19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 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 | 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 | ||
| 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 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 | ||
| 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 | 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 | ||
| 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 | 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 | ||
| 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 | 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 | ||
| 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 | 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 | ||
| 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 | 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 | ||
| 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 | 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 | ||
| 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 | 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 | 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 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 | 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 | 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 | ||
| 11 | EUCTR2010-023953-12-IT (EUCTR) | 28/01/2014 | 24/09/2013 | Safety, Efficacy, and Pharmacokinetics Study of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase Deficiency | A 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 Corporation | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 2 | United States;France;Brazil;Chile;Tunisia;Germany;United Kingdom;Italy | ||
| 12 | EUCTR2010-023953-12-FR (EUCTR) | 23/12/2013 | 29/09/2015 | Safety, Efficacy, and Pharmacokinetics Study of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase Deficiency | A 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 Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 15 | Phase 2 | United States;France;Brazil;Chile;Germany;Tunisia;Italy;United Kingdom | ||
| 13 | 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 | ||
| 14 | EUCTR2010-023953-12-GB (EUCTR) | 16/12/2013 | 26/07/2013 | Safety, Efficacy, and Pharmacokinetics Study of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase Deficiency | A 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 Corporation | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 2 | France;United States;Brazil;Chile;Germany;Tunisia;Italy;United Kingdom | ||
| 15 | NCT01722526 (ClinicalTrials.gov) | March 2013 | 5/11/2012 | Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients | An 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 Deficiency | Drug: Recombinant human acid sphingomyelinase | Genzyme, a Sanofi Company | NULL | Completed | 18 Years | 65 Years | Both | 5 | Phase 1 | United States;United Kingdom |
| 16 | NCT00410566 (ClinicalTrials.gov) | December 2006 | 11/12/2006 | Safety 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 Disease | Drug: rhASM | Genzyme, a Sanofi Company | NULL | Terminated | 18 Years | 65 Years | Both | 11 | Phase 1 | United States |
| 17 | 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 | |||
| 18 | EUCTR2014-003198-40-FR (EUCTR) | 25/06/2015 | 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 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 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 | United States;France;Brazil;Chile;Germany;Italy;United Kingdom |