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 | NCT02678689 (ClinicalTrials.gov) | February 2016 | 15/1/2016 | A Safety, Tolerability, and Efficacy Study of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease | 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 | Jansky-Bielschowsky Disease;Batten Disease;Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2;CLN2 Disease;CLN2 Disorder | Biological: BMN190 recombinant human tripeptidyl peptidase-1 (rhTPP1);Device: Intraventricular access device | BioMarin Pharmaceutical | NULL | Completed | 0 Years | 17 Years | All | 14 | Phase 2 | United States;Germany;Italy;United Kingdom |
| 2 | EUCTR2014-003480-37-IT (EUCTR) | 06/05/2015 | 07/01/2015 | 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 (CLN2). MedDRA version: 17.1;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: BMN 190 INN or Proposed INN: TBD Other descriptive name: RECOMBINANT HUMAN TRIPEPTIDYL PEPTIDASE-1 (RHTPP1) | BioMarin Pharmaceutical Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 22 | United States;Germany;United Kingdom;Japan;Italy | |||
| 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 |