T1401 ( DrugBank: - )


1 disease
IDDisease name (Link within this page)Number of trials
240Phenylketonuria17

240. Phenylketonuria


Clinical trials : 143 Drugs : 90 - (DrugBank : 10) / Drug target gene : 1 - Drug target pathways : 5
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2006-000839-10-ES
(EUCTR)
06/10/200613/07/2006A 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-006A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
128Phase 3United Kingdom;Germany;Ireland;Spain;Italy
2EUCTR2006-000839-10-IE
(EUCTR)
02/09/200612/06/2006A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 - NAA 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.NULLNot RecruitingFemale: yes
Male: yes
128Phase 3United Kingdom;Germany;Spain;Ireland;Italy
3EUCTR2005-003777-24-GB
(EUCTR)
17/08/200620/04/2006A 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 - NAA 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.NULLNot 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): noSpain;Germany;United Kingdom
4EUCTR2006-000839-10-GB
(EUCTR)
14/08/200620/09/2006A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 - NAA 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.NULLNot 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): noSpain;Ireland;Germany;Italy;United Kingdom
5EUCTR2006-000839-10-DE
(EUCTR)
09/08/200626/05/2006A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 - NAA 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.NULLNot RecruitingFemale: yes
Male: yes
128Phase 3United Kingdom;Germany;Spain;Ireland;Italy
6EUCTR2005-003778-13-DE
(EUCTR)
13/07/200614/06/2006A Phase 2, Multicenter, Open-label Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Hyperphenylalaninemia Due to Primary BH4 DeficiencyA 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 IncNULLNot RecruitingFemale: yes
Male: yes
15Phase 2Germany
7EUCTR2005-003777-24-DE
(EUCTR)
24/03/200614/12/2005A 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 - NAA 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.NULLNot RecruitingFemale: yes
Male: yes
300Phase 3Germany;Spain
8EUCTR2005-003777-24-ES
(EUCTR)
01/03/200619/01/2006Estudio 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.NULLNot RecruitingFemale: yes
Male: yes
300Phase 3Spain;Germany;United Kingdom
9EUCTR2004-004513-41-DE
(EUCTR)
27/10/200512/05/2006A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine LevelsA 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.NULLNot RecruitingFemale: yes
Male: yes
100Phase 3Ireland;Germany;Italy;United Kingdom
10EUCTR2004-004513-41-GB
(EUCTR)
25/10/200502/06/2005A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine LevelsA 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.NULLNot 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
11EUCTR2004-004513-41-IE
(EUCTR)
19/08/200510/06/2005A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Have Elevated Phenylalanine LevelsA 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 hydrochloride
BioMarin Pharmaceutical Inc.NULLNot 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
12EUCTR2004-004512-23-GB
(EUCTR)
15/08/200523/02/2005A 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 - NAA 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.NULLNot 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
13EUCTR2004-004512-23-IE
(EUCTR)
22/05/200515/02/2005A 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 - NAA 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.NULLNot 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
14EUCTR2004-002071-16-GB
(EUCTR)
18/03/200522/02/2005A 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 - NAA 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.NULLNot Recruiting Female: yes
Male: yes
700 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noIreland;Germany;Italy;United Kingdom
15EUCTR2004-002071-16-DE
(EUCTR)
28/01/200514/10/2004A 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 - NAA 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.NULLNot RecruitingFemale: yes
Male: yes
700Phase 2United Kingdom;Germany;Italy
16EUCTR2004-004512-23-DE
(EUCTR)
17/03/2005A 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 - NAA 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.NULLNot RecruitingFemale: yes
Male: yes
100Phase 3United Kingdom;Germany;Ireland;Italy
17EUCTR2004-002071-16-IE
(EUCTR)
13/10/2004A 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 - NAA 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.NULLNot Recruiting Female: yes
Male: yes
400 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noIreland;Germany;Italy;United Kingdom