256. Muscle glycogenosis Clinical trials / Disease details


Clinical trials : 180 Drugs : 153 - (DrugBank : 30) / Drug target genes : 31 - Drug target pathways : 134

  
2 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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size
PhaseCountries
1NCT02185651
(ClinicalTrials.gov)
October 20161/7/2014A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated ReactionA Pilot Study of the Effects of Oral Administration of Zavesca® on Anti-rhGAA Immune Response in Subjects With Pompe Disease Receiving rhGAA Enzyme Replacement TherapyPompe Disease;Hypersensitivity ReactionDrug: Zavesca® PrescriptionUniversity of FloridaAmicus TherapeuticsTerminated18 Years65 YearsAll2Phase 1United States
2EUCTR2010-024647-32-IT
(EUCTR)
18/03/201109/05/2011Combined enzyme enhancement therapy (EET) and enzyme replacement therapy (ERT) in patients with Pompe disease - NDCombined enzyme enhancement therapy (EET) and enzyme replacement therapy (ERT) in patients with Pompe disease - ND Patients with Pompe Disease (Type II Glycogenosis) on therapy with Enzyme Replacement Therapy (ERT) for at least 6 months
MedDRA version: 13.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders
Trade Name: ZAVESCA
INN or Proposed INN: Miglustat
UNIVERSITA` DEGLI STUDI DI NAPOLI FEDERICO IINULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Italy