19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 854 / Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191
| 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 | EUCTR2017-002158-35-PT (EUCTR) | 14/01/2019 | 23/07/2018 | Study 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 VI | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | France;Portugal;Germany;United Kingdom | ||
| 2 | EUCTR2017-002158-35-DE (EUCTR) | 03/12/2018 | 16/08/2017 | Study 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 VI | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | Portugal;France;Germany;United Kingdom | ||
| 3 | NCT03370653 (ClinicalTrials.gov) | December 30, 2017 | 23/11/2017 | A Study in MPS VI to Assess Safety and Efficacy of Odiparcil | A Phase IIa Study to Investigate Safety, Pharmacokinetics, and Efficacy of Odiparcil in Patients 16 Years and Above With Mucopolysaccharidosis (MPS) Type VI | Mucopolysaccharidosis VI | Drug: Odiparcil;Other: Placebo | Inventiva Pharma | NULL | Completed | 16 Years | N/A | All | 20 | Phase 2 | France;Germany;Portugal;United Kingdom |
| 4 | EUCTR2017-002158-35-GB (EUCTR) | 16/11/2017 | 08/08/2017 | Study 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 VI | A 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. | NULL | Not 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): no | Portugal;France;Germany;United Kingdom | ||
| 5 | EUCTR2017-002158-35-FR (EUCTR) | 07/03/2018 | Study 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 VI | A 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. | NULL | Not 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): no | Portugal;France;Germany;United Kingdom |