RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM) ( 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18

19. ライソゾーム病


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
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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agemax
Inclusion_
gender
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size
PhaseCountries
1EUCTR2013-000051-40-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
2EUCTR2013-000051-40-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
3EUCTR2015-000371-26-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
4EUCTR2015-000371-26-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
5EUCTR2015-000371-26-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
6EUCTR2015-000371-26-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
7EUCTR2014-003198-40-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
8EUCTR2015-000371-26-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
9EUCTR2014-003198-40-IT
(EUCTR)
27/02/201512/12/2014Safety, 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 Deficiency Patients with acid sphingomyelinase deficiency (Niemann-Pick disease)
MedDRA version: 17.1;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: rhASM
Product Code: GZ402665
Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)
Genzyme CorporationNULLNot Recruiting Female: yes
Male: yes
12 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;United States;Brazil;Chile;Germany;United Kingdom;Italy
10EUCTR2014-003198-40-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
11EUCTR2010-023953-12-IT
(EUCTR)
28/01/201424/09/2013Safety, Efficacy, and Pharmacokinetics Study of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase DeficiencyA Phase 2, Multi-Center, Randomized, Open-Label, Repeat Dose, Dose-Comparison Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase Deficiency - ASCEND Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease)
MedDRA version: 16.0;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: rhASM
Product Code: GZ402665
Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)
Genzyme CorporationNULLNot RecruitingFemale: yes
Male: yes
15Phase 2United States;France;Brazil;Chile;Tunisia;Germany;United Kingdom;Italy
12EUCTR2010-023953-12-FR
(EUCTR)
23/12/201329/09/2015Safety, Efficacy, and Pharmacokinetics Study of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase DeficiencyA Phase 2, Multi-Center, Randomized, Open-Label, Repeat Dose, Dose-Comparison Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase Deficiency 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: rhASM
Product Code: GZ402665
Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
15Phase 2United States;France;Brazil;Chile;Germany;Tunisia;Italy;United Kingdom
13EUCTR2013-000051-40-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
14EUCTR2010-023953-12-GB
(EUCTR)
16/12/201326/07/2013Safety, Efficacy, and Pharmacokinetics Study of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase DeficiencyA Phase 2, Multi-Center, Randomized, Open-Label, Repeat Dose, Dose-Comparison Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase Deficiency Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease)
MedDRA version: 17.1;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: rhASM
Product Code: GZ402665
Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)
Genzyme CorporationNULLNot RecruitingFemale: yes
Male: yes
15Phase 2France;United States;Brazil;Chile;Germany;Tunisia;Italy;United Kingdom
15NCT01722526
(ClinicalTrials.gov)
March 20135/11/2012Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency PatientsAn Open-label, Multicenter, Ascending Dose Study of the Tolerability and Safety of Recombinant Human Acid Sphingomyelinase (rhASM) in Patients With Acid Sphingomyelinase Deficiency (ASMD)Human Acid Sphingomyelinase DeficiencyDrug: Recombinant human acid sphingomyelinaseGenzyme, a Sanofi CompanyNULLCompleted18 Years65 YearsBoth5Phase 1United States;United Kingdom
16NCT00410566
(ClinicalTrials.gov)
December 200611/12/2006Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)A Phase I, Single-Center, Single Dose, Dose Escalation Study of Recombinant Human Acid Sphingomyelinase (rhASM) in Adults With Acid Sphingomyelinase Deficiency (ASMD)Acid Sphingomyelinase Deficiency;Niemann-Pick DiseaseDrug: rhASMGenzyme, a Sanofi CompanyNULLTerminated18 Years65 YearsBoth11Phase 1United States
17EUCTR2013-000051-40-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
18EUCTR2014-003198-40-FR
(EUCTR)
25/06/2015Safety, 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 Deficiency Patients with acid sphingomyelinase deficiency (Niemann-Pick 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: rhASM
Product Code: GZ402665
Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)
Genzyme CorporationNULLNot Recruiting Female: yes
Male: yes
12 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;France;Brazil;Chile;Germany;Italy;United Kingdom