Sapropterin ( DrugBank: Sapropterin )
3 diseases
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 86 | 肺動脈性肺高血圧症 | 1 |
| 124 | 皮質下梗塞と白質脳症を伴う常染色体優性脳動脈症 | 1 |
| 240 | フェニルケトン尿症 | 65 |
86. 肺動脈性肺高血圧症
臨床試験数 : 1,181 / 薬物数 : 701 - (DrugBank : 126) / 標的遺伝子数 : 105 - 標的パスウェイ数 : 192
Showing 1 to 1 of 1 diseases
| 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 | NCT00435331 (ClinicalTrials.gov) | March 2008 | 12/2/2007 | 6R-BH4 Pulmonary Arterial Hypertension Study | A Phase 1, Multicenter, Open-label, Dose-escalation Study to Evaluate the Safety and Efficacy of 6R-BH4 in Subjects With Pulmonary Arterial Hypertension A Phase 1, Multicenter, Open-label, Dose-escalation Study to Evaluate the Safety and Efficacy of 6R- ... | Pulmonary Arterial Hypertension | Drug: sapropterin dihydrochloride (6R-BH4) | Vanderbilt University | National Institutes of Health (NIH);BioMarin Pharmaceutical | Completed | 18 Years | N/A | Both | 14 | Phase 1 | United States |
124. 皮質下梗塞と白質脳症を伴う常染色体優性脳動脈症
臨床試験数 : 12 / 薬物数 : 14 - (DrugBank : 5) / 標的遺伝子数 : 6 - 標的パスウェイ数 : 23
Showing 1 to 1 of 1 diseases
| 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 | EUCTR2007-004370-55-IT (EUCTR) | 14/12/2007 | 11/12/2007 | Effects of tetrahydrobiopterin (6R-BH4) on flow-mediated dilation in CADASIL patients: a randomised controlled trial - CADASIL BH4 Effects of tetrahydrobiopterin (6R-BH4) on flow-mediated dilation in CADASIL patients: a randomised ... | Effects of tetrahydrobiopterin (6R-BH4) on flow-mediated dilation in CADASIL patients: a randomised controlled trial - CADASIL BH4 Effects of tetrahydrobiopterin (6R-BH4) on flow-mediated dilation in CADASIL patients: a randomised ... | CADASIL (Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy), is a rare autosomal dominant disorder characterized by recurrent strokes starting in mid-adulthood and leading in some to severe motor disability with pseudobulbar palsy and dementia of the subcortical type MedDRA version: 9.1;Level: HLGT;Classification code 10008804;Term: Chromosomal abnormalities and abnormal gene carriers CADASIL (Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy) ... | Product Name: Phenoptin Product Code: 6R-BH4 INN or Proposed INN: sapropterin | AZIENDA OSPEDALIERA OSPEDALE NIGUARDA CA' GRANDA (A.O. DI RILIEVO NAZIONALE) | NULL | Not Recruiting | Female: yes Male: yes | Italy |
240. フェニルケトン尿症
臨床試験数 : 138 / 薬物数 : 106 - (DrugBank : 11) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 5
Showing 1 to 10 of 65 diseases
| 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 | NCT03820804 (ClinicalTrials.gov) | January 7, 2019 | 19/10/2018 | Nutritional Status in Phenylketonuria | Trends in Nutritional Status of Patients With Phenylketonuria | Phenylketonurias | Other: Diet;Drug: Sapropterin | Universidade do Porto | Centro Hospitalar do Porto;BioMarin Pharmaceutical | Completed | 3 Years | N/A | All | 94 | Portugal | |
| 2 | 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 clinical study, carried out in different centers and using a placebo, to assess the effect of a me ... | 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) A Multicenter, Double-Blind, Placebo Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing t ... | 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] Phenylketonuria MedDRA version: 16.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria ( ... | Trade Name: Kuvan® INN or Proposed INN: Sapropterin Other descriptive name: SAPROPTERIN DIHYDROCHLORIDE Trade Name: Kuvan® INN or Proposed INN: Sapropterin Other descriptive name: SAPROPTERINDIHYDROCHLORIDE ... | Merck KGaA | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | France;Spain;Belgium;Germany;Netherlands;Italy;Switzerland | ||
| 3 | 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 Effect of sapropterinon variations of blood phenylalanine and tyrosine over 24 hours and from day to ... | 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] Phenylketonuria (PKU) is an autosomal recessive metabolic genetic disorder by a mutation in the gene ... | Trade Name: Kuvan | University Medical Center Groningen | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Phase 4 | Netherlands | |||
| 4 | NCT01977820 (ClinicalTrials.gov) | February 2014 | 31/10/2013 | Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria | 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 A Multicenter, Double-Blind, Placebo-Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing t ... | Phenylketonuria | Drug: Sapropterin;Drug: Placebo | BioMarin Pharmaceutical | NULL | Terminated | 18 Years | 29 Years | All | 2 | Phase 2 | Italy;Switzerland;Belgium;Germany;Netherlands;Spain |
| 5 | 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 clinical study, carried out in different centers and using a placebo, to assess the effect of a me ... | 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) A Multicenter, Double-Blind, Placebo Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing t ... | 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] Phenylketonuria MedDRA version: 16.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria ( ... | Trade Name: Kuvan® INN or Proposed INN: Sapropterin Other descriptive name: SAPROPTERIN DIHYDROCHLORIDE Trade Name: Kuvan® INN or Proposed INN: Sapropterin Other descriptive name: SAPROPTERINDIHYDROCHLORIDE ... | Merck KGaA | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | France;Belgium;Spain;Netherlands;Germany;Italy;Switzerland | ||
| 6 | 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 clinical study, carried out in different centers and using a placebo, to assess the effect of a me ... | 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) A Multicenter, Double-Blind, Placebo Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing t ... | 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] Phenylketonuria MedDRA version: 16.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria ( ... | Trade Name: Kuvan® INN or Proposed INN: Sapropterin Other descriptive name: SAPROPTERIN DIHYDROCHLORIDE Trade Name: Kuvan® INN or Proposed INN: Sapropterin Other descriptive name: SAPROPTERINDIHYDROCHLORIDE ... | Merck KGaA | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | France;Spain;Belgium;Netherlands;Germany;Italy;Switzerland | ||
| 7 | 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 clinical study, carried out in different centers and using a placebo, to assess the effect of a me ... | 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) A Multicenter, Double-Blind, Placebo Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing t ... | 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] Phenylketonuria MedDRA version: 16.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria ( ... | Trade Name: Kuvan® INN or Proposed INN: Sapropterin Other descriptive name: SAPROPTERIN DIHYDROCHLORIDE Trade Name: Kuvan® INN or Proposed INN: Sapropterin Other descriptive name: SAPROPTERINDIHYDROCHLORIDE ... | Merck KGaA | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | France;Belgium;Spain;Netherlands;Germany;Italy;Switzerland | ||
| 8 | 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 Study of the Long-term Effects on Mental Abilities of 4 to 5 Year-old Children with a Disease Caus ... | 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) A Phase IV Open-Label, Single-Cohort Study of the Long-Term Neurocognitive Outcomes in 4 to 5 Year-O ... | 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] Phenylketonuria MedDRA version: 16.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria ( ... | 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 | ||
| 9 | 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 A Phase 4 Open-Label, Single-Cohort Study of the Long-Term Neurocognitive Outcomes in 4 to 5 Year-Ol ... | Phenylketonuria | Drug: Kuvan® | BioMarin Pharmaceutical | NULL | Active, not recruiting | 4 Years | 5 Years | All | 34 | Phase 4 | Germany;Italy;Spain;United Kingdom |
| 10 | 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 clinical study, carried out in different centers and using a placebo, to assess the effect of a me ... | 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) A Multicenter, Double-Blind, Placebo Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing t ... | 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] Phenylketonuria MedDRA version: 14.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria ( ... | Trade Name: Kuvan® INN or Proposed INN: Sapropterin Other descriptive name: SAPROPTERIN DIHYDROCHLORIDE Trade Name: Kuvan® INN or Proposed INN: Sapropterin Other descriptive name: SAPROPTERINDIHYDROCHLORIDE ... | Merck KGaA | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | France;Belgium;Spain;Netherlands;Germany;Switzerland;Italy |