19. Lysosomal storage disease Clinical trials / Disease details


Clinical trials : 854 Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191

  
5 trials found
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
1EUCTR2017-002158-35-PT
(EUCTR)
14/01/201923/07/2018Study to investigate the fate of odiparcil since its administration up to the point is completely eliminated, its safety and efficacy in patients 16 years and above with mucopolysaccharidosis (MPS) type VIA phase IIa study to investigate safety, pharmacokinetics, and efficacy of odiparcil in patients 16 years and above with mucopolysaccharidosis (MPS) type VI. Mucopolysaccharidosis (MPS) type VI.
MedDRA version: 20.1;Level: PT;Classification code 10056892;Term: Mucopolysaccharidosis VI;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Product Name: Odiparcil
Product Code: IVA 336
INN or Proposed INN: Odiparcil
Inventiva S.A.NULLNot RecruitingFemale: yes
Male: yes
20Phase 2France;Portugal;Germany;United Kingdom
2EUCTR2017-002158-35-DE
(EUCTR)
03/12/201816/08/2017Study to investigate the fate of odiparcil since its administration up to the point is completely eliminated, its safety and efficacy in patients 16 years and above with mucopolysaccharidosis (MPS) type VIA phase IIa study to investigate safety, Pharmacokinetics, and efficacy of odiparcil in patients 16 years and above with mucopolysaccharidosis (MPS) type VI. Mucopolysaccharidosis (MPS) type VI.
MedDRA version: 20.1;Level: PT;Classification code 10056892;Term: Mucopolysaccharidosis VI;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Product Name: Odiparcil
Product Code: IVA 336
INN or Proposed INN: Odiparcil
Inventiva S.A.NULLNot RecruitingFemale: yes
Male: yes
20Phase 2Portugal;France;Germany;United Kingdom
3NCT03370653
(ClinicalTrials.gov)
December 30, 201723/11/2017A Study in MPS VI to Assess Safety and Efficacy of OdiparcilA Phase IIa Study to Investigate Safety, Pharmacokinetics, and Efficacy of Odiparcil in Patients 16 Years and Above With Mucopolysaccharidosis (MPS) Type VIMucopolysaccharidosis VIDrug: Odiparcil;Other: PlaceboInventiva PharmaNULLCompleted16 YearsN/AAll20Phase 2France;Germany;Portugal;United Kingdom
4EUCTR2017-002158-35-GB
(EUCTR)
16/11/201708/08/2017Study to investigate the fate of odiparcil since its administration up to the point is completely eliminated, its safety and efficacy in patients 16 years and above with mucopolysaccharidosis (MPS) type VIA phase IIa study to investigate safety, Pharmacokinetics, and efficacy of odiparcil in patients 16 years and above with mucopolysaccharidosis (MPS) type VI. Mucopolysaccharidosis (MPS) type VI.
MedDRA version: 20.1;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Product Name: Odiparcil
Product Code: IVA 336
INN or Proposed INN: Odiparcil
Inventiva S.A.NULLNot Recruiting Female: yes
Male: yes
20 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noPortugal;France;Germany;United Kingdom
5EUCTR2017-002158-35-FR
(EUCTR)
07/03/2018Study to investigate the fate of odiparcil since its administration up to the point is completely eliminated, its safety and efficacy in patients 16 years and above with mucopolysaccharidosis (MPS) type VIA phase IIa study to investigate safety, Pharmacokinetics, and efficacy of odiparcil in patients 16 years and above with mucopolysaccharidosis (MPS) type VI. - iMProveS Mucopolysaccharidosis (MPS) type VI.
MedDRA version: 20.1;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Product Name: Odiparcil
Product Code: IVA 336
INN or Proposed INN: Odiparcil
Inventiva S.A.NULLNot Recruiting Female: yes
Male: yes
24 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noPortugal;France;Germany;United Kingdom