated with Kuvan® (a Medicinal Product aimed at Restoring the Defect) for 7 Years. | A Phase IV Open-Label, Single-Cohort Study of the Long-Term Neurocognitive Outcomes in 4 to 5 Year-Old Children with Phenylketonuria Treated with Sapropterin Dihydrochloride (Kuvan®) for 7 Years. - KOGNITO (Kuvan®'s effect on the cOGNITion of children with phenylketOnuria) | Phenylketonuria MedDRA version: 16.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Kuvan INN or Proposed INN: SAPROPTERIN Other descriptive name: tetrahydrobiopterin | Merck KGaA | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 4 | Spain;Germany;United Kingdom | ||
| 6 | NCT01965912 (ClinicalTrials.gov) | October 2013 | 16/10/2013 | Kuvan®'s Effect on the Cognition of Children With Phenylketonuria | A Phase 4 Open-Label, Single-Cohort Study of the Long-Term Neurocognitive Outcomes in 4 to 5 Year-Old Children With Phenylketonuria Treated With Sapropterin Dihydrochloride (Kuvan®) for 7 Years | Phenylketonuria | Drug: Kuvan® | BioMarin Pharmaceutical | NULL | Active, not recruiting | 4 Years | 5 Years | All | 34 | Phase 4 | Germany;Italy;Spain;United Kingdom |
| 7 | EUCTR2010-024311-13-IT (EUCTR) | 21/09/2013 | 26/07/2013 | A clinical study, carried out in different centers and using a placebo, to assess the effect of a medicinal product, containing sapropterin as the active substance, on mental abilities in young adults suffering from phenylketonuria, a genetic disease in which an enzyme defect leads to accumulation of a waste product (phenylketone) which can be detected in urine. | A Multicenter, Double-Blind, Placebo Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing the Effect of Sapropterin on Cognitive Abilities in Young Adults with Phenylketonuria - SIGNAL (SapropterIn on coGnitive abilities in youNg Adults with phenyLketonuria) | Phenylketonuria MedDRA version: 14.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Kuvan® INN or Proposed INN: Sapropterin Other descriptive name: SAPROPTERIN DIHYDROCHLORIDE | Merck KGaA | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | France;Belgium;Spain;Netherlands;Germany;Switzerland;Italy | ||
| 8 | EUCTR2009-015844-41-DE (EUCTR) | 10/09/2013 | 13/06/2013 | A Study of the Long-term Effects on Mental Abilities of 4 to 5 Year-old Children with a Disease Caused by an Enzyme Defect (Phenylketonuria) that Have Been Treated with Kuvan® (a Medicinal Product aimed at Restoring the Defect) for 7 Years. | A Phase IV Open-Label, Single-Cohort Study of the Long-Term Neurocognitive Outcomes in 4 to 5 Year-Old Children with Phenylketonuria Treated with Sapropterin Dihydrochloride (Kuvan®) for 7 Years. - KOGNITO (Kuvan®’s effect on the cOGNITion of children with phenylketOnuria) | Phenylketonuria MedDRA version: 20.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Kuvan INN or Proposed INN: SAPROPTERIN Other descriptive name: tetrahydrobiopterin | BioMarin International Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): yes | Spain;Germany;Italy;United Kingdom | ||
| 9 | EUCTR2009-015844-41-IT (EUCTR) | 02/09/2013 | 25/06/2013 | A Study of the Long-term Effects on Mental Abilities of 4 to 5 Year-old Children with a Disease Caused by an Enzyme Defect (Phenylketonuria) that Have Been Treated with Kuvan® (a Medicinal Product aimed at Restoring the Defect) for 7 Years. | A Phase IV Open-Label, Single-Cohort Study of the Long-Term Neurocognitive Outcomes in 4 to 5 Year-Old Children with Phenylketonuria Treated with Sapropterin Dihydrochloride (Kuvan®) for 7 Years. - KOGNITO (Kuvan®’s effect on the cOGNITion of children with phenylketOnuria) | Phenylketonuria MedDRA version: 14.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Kuvan INN or Proposed INN: SAPROPTERIN Other descriptive name: tetrahydrobiopterin | Merck KGaA | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 4 | Spain;Germany;United Kingdom;Italy | ||
| 10 | EUCTR2009-015844-41-GB (EUCTR) | 25/07/2013 | 13/06/2013 | A Study of the Long-term Effects on Mental Abilities of 4 to 5 Year-old Children with a Disease Caused by an Enzyme Defect (Phenylketonuria) that Have Been Treated with Kuvan® (a Medicinal Product aimed at Restoring the Defect) for 7 Years. | A Phase IV Open-Label, Single-Cohort Study of the Long-Term Neurocognitive Outcomes in 4 to 5 Year-Old Children with Phenylketonuria Treated with Sapropterin Dihydrochloride (Kuvan®) for 7 Years. - KOGNITO (Kuvan®’s effect on the cOGNITion of children with phenylketOnuria) | Phenylketonuria MedDRA version: 19.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Kuvan Product Name: Kuvan INN or Proposed INN: SAPROPTERIN Other descriptive name: tetrahydrobiopterin | BioMarin International Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 4 | Spain;Germany;Italy;United Kingdom | ||
| 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 | NCT01395394 (ClinicalTrials.gov) | June 2011 | 23/6/2011 | Phenylketonuria, Oxidative Stress, and BH4 | The Ability of Kuvan® (Sapropterin Dihydrochloride) to Prevent Meal-induced Lipid Peroxidation and Endothelial Dysfunction in Patients With Phenylketonuria: a Pilot Study | Phenylketonuria | Drug: Kuvan;Other: Meal Challenge | Emory University | NULL | Terminated | 10 Years | 45 Years | All | 12 | Phase 2 | United States |
| 13 | 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 |
| 14 | 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 | ||
| 15 | 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 | ||
| 16 | EUCTR2009-015768-33-NL (EUCTR) | 03/05/2011 | 14/02/2011 | 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: 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 Dihydrochloride 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;Germany;Netherlands;Italy;United Kingdom | ||
| 17 | NCT01412437 (ClinicalTrials.gov) | April 2011 | 1/8/2011 | Neuroimaging and Neurocognitive Assessment and Response to Sapropterin Dihydrochloride Treatment in Phenylketonuria | Multimodal Neuroimaging and Neurocognitive Assessment of Biomarkers and Response to Sapropterin Dihydrochloride Treatment in Phenylketonuria | PKU | Dietary Supplement: diet;Drug: sapropterin dihydrochloride | Children's Research Institute | Georgetown University | Withdrawn | 18 Years | 40 Years | Both | 0 | N/A | United States |
| 18 | EUCTR2009-015768-33-IT (EUCTR) | 15/03/2011 | 28/12/2011 | SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan) | A Phase IIIb, Multicenter, Open-Label, Randomized, Controlled Study of the Efficacy, Safety, and Population Pharmacokinetics of Sapropterin Dihydrochloride (Kuvan) in Phenylketonuria (PKU) Patients <4 Years Old - SPARK | Phenylketonuria MedDRA version: 14.1;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1;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] | Trade Name: KUVAN INN or Proposed INN: SAPROPTERIN | MERCK SERONO SA | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | Portugal;Czech Republic;Slovakia;Belgium;Turkey;Austria;Netherlands;Germany;United Kingdom;Italy | ||
| 19 | 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 | ||
| 20 | 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 | ||
| 21 | 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 | ||
| 22 | 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 | ||
| 23 | NCT01274026 (ClinicalTrials.gov) | January 2011 | 10/1/2011 | Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression Kuvan Nonresponders | Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression in PKU Nonresponders to Kuvan® (Sapropterin Dihydrochloride) | Phenylketonuria;Behavior and Behavior Mechanisms;PAH Gene Expression | Drug: sapropterin dihydrochloride | Tulane University School of Medicine | BioMarin Pharmaceutical | Completed | 2 Years | N/A | All | 21 | United States | |
| 24 | 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 | ||
| 25 | NCT01114737 (ClinicalTrials.gov) | August 2010 | 27/4/2010 | Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients | A Double-blind, Placebo-controlled, Randomized Study to Evaluate the Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Subjects With Phenylketonuria | Phenylketonuria | Drug: Sapropterin dihydrochloride;Drug: Placebo | BioMarin Pharmaceutical | NULL | Completed | 8 Years | 65 Years | All | 206 | Phase 3 | United States;Canada |
| 26 | 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 |
| 27 | NCT00789568 (ClinicalTrials.gov) | October 2008 | 10/11/2008 | A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects | A Phase 1, Randomized, Placebo- and Active-controlled Crossover Study to Evaluate the Effects of Sapropterin Dihydrochloride Oral Administration on QTc Intervals in Healthy Adult Subjects | Phenylketonuria | Drug: sapropterin dihydrochloride;Drug: Moxifloxacin;Drug: Moxifloxacin placebo | BioMarin Pharmaceutical | NULL | Completed | 18 Years | 50 Years | All | 56 | Phase 1 | United States |
| 28 | NCT00688844 (ClinicalTrials.gov) | October 2008 | 29/5/2008 | Nutritional and Neurotransmitter Changes in PKU Subjects on BH4 | Baseline Evaluation and Long-term Follow-up of Nutritional Status and Neurotransmitter Concentrations in Phenylketonuria Patients Initiating Treatment With Sapropterin Dihydrochloride (KuvanTM), a Tetrahydrobiopterin Analog. | Phenylketonuria | Drug: KuvanTM Therapy | Emory University | BioMarin Pharmaceutical;Atlanta Clinical and Translational Science Institute | Completed | 4 Years | N/A | All | 58 | N/A | United States |
| 29 | NCT00484991 (ClinicalTrials.gov) | February 2007 | 8/6/2007 | Sapropterin Expanded Access Program | Sapropterin Expanded Access Program | Phenylketonuria | Drug: Sapropterin dihydrochloride | BioMarin Pharmaceutical | NULL | Approved for marketing | 9 Years | N/A | Both | N/A | United States | |
| 30 | EUCTR2006-000839-10-ES (EUCTR) | 06/10/2006 | 13/07/2006 | A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006Estudio de extensión fase 3b, multicéntrico y abierto de PhenoptinTM en sujetos fenilcetonúricos que participaron en los estudios PKU-004 o PKU-006 | A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006Estudio de extensión fase 3b, multicéntrico y abierto de PhenoptinTM en sujetos fenilcetonúricos que participaron en los estudios PKU-004 o PKU-006 | Fenilcetonuria (PKU) MedDRA version: 8.1;Level: LLT;Classification code 10034872;Term: Phenylketonuria | Product Name: Phenoptin Product Code: T1401 INN or Proposed INN: Sapropterin Other descriptive name: sapropterin dihydrochloride | BioMarin Pharmaceutical Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 128 | Phase 3 | United Kingdom;Germany;Ireland;Spain;Italy | ||
| 31 | EUCTR2006-000839-10-IE (EUCTR) | 02/09/2006 | 12/06/2006 | A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 - NA | A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 - NA | Phenylketonuria (PKU) MedDRA version: 8.1;Level: LLT;Classification code 10034872;Term: Phenylketonuria | 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 | 128 | Phase 3 | United Kingdom;Germany;Spain;Ireland;Italy | ||
| 32 | 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 | ||
| 33 | EUCTR2006-000839-10-GB (EUCTR) | 14/08/2006 | 20/09/2006 | A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 - NA | A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 - NA | Phenylketonuria (PKU) MedDRA version: 8.1;Level: LLT;Classification code 10034872;Term: Phenylketonuria | 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 | 128 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Spain;Ireland;Germany;Italy;United Kingdom | ||
| 34 | EUCTR2006-000839-10-DE (EUCTR) | 09/08/2006 | 26/05/2006 | A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 - NA | A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 - 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 | 128 | Phase 3 | United Kingdom;Germany;Spain;Ireland;Italy | ||
| 35 | NCT00332189 (ClinicalTrials.gov) | July 2006 | 30/5/2006 | Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 | A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 | Phenylketonuria | Drug: sapropterin dihydrochloride | BioMarin Pharmaceutical | NULL | Completed | 4 Years | N/A | All | 111 | Phase 3 | United States |
| 36 | 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 | ||
| 37 | 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 | ||
| 38 | 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 |
| 39 | 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 | ||
| 40 | 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 | ||
| 41 | 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 |
| 42 | 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 |
| 43 | 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 | |||
| 44 | EUCTR2021-003777-63-NL (EUCTR) | 24/11/2021 | BH4 (Kuvan©) treatment in Phenylketonuria - comparing different practices of dosing regimens | Tetrahydrobiopterin (BH4) treatment in Phenylketonuria - comparing different practices of dosing regimen - BH4 dose | Phenylketonuria (PKU; OMIM 261600);Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Kuvan® (sapropterin dihydrochloride) Product Name: Kuvan® (sapropterin dihydrochloride) Product Code: A16AX07 | University Medical Center Groningen | NULL | NA | Female: yes Male: yes | 28 | Phase 4 | Netherlands | |||
| 45 | 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 |