19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 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 | EUCTR2017-002806-10-NL (EUCTR) | 29/11/2019 | 08/11/2018 | An open, non-controlled, parallel, ascending multiple-dose, multicenter study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of SOBI003 in pediatric MPS IIIA patients | An open, non-controlled, parallel, ascending multiple-dose, multicenter study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of SOBI003 in pediatric MPS IIIA patients | Mucopolysaccharidosis Type IIIA or Sanfilippo Syndrome MedDRA version: 20.1;Level: LLT;Classification code 10028094;Term: Mucopolysaccharidosis IH;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Chemically modified recombinant human sulfamidase Product Code: SOBI003 INN or Proposed INN: not applicable Other descriptive name: CHEMICALLY MODIFIED RECOMBINANT HUMAN SULFAMIDASE | Swedish Orphan Biovitrum AB (publ) | NULL | Not Recruiting | Female: yes Male: yes | 9 | Phase 1 | United States;Turkey;Germany;Netherlands | ||
| 2 | NCT03811028 (ClinicalTrials.gov) | January 19, 2019 | 17/12/2018 | A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients | An Open, Single-arm, Multicenter Extension Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients | Sanfilippo Syndrome Type A (MPS IIIA) | Drug: SOBI003 | Swedish Orphan Biovitrum | NULL | Completed | 18 Months | 78 Months | All | 6 | Phase 1/Phase 2 | United States;Turkey |
| 3 | NCT03423186 (ClinicalTrials.gov) | June 19, 2018 | 29/1/2018 | A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients | An Open, Non-controlled, Parallel, Ascending Multiple-dose, Multicenter Study to Assess Safety and Tolerability, Pharmacokinetics and Pharmacodynamics of SOBI003 in Pediatric MPS IIIA Patients | Sanfilippo Syndrome Type A (MPS IIIA) | Drug: SOBI003 | Swedish Orphan Biovitrum | NULL | Completed | 12 Months | 72 Months | All | 6 | Phase 1/Phase 2 | United States;Germany;Turkey |
| 4 | EUCTR2017-002806-10-DE (EUCTR) | 01/10/2018 | An open, non-controlled, parallel, ascending multiple-dose, multicenter study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of SOBI003 in pediatric MPS IIIA patients | An open, non-controlled, parallel, ascending multiple-dose, multicenter study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of SOBI003 in pediatric MPS IIIA patients | Mucopolysaccharidosis Type IIIA or Sanfilippo Syndrome MedDRA version: 20.1;Level: LLT;Classification code 10028094;Term: Mucopolysaccharidosis IH;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Chemically modified recombinant human sulfamidase Product Code: SOBI003 Other descriptive name: CHEMICALLY MODIFIED RECOMBINANT HUMAN SULFAMIDASE | Swedish Orphan Biovitrum AB (publ) | NULL | Not Recruiting | Female: yes Male: yes | 9 | Phase 1 | United States;Turkey;Netherlands;Germany |