VAL-1221 ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
256筋型糖原病2

256. 筋型糖原病


臨床試験数 : 180 薬物数 : 133 - (DrugBank : 29) / 標的遺伝子数 : 25 - 標的パスウェイ数 : 105
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT02898753
(ClinicalTrials.gov)
June 21, 20178/9/2016VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe DiseaseA 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 PatientsPompe DiseaseDrug: VAL-1221;Drug: RhGAAValerion Therapeutics, LLCNULLTerminated18 YearsN/AAll12Phase 1/Phase 2United States;United Kingdom
2EUCTR2016-004578-16-GB
(EUCTR)
19/06/201729/03/2017A Study to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 in Ambulatory and Ventilator-free Patients with Pompe DiseaseA 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, LLCNULLNot RecruitingFemale: yes
Male: yes
12Phase 1;Phase 2United States;United Kingdom