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

  
7 trials found
No.TrialIDDate_
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agemin
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PhaseCountries
1NCT05152914
(ClinicalTrials.gov)
November 1, 202129/11/2021Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2Intravitreal Enzyme Replacement Therapy to Prevent Retinal Disease Progression in Children With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)Neuronal Ceroid Lipofuscinosis Type 2Drug: Cerliponase AlfaDavid L Rogers, MDNULLEnrolling by invitation24 Months72 MonthsAll5Phase 1/Phase 2United States
2NCT04476862
(ClinicalTrials.gov)
August 19, 202015/7/2020Cerliponase Alfa Observational Study in the USCerliponase Alfa Observational StudyLate-Infantile Neuronal Ceroid Lipofuscinosis Type 2Drug: Cerliponase Alfa;Device: Administration KitBioMarin PharmaceuticalNULLRecruitingN/AN/AAll35United States
3EUCTR2015-000891-85-GB
(EUCTR)
05/09/201706/03/2017Phase 2 study to evaluate safety and efficacy of BMN 190 in patients with CLN2A 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 IncNULLAuthorised-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): noUnited States;Germany;Italy;United Kingdom
4EUCTR2015-000891-85-DE
(EUCTR)
19/01/201604/11/2015Phase 2 study to evaluate safety and efficacy of BMN 190 in patients with CLN2A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
10Phase 2Germany;Italy;United Kingdom
5EUCTR2014-003480-37-GB
(EUCTR)
10/03/201520/10/2014An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 DiseaseA 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.NULLAuthorised-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): noUnited States;Germany;Italy;United Kingdom
6EUCTR2014-003480-37-DE
(EUCTR)
23/01/201503/11/2014An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 DiseaseA 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.NULLNot RecruitingFemale: yes
Male: yes
23Phase 2United States;Germany;Italy;United Kingdom
7EUCTR2012-005430-11-GB
(EUCTR)
15/08/201308/04/2013Phase 1/2 study to evaluate safety and efficacy of BMN 190 in patients with CLN2A 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: 18.0;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: cerliponase alfa
Other descriptive name: recombinant human tripeptidyl peptidase-1
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
24 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Germany;Italy;United Kingdom