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

  
18 trials found
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1NCT05359276
(ClinicalTrials.gov)
June 10, 202228/4/2022Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in FranceAcid Sphingomyelinase Deficiency (ASMD): Data Analysis of Adult and Pediatric Patients on Early Access to Olipudase Alfa in FranceAcid Sphingomyelinase Deficiency (ASMD)Drug: Olipudase alfaSanofiNULLRecruitingN/AN/AAll55France
2JPRN-jRCT2031210279
15/09/202127/08/2021An open label interventional single-patient study to evaluate the safety of olipudase alfa enzyme replacement therapy for the patient in Japan with acid sphingomyelinase deficiency (ASMD) who has completed Study DFI12712An open label interventional single-patient study to evaluate the safety of olipudase alfa enzyme replacement therapy for the patient in Japan with acid sphingomyelinase deficiency (ASMD) who has completed Study DFI12712 Acid sphingomyelinase deficiencyDrug: Olipudase alfa (GZ402665)
Pharmaceutical form: lyophilized powder, Route of administration: Intravenous (IV) infusion.
Tanaka TomoyukiNULLNot Recruiting>= 20age oldNot applicableBoth1Phase 2-3Japan
3NCT04877132
(ClinicalTrials.gov)
July 21, 20213/5/2021Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)Sphingomyelin LipidosisDrug: olipudase alfa (GZ402665)SanofiNULLApproved for marketing3 YearsN/AAllNULL
4EUCTR2013-000051-40-DE
(EUCTR)
14/11/201801/10/2018A Long-Term Study of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase DeficiencyA Long-Term Study to Assess the Ongoing Safety and Efficacy of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease)
MedDRA version: 20.1;Level: LLT;Classification code 10041515;Term: Sphingomyelin lipidosis;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Olipudase alfa (rhASM)
Product Code: GZ402665
INN or Proposed INN: Olipudase alfa
Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
25Phase 2France;United States;Belgium;Brazil;Germany;United Kingdom;Italy
5EUCTR2013-000051-40-IT
(EUCTR)
19/08/201625/05/2016A Long-Term Study of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase DeficiencyA Long-Term Study to Assess the Ongoing Safety and Efficacy of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase Deficiency Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease)
MedDRA version: 19.0;Level: LLT;Classification code 10041515;Term: Sphingomyelin lipidosis;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Olipudase alfa (rhASM)
Product Code: GZ402665
INN or Proposed INN: Olipudase alfa
Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
17Phase 2France;United States;Brazil;Germany;United Kingdom;Italy
6EUCTR2015-000371-26-IT
(EUCTR)
08/03/201606/02/2018Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients with Acid Sphingomyelinase DeficiencyA phase 2/3, multicenter, randomized, double-blinded, placebo-controlled, repeatdose, dose-comparison study to evaluate the efficacy, safety, pharmacodynamics, and pharmacokinetics of olipudase alfa in patients with acid sphingomyelinase deficiency - ASCEND Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease)
MedDRA version: 20.1;Level: LLT;Classification code 10041515;Term: Sphingomyelin lipidosis;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: OLIPUDASE ALFA
Product Code: GZ402665
INN or Proposed INN: OLIPUDASE ALFA
GENZYME CORPORATIONNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
35Phase 2;Phase 3Portugal;United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;France;Argentina;Brazil;Australia;Netherlands;Tunisia;Germany;Japan
7EUCTR2015-000371-26-PT
(EUCTR)
15/02/201608/09/2015Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients with Acid Sphingomyelinase DeficiencyA Phase 2/3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Repeat Dose Study to Evaluate the Efficacy, Safety, Pharmacodynamics and Pharmacokinetics of olipudase alfa in Patients With Acid Sphingomyelinase Deficiency - ASCEND Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease)
MedDRA version: 20.1;Level: LLT;Classification code 10041515;Term: Sphingomyelin lipidosis;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Olipudase alfa
Product Code: GZ402665
INN or Proposed INN: Olipudase alfa
Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)
Genzyme CorporationNULLNot Recruiting Female: yes
Male: yes
36 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noPortugal;United States;Spain;Turkey;Chile;United Kingdom;Italy;France;Argentina;Brazil;Australia;Netherlands;Tunisia;Germany;Japan
8EUCTR2015-000371-26-NL
(EUCTR)
19/01/201620/07/2015Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients with Acid Sphingomyelinase DeficiencyA Phase 2/3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Repeat Dose Study to Evaluate the Efficacy, Safety, Pharmacodynamics and Pharmacokinetics of olipudase alfa in Patients With Acid Sphingomyelinase Deficiency - ASCEND Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease)
MedDRA version: 20.1;Level: LLT;Classification code 10041515;Term: Sphingomyelin lipidosis;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Olipudase alfa
Product Code: GZ402665
INN or Proposed INN: Olipudase alfa
Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
36Phase 2;Phase 3Portugal;United States;Spain;Turkey;Chile;United Kingdom;Italy;France;Argentina;Brazil;Australia;Bulgaria;Netherlands;Tunisia;Germany;Japan
9EUCTR2015-000371-26-ES
(EUCTR)
18/12/201505/10/2015Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients with Acid Sphingomyelinase DeficiencyA phase 2/3, multicenter, randomized, double-blinded, placebo-controlled, repeatdose, dose-comparison study to evaluate the efficacy, safety, pharmacodynamics, and pharmacokinetics of olipudase alfa in patients with acid sphingomyelinase deficiency - ASCEND Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease)
MedDRA version: 18.0;Level: LLT;Classification code 10041515;Term: Sphingomyelin lipidosis;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Olipudase alfa
Product Code: GZ402665
INN or Proposed INN: Olipudase alfa
Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
35Phase 2/3Portugal;United States;Spain;Turkey;Chile;Israel;United Kingdom;Italy;France;Argentina;Brazil;Australia;Netherlands;Tunisia;Germany;Japan
10NCT02004691
(ClinicalTrials.gov)
December 18, 201526/11/2013Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase DeficiencyA Phase 2/3, Multicenter, Randomized, Double-blinded, Placebo-controlled, Repeat-dose Study to Evaluate the Efficacy, Safety, Pharmacodynamics, and Pharmacokinetics of Olipudase Alfa in Patients With Acid Sphingomyelinase DeficiencySphingomyelin LipidosisDrug: placebo (saline);Drug: GZ402665Genzyme, a Sanofi CompanyNULLActive, not recruiting18 YearsN/AAll36Phase 2/Phase 3United States;Argentina;Australia;Belgium;Brazil;Bulgaria;Chile;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom
11EUCTR2015-000371-26-GB
(EUCTR)
27/10/201502/06/2015Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients with Acid Sphingomyelinase DeficiencyA phase 2/3, multicenter, randomized, double-blinded, placebo-controlled, repeat dose study to evaluate the efficacy, safety, pharmacodynamics, and pharmacokinetics of olipudase alfa in patients with acid sphingomyelinase deficiency - ASCEND Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease)
MedDRA version: 20.1;Level: LLT;Classification code 10041515;Term: Sphingomyelin lipidosis;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Olipudase alfa
Product Code: GZ402665
INN or Proposed INN: Olipudase alfa
Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
36Phase 2;Phase 3United States;Portugal;Spain;Turkey;Chile;United Kingdom;Italy;France;Argentina;Brazil;Australia;Bulgaria;Tunisia;Germany;Netherlands;Japan
12EUCTR2014-003198-40-DE
(EUCTR)
06/10/201507/08/2015Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of rhASM in Pediatric Patients <18 Years of Age with Acid Sphingomyelinase DeficiencyA Phase 1/2, Multi-Center, Open-Label, Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Exploratory Efficacy of Recombinant Human Acid Sphingomyelinase in Pediatric Patients Aged <18 Years With Acid Sphingomyelinase DeficiencyRevised title further to the protocol amendment 1A phase 1/2, multi-center, open-label, ascending dose study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and exploratory efficacy of olipudase afa in pediatric patients aged <18 years with acid sphingomyelinase deficiency Patients with acid sphingomyelinase deficiency (Niemann-Pick disease)
MedDRA version: 20.1;Level: LLT;Classification code 10041515;Term: Sphingomyelin lipidosis;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Olipudase alfa (rhASM)
Product Code: GZ402665
INN or Proposed INN: Olipudase alfa
Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)
Genzyme CorporationNULLNot RecruitingFemale: yes
Male: yes
20Phase 2United States;France;Brazil;Germany;United Kingdom;Italy
13EUCTR2015-000371-26-DE
(EUCTR)
14/08/201502/06/2015Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients with Acid Sphingomyelinase DeficiencyA Phase 2/3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled,Repeat Dose Study to Evaluate the Efficacy, Safety, Pharmacodynamics andPharmacokinetics of olipudase alfa in Patients With Acid SphingomyelinaseDeficiency - ASCEND Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease)
MedDRA version: 20.1;Level: LLT;Classification code 10041515;Term: Sphingomyelin lipidosis;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Olipudase alfa
Product Code: GZ402665
INN or Proposed INN: Olipudase alfa
Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
36Phase 2;Phase 3United States;Portugal;Spain;Turkey;Chile;United Kingdom;Italy;France;Argentina;Brazil;Australia;Bulgaria;Germany;Tunisia;Netherlands;Japan
14NCT02292654
(ClinicalTrials.gov)
May 1, 20157/11/2014Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase DeficiencyA Phase 1/2, Multi-Center, Open-Label, Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Exploratory Efficacy of Olipudase Alfa in Pediatric Patients Aged <18 Years With Acid Sphingomyelinase DeficiencySphingomyelin LipidosisDrug: Olipudase alfaGenzyme, a Sanofi CompanyNULLCompletedN/A17 YearsAll20Phase 1/Phase 2United States;Brazil;France;Germany;Italy;United Kingdom
15EUCTR2014-003198-40-GB
(EUCTR)
19/02/201526/11/2014Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age with Acid Sphingomyelinase DeficiencyA phase 1/2, multi-center, open-label, ascending dose study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and exploratory efficacy of olipudase alfa in pediatric patients Aged <18 Years With acid sphingomyelinase deficiency - ASCEND-Peds Patients with acid sphingomyelinase deficiency (Niemann-Pick disease)
MedDRA version: 20.1;Level: LLT;Classification code 10041515;Term: Sphingomyelin lipidosis;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Olipudase alfa (rhASM)
Product Code: GZ402665
INN or Proposed INN: Olipudase alfa
Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)
Genzyme CorporationNULLNot RecruitingFemale: yes
Male: yes
20Phase 2United States;France;Brazil;Germany;Italy;United Kingdom
16EUCTR2013-000051-40-GB
(EUCTR)
16/12/201322/07/2013A Long-Term Study of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase DeficiencyA Long-Term Study to Assess the Ongoing Safety and Efficacy of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease)
MedDRA version: 20.1;Level: LLT;Classification code 10041515;Term: Sphingomyelin lipidosis;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Olipudase alfa (rhASM)
Product Code: GZ402665
INN or Proposed INN: Olipudase alfa
Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
25Phase 2United States;France;Belgium;Brazil;Germany;Italy;United Kingdom
17NCT02004704
(ClinicalTrials.gov)
December 4, 201326/11/2013A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase DeficiencyA Long-Term Study to Assess the Ongoing Safety and Efficacy of Olipudase Alfa in Patients With Acid Sphingomyelinase DeficiencySphingomyelin LipidosisDrug: GZ402665Genzyme, a Sanofi CompanyNULLActive, not recruitingN/AN/AAll25Phase 2United States;Belgium;Brazil;France;Germany;Italy;United Kingdom
18EUCTR2013-000051-40-BE
(EUCTR)
14/08/2019A Long-Term Study of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase Deficiency A Long-Term Study to Assess the Ongoing Safety and Efficacy of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease)
MedDRA version: 20.1;Level: LLT;Classification code 10041515;Term: Sphingomyelin lipidosis;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Olipudase alfa (rhASM)
Product Code: GZ402665
INN or Proposed INN: Olipudase alfa
Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)
Genzyme CorporationNULLNA Female: yes
Male: yes
25 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;United States;Brazil;Belgium;Germany;Italy;United Kingdom