19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 854 / 薬物数 : 716 - (DrugBank : 105) / 標的遺伝子数 : 70 - 標的パスウェイ数 : 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 | EUCTR2015-000891-85-GB (EUCTR) | 05/09/2017 | 06/03/2017 | Phase 2 study to evaluate safety and efficacy of BMN 190 in patients with CLN2 | A Phase 2, Open-Label, Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Intracerebroventricular BMN 190 in Pediatric Patients < 18 years of age with CLN2 Disease | Neuronal Ceroid Lipofuscinosis type 2 (CLN2) disease MedDRA version: 20.1;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: Brineura Product Name: cerliponase alfa Product Code: BMN 190 INN or Proposed INN: cerliponase alfa Other descriptive name: recombinant human tripeptidyl peptidase-1 | BioMarin Pharmaceutical Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 10 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Germany;Italy;United Kingdom | ||
| 2 | EUCTR2015-000891-85-DE (EUCTR) | 19/01/2016 | 04/11/2015 | Phase 2 study to evaluate safety and efficacy of BMN 190 in patients with CLN2 | A Phase 2 Open-Label Study to Evaluate Safety, Tolerability, and Efficacy of Intracerebroventricular BMN 190 in Pediatric Patients < 18 years of age with CLN2 Disease | Neuronal Ceroid Lipofuscinosis type 2 (CLN2) disease MedDRA version: 20.0;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000157084;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: Brineura Product Name: cerliponase alfa Product Code: BMN 190 INN or Proposed INN: cerliponase alfa Other descriptive name: recombinant human tripeptidyl peptidase-1 | BioMarin Pharmaceutical Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 10 | Phase 2 | Germany;Italy;United Kingdom | ||
| 3 | EUCTR2014-003480-37-GB (EUCTR) | 10/03/2015 | 20/10/2014 | An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 Disease | A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 Disease | Late-Infantile Neuronal Ceroid Lipofuscinosis type 2 (CLN2). MedDRA version: 20.1;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: Brineura Product Code: BMN 190 INN or Proposed INN: cerliponase alfa Other descriptive name: RECOMBINANT HUMAN TRIPEPTIDYL PEPTIDASE-1 (RHTPP1) | BioMarin Pharmaceutical Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 23 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Germany;Italy;United Kingdom | ||
| 4 | EUCTR2014-003480-37-DE (EUCTR) | 23/01/2015 | 03/11/2014 | An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 Disease | A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 Disease | Late-Infantile Neuronal Ceroid Lipofuscinosis type 2(CLN2). MedDRA version: 20.1;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: Brineura Product Code: BMN 190 INN or Proposed INN: cerliponase alfa Other descriptive name: RECOMBINANT HUMAN TRIPEPTIDYL PEPTIDASE-1 (RHTPP1) | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 23 | Phase 2 | United States;Germany;Italy;United Kingdom |