256. 筋型糖原病 Muscle glycogenosis Clinical trials / Disease details
臨床試験数 : 180 / 薬物数 : 153 - (DrugBank : 30) / 標的遺伝子数 : 31 - 標的パスウェイ数 : 134
| 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 | NCT02898753 (ClinicalTrials.gov) | June 21, 2017 | 8/9/2016 | VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease | A Three-Month, Open-Label, Randomized, Dose-escalation Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 Versus Myozyme®/Lumizyme® in Patients With Late-Onset GSD-II (Pompe Disease) Followed by Open-Label Treatment With VAL-1221 in All Patients | Pompe Disease | Drug: VAL-1221;Drug: RhGAA | Valerion Therapeutics, LLC | NULL | Terminated | 18 Years | N/A | All | 12 | Phase 1/Phase 2 | United States;United Kingdom |
| 2 | EUCTR2016-004578-16-GB (EUCTR) | 19/06/2017 | 29/03/2017 | A Study to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 in Ambulatory and Ventilator-free Patients with Pompe Disease | A Three-month, Open-Label, Randomized, Dose-escalation Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 versus Myozyme®/Lumizyme® in Patients with Late-Onset GSD-II (Pompe Disease) Followed by Open-Label Treatment with VAL-1221 in all Patients | Late-Onset GSD-II (Pompe Disease) MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850 MedDRA version: 20.1;Classification code 10045253;Term: Type II glycogen storage disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: VAL-1221 Product Code: VAL-1221 INN or Proposed INN: VAL-1221 Trade Name: Myozyme Product Name: Myozyme INN or Proposed INN: ALGLUCOSIDASE ALFA Other descriptive name: rhGAA | Valerion Therapeutics, LLC | NULL | Not Recruiting | Female: yes Male: yes | 12 | Phase 1;Phase 2 | United States;United Kingdom |