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 | EUCTR2021-005356-10-NL (EUCTR) | 18/01/2022 | A pivotal study of N-Acetyl-L-Leucine on Niemann-Pick disease type C | Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study | Niemann-Pick Disease type C (NPC) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N-acetyl-L-leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L Leucine Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID | IntraBio Ltd | NULL | NA | Female: yes Male: yes | 53 | Phase 3 | United States;Czechia;Slovakia;Spain;Australia;Germany;Netherlands;United Kingdom;Switzerland |