Phenoptin (DrugBank: -)
2 diseases| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 124 | Cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy | 1 |
| 240 | Phenylketonuria | 26 |
| 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 controlled trial - CADASIL BH4 | 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 | 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 |
| 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 | 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 | ||
| 2 | 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 | ||
| 3 | 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 | ||
| 4 | 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 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 300 | Phase 3 | Spain;Germany;United Kingdom | |||
| 5 | 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 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 128 | Phase 3 | Spain;Ireland;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 |
| 6 | 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 | ||
| 7 | 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 |
| 8 | 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 | ||
| 9 | 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 |
| 10 | 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 | ||
| 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 | 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 | |||
| 12 | 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 |
| 13 | 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 | ||
| 14 | 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 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | Ireland;Germany;Italy;United Kingdom | |||
| 15 | EUCTR2004-004513-41-IT (EUCTR) | 23/09/2005 | 11/11/2005 | A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels | A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels | Treatment of phenylketonuria MedDRA version: 6.1;Level: SOC;Classification code 10027433 | Product Name: Phenoptin | BIOMARIN | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | Germany;United Kingdom;Ireland;Italy | ||
| 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 | EUCTR2004-004513-41-IE (EUCTR) | 19/08/2005 | 10/06/2005 | A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels | A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels | Phenylketonuria (PKU) MedDRA version: 6.1;Classification code 10034872 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | Ireland;Germany;Italy;United Kingdom | |||
| 17 | EUCTR2004-004512-23-GB (EUCTR) | 15/08/2005 | 23/02/2005 | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA | Phenylketonuria (PKU) MedDRA version: 6.1;Classification code 10034872 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | Ireland;Germany;Italy;United Kingdom | |||
| 18 | EUCTR2004-004512-23-IT (EUCTR) | 09/06/2005 | 22/06/2005 | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels | Treatment of phenylketonuria MedDRA version: 6.1;Level: SOC;Classification code 10027433 | Product Name: Phenoptin | BIOMARIN | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | Germany;United Kingdom;Ireland;Italy | ||
| 19 | EUCTR2004-004512-23-IE (EUCTR) | 22/05/2005 | 15/02/2005 | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA | Phenylketonuria (PKU) MedDRA version: 6.1;Classification code 10034872 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | Ireland;Germany;Italy;United Kingdom | |||
| 20 | EUCTR2004-002071-16-GB (EUCTR) | 18/03/2005 | 22/02/2005 | A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA | A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA | Phenylketonuria (PKU) MedDRA version: 6.1;Classification code 10034872 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 700 | Phase 2 | Ireland;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 |
| 21 | 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 | |||
| 22 | 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 |
| 23 | 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 | ||
| 24 | 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 |
| 25 | EUCTR2004-002071-16-IE (EUCTR) | 13/10/2004 | A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA | A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects with Phenylketonuria Who Have Elevated Phenylalanine Levels - NA | Phenylketonuria (PKU) MedDRA version: 6.1;Classification code 10034872 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 400 | Phase 2 | Ireland;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 |
| 26 | 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 |