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
| 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 | NCT05359276 (ClinicalTrials.gov) | June 10, 2022 | 28/4/2022 | Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France | Acid Sphingomyelinase Deficiency (ASMD): Data Analysis of Adult and Pediatric Patients on Early Access to Olipudase Alfa in France | Acid Sphingomyelinase Deficiency (ASMD) | Drug: Olipudase alfa | Sanofi | NULL | Recruiting | N/A | N/A | All | 55 | France | |
| 2 | JPRN-jRCT2031210279 | 15/09/2021 | 27/08/2021 | An 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 | An 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 deficiency | Drug: Olipudase alfa (GZ402665) Pharmaceutical form: lyophilized powder, Route of administration: Intravenous (IV) infusion. | Tanaka Tomoyuki | NULL | Not Recruiting | >= 20age old | Not applicable | Both | 1 | Phase 2-3 | Japan |
| 3 | NCT04877132 (ClinicalTrials.gov) | July 21, 2021 | 3/5/2021 | Compassionate 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 Lipidosis | Drug: olipudase alfa (GZ402665) | Sanofi | NULL | Approved for marketing | 3 Years | N/A | All | NULL | ||
| 4 | EUCTR2013-000051-40-DE (EUCTR) | 14/11/2018 | 01/10/2018 | A 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 Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 25 | Phase 2 | France;United States;Belgium;Brazil;Germany;United Kingdom;Italy | ||
| 5 | EUCTR2013-000051-40-IT (EUCTR) | 19/08/2016 | 25/05/2016 | A 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 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 Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 17 | Phase 2 | France;United States;Brazil;Germany;United Kingdom;Italy | ||
| 6 | EUCTR2015-000371-26-IT (EUCTR) | 08/03/2016 | 06/02/2018 | Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients with Acid Sphingomyelinase Deficiency | A 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 CORPORATION | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 35 | Phase 2;Phase 3 | Portugal;United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;France;Argentina;Brazil;Australia;Netherlands;Tunisia;Germany;Japan | ||
| 7 | EUCTR2015-000371-26-PT (EUCTR) | 15/02/2016 | 08/09/2015 | Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients with Acid Sphingomyelinase Deficiency | A 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 Corporation | NULL | Not 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): no | Portugal;United States;Spain;Turkey;Chile;United Kingdom;Italy;France;Argentina;Brazil;Australia;Netherlands;Tunisia;Germany;Japan | ||
| 8 | EUCTR2015-000371-26-NL (EUCTR) | 19/01/2016 | 20/07/2015 | Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients with Acid Sphingomyelinase Deficiency | A 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 Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 36 | Phase 2;Phase 3 | Portugal;United States;Spain;Turkey;Chile;United Kingdom;Italy;France;Argentina;Brazil;Australia;Bulgaria;Netherlands;Tunisia;Germany;Japan | ||
| 9 | EUCTR2015-000371-26-ES (EUCTR) | 18/12/2015 | 05/10/2015 | Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients with Acid Sphingomyelinase Deficiency | A 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 Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 35 | Phase 2/3 | Portugal;United States;Spain;Turkey;Chile;Israel;United Kingdom;Italy;France;Argentina;Brazil;Australia;Netherlands;Tunisia;Germany;Japan | ||
| 10 | NCT02004691 (ClinicalTrials.gov) | December 18, 2015 | 26/11/2013 | Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency | A 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 | Sphingomyelin Lipidosis | Drug: placebo (saline);Drug: GZ402665 | Genzyme, a Sanofi Company | NULL | Active, not recruiting | 18 Years | N/A | All | 36 | Phase 2/Phase 3 | United States;Argentina;Australia;Belgium;Brazil;Bulgaria;Chile;France;Germany;Italy;Japan;Netherlands;Portugal;Spain;Tunisia;Turkey;United Kingdom |
| 11 | EUCTR2015-000371-26-GB (EUCTR) | 27/10/2015 | 02/06/2015 | Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients with Acid Sphingomyelinase Deficiency | A 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 Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 36 | Phase 2;Phase 3 | United States;Portugal;Spain;Turkey;Chile;United Kingdom;Italy;France;Argentina;Brazil;Australia;Bulgaria;Tunisia;Germany;Netherlands;Japan | ||
| 12 | EUCTR2014-003198-40-DE (EUCTR) | 06/10/2015 | 07/08/2015 | Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of rhASM in Pediatric Patients <18 Years of Age with Acid Sphingomyelinase Deficiency | A 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 Corporation | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | United States;France;Brazil;Germany;United Kingdom;Italy | ||
| 13 | EUCTR2015-000371-26-DE (EUCTR) | 14/08/2015 | 02/06/2015 | Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients with Acid Sphingomyelinase Deficiency | A 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 Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 36 | Phase 2;Phase 3 | United States;Portugal;Spain;Turkey;Chile;United Kingdom;Italy;France;Argentina;Brazil;Australia;Bulgaria;Germany;Tunisia;Netherlands;Japan | ||
| 14 | NCT02292654 (ClinicalTrials.gov) | May 1, 2015 | 7/11/2014 | Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency | A 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 | Sphingomyelin Lipidosis | Drug: Olipudase alfa | Genzyme, a Sanofi Company | NULL | Completed | N/A | 17 Years | All | 20 | Phase 1/Phase 2 | United States;Brazil;France;Germany;Italy;United Kingdom |
| 15 | EUCTR2014-003198-40-GB (EUCTR) | 19/02/2015 | 26/11/2014 | Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age with Acid Sphingomyelinase Deficiency | A 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 Corporation | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | United States;France;Brazil;Germany;Italy;United Kingdom | ||
| 16 | EUCTR2013-000051-40-GB (EUCTR) | 16/12/2013 | 22/07/2013 | A 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 Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 25 | Phase 2 | United States;France;Belgium;Brazil;Germany;Italy;United Kingdom | ||
| 17 | NCT02004704 (ClinicalTrials.gov) | December 4, 2013 | 26/11/2013 | A Long-Term Study of Olipudase Alfa 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 | Sphingomyelin Lipidosis | Drug: GZ402665 | Genzyme, a Sanofi Company | NULL | Active, not recruiting | N/A | N/A | All | 25 | Phase 2 | United States;Belgium;Brazil;France;Germany;Italy;United Kingdom |
| 18 | EUCTR2013-000051-40-BE (EUCTR) | 14/08/2019 | A 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 Corporation | NULL | NA | Female: yes Male: yes | 25 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;United States;Brazil;Belgium;Germany;Italy;United Kingdom |