RhASM ( DrugBank: - )
1 disease
| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 19 | Lysosomal storage disease | 18 |
19. Lysosomal storage disease
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182
Showing 1 to 10 of 18 diseases
| 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 | 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 of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase ... | A Long-Term Study to Assess the Ongoing Safety and Efficacy 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 ... | 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] Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease) MedDRA version: 20.1;Le ... | Product Name: Olipudase alfa (rhASM) Product Code: GZ402665 INN or Proposed INN: Olipudase alfa Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM) Product Name: Olipudase alfa (rhASM) Product Code: GZ402665 INN or Proposed INN: Olipudase alfa Other d ... | 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 | ||
| 2 | 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 of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase ... | A Long-Term Study to Assess the Ongoing Safety and Efficacy 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 Sphingomyelina ... | 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] Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease) MedDRA version: 19.0;Le ... | Product Name: Olipudase alfa (rhASM) Product Code: GZ402665 INN or Proposed INN: Olipudase alfa Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM) Product Name: Olipudase alfa (rhASM) Product Code: GZ402665 INN or Proposed INN: Olipudase alfa Other d ... | 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 | ||
| 3 | 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 Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients with Aci ... | 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 A Phase 2/3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Repeat Dose Study to Evalua ... | 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] Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease) MedDRA version: 20.1;Le ... | Product Name: Olipudase alfa Product Code: GZ402665 INN or Proposed INN: Olipudase alfa Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM) Product Name: Olipudase alfa Product Code: GZ402665 INN or Proposed INN: Olipudase alfa Other descripti ... | 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 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - ( ... | Portugal;United States;Spain;Turkey;Chile;United Kingdom;Italy;France;Argentina;Brazil;Australia;Netherlands;Tunisia;Germany;Japan Portugal;United States;Spain;Turkey;Chile;United Kingdom;Italy;France;Argentina;Brazil;Australia;Net ... | ||
| 4 | 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 Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients with Aci ... | 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 A Phase 2/3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Repeat Dose Study to Evalua ... | 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] Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease) MedDRA version: 20.1;Le ... | Product Name: Olipudase alfa Product Code: GZ402665 INN or Proposed INN: Olipudase alfa Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM) Product Name: Olipudase alfa Product Code: GZ402665 INN or Proposed INN: Olipudase alfa Other descripti ... | 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 Portugal;United States;Spain;Turkey;Chile;United Kingdom;Italy;France;Argentina;Brazil;Australia;Bul ... | ||
| 5 | 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 Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients with Aci ... | 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 A phase 2/3, multicenter, randomized, double-blinded, placebo-controlled, repeatdose, dose-compariso ... | 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] Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease) MedDRA version: 18.0;Le ... | Product Name: Olipudase alfa Product Code: GZ402665 INN or Proposed INN: Olipudase alfa Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM) Product Name: Olipudase alfa Product Code: GZ402665 INN or Proposed INN: Olipudase alfa Other descripti ... | 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 Portugal;United States;Spain;Turkey;Chile;Israel;United Kingdom;Italy;France;Argentina;Brazil;Austra ... | ||
| 6 | 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 Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients with Aci ... | 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 A phase 2/3, multicenter, randomized, double-blinded, placebo-controlled, repeat dose study to evalu ... | 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] Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease) MedDRA version: 20.1;Le ... | Product Name: Olipudase alfa Product Code: GZ402665 INN or Proposed INN: Olipudase alfa Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM) Product Name: Olipudase alfa Product Code: GZ402665 INN or Proposed INN: Olipudase alfa Other descripti ... | 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 United States;Portugal;Spain;Turkey;Chile;United Kingdom;Italy;France;Argentina;Brazil;Australia;Bul ... | ||
| 7 | 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 Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of rhASMin Pediatric Pat ... | 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 A Phase 1/2, Multi-Center, Open-Label, Ascending Dose Study to Evaluate the Safety, Tolerability, Ph ... | 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] Patients with acid sphingomyelinase deficiency (Niemann-Pick disease) MedDRA version: 20.1;Level: LL ... | Product Name: Olipudase alfa (rhASM) Product Code: GZ402665 INN or Proposed INN: Olipudase alfa Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM) Product Name: Olipudase alfa (rhASM) Product Code: GZ402665 INN or Proposed INN: Olipudase alfa Other d ... | Genzyme Corporation | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | United States;France;Brazil;Germany;United Kingdom;Italy | ||
| 8 | 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 Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients with Aci ... | 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 A Phase 2/3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled,Repeat Dose Study to Evaluat ... | 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] Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease) MedDRA version: 20.1;Le ... | Product Name: Olipudase alfa Product Code: GZ402665 INN or Proposed INN: Olipudase alfa Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM) Product Name: Olipudase alfa Product Code: GZ402665 INN or Proposed INN: Olipudase alfa Other descripti ... | 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 United States;Portugal;Spain;Turkey;Chile;United Kingdom;Italy;France;Argentina;Brazil;Australia;Bul ... | ||
| 9 | EUCTR2014-003198-40-IT (EUCTR) | 27/02/2015 | 12/12/2014 | Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of rhASM in Pediatric Patients <18 Years of Age with Acid Sphingomyelinase Deficiency Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of rhASMin Pediatric Pat ... | 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 Deficiency A Phase 1/2, Multi-Center, Open-Label, Ascending Dose Study to Evaluate the Safety, Tolerability, Ph ... | 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] Patients with acid sphingomyelinase deficiency (Niemann-Pick disease) MedDRA version: 17.1;Level: LL ... | Product Name: rhASM Product Code: GZ402665 Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM) Product Name: rhASM Product Code: GZ402665 Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELI ... | Genzyme Corporation | NULL | Not 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): no Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - ( ... | France;United States;Brazil;Chile;Germany;United Kingdom;Italy | ||
| 10 | 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 Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Ped ... | 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 A phase 1/2, multi-center, open-label, ascending dose study to evaluate the safety, tolerability, ph ... | 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] Patients with acid sphingomyelinase deficiency (Niemann-Pick disease) MedDRA version: 20.1;Level: LL ... | Product Name: Olipudase alfa (rhASM) Product Code: GZ402665 INN or Proposed INN: Olipudase alfa Other descriptive name: RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM) Product Name: Olipudase alfa (rhASM) Product Code: GZ402665 INN or Proposed INN: Olipudase alfa Other d ... | Genzyme Corporation | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | United States;France;Brazil;Germany;Italy;United Kingdom |