256. Muscle glycogenosis Clinical trials / Disease details
Clinical trials : 180 / Drugs : 133 - (DrugBank : 29) / Drug target genes : 25 - Drug target pathways : 105
| 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 | EUCTR2019-001283-30-DK (EUCTR) | 03/11/2021 | 05/10/2021 | A study of safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease (type II glycogen storage disease) | Phase 1/2, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease | Pompe Disease (also known as glycogen storage disease type II) MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Code: SPK-3006 INN or Proposed INN: SPK-3006 | Spark Therapeutics | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 1;Phase 2 | France;United States;Spain;Denmark;Netherlands;Germany;United Kingdom;Italy | ||
| 2 | EUCTR2019-001283-30-NL (EUCTR) | 06/01/2021 | 08/11/2020 | A study of safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease (type II glycogen storage disease) | Phase 1/2, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease | Pompe Disease (also known as glycogen storage disease type II) MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Code: SPK-3006 INN or Proposed INN: SPK-3006 | Spark Therapeutics | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 1;Phase 2 | United States;France;Germany;Netherlands;United Kingdom;Italy | ||
| 3 | EUCTR2019-001283-30-DE (EUCTR) | 20/08/2020 | 20/01/2020 | A study of safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease (type II glycogen storage disease) | Phase 1/2, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease | Pompe Disease (also known as glycogen storage disease type II) MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Code: SPK-3006 INN or Proposed INN: SPK-3006 | Spark Therapeutics | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 1;Phase 2 | France;United States;Spain;Denmark;Netherlands;Germany;United Kingdom;Italy | ||
| 4 | EUCTR2019-001283-30-FR (EUCTR) | 16/06/2020 | 18/09/2019 | A study of safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease (type II glycogen storage disease) | Phase 1/2, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease | Pompe Disease (also known as glycogen storage disease type II) MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Code: SPK-3006 INN or Proposed INN: SPK-3006 | Spark Therapeutics | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 1;Phase 2 | United States;France;Denmark;Netherlands;Germany;United Kingdom;Italy |