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 | EUCTR2014-003198-40-DE (EUCTR) | 06/10/2015 | 07/08/2015 | Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of rhASM in Pediatric Patients <18 Years of Age with Acid Sphingomyelinase Deficiency | A Phase 1/2, Multi-Center, Open-Label, Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Exploratory Efficacy of Recombinant Human Acid Sphingomyelinase in Pediatric Patients Aged <18 Years With Acid Sphingomyelinase DeficiencyRevised title further to the protocol amendment 1A phase 1/2, multi-center, open-label, ascending dose study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and exploratory efficacy of olipudase afa in pediatric patients aged <18 years with acid sphingomyelinase deficiency | Patients with acid sphingomyelinase deficiency (Niemann-Pick disease) MedDRA version: 20.1;Level: LLT;Classification code 10041515;Term: Sphingomyelin lipidosis;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Olipudase alfa (rhASM) Product Code: GZ402665 INN or Proposed INN: Olipudase alfa Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM) | Genzyme Corporation | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | United States;France;Brazil;Germany;United Kingdom;Italy | ||
| 2 | EUCTR2014-003198-40-GB (EUCTR) | 19/02/2015 | 26/11/2014 | Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age with Acid Sphingomyelinase Deficiency | A phase 1/2, multi-center, open-label, ascending dose study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and exploratory efficacy of olipudase alfa in pediatric patients Aged <18 Years With acid sphingomyelinase deficiency - ASCEND-Peds | Patients with acid sphingomyelinase deficiency (Niemann-Pick disease) MedDRA version: 20.1;Level: LLT;Classification code 10041515;Term: Sphingomyelin lipidosis;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Olipudase alfa (rhASM) Product Code: GZ402665 INN or Proposed INN: Olipudase alfa Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM) | Genzyme Corporation | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | United States;France;Brazil;Germany;Italy;United Kingdom |