240. Phenylketonuria
125 clinical trials, 95 drugs (DrugBank: 11 drugs), 1 drug target gene, 5 drug target pathways
Searched query = "Phenylketonuria", "PKU", "Phenylalanine hydroxylase deficiency", "PAH deficiency", "Tetrahydrobiopterin deficiency", "BH4 deficiency", "BH4 reactive hyper pheemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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 | NCT04014712 (ClinicalTrials.gov) | August 2019 | 3/7/2019 | O2 Transport and Utilization in Health and Lung Disease | Role of Nitric Oxide Coupling in Muscle Dysfunction With COPD | COPD;Tetrahydrobiopterin Deficiency;Oxidative Stress | Drug: Tetrahydrobiopterin;Drug: Placebo oral tablet | University of Massachusetts, Amherst | NULL | Not yet recruiting | 18 Years | 85 Years | All | 30 | Phase 1 | United States |
| 2 | 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 | |
| 3 | NCT03519711 (ClinicalTrials.gov) | June 24, 2018 | 11/4/2018 | A Study of CNSA-001 in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia | A Phase 1/2, Open-Label, Randomized Parallel Arm, Intra-patient Dose Escalation Study to Evaluate the Safety, Pharmacokinetics and Preliminary Efficacy of CNSA-001(Sepiapterin) in Primary Tetrahydrobiopterin Deficient Patients With Hyperphenylalaninemia | BH4 Deficiency;Hyperphenylalaninemia | Drug: CNSA-001 | PTC Therapeutics | NULL | Active, not recruiting | 12 Months | N/A | All | 6 | Phase 1;Phase 2 | United States;Germany |
| 4 | NCT03864029 (ClinicalTrials.gov) | October 10, 2017 | 22/2/2019 | Retrospective Observational Safety Effectiveness With Kuvan in hpA | An Observational Study Research to Collect the Effectiveness and Safety Data of KUVAN® Retrospectively in Chinese Subjects With Hyperphenylalaninemia (HPA) Caused by Tetrahydrobiopterin (BH4) Deficiency | Tetrahydrobiopterin Deficiency | Drug: KUVAN | BioMarin Pharmaceutical | Quintiles, Inc. | Completed | N/A | N/A | All | 26 | China | |
| 5 | 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 2a | France;Spain;Belgium;Germany;Netherlands;Italy;Switzerland | ||
| 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 |
| 6 | 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 | |||
| 7 | 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 | Phenylketonuria | Drug: Sapropterin;Drug: Placebo | BioMarin Pharmaceutical | NULL | Terminated | 18 Years | 29 Years | All | 2 | Phase 2 | Italy;Switzerland;Belgium;Germany;Netherlands;Spain |
| 8 | 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 2a | France;Belgium;Spain;Netherlands;Germany;Italy;Switzerland | ||
| 9 | 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 2a | France;Spain;Belgium;Netherlands;Germany;Italy;Switzerland | ||
| 10 | 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 2a | France;Belgium;Spain;Netherlands;Germany;Italy;Switzerland | ||
| 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 |
| 11 | 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 | ||
| 12 | 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 |
| 13 | 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 2a | France;Belgium;Spain;Netherlands;Germany;Switzerland;Italy | ||
| 14 | 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] | BioMarin International Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 4 | Spain;Germany;Italy;United Kingdom | |||
| 15 | 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 | ||
| 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 |
| 16 | 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 | ||
| 17 | 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 |
| 18 | NCT01617070 (ClinicalTrials.gov) | May 2012 | 8/6/2012 | Effects of Kuvan on Melatonin Secretion | Pilot Study to Evaluate Melatonin Secretion as a Marker of Decreased Serotonin in Individuals With PKU: Evaluation of the CNS Effects of Tetrahydrobiopterin | Phenylketonuria (PKU) | Drug: Kuvan;Dietary Supplement: Large Neutral Amino Acid Therapy | University of Southern California | BioMarin Pharmaceutical | Completed | 18 Years | N/A | All | 10 | Phase 4 | United States |
| 19 | NCT01541397 (ClinicalTrials.gov) | June 2011 | 11/7/2011 | Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy | Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy | Hyperphenylalaninemia;Phenylketonuria | Drug: Sapropterin | The University of Texas Health Science Center, Houston | BioMarin Pharmaceutical | Terminated | 18 Years | 50 Years | All | 6 | N/A | United States |
| 20 | 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 |
| 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 |
| 21 | 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 |
| 22 | 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 | ||
| 23 | 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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 50 | Phase 3b | Portugal;Czech Republic;Germany;Slovakia;United Kingdom;Turkey;Netherlands;Belgium;Italy;Austria | ||
| 24 | 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 | ||
| 25 | 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 |
| 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 |
| 26 | 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 | ||
| 27 | 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 | ||
| 28 | 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 | ||
| 29 | 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 | ||
| 30 | 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] | 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 | |||
| 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 |
| 31 | 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 | |
| 32 | 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 | ||
| 33 | 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 |
| 34 | EUCTR2009-018168-81-DK (EUCTR) | 16/04/2010 | 03/03/2010 | ENDURE: A Phase IV, prospective, open-label, uncontrolled, multi-centre cohort trial to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 28 days - ENDURE | ENDURE: A Phase IV, prospective, open-label, uncontrolled, multi-centre cohort trial to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 28 days - ENDURE | Subjects diagnosed with Phenylketonuria (PKU) (classic or mild PKU, or mild hyperphenylalaninaemia (HPA)). MedDRA version: 14.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 10010331 - Congenital, familial and genetic disorders | Trade Name: Kuvan INN or Proposed INN: Sapropterindihydrochloride Other descriptive name: SAPROPTERIN HYDROCHLORIDE | Merck Serono Norway | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 4 | Denmark | ||
| 35 | NCT00986973 (ClinicalTrials.gov) | March 2010 | 28/9/2009 | Fluorodeoxyglucose Positron Emission Tomography (FDG PET) Findings in Patients With Phenylketonuria Before and After KUVAN Therapy | A Pilot Study of FDG PET Findings in Patients With Phenylketonuria Before and After BH4 Supplementation | Phenylketonuria | Drug: Sapropterin | Children's Hospital of Philadelphia | NULL | Completed | 18 Years | 50 Years | All | 6 | N/A | United States |
| 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 |
| 36 | NCT00964236 (ClinicalTrials.gov) | August 2009 | 21/8/2009 | The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU) | The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU) | Phenylketonuria | Drug: Sapropterin | University of Missouri-Columbia | BioMarin Pharmaceutical | Completed | 6 Years | N/A | All | 20 | United States | |
| 37 | 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 |
| 38 | 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 |
| 39 | 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 | BioMarin Pharmaceutical | NULL | Completed | 18 Years | 50 Years | Both | 56 | Phase 1 | United States |
| 40 | NCT00730080 (ClinicalTrials.gov) | July 2008 | 1/8/2008 | Sapropterin in Individuals With Phenylketonuria | Effects of Sapropterin on Brain and Cognition in Individuals With Phenylketonuria | Phenylketonuria | Drug: Sapropterin (Kuvan) | Washington University School of Medicine | BioMarin Pharmaceutical;University of Missouri-Columbia | Completed | 6 Years | 50 Years | All | 45 | United States | |
| 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 |
| 41 | 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 | |
| 42 | 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 |
| 43 | 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 3b | United Kingdom;Germany;Ireland;Spain;Italy | ||
| 44 | EUCTR2006-000839-10-IT (EUCTR) | 07/09/2006 | 02/08/2007 | A Phase 3b, Multicenter, Open?Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Studies PKU?004 or PKU?006 - ND | A Phase 3b, Multicenter, Open?Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Studies PKU?004 or PKU?006 - ND | phenylketonuria Level: PTClassification code 10034872 | Trade Name: Phenoptin INN or Proposed INN: Sapropterin | BIOMARIN | NULL | Not Recruiting | Female: yes Male: yes | 128 | Phase 3b | United Kingdom;Germany;Spain;Ireland;Italy | ||
| 45 | 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 3b | United Kingdom;Germany;Spain;Ireland;Italy | ||
| 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 |
| 46 | 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 3b | United Kingdom;Germany;Spain;Ireland;Italy | ||
| 47 | NCT00355264 (ClinicalTrials.gov) | August 2006 | 19/7/2006 | Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency | Phase 2, Multicenter, Open Label Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to Primary BH4 Deficiency | Tetrahydrobiopterin Deficiencies;Hyperphenylalaninemia, Non-Phenylketonuric | Drug: Phenoptin | BioMarin Pharmaceutical | NULL | Completed | N/A | N/A | All | 12 | Phase 2 | United States;Germany |
| 48 | EUCTR2005-003778-13-DE (EUCTR) | 13/07/2006 | 14/06/2006 | A Phase 2, Multicenter, Open-label Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Hyperphenylalaninemia Due to Primary BH4 Deficiency | A Phase 2, Multicenter, Open-label Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Hyperphenylalaninemia Due to Primary BH4 Deficiency | 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: saproterin dihydrochloride | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 2 | Germany | ||
| 49 | 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 |
| 50 | 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 | |||
| 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 |
| 51 | 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 | ||
| 52 | 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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 300 | Germany;Spain | |||
| 53 | 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 |
| 54 | 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 | ||
| 55 | NCT00244218 (ClinicalTrials.gov) | April 2005 | 25/10/2005 | Response to Phenylketonuria to Tetrahydrobiopterin (BH4) | Response to Phenylketonuria to Tetrahydrobiopterin (BH4) | Phenylketonuria | Drug: tetrahydrobiopterin (BH4) | FDA Office of Orphan Products Development | NULL | Recruiting | 10 Years | N/A | Both | 36 | Phase 1 | United States |
| 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 |
| 56 | 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 |
| 57 | 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 | |||
| 58 | 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 |
| 59 | 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 | ||
| 60 | 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 |
| 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 |
| 61 | 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 | ||||
| 62 | 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 | ||||
| 63 | 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 | |||
| 64 | 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 | |||
| 65 | EUCTR2009-018168-81-NO (EUCTR) | 10/03/2010 | ENDURE: A Phase IV, prospective, open-label, uncontrolled, multi-centre cohort trial to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 28 days - ENDURE | ENDURE: A Phase IV, prospective, open-label, uncontrolled, multi-centre cohort trial to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 28 days - ENDURE | Subjects diagnosed with Phenylketonuria (PKU) (classic or mild PKU, or mild hyperphenylalaninaemia (HPA)). MedDRA version: 12.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU) | Trade Name: Kuvan INN or Proposed INN: Sapropterindihydrochloride Other descriptive name: SAPROPTERIN HYDROCHLORIDE | Merck Serono Norway | NULL | NA | Female: yes Male: yes | 150 | Phase 4 | Denmark;Norway |