19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182
| 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 | EUCTR2017-003083-13-ES (EUCTR) | 27/12/2017 | 27/11/2017 | Phase 2 study to evaluate Long-Term Safety and Efficacy of BMN 250 in Patients with MPS Type IIIB | A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Safety and Efficacy of Intracerebroventricular BMN 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) | Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPSIIIB) MedDRA version: 20.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N/A Product Code: BMN 250 INN or Proposed INN: Not available Other descriptive name: RHNAGLU-IGF2 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 33 | Phase 2 | Germany;Colombia;United Kingdom;United States;Taiwan;Argentina;Spain;Turkey;Australia | ||
| 2 | EUCTR2015-001985-25-ES (EUCTR) | 02/03/2016 | 13/01/2016 | Phase 1/2 Study to Evaluate the Safety and Efficacy of BMN 250 in Patients with MPS IIIB | A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) | Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPS IIIB) MedDRA version: 18.1;Level: LLT;Classification code 10056918;Term: Sanfilippo's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1;Level: PT;Classification code 10056890;Term: Mucopolysaccharidosis III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N/A Product Code: BMN 250 INN or Proposed INN: Not available Other descriptive name: RHNAGLU-IGF2 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 33 | Phase 1;Phase 2 | Taiwan;Brazil;Spain;Turkey;Australia;Germany;Colombia;United Kingdom |