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

  
5 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT02525172
(ClinicalTrials.gov)
August 201510/8/2015Immune Modulation Therapy for Pompe DiseaseImmune Modulation Therapy for ERT-naïve or ERT-treated Pompe Disease PatientsPompe DiseaseDrug: Rituximab;Drug: intravenous immune globulin;Drug: Bortezomib;Drug: MethotrexateNational Taiwan University HospitalNULLRecruitingN/AN/ABoth8Phase 4Taiwan
2NCT00701129
(ClinicalTrials.gov)
October 200917/6/2008An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe DiseaseAn Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme®-Naive, CRIM(-) Patients With Infantile-onset Pompe DiseasePompe Disease;Glycogen Storage Disease Type IIBiological: Alglucosidase Alfa;Drug: Methotrexate;Drug: RituximabGenzyme, a Sanofi CompanyNULLCompletedN/AN/AAll4Phase 4United States;Israel
3NCT01451879
(ClinicalTrials.gov)
October 200815/9/2011Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation TherapiesEffects of Immunomodulation Therapy on Anti-rhGAA Immune Response in Subjects With Pompe Disease Receiving rhGAA Enzyme Replacement TherapyPompe DiseaseDrug: Rituximab;Drug: MiglustatUniversity of FloridaNULLCompletedN/A65 YearsAll11N/AUnited States
4EUCTR2015-000584-14-Outside-EU/EEA
(EUCTR)
15/04/2015An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe DiseaseAn Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naïve CRIM(-) Patients with Infantile-Onset Pompe Disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 17.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Product Name: Rituximab
INN or Proposed INN: RITUXIMAB
Product Name: Methotrexate
INN or Proposed INN: METHOTREXATE SODIUM
Other descriptive name: METHOTREXATE SODIUM
Genzyme Corporation IncNULLNAFemale: yes
Male: yes
4United States
5EUCTR2015-000583-34-Outside-EU/EEA
(EUCTR)
15/04/2015Immune Tolerance Induction StudyAn Exploratory Study of the Safety and Efficacy of Immune Tolerance Induction (ITI) in Patients with Pompe Disease Who Have Previously Received Myozyme Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 17.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Alglucosidase alfa
Product Code: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Product Name: Cyclophosphamide
INN or Proposed INN: CYCLOPHOSPHAMIDE MONOHYDRATE
Other descriptive name: CYCLOPHOSPHAMIDE MONOHYDRATE
Product Name: Rituximab
INN or Proposed INN: RITUXIMAB
Product Name: Methotrexate
INN or Proposed INN: METHOTREXATE SODIUM
Other descriptive name: METHOTREXATE SODIUM
Genzyme Corporation IncNULLNAFemale: yes
Male: yes
9United States;Israel