Recombinant human tripeptidyl peptidase-1 (DrugBank: -)
1 disease| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 19 | ライソゾーム病 | 5 |
| 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 | Active, not recruiting | N/A | 17 Years | All | 14 | Phase 2 | 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-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 | |||
| 4 | EUCTR2012-005430-11-IT (EUCTR) | 14/05/2014 | 20/01/2014 | Phase 1/2 study to evaluate safety and efficacy of BMN 190 in patients with CLN2 | A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients with Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease | Neuronal Ceroid Lipofuscinosis Type 2 (CLN2). MedDRA version: 16.1;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: N/A Product Code: BMN 190 INN or Proposed INN: TBD Other descriptive name: recombinant human tripeptidyl peptidase-1 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 22 | Phase 1;Phase 2 | Germany;United Kingdom;Italy | ||
| 5 | EUCTR2012-005430-11-DE (EUCTR) | 11/09/2013 | 10/04/2013 | Phase 1/2 study to evaluate safety and efficacy of BMN 190 in patients with CLN2 | A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients with Late-Infantile Neuronal Ceroid Lipofuscinosis (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 Name: N/A Product Code: BMN 190 INN or Proposed INN: TBD Other descriptive name: recombinant human tripeptidyl peptidase-1 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 22 | Phase 1;Phase 2 | Germany;United Kingdom |