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 | EUCTR2014-000350-11-DE (EUCTR) | 28/05/2014 | 10/03/2014 | Treatment of patients, who have Mucopolysaccharidosis Type I, receiving pentosan polysulfate subcutaneous injections weekly | An open label, randomized study to investigate the safety of weekly pentosan polysulfate injections in adult patients with Mucopolysaccharidosis Type I receiving enzyme replacement therapy. | Mucopolysaccharidosis type I (alpha-L-Iduronidase deficiency) MedDRA version: 17.0;Level: LLT;Classification code 10056888;Term: Mucopolysaccharidosis IS;System Organ Class: 100000004850 MedDRA version: 17.0;Classification code 10056887;Term: Mucopolysaccharidosis IH/S;Classification code 10028094;Term: Mucopolysaccharidosis IH;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Pentosan Polysulfat SP 54® injection solution Product Name: Pentosan Polysulphate SP54® INN or Proposed INN: PENTOSAN POLYSULFATE SODIUM Other descriptive name: Pentosan Polysulphate SP54 | Multiplex Pharma Holdings LLC | NULL | Not Recruiting | Female: yes Male: yes | 6 | Germany |