Nitisinone ( DrugBank: Nitisinone )


3 diseases
IDDisease name (Link within this page)Number of trials
6Parkinson disease2
164Oculocutaneous albinism1
241Hypertyrosinemia type I12

6. Parkinson disease


Clinical trials : 2,307 Drugs : 2,007 - (DrugBank : 349) / Drug target genes : 188 - Drug target pathways : 199
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2009-013670-41-CZ
(EUCTR)
18/11/200907/09/2009A Multi-center, Double-blind, Randomized, Placebo-controlled, Parallel-group, Two-arm Safety and Efficacy Study of SYN118 as Adjunctive Therapy in Subjects with Parkinson?s DiseaseA Multi-center, Double-blind, Randomized, Placebo-controlled, Parallel-group, Two-arm Safety and Efficacy Study of SYN118 as Adjunctive Therapy in Subjects with Parkinson?s Disease Parkinson´s disease inadequately controlled on a stable regimen of anti-Parkinsonian medications.
MedDRA version: 12.0;Level: LLT;Classification code 10013113;Term: Disease Parkinson's
Trade Name: Orfadin
INN or Proposed INN: NITISINONE
Synosia Therapeutics AGNULLNot RecruitingFemale: yes
Male: yes
126Czech Republic;France
2EUCTR2009-013670-41-FR
(EUCTR)
16/10/200911/09/2009A Multi-center, Double-blind, Randomized, Placebo-controlled, Parallel-group, Two-arm Safety and Efficacy Study of SYN118 as Adjunctive Therapy in Subjects with Parkinson?s DiseaseA Multi-center, Double-blind, Randomized, Placebo-controlled, Parallel-group, Two-arm Safety and Efficacy Study of SYN118 as Adjunctive Therapy in Subjects with Parkinson?s Disease Parkinson´s disease inadequately controlled on a stable regimen of anti-Parkinsonian medications.
MedDRA version: 12.0;Level: LLT;Classification code 10013113;Term: Disease Parkinson's
Trade Name: Orfadin
INN or Proposed INN: NITISINONE
Synosia Therapeutics AGNULLNot RecruitingFemale: yes
Male: yes
126Phase 2France;Czech Republic

164. Oculocutaneous albinism


Clinical trials : 15 Drugs : 54 - (DrugBank : 34) / Drug target genes : 35 - Drug target pathways : 141
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT01838655
(ClinicalTrials.gov)
April 16, 201320/4/2013Nitisinone for Type 1B Oculocutaneous AlbinismA Pilot Study of Nitisinone in the Treatment of Oculocutaneous Albinism, Type 1BAlbinism;Vision LossDrug: NitisinoneNational Eye Institute (NEI)National Human Genome Research Institute (NHGRI)Completed18 YearsN/AAll5Phase 1/Phase 2United States

241. Hypertyrosinemia type I


Clinical trials : 14 Drugs : 7 - (DrugBank : 1) / 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
1NCT04113772
(ClinicalTrials.gov)
November 1, 20191/10/2019Bio Equivalency 20 Mgm Orfadin and 20 Mgm of NitisonineOrfadin and Nitinosine StudyHereditary Tyrosinemia, Type IDrug: Nitisinone;Drug: OrfadinSutphin DrugsNULLNot yet recruiting18 Years50 YearsAll4N/AIndia
2NCT02750345
(ClinicalTrials.gov)
March 201621/4/2016Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1)A Three-Period Crossover Study to Determine the Bioequivalence of Two Oral Formulations Containing Nitisinone 10 mg Compared to Reference Formulation Orfadin In Healthy Subjects Under Fasting ConditionsHereditary Tyrosinemia, Type IDrug: Nitisinone;Drug: Nitisinone Baked Tablet;Drug: OrfadinCycle Pharmaceuticals Ltd.ParexelCompleted18 Years55 YearsAll24Phase 1South Africa
3NCT02750332
(ClinicalTrials.gov)
November 201521/4/2016Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1)A Single Center, Single-Dose, Open-Label, Randomized Study to Compare the Bioavailability of an Oral Test Formulation Containing Nitisinone 10 mg in at Least 16 Healthy Male and Female Subjects Under Fasting and Fed ConditionsHereditary Tyrosinemia, Type IDrug: NitisinoneCycle Pharmaceuticals Ltd.ParexelCompleted18 Years55 YearsAll20Phase 1South Africa
4NCT02750709
(ClinicalTrials.gov)
October 201520/4/2016Bioequivalence Study of Two Nitisinone Formulations Compared to OrfadinA Single Center, Single-Dose, Open-Label, Laboratory-Blind, Randomized, Three-Period Crossover Study to Determine the Bioequivalence of Two Oral Formulations Containing of Nitisinone 10 mg Compared to the Reference Formulation Orfadin 10 mg in at Least 18 Healthy Male and Female Subjects Under Fasting ConditionsHereditary Tyrosinemia, Type IDrug: Nitisinone;Drug: Nitisinone 10 mg Tablet High Compritol;Drug: OrfadinCycle Pharmaceuticals Ltd.ParexelCompleted18 Years55 YearsAll24Phase 1South Africa
5EUCTR2013-004132-29-DK
(EUCTR)
16/12/201425/08/2014An open-label, non-randomized, sequential, multicenter study to evaluate the pharmacokinetics, efficacy and safety of once daily dosing compared to twice daily dosing of Orfadin in patients diagnosed with hereditary tyrosinemia type 1An open-label, non-randomized, sequential, multicenter study to evaluate the pharmacokinetics, efficacy and safety of once daily dosing compared to twice daily dosing of Orfadin in patients diagnosed with hereditary tyrosinemia type 1 hereditary tyrosinemia type 1
MedDRA version: 17.1;Level: LLT;Classification code 10069462;Term: Tyrosinemia type I;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Trade Name: Orfadin
INN or Proposed INN: NITISINONE
Swedish Orphan Biovitrum AB (Publ)NULLNot RecruitingFemale: yes
Male: yes
20Belgium;Denmark;Sweden
6NCT02323529
(ClinicalTrials.gov)
December 201418/12/2014Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1Open-label, Multicentre, Multiple-dose Trial to Evaluate Pharmacokinetics, Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Orfadin in Patients Diagnosed With Hereditary Tyrosinemia Type 1Hereditary Tyrosinemia, Type IDrug: NitisinoneSwedish Orphan BiovitrumNULLCompletedN/AN/ABoth18Phase 3Belgium;Denmark;France;Germany;Sweden
7EUCTR2013-004132-29-BE
(EUCTR)
06/11/201415/10/2014An open-label, non-randomized, sequential, multicenter study to evaluate the pharmacokinetics, efficacy and safety of once daily dosing compared to twice daily dosing of Orfadin in patients diagnosed with hereditary tyrosinemia type 1An open-label, non-randomized, sequential, multicenter study to evaluate the pharmacokinetics, efficacy and safety of once daily dosing compared to twice daily dosing of Orfadin in patients diagnosed with hereditary tyrosinemia type 1 hereditary tyrosinemia type 1
MedDRA version: 17.0;Level: LLT;Classification code 10069462;Term: Tyrosinemia type I;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Trade Name: Orfadin
INN or Proposed INN: NITISINONE
Trade Name: Orfadin
INN or Proposed INN: NITISINONE
Trade Name: Orfadin
INN or Proposed INN: NITISINONE
Swedish Orphan Biovitrum AB (Publ)NULLNot RecruitingFemale: yes
Male: yes
20Phase 4Belgium;Denmark;Sweden
8EUCTR2013-004132-29-SE
(EUCTR)
15/10/201418/08/2014An open-label, non-randomized, sequential, multicenter study to evaluate the pharmacokinetics, efficacy and safety of once daily dosing compared to twice daily dosing of Orfadin in patients diagnosed with hereditary tyrosinemia type 1An open-label, non-randomized, sequential, multicenter study to evaluate the pharmacokinetics, efficacy and safety of once daily dosing compared to twice daily dosing of Orfadin in patients diagnosed with hereditary tyrosinemia type 1 hereditary tyrosinemia type 1
MedDRA version: 17.1;Level: LLT;Classification code 10069462;Term: Tyrosinemia type I;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Trade Name: Orfadin
INN or Proposed INN: NITISINONE
Swedish Orphan Biovitrum AB (Publ)NULLNot RecruitingFemale: yes
Male: yes
20Belgium;Denmark;Sweden
9NCT02320084
(ClinicalTrials.gov)
September 201323/10/2014Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical CareA Non-interventional Post Authorization Study (PASS) to Evaluate Long-term Safety of Orfadin Treatment in Hypertyrosinemia Type 1 (HT-1) Patients in Standard CareHereditary Tyrosinemia, Type IDrug: NitisinoneSwedish Orphan BiovitrumNULLCompletedN/AN/AAll315Austria;Belgium;Czechia;Denmark;Finland;France;Germany;Hungary;Ireland;Italy;Netherlands;Norway;Poland;Portugal;Spain;Sweden;United Kingdom;Croatia;Czech Republic;Greece;Kosovo;Lithuania;Macedonia, The Former Yugoslav Republic of;Romania;Slovakia;Switzerland
10EUCTR2012-002286-36-DE
(EUCTR)
19/11/201203/08/2012Taste and palatability of Orfadin suspension.Taste and palatability of Orfadin suspension. An open, non-controlled 3-day study in pediatric patients with hereditary tyrosinemia type 1 treated with Orfadin. Hereditary tyrosinemia type 1 (HT-1)
MedDRA version: 14.1;Level: LLT;Classification code 10069459;Term: Tyrosinaemia type I;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: Orfadin oral suspension 4mg/ml
INN or Proposed INN: Nitisinone
Swedish Orphan Biovitrum ABNULLNot RecruitingFemale: yes
Male: yes
18Germany;United Kingdom
11EUCTR2012-002286-36-GB
(EUCTR)
04/10/201210/08/2012Taste and palatability of Orfadin suspension.Taste and palatability of Orfadin suspension. An open, non-controlled 3-day study in pediatric patients with hereditary tyrosinemia type 1 treated with Orfadin. Hereditary tyrosinemia type 1 (HT-1)
MedDRA version: 14.1;Level: LLT;Classification code 10069459;Term: Tyrosinaemia type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: Orfadin oral suspension 4mg/ml
INN or Proposed INN: NITISINONE
Swedish Orphan Biovitrum ABNULLNot RecruitingFemale: yes
Male: yes
18Germany;United Kingdom
12NCT01734889
(ClinicalTrials.gov)
October 201222/11/2012Taste and Palatability of Orfadin SuspensionTaste and Palatability of Orfadin Suspension. An Open, Non-controlled 3 Day Study in Pediatric Patients With Hereditary Tyrosinemia Type 1 Treated With Orfadin.Hereditary Tyrosinemia, Type IDrug: NitisinoneSwedish Orphan BiovitrumNULLCompleted1 Month17 YearsAll18Phase 1France;Germany;United Kingdom