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.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
1NCT04094948
(ClinicalTrials.gov)
January 1, 202317/9/2019Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe DiseasePhase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease Stably Treated With Enzyme Replacement TherapyPompe Disease (Late-onset)Drug: Clenbuterol;Drug: PlacebosDuke UniversityNULLNot yet recruiting18 YearsN/AAll40Phase 2United States
2NCT04808505
(ClinicalTrials.gov)
August 202210/3/2021Rossella: A Study to Evaluate the Safety, PK, Efficacy, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18An Open-label Study to Evaluate the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 YearsGlycogen Storage Disease Type II Infantile OnsetBiological: Cipaglucosidase alfa (ATB200);Drug: Miglustat (AT2221)Amicus TherapeuticsNULLRecruitingN/A17 YearsAll22Phase 3United States
3NCT05272969
(ClinicalTrials.gov)
March 31, 202228/1/2022Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe DiseasePompe & Pain - Observational Study to Assess Musculoskeletal Pain in Late-onset Pompe Disease (LOPD)Pompe Disease (Late-onset);Inclusion Body Myositis;Spinal Muscular Atrophy Type 3;FSHDDiagnostic Test: Beck depression inventory fast screen (Questionnaire);Diagnostic Test: Brief Pain Inventory (BPI) (Questionnaire);Diagnostic Test: German Pain Inventory (Questionnaire);Diagnostic Test: Fatigue Severity and Disability Scale (FSS) (Questionnaire);Diagnostic Test: Rotterdam Handicap Scale (RHS) (Questionnaire);Diagnostic Test: R-PAct (Questionnaire);Diagnostic Test: Quick Motor Function Test;Diagnostic Test: Handheld Dynamometry (HHD);Diagnostic Test: Six-minute walk test (6MWT);Diagnostic Test: Pressure pain threshold;Diagnostic Test: Muscle ultrasound;Diagnostic Test: Vital signs;Diagnostic Test: Borg Scale;Diagnostic Test: Laboratory assessment: Creatine kinase;Diagnostic Test: Laboratory assessment: Vitamin D Level;Diagnostic Test: Laboratory assessment: calcium;Diagnostic Test: Laboratory assessment: magnesium;Diagnostic Test: Laboratory assessment: phosphate;Genetic: Genetic test: ACE polymorphism;Genetic: Genetic test: ACTN3 polymorphism;Genetic: Blood draw for optional genetic exome sequencingLMU KlinikumNULLNot yet recruiting18 YearsN/AAll95Germany
4NCT05164055
(ClinicalTrials.gov)
February 15, 202216/11/2021A French Multicenter Phase 4 Open Label Extension Study of Long Term Safety and Efficacy in Patients With Pompe Disease Who Previously Participated in Avalglucosidase Development Studies in FranceA French Multicenter Phase 4 Open Label Extension Study of Long Term Safety and Efficacy in Patients With Pompe Disease Who Previously Participated in Avalglucosidase Development Studies in FranceGlycogen Storage Disease Type IIDrug: Avalglucosidase alfa (GZ402666)Genzyme, a Sanofi CompanyNULLNot yet recruiting6 MonthsN/AAll18Phase 4NULL
5NCT04476550
(ClinicalTrials.gov)
February 1, 202226/2/2020Clinical Specimen Collection From Pompe Disease PatientsDeveloping a Potential Cure for Pompe Disease: Clinical Specimen Collection From Individuals With Pompe DiseasePompe DiseaseDrug: FilgrastimSerhat Gumrukcu, MD PhDFrida Therapeutics LLCRecruiting3 Years30 YearsAll12United States
6EUCTR2019-001283-30-DK
(EUCTR)
03/11/202105/10/2021A 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 TherapeuticsNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 1;Phase 2France;United States;Spain;Denmark;Netherlands;Germany;United Kingdom;Italy
7EUCTR2020-004686-39-NL
(EUCTR)
04/10/202104/05/2021Clinical Study for IOPD Participants Less Than or Equal to 6 Months of age to Evaluate Efficacy and Safety of enzyme replacement therapy (ERT) With Avalglucosidase AlfaAn Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of avalglucosidase alfa in Treatment naïve Pediatric Participants Less than or Equal to 6 Months of Age with Infantile-Onset Pompe Disease (IOPD) - Baby-COMET 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: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Avalglucosidase Alfa
Product Code: GZ402666
INN or Proposed INN: ALGLUCOSIDASE ALFA
Other descriptive name: RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN
Sanofi-Aventis Recherche & DéveloppementNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 3France;United States;Taiwan;Belgium;Germany;Netherlands;United Kingdom;Italy;China
8NCT04910776
(ClinicalTrials.gov)
September 1, 202125/5/2021Clinical Study for IOPD Participants Less Than or Equal to 6 Months of Age to Evaluate Efficacy and Safety of Enzyme Replacement Therapy (ERT) With Avalglucosidase AlfaAn Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Avalglucosidase Alfa in Treatment naïve Pediatric Participants Less Than or Equal to 6 Months of Age With Infantile-Onset Pompe Disease (IOPD)Glycogen Storage Disease Type IIDrug: avalglucosidase alfaSanofiNULLRecruitingN/A6 MonthsAll16Phase 3Belgium;Germany;Italy;Netherlands;Taiwan;United Kingdom
9NCT05017402
(ClinicalTrials.gov)
September 1, 20215/8/2021Higher Dose of Alglucosidase Alpha for Pompe DiseaseHigher Dose of Alglucosidase Alpha for Pompe Disease: Long-term Follow-up StudyGlycogen Storage Disease Type IIDrug: Alglucosidase AlfaTaipei Veterans General Hospital, TaiwanNULLNot yet recruitingN/A60 YearsAll36NULL
10NCT04848779
(ClinicalTrials.gov)
June 10, 202129/3/2021A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients Upto 6 Months of Age With Infantile-onset Pompe Disease (IOPD)A Prospective Observational Study to Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients =6 Months of Age With Infantile-onset Pompe Disease (IOPD)Glycogen Storage Disease Type IIDrug: Alglucosidase alfa GZ419829 (Myozyme)SanofiNULLRecruitingN/A6 MonthsAll16Belgium;Germany;Italy;Taiwan
11EUCTR2020-004686-39-IT
(EUCTR)
27/04/202107/06/2021Clinical Study for IOPD Participants Less Than or Equal to 6 Months of age to Evaluate Efficacy and Safety of enzyme replacement therapy (ERT) WithAvalglucosidase Alfa(Baby-COMET)An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of avalglucosidase alfa inTreatment-naïve Pediatric Participants Less than or Equal to 6 Months of Age with Infantile-Onset Pompe Disease (IOPD) - Baby-COMET 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: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Avalglucosidase Alfa
Product Code: [GZ402666]
INN or Proposed INN: avalglucosidasi alfa
Other descriptive name: RECOMBINANT HUMAN ALFA-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN
SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENTNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 3France;United States;Taiwan;Belgium;Netherlands;Germany;United Kingdom;China;Italy
12NCT04532047
(ClinicalTrials.gov)
April 7, 202119/8/2020In Utero Enzyme Replacement Therapy for Lysosomal Storage DiseasesIn Utero Enzyme Replacement Therapy (ERT) for Prenatally Diagnosed Lysosomal Storage Disorders (LSDs).MPS I;MPS II;MPS IVA;MPS VI;Mps VII;Gaucher Disease, Type 2;Gaucher Disease, Type 3;Pompe Disease Infantile-Onset;Wolman DiseaseDrug: Aldurazyme (laronidase)University of California, San FranciscoDuke UniversityRecruiting18 Years50 YearsFemale10Phase 1United States
13NCT04676373
(ClinicalTrials.gov)
March 10, 202115/12/2020Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa TreatmenA Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa TreatmentPompe's DiseaseDrug: ALGLUCOSIDASE ALFAGenzyme, a Sanofi CompanyNULLRecruiting3 YearsN/AAll40Phase 4China
14EUCTR2019-001283-30-NL
(EUCTR)
06/01/202108/11/2020A 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 TherapeuticsNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 1;Phase 2United States;France;Germany;Netherlands;United Kingdom;Italy
15EUCTR2019-003595-38-GB
(EUCTR)
20/11/202003/07/2020FORTIS: AAV8-Delivered Gene Therapy in Late Onset Pompe DiseaseFORTIS: A Phase 1/2, Open-Label, Ascending-Dose Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT845, an AAV8-Delivered Gene Transfer Therapy in Patients with Late Onset Pompe Disease - FORTIS Late Onset Pompe Disease
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: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: AT845
Product Code: AT845
Audentes Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
8Phase 1;Phase 2Germany;Italy;United Kingdom
16EUCTR2019-001283-30-DE
(EUCTR)
20/08/202020/01/2020A 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 TherapeuticsNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 1;Phase 2France;United States;Spain;Denmark;Netherlands;Germany;United Kingdom;Italy
17EUCTR2019-000954-67-AT
(EUCTR)
19/06/202013/03/2020A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe diseaseA Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 24.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: cipaglucosidase alfa
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Other descriptive name: AT2221 65 mg Formulated Capsules
Amicus Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3Australia;Netherlands;Korea, Republic of;Bosnia and Herzegovina;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden;United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark
18EUCTR2019-001283-30-FR
(EUCTR)
16/06/202018/09/2019A 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 TherapeuticsNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 1;Phase 2United States;France;Netherlands;Germany;United Kingdom;Italy
19EUCTR2019-000954-67-SI
(EUCTR)
03/06/202010/03/2020A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe diseaseA Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: cipaglucosidase alfa
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Amicus Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 3United States;Taiwan;Slovenia;Greece;Spain;Austria;United Kingdom;Italy;France;Hungary;Canada;Argentina;Poland;Belgium;Australia;Denmark;Netherlands;Germany;Japan;Sweden;Bosnia and Herzegovina;Korea, Republic of
20EUCTR2019-000954-67-DE
(EUCTR)
17/04/202023/12/2019A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe diseaseA Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 24.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: cipaglucosidase alfa
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Amicus Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3Germany;Japan;Sweden;Bosnia and Herzegovina;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Korea, Republic of
21EUCTR2019-000954-67-GB
(EUCTR)
08/04/202013/12/2019A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe diseaseA Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: cipaglucosidase alfa
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Amicus Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
22EUCTR2019-000954-67-GR
(EUCTR)
02/04/202013/02/2020A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe diseaseA Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 24.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: cipaglucosidase alfa
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Amicus Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Korea, Republic of;Bosnia and Herzegovina;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
23EUCTR2019-000954-67-IT
(EUCTR)
01/04/202017/06/2021A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe diseaseA Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease - AMICUS ATB200-07 Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 24.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: [ATB200]
INN or Proposed INN: cipaglucosidasi alfa
Product Name: AT2221
Product Code: [AT2221]
INN or Proposed INN: MIGLUSTAT
AMICUS THERAPEUTICS, INC.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 3United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Korea, Republic of;Bosnia and Herzegovina;Slovenia;Korea, Democratic People's Republic of;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
24EUCTR2019-000954-67-DK
(EUCTR)
16/03/202020/01/2020A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe diseaseA Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 24.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: cipaglucosidase alfa
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Amicus Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Bosnia and Herzegovina;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
25EUCTR2019-000954-67-FR
(EUCTR)
14/03/202020/01/2020A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe diseaseA Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: cipaglucosidase alfa
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Amicus Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 3United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
26NCT03911505
(ClinicalTrials.gov)
February 13, 20209/4/2019ZIP Study - A Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of ATB200/AT2221 in Pediatric Subjects Aged 0 to < 18 Years With Pompe DiseaseAn Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of ATB200/AT2221 in Pediatric Subjects Aged 0 to < 18 Years With Pompe DiseasePompe Disease (Late-onset)Biological: Cipaglucosidase Alfa;Drug: MiglustatAmicus TherapeuticsNULLRecruitingN/A17 YearsAll22Phase 3United States;Canada;Japan;Taiwan
27EUCTR2019-000954-67-ES
(EUCTR)
11/02/202020/12/2019A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe diseaseA Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: N/A
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Amicus Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
120 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Taiwan;Slovakia;Greece;Spain;Italy;France;Australia;Denmark;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
28EUCTR2019-000954-67-BE
(EUCTR)
31/01/202006/02/2020A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe diseaseA Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 24.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: cipaglucosidase alfa
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Amicus Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Korea, Republic of;Bosnia and Herzegovina;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
29NCT04138277
(ClinicalTrials.gov)
December 18, 201918/10/2019A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPDA Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe DiseasePompe Disease (Late-onset)Drug: AT2221;Biological: ATB200Amicus TherapeuticsNULLActive, not recruiting18 YearsN/AAll110Phase 3United States;Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Poland;Slovenia;Spain;Sweden;Taiwan;United Kingdom
30NCT04226274
(ClinicalTrials.gov)
December 10, 20199/1/2020A Study of the Safety of REN001 in Patients With McArdle DiseaseA Phase 1b, Open-label Study to Evaluate the Safety and Tolerability of 12 Weeks Treatment With Oral REN001 in Patients With McArdle Disease (Glycogen Storage Disorder 5)McArdle DiseaseDrug: REN001Reneo Pharma LtdNULLCompleted18 YearsN/AAll19Phase 1Spain;United Kingdom
31EUCTR2018-000755-40-AT
(EUCTR)
25/09/201904/06/2019A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared withAlglucosidase Alfa/Placebo.A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: Cipaglucosidase alfa
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Trade Name: Myozyme
INN or Proposed INN: ALGLUCOSIDASE ALFA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
110Phase 3United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
32JPRN-JapicCTI-194887
17/9/201926/07/2019ATB200/AT2221phase3 double blind randomized studyA phase 3 double-blind randomized study to assess the efficacy and safety of intravenous ATB200 co-administered with oral AT2221 in adult subjects with late- onset pompe disease compared with alglucosidase alfa/placebo Pompe diseaseIntervention name : Recombinant human acid alfa-glucosidase (ATB200)
INN of the intervention : Recombinant human acid alfa-glucosidase
Dosage And administration of the intervention : 20 mg/kg IV infusion over a 4-hour duration 1 hour after AT2221(Miglustat) administration every 2 weeks
Intervention name : Miglustat (AT2221)
INN of the intervention : Miglustat
Dosage And administration of the intervention : Subjects weighing >= 50 kg, 260mg (4 oral capsules of AT2221) 1hour prior to ATB200 infusion every 2 weeks. Subjects weighing >= 40 kg to < 50 kg, 195 mg (3 oral capsules) 1 hour prior to ATB200 infusion every 2 weeks.
Control intervention name : Alglucosidase Alfa (Genetical Recombination)
INN of the control intervention : Alglucosidase Alfa (Genetical Recombination)
Dosage And administration of the control intervention : 20 mg/kg IV infusion over a 4-hour duration 1 hour after placebo administration every 2 weeks
Control intervention name : Plecebo
INN of the control intervention : -
Dosage And administration of the control intervention : -
Amicus Therapeutics, Inc./CMIC Co., Ltd.NULLcomplete18BOTH110Phase 3Japan, Asia except Japan, North America, South America, Europe, Oceania
33EUCTR2018-000755-40-NL
(EUCTR)
28/08/201924/01/2019A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared withAlglucosidase Alfa/Placebo.A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: Cipaglucosidase alfa
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Trade Name: Myozyme
INN or Proposed INN: ALGLUCOSIDASE ALFA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
110Phase 3United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
34EUCTR2018-000755-40-GR
(EUCTR)
28/08/201921/05/2019A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared withAlglucosidase Alfa/Placebo.A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: Cipaglucosidase alfa
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Trade Name: Myozyme
INN or Proposed INN: ALGLUCOSIDASE ALFA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
110Phase 3United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
35NCT04327973
(ClinicalTrials.gov)
August 15, 201927/3/2020Expanded Access for ATB200/AT2221 for the Treatment of IOPDExpanded Use Of Recombinant Human Acid Alpha-Glucosidase/N-butyl-deoxynojirimycin (ATB200/AT2221) For Patients With Infantile-Onset Pompe DiseasePompe Disease Infantile-OnsetBiological: ATB200;Drug: AT2221Amicus TherapeuticsNULLAvailableN/A17 YearsAllUnited States;Italy;Taiwan
36NCT04044508
(ClinicalTrials.gov)
August 3, 201912/12/2018Modified Ketogenic Diet in Patients With McArdle Disease Part BOdified Ketogenic Diet in Patients With McArdle Disease Part B - a Placebo-controlled, Cross-over StudyMcArdle DiseaseDietary Supplement: Ketocal 4:1 liquid Nutricia (intervention);Dietary Supplement: Fortini multifibre Nutrica (placebo)Rigshospitalet, DenmarkUniversity College, LondonRecruiting18 Years80 YearsAll18N/ADenmark;United Kingdom
37EUCTR2018-000755-40-SI
(EUCTR)
22/05/201903/04/2019A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared withAlglucosidase Alfa/Placebo.A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: cipaglucosidase alfa
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Trade Name: Myozyme
INN or Proposed INN: ALGLUCOSIDASE ALFA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
110Phase 3United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
38EUCTR2018-000755-40-IT
(EUCTR)
17/05/201925/01/2021A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared with Alglucosidase Alfa/Placebo.A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo - AMICUS ATB200-03 Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: [ATB200]
INN or Proposed INN: non applicabile
Product Name: AT2221
Product Code: [AT2221]
INN or Proposed INN: MIGLUSTAT
Trade Name: Myozyme
Product Name: Myozyme
Product Code: [Myozyme]
INN or Proposed INN: ALGLUCOSIDASI ALFA UMANA RICOMBINANTE
AMICUS THERAPEUTICS, INC.NULLNot RecruitingFemale: yes
Male: yes
110Phase 3United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Bosnia and Herzegovina;Slovenia;Korea, Democratic People's Republic of;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
39NCT03980483
(ClinicalTrials.gov)
May 16, 201915/5/2019Efficacy and Safety of GSK3196165 Versus Placebo and Tofacitinib in Participants With Moderately to Severely Active Rheumatoid Arthritis Who Have an Inadequate Response to MethotrexateA 52-week, Phase 3, Multicentre, Randomised, Double Blind, Efficacy and Safety Study Comparing GSK3196165 With Placebo and With Tofacitinib, in Combination With Methotrexate in Participants With Moderately to Severely Active Rheumatoid Arthritis Who Have an Inadequate Response to MethotrexateArthritis, Rheumatoid;Glycogen Storage Disease Type IIBiological: GSK3196165 (Otilimab);Drug: Tofacitinib 5 mg;Drug: Placebo to GSK3196165;Drug: Placebo to tofacitinib;Drug: Methotrexate;Drug: Folic (or folinic) acidGlaxoSmithKlineIqvia Pty LtdActive, not recruiting18 YearsN/AAll1537Phase 3United States;Argentina;Canada;China;Czechia;Hungary;India;Italy;Latvia;Lithuania;Malaysia;Mexico;Poland;Russian Federation;Serbia;South Africa;Spain;Ukraine;United Kingdom
40NCT03945370
(ClinicalTrials.gov)
May 6, 201918/2/2019Oral Ketone Body Supplementation in Patients With McArdle DiseaseOral Ketone Body Supplementation in Patients With McArdle DiseaseMcArdle DiseaseDietary Supplement: ß-hydroxybuturate esters;Dietary Supplement: Placebo drinkRigshospitalet, DenmarkNULLCompleted18 YearsN/AAll8N/ADenmark
41EUCTR2018-000755-40-BG
(EUCTR)
16/04/201928/02/2019 A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared with Alglucosidase Alfa/Placebo.A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: N/A
Product Name: Miglustat
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Trade Name: Myozyme
INN or Proposed INN: ALGLUCOSIDASE ALFA
Amicus Therapeutics, Inc.NULLNot Recruiting Female: yes
Male: yes
110 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Taiwan;Slovakia;Greece;Spain;Italy;France;Australia;Denmark;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
42EUCTR2018-000755-40-DE
(EUCTR)
08/04/201929/11/2018A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared withAlglucosidase Alfa/Placebo.A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: Cipaglucosidase alfa
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Trade Name: Myozyme
INN or Proposed INN: ALGLUCOSIDASE ALFA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
110Phase 3United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
43NCT04292938
(ClinicalTrials.gov)
March 25, 201928/2/2020McArdle Disease Treatment by Ketogenic DietKetogenic Diet in McArdle Disease: a Multicentric Single Blind Controlled TrialGlycogen Storage DiseaseDietary Supplement: Low carbohydrate ketogenic dietIRCCS Eugenio MedeaUniversity of Pisa;University of MessinaCompleted18 YearsN/AAll16N/AItaly
44EUCTR2018-000755-40-GB
(EUCTR)
18/03/201906/12/2018A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared withAlglucosidase Alfa/Placebo.A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: N/A
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Trade Name: Myozyme
INN or Proposed INN: ALGLUCOSIDASE ALFA
Amicus Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
45EUCTR2018-000755-40-BE
(EUCTR)
11/03/201903/01/2019A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared withAlglucosidase Alfa/Placebo.A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: Cipaglucosidase alfa
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Trade Name: Myozyme
INN or Proposed INN: ALGLUCOSIDASE ALFA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
110Phase 3United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
46EUCTR2018-000755-40-SK
(EUCTR)
04/03/201908/01/2019A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared withAlglucosidase Alfa/Placebo.A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: N/A
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Trade Name: Myozyme
INN or Proposed INN: ALGLUCOSIDASE ALFA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
110Phase 3United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
47EUCTR2018-000755-40-HU
(EUCTR)
28/02/201928/12/2018A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared withAlglucosidase Alfa/Placebo.A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: ALGLUCOSIDASE ALFA
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Trade Name: Myozyme
INN or Proposed INN: ALGLUCOSIDASE ALFA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
110Phase 3United States;Taiwan;Slovakia;Greece;Spain;Israel;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Brazil;Belgium;Poland;Romania;Bulgaria;Germany;Japan;New Zealand;Sweden
48EUCTR2018-000755-40-ES
(EUCTR)
12/02/201918/01/2019 A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared with Alglucosidase Alfa/Placebo.A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: N/A
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Trade Name: Myozyme
INN or Proposed INN: ALGLUCOSIDASE ALFA
Amicus Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
110 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Taiwan;Slovakia;Greece;Spain;Italy;France;Australia;Denmark;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
49EUCTR2018-000755-40-DK
(EUCTR)
06/02/201930/11/2018A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared withAlglucosidase Alfa/Placebo.A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: Cipaglucosidase alfa
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Trade Name: Myozyme
INN or Proposed INN: ALGLUCOSIDASE ALFA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
110Phase 3United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Australia;Denmark;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
50EUCTR2018-000755-40-SE
(EUCTR)
30/01/201912/12/2018A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared withAlglucosidase Alfa/Placebo.A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: Cipaglucosidase alfa
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Trade Name: Myozyme
INN or Proposed INN: ALGLUCOSIDASE ALFA
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
110Phase 3United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
51EUCTR2016-000942-77-HU
(EUCTR)
29/01/201904/12/2018Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease A phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and safety of repeated biweekly infusions of neoGAA (GZ402666) and alglucosidase alfa in treatment-naïve patients with late onset Pompe disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA
Product Code: GZ402666
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
96 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noPortugal;United States;Taiwan;Spain;Russian Federation;Colombia;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Poland;Brazil;Belgium;Bulgaria;Germany;Japan;Sweden
52NCT03865836
(ClinicalTrials.gov)
January 15, 20195/3/2019Expanded Access for ATB200/AT2221 for the Treatment of Pompe DiseaseNamed Patient or Compassionate Use for Treatment Use of ATB200/AT2221 for Patients With Pompe DiseasePompe DiseaseBiological: ATB200;Drug: AT2221Amicus TherapeuticsNULLAvailableN/AN/AAllNULL
53NCT03729362
(ClinicalTrials.gov)
December 4, 201810/10/2018PROPEL Study - A Study Comparing ATB200/AT2221 With Alglucosidase/Placebo in Adult Subjects With LOPDA Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/PlaceboPompe Disease (Late-onset)Drug: AT2221;Biological: alglucosidase alfa;Biological: ATB200Amicus TherapeuticsNULLCompleted18 YearsN/AAll123Phase 3United States;Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Poland;Slovenia;Spain;Sweden;Taiwan;United Kingdom
54NCT03687333
(ClinicalTrials.gov)
December 4, 201824/9/2018Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa TreatmentA Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa TreatmentGlycogen Storage Disease Type IIDrug: ALGLUCOSIDASE ALFA (MYOZYME)Genzyme, a Sanofi CompanyNULLCompletedN/A12 MonthsAll10Phase 4China
55ChiCTR1800018514
2018-11-062018-09-21A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients with Infantile-Onset Pompe Disease with One Year Alglucosidase Alfa TreatmentA Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients with Infantile-Onset Pompe Disease with One Year Alglucosidase Alfa Treatment Genetic and MetabolismCase series:Alglucosidase Alfa treatment;Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of MedicineNULLPending01BothCase series:10;4 (Phase 4 study)China
56NCT03533673
(ClinicalTrials.gov)
November 1, 201813/4/2018AAV2/8-LSPhGAA in Late-Onset Pompe DiseaseA Phase 1 Study of the Safety of AAV2/8-LSPhGAA in Late-onset Pompe DiseasePompe DiseaseBiological: AAV2/8LSPhGAAAsklepios Biopharmaceutical, Inc.Duke University;National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)Recruiting18 YearsN/AAll8Phase 1/Phase 2United States
57EUCTR2016-000942-77-BG
(EUCTR)
23/10/201824/07/2018Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseA phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and safety of repeated biweekly infusions of neoGAA (GZ402666) and alglucosidase alfa in treatment-naïve patients with late onset Pompe disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 24.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA
Product Code: GZ402666
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
96Phase 3United States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Italy;Switzerland;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Germany;Japan;Sweden
58EUCTR2017-004370-34-HU
(EUCTR)
29/08/201826/03/2018A study to evaluate changes in function such as walking and/or breathing in subjects taking enzyme replacement therapy (ERT) for late-onset Pompe diseaseA Prospective Study in Subjects with Late-onset Pompe Disease who are Currently Being Treated with Enzyme Replacement Therapy Late-onset Pompe disease (LOPD) in subjects receiving standard-of-care enzyme replacement therapy (ERT)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
INN or Proposed INN: ALGLUCOSIDASE ALFAAmicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
125Phase 4United States;Taiwan;Slovakia;Spain;Korea, Democratic People's Republic of;Italy;United Kingdom;France;Hungary;Canada;Poland;Belgium;Brazil;Romania;Australia;Denmark;Bulgaria;Netherlands;Germany;Japan;Sweden
59NCT03642860
(ClinicalTrials.gov)
August 15, 201820/8/2018The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With GlycogenosesTriheptanoin's Effect on Fatty Acid Oxidation and Exercise Tolerance in Patients With Debrancher Deficiency, Glycogenin-1 Deficiency and Phosphofructoinase Deficiency at Rest and During Exercise. A Randomized, Double-blind, Placebo-controlled, Cross-over StudyTarui Disease;Debrancher Deficiency;GYG1 DEFICIENCYDrug: Triheptanoin;Drug: Placebo OilRigshospitalet, DenmarkUltragenyx Pharmaceutical IncTerminated15 Years85 YearsAll3Phase 2Denmark
60EUCTR2016-000942-77-PT
(EUCTR)
24/07/201812/04/2018Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseA Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase Alfa in Treatment naïve Patients with Late-onset Pompe Disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 24.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA or avalglucosidase alfa
Product Code: GZ402666
INN or Proposed INN: neoGAA
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 3United States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Italy;Switzerland;France;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Germany;Japan;Sweden
61EUCTR2017-004153-17-DK
(EUCTR)
03/05/201826/03/2018The effect of Triheptanoin on excercise in adults and adolescence with glycogenosesEvaluation of the effect of Triheptanoin on fatty acid oxidation and exercise tolerance in patients with debrancher deficiency, glycogenin-1 deficiency and phosphofructokinase deficiency at rest and during exercise. A randomized, double-blind, placebo-controlled, cross-over study. - Triheptanoin in Glycogenoses Cori Forbe's Disease Also called: glycogen storage disease Type III or debrancher deficiency.Tarui's diseaseAlso called: glycogen storage disease Type VII or phosphofructokinase deficiency.Glycogenin-1 deficiency or glycogen storage disease Type XV.
MedDRA version: 20.1;Level: PT;Classification code 10053241;Term: Glycogen storage disease type VII;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.1;Classification code 10053250;Term: Glycogen storage disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10053255;Term: Tarui disease;Classification code 10016983;Term: Forbes' disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Triheptanoin
Product Code: UX007
INN or Proposed INN: Not available
Other descriptive name: TRIHEPTANOIN
Copenhagen Neuromuscular CenterNULLNot RecruitingFemale: yes
Male: yes
20Phase 2Denmark
62EUCTR2017-004370-34-BE
(EUCTR)
09/04/201815/02/2018A study to evaluate changes in function such as walking and/or breathing in subjects taking enzyme replacement therapy (ERT) for late-onset Pompe diseaseA Prospective Study in Subjects with Late-onset Pompe Disease who are Currently Being Treated with Enzyme Replacement Therapy Late-onset Pompe disease (LOPD) in subjects receiving standard-of-care enzyme replacement therapy (ERT)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
INN or Proposed INN: ALGLUCOSIDASE ALFAAmicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
125Phase 4United States;Taiwan;Spain;Korea, Democratic People's Republic of;Italy;United Kingdom;France;Hungary;Canada;Belgium;Brazil;Australia;Denmark;Netherlands;Germany;Japan;Sweden
63EUCTR2010-022231-11-GB
(EUCTR)
07/11/201717/08/2017Pharmacokinetics of Alglucosidase Alfa in Patients with Pompe DiseaseA Phase 3/4 Prospective Study to Characterize the Pharmacokinetics of Alglucosidase Alfa in Patients with Pompe Disease - PAPAYA Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000011505 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Myozyme
Product Name: Myozyme
INN or Proposed INN: ALGLUCOSIDASE ALFA
Other descriptive name: rhGAA
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
20 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): yesUnited States;Ukraine;Russian Federation;Bulgaria;Germany;United Kingdom;India
64NCT02240407
(ClinicalTrials.gov)
October 17, 201711/9/2014Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe DiseaseEvaluation of Re-administration of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV9-DES-hGAA) in Patients With Late-Onset Pompe Disease (LOPD)Pompe DiseaseGenetic: Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase;Drug: Rapamycin;Other: saline;Drug: Rituxan;Drug: Diphenhydramine;Drug: Acetaminophen;Drug: Lidocaine;Drug: LMX 4 Topical CreamUniversity of FloridaNULLActive, not recruiting18 Years50 YearsAll2Phase 1United States
65NCT03019406
(ClinicalTrials.gov)
October 12, 201720/12/2016A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase AlfaAn Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of Avalglucosidase Alfa (NeoGAA, GZ402666) in Patients With Infantile-onset Pompe Disease Treated With Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical ResponseGlycogen Storage Disease Type II-Pompe's DiseaseDrug: avalglucosidase alfa GZ402666;Drug: alglucosidase alfa GZ419829Genzyme, a Sanofi CompanyNULLActive, not recruiting6 Months17 YearsAll22Phase 2United States;France;Japan;Taiwan;United Kingdom
66NCT02898753
(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
67EUCTR2016-004578-16-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
68EUCTR2016-003475-21-GB
(EUCTR)
17/05/201703/01/2017A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated with Alglucosidase AlfaAn Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of Avalglucosidase Alfa (NeoGAA, GZ402666) in Patients with Infantile-onset Pompe Disease Treated with Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders
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: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Avalglucosidase Alfa (NeoGAA)
Product Code: GZ402666
INN or Proposed INN: Avalglucosidase Alfa (NeoGAA)
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan Avalglucosidase Alfa (NeoGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
34Phase 2United States;France;Taiwan;Germany;Japan;United Kingdom
69NCT03045042
(ClinicalTrials.gov)
April 201727/1/2017Search for Serum/Plasma Biomarkers in Pompe's DiseaseSearch for Serum/Plasma Biomarkers in Pompe's DiseaseLate Onset Pompe Disease;Pompe DiseaseDrug: Enzyme Replacement AgentFundació Institut de Recerca de l'Hospital de la Santa Creu i Sant PauGenzyme, a Sanofi CompanyNot yet recruiting2 YearsN/AAll35N/ASpain
70EUCTR2016-000942-77-NL
(EUCTR)
21/03/201715/06/2016Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseA phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and safety of repeated biweekly infusions of neoGAA (GZ402666) and alglucosidase alfa in treatment-naïve patients with late onset Pompe disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA
Product Code: GZ402666
INN or Proposed INN: NEO-GAA
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
96Phase 3United States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Italy;Switzerland;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Germany;Japan;Sweden
71EUCTR2016-000942-77-AT
(EUCTR)
06/02/201731/10/2016Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseA phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and safety of repeated biweekly infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and alglucosidase alfa in treatment-naïve patients with late onset Pompe disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 24.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA or avalglucosidase alfa
Product Code: GZ402666
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 3Portugal;United States;Taiwan;Spain;Russian Federation;Colombia;Italy;Switzerland;France;Denmark;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Belgium;Brazil;Poland;Bulgaria;Germany;Japan;Sweden
72EUCTR2016-000942-77-DE
(EUCTR)
18/11/201627/06/2016Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseA Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase Alfa in Treatment naïve Patients with Late-onset Pompe Disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 24.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA or avalglucosidase alfa
Product Code: GZ402666
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 3Portugal;United States;Taiwan;Spain;Russian Federation;Colombia;Italy;Switzerland;France;Denmark;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Belgium;Brazil;Poland;Bulgaria;Germany;Japan;Sweden
73EUCTR2016-000942-77-ES
(EUCTR)
20/10/201622/06/2016Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseA Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of NeoGAA (GZ402666) and Alglucosidase Alfa in Treatmentnaïve Patients with Late-onset Pompe Disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 19.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA
Product Code: GZ402666
INN or Proposed INN: NeoGAA
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
90Phase 3United States;Taiwan;Spain;Turkey;Austria;Russian Federation;Colombia;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Canada;Argentina;Belgium;Brazil;Denmark;Netherlands;Germany;Japan;Sweden;Korea, Republic of
74EUCTR2016-000942-77-BE
(EUCTR)
14/10/201601/07/2016Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseA Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of NeoGAA (GZ402666) and Alglucosidase Alfa in Treatmentnaïve Patients with Late-onset Pompe Disease - COMET Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA
Product Code: GZ402666
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 3United States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Switzerland;Italy;France;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Germany;Japan;Sweden
75EUCTR2016-000942-77-CZ
(EUCTR)
05/10/201627/06/2016Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseA phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and safety of repeated biweekly infusions of neoGAA (GZ402666) and alglucosidase alfa in treatment-naïve patients with late onset Pompe disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA
Product Code: GZ402666
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
96Phase 3Portugal;United States;Taiwan;Spain;Russian Federation;Colombia;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Germany;Japan;Sweden
76NCT02919631
(ClinicalTrials.gov)
October 201628/9/2016Triheptanoin in Mc ArdleThe Effect of Triheptanoin in Adults With Mc Ardle Disease (Glycogen Storage Disease Type V)Glycogen Storage Disease Type VDrug: Triheptanoin;Drug: Placebo oilInstitut National de la Santé Et de la Recherche Médicale, FranceRigshospitalet, Denmark;Rigshospitalet, DenmarkNot yet recruiting18 Years65 YearsBoth12Phase 2NULL
77NCT02185651
(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
78NCT02782741
(ClinicalTrials.gov)
October 201623/5/2016Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseA Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase Alfa in Treatment naïve Patients With Late-onset Pompe DiseaseGlycogen Storage Disease Type II;Pompe's DiseaseDrug: avalglucosidase alfa(GZ402666);Drug: alglucosidase alfa (GZ419829)SanofiNULLActive, not recruiting3 YearsN/AAll102Phase 3United States;Argentina;Australia;Austria;Belgium;Brazil;Canada;Colombia;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Switzerland;Taiwan;Turkey;United Kingdom;Bulgaria;Czech Republic;Sweden
79EUCTR2015-004798-34-DE
(EUCTR)
30/08/201631/03/2016This is an open-label, fixed-sequence, ascending-dose, first-in-human study to evaluate the safety, tolerability, PK, PD and efficacy of intravenous (IV) ATB200 when co-administered with oral AT2221.AN OPEN-LABEL, FIXED-SEQUENCE, ASCENDING-DOSE, FIRST-IN-HUMAN STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF INTRAVENOUS INFUSIONS OF ATB200 CO-ADMINISTERED WITH ORAL AT2221 IN ADULT SUBJECTS WITH POMPE DISEASE Pompe Disease - acid maltase deficiency or glycogen storage disease type II.
MedDRA version: 24.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: cipaglucosidase alfa
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Amicus Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
32Phase 1;Phase 2United States;Australia;Netherlands;Germany;United Kingdom
80EUCTR2016-000942-77-IT
(EUCTR)
26/08/201606/02/2018Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseA Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of NeoGAA (GZ402666) and Alglucosidase Alfa in Treatmentnaïve Patients with Late-onset Pompe Disease - COMET Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA
Product Code: GZ402666
Other descriptive name: a-glucosidasi acida umana ricombinante coniugata con multiple copie di bis-mannosio-6- fosfato-tetra-mannosio glicano sintetico (NEOGAA)
Trade Name: MYOZYME®
INN or Proposed INN: ALGLUCOSIDASE ALFA
GENZYME CORPORATIONNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
90Phase 3Czech Republic;Poland;Belgium;Spain;Austria;Denmark;Germany;Netherlands;United Kingdom;Italy;Sweden
81EUCTR2016-000942-77-DK
(EUCTR)
25/08/201602/06/2016Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseA Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of NeoGAA (GZ402666) and Alglucosidase Alfa in Treatmentnaïve Patients with Late-onset Pompe Disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 19.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA
Product Code: GZ402666
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLNot Recruiting Female: yes
Male: yes
90 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Switzerland;Italy;France;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Germany;Japan;Sweden
82EUCTR2016-000942-77-SE
(EUCTR)
24/08/201608/06/2016Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseA Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of NeoGAA (GZ402666) and Alglucosidase Alfa in Treatmentnaïve Patients with Late-onset Pompe Disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA
Product Code: GZ402666
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLNot Recruiting Female: yes
Male: yes
90 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noCanada;Argentina;Brazil;Belgium;Poland;Bulgaria;Germany;Japan;Sweden;Mexico;Czech Republic;Hungary;United Kingdom;Austria;Turkey;Korea, Republic of;Netherlands;Denmark;France;Italy;Switzerland;Colombia;Russian Federation;Spain;Taiwan;Portugal;United States
83EUCTR2015-004798-34-GB
(EUCTR)
01/08/201604/01/2016This is an open-label, fixed-sequence, ascending-dose, first-in-human study to evaluate the safety, tolerability, PK, PD and efficacy of intravenous (IV) ATB200 when co-administered with oral AT2221.AN OPEN-LABEL, FIXED-SEQUENCE, ASCENDING-DOSE, FIRST-IN-HUMAN STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF INTRAVENOUS INFUSIONS OF ATB200 CO-ADMINISTERED WITH ORAL AT2221 IN ADULT SUBJECTS WITH POMPE DISEASE Pompe Disease - acid maltase deficiency or glycogen storage disease type II.
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: cipaglucosidase alfa
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Amicus Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
32Phase 1;Phase 2United States;Australia;Netherlands;Germany;United Kingdom
84EUCTR2015-004798-34-NL
(EUCTR)
25/07/201625/04/2016This is an open-label, fixed-sequence, ascending-dose, first-in-human study to evaluate the safety, tolerability, PK, PD and efficacy of intravenous (IV) ATB200 when co-administered with oral AT2221. AN OPEN-LABEL, FIXED-SEQUENCE, ASCENDING-DOSE, FIRST-IN-HUMAN STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF INTRAVENOUS INFUSIONS OF ATB200 CO-ADMINISTERED WITH ORAL AT2221 IN ADULT SUBJECTS WITH POMPE DISEASE Pompes Disease - acid maltase deficiency or glycogen storage disease type II.
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850 ;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: N/A
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Amicus Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
32 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Australia;Germany;Netherlands;New Zealand;United Kingdom
85EUCTR2016-000942-77-GB
(EUCTR)
15/07/201604/05/2016Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseA Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase Alfa in Treatment naïve Patients with Late-onset Pompe Disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA or avalglucosidase alfa
Product Code: GZ402666
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
100 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Switzerland;Italy;France;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Germany;Japan;Sweden
86NCT02824068
(ClinicalTrials.gov)
June 1, 201620/6/2016Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (STIG-Pompe-Study)Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 MonthsPompe DiseaseDrug: glucosidase alfaKlinikum der Universitaet MuenchenGenzyme, a Sanofi CompanyCompleted8 YearsN/AAll112Germany;Italy;Taiwan;Argentina;Brazil
87EUCTR2013-001768-48-AT
(EUCTR)
21/02/201612/01/2016A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe DiseaseA Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease Pompe disease
MedDRA version: 18.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: reveglucosidase alpha
Other descriptive name: GILT-rhGAA
Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: reveglucosidase alpha
Other descriptive name: GILT-rhGAA
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
70Phase 3Portugal;France;United States;Belgium;Austria;Netherlands;Germany;Japan;Italy;United Kingdom;Switzerland
88NCT02675465
(ClinicalTrials.gov)
January 201626/1/2016First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221An Open-Label, Fixed-Sequence, Ascending-Dose, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Intravenous Infusions of ATB200 Co-Administered With Oral AT2221 in Adult Subjects With Pompe DiseasePompe DiseaseDrug: ATB200;Drug: AT2221Amicus TherapeuticsNULLActive, not recruiting18 Years75 YearsAll32Phase 1/Phase 2United States;Australia;Germany;Netherlands;New Zealand;United Kingdom
89NCT02357225
(ClinicalTrials.gov)
August 201523/1/2015A Pilot Study of Pyridostigmine in Pompe DiseaseEvaluation of Respiratory and Skeletal Muscle Functions in Response to Acetylcholinesterase Inhibitors in Pompe DiseasePompe DiseaseDrug: Pyridostigmine BromideUniversity of FloridaNULLTerminated8 Years60 YearsAll2Early Phase 1United States
90NCT02525172
(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
91NCT02432768
(ClinicalTrials.gov)
April 20154/11/2014The Effect of Triheptanoin in Adults With McArdle Disease (Glycogen Storage Disease Type V)The Effect of Triheptanoin in Adults With McArdle Disease (Glycogen Storage Disease Type V)Glycogen Storage Disease Type VDrug: Triheptanoin;Other: Placebo oilRigshospitalet, DenmarkGroupe Hospitalier Pitie-Salpetriere;University of Texas Southwestern Medical Center;Ultragenyx Pharmaceutical IncCompleted18 Years65 YearsAll22Phase 2Denmark
92NCT01710813
(ClinicalTrials.gov)
March 20, 201517/10/2012Alglucosidase Alfa Pompe Safety Sub-RegistryA Prospective Safety Sub-Registry to Assess Anaphylaxis and Severe Allergic Reactions, and Severe Cutaneous and Systemic Immune Complex Mediated Reactions With Alglucosidase Alfa TreatmentPompe DiseaseBiological: alglucosidase alfaGenzyme, a Sanofi CompanyNULLCompletedN/AN/AAll110Germany;Italy;Taiwan;United States;Czech Republic;Belgium;Czechia
93EUCTR2014-003644-12-DK
(EUCTR)
20/02/201504/11/2014The effect of Triheptanoin in adults with McArdle Disease (Glycogen Storage Disease Type V)The effect of Triheptanoin in adults with McArdle Disease (Glycogen Storage Disease Type V) - Triheptanoin in McArdle McArdle Disease Also called: Glycogen Storage Disease Type V or Myophosphorylase Deficiency
MedDRA version: 19.0;Level: LLT;Classification code 10026970;Term: McArdles disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 19.0;Classification code 10026969;Term: McArdle's disease;Level: PT;Classification code 10018462;Term: Glycogen storage disease type V;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Triheptanoin
Product Code: UX007
INN or Proposed INN: Not available
Other descriptive name: TRIHEPTANOIN
Neuromuscular Research Unit, RigshospitaletNULLNot RecruitingFemale: yes
Male: yes
28Phase 2Denmark
94EUCTR2013-001768-48-PT
(EUCTR)
30/01/201502/07/2014A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe DiseaseA Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease Pompe disease
MedDRA version: 18.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: GILT-rhGAA
Other descriptive name: GILT-rhGAA
Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: GILT-rhGAA
Other descriptive name: GILT-rhGAA
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
50Phase 3France;United States;Portugal;Argentina;Belgium;Austria;Netherlands;Germany;Colombia;Japan;Italy;United Kingdom
95NCT03112889
(ClinicalTrials.gov)
January 201511/8/2015Sodium Valproate for GSDVA Phase II Pilot Study to Explore Treatment With Sodium Valproate in Adults With McArdle Disease (Glycogen Storage Disorder Type V, GSDV)Glycogen Storage Disease Type V;McArdle DiseaseDrug: Sodium ValproateUniversity College, LondonNULLCompleted18 Years64 YearsAll8Phase 2Denmark;United Kingdom
96EUCTR2013-003321-28-DK
(EUCTR)
14/11/201418/09/2014Avalglucosidase Alfa Extension StudyAn open-label, multicenter, multinational extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of Avalglucosidase Alfa (NeoGAA, GZ402666) in patients with Pompe disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders
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: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Avalglucosidase Alfa
Product Code: GZ402666
Other descriptive name: RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
21Phase 2United States;France;Belgium;Denmark;Netherlands;Germany;United Kingdom;Italy
97NCT01410890
(ClinicalTrials.gov)
November 3, 20142/8/2011Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe DiseaseA Phase 3/4 Prospective Study to Characterize the Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe DiseasePompe Disease;Glycogen Storage Disease Type II (GSD II)Biological: alglucosidase alfaGenzyme, a Sanofi CompanyNULLCompletedN/AN/AAll21Phase 4United States;Bulgaria;India;Russian Federation;Ukraine;United Kingdom;Germany
98EUCTR2013-003321-28-DE
(EUCTR)
20/10/201425/09/2014Avalglucosidase Alfa Extension StudyAn open-label, multicenter, multinational extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of Avalglucosidase Alfa (NeoGAA, GZ402666) in patients with Pompe disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 24.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders
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: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Avalglucosidase Alfa
Product Code: GZ402666
Other descriptive name: RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
21Phase 2France;United States;Belgium;Denmark;Netherlands;Germany;United Kingdom;Italy
99EUCTR2013-003321-28-NL
(EUCTR)
25/09/201416/07/2014NeoGAA Extension StudyAn open-label, multicenter, multinational extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of neoGAA in patients with Pompe disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders
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: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA
Product Code: GZ402666
INN or Proposed INN: GZ402666
Other descriptive name: RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
21Phase 2France;United States;Belgium;Denmark;Germany;Netherlands;United Kingdom;Italy
100EUCTR2013-001768-48-DE
(EUCTR)
23/09/201422/05/2014A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe DiseaseA Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease Pompe disease
MedDRA version: 18.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: reveglucosidase alpha
Other descriptive name: GILT-rhGAA
Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: reveglucosidase alpha
Other descriptive name: GILT-rhGAA
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
50Phase 3Portugal;France;United States;Argentina;Belgium;Austria;Netherlands;Colombia;Germany;Japan;Italy;United Kingdom
101EUCTR2013-001768-48-IT
(EUCTR)
22/09/201401/07/2014A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe DiseaseA Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease Pompe disease
MedDRA version: 17.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: GILT-rhGAA
Other descriptive name: GILT-rhGAA
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
50Phase 3Portugal;France;United States;Argentina;Belgium;Austria;Netherlands;Germany;Colombia;Japan;United Kingdom;Italy
102EUCTR2013-001768-48-BE
(EUCTR)
08/09/201412/05/2014A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe DiseaseA Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease Pompe disease
MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: reveglucosidase alpha
Other descriptive name: GILT-rhGAA
Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: reveglucosidase alpha
Other descriptive name: GILT-rhGAA
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
50Phase 3Portugal;France;United States;Argentina;Belgium;Austria;Netherlands;Germany;Colombia;Japan;Italy;United Kingdom
103EUCTR2014-001637-88-DK
(EUCTR)
12/08/201424/06/2014Effects on muscle function, in persons with McArdle disease, when treated with the drug Valproate.A phase 2a study to explore treatment with Sodium Valproate in adults with McArdle Disease (Glycogen Storage Disorder Type V, GSDV) - Valproate treatment in McArdle disease McArdle disease (Glycogen storage disorder type V)
MedDRA version: 17.0;Level: LLT;Classification code 10026969;Term: McArdle's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Deprakine
INN or Proposed INN: SODIUM VALPROATE
John VissingNULLNot RecruitingFemale: yes
Male: yes
15Phase 2Denmark
104EUCTR2013-003321-28-BE
(EUCTR)
12/05/201417/02/2014Avalglucosidase Alfa Extension StudyAn Open-Label, Multicenter, Multinational Extension Study Of The Long-Term Safety And Pharmacokinetics Of Repeated Biweekly Infusions Of Avalglucosidase Alfa (NeoGAA, GZ402666) In Patients With Pompe Disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders
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: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Avalglucosidase Alfa
Product Code: GZ402666
Other descriptive name: RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
21Phase 2France;United States;Belgium;Denmark;Netherlands;Germany;Italy;United Kingdom
105EUCTR2012-002933-12-GB
(EUCTR)
07/04/201404/04/2014A Phase II Pilot Study to Assess Safety and Efficacy of Sodium Valproate in Adults with McArdle DiseaseA Phase II pilot study to explore treatment with Sodium Valproate in Adults with McArdle Disease (Glycogen Storage Disorder Type V, GSDV) - Sodium Valproate for GSDV Version 1.0 13th January 2014 McArdle disease (Glycogen storage disease type V, GSDV). The condition is an inherited disorder of skeletal muscle that causes exercise intolerance. The condition can give way to potential rhabdomyolysis which can cause acute renal failure and from middle age muscle wasting and weakness. Affected patients are unable to produce lactate during ischaemic exercise due to a congenital lack of the enzyme muscle glycogen phosphorylase which is essential for glycogen metabolism.
MedDRA version: 16.1;Level: LLT;Classification code 10026969;Term: McArdle's disease;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Epilim Chrono 200 Controlled Released tablets
Product Name: Epilim Chrono 200 Controlled Released tablets
INN or Proposed INN: Sodium valproate
Other descriptive name: Valproic acid
Trade Name: Epilim Chrono 300 Controlled Released tablets
Product Name: Epilim Chrono 300 Controlled Released tablets
INN or Proposed INN: Sodium valproate
Other descriptive name: valproic acid
Trade Name: Epilim Chrono 500 Controlled Released tablets
Product Name: Epilim Chrono 500 Controlled Released tablets
INN or Proposed INN: Sodium valproate
Other descriptive name: valproic acid
University College London,NULLNot Recruiting Female: yes
Male: yes
8 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Denmark;United Kingdom
106NCT01924845
(ClinicalTrials.gov)
April 201413/8/2013BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects With Late-onset Pompe DiseaseLate-onset Pompe DiseaseDrug: BMN 701BioMarin PharmaceuticalNULLTerminated18 YearsN/AAll24Phase 3United States;Belgium;France;Germany;Italy;Netherlands;Portugal;United Kingdom
107EUCTR2013-003321-28-FR
(EUCTR)
19/03/201417/06/2015NeoGAA Extension StudyAn open-label, multicenter, multinational extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of neoGAA in patients with Pompe disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA
Product Code: GZ402666
Other descriptive name: RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
18United States;France;Belgium;Denmark;Netherlands;Germany;Italy;United Kingdom
108NCT02032524
(ClinicalTrials.gov)
February 27, 20144/12/2013Avalglucosidase Alfa Extension StudyAn Open-Label, Multicenter, Multinational Extension Study Of The Long-Term Safety And Pharmacokinetics Of Repeated Biweekly Infusions Of Avalglucosidase Alfa In Patients With Pompe DiseaseGlycogen Storage Disease Type II Pompe DiseaseDrug: GZ402666Genzyme, a Sanofi CompanyNULLActive, not recruitingN/AN/AAll19Phase 2/Phase 3United States;Belgium;Denmark;France;Germany;Netherlands;United Kingdom
109EUCTR2013-001768-48-GB
(EUCTR)
27/11/201322/10/2013A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe DiseaseA Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease Pompe disease
MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: reveglucosidase alfa
Other descriptive name: GILT-rhGAA
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
70Phase 3Portugal;France;United States;Belgium;Austria;Denmark;Netherlands;Germany;Colombia;Japan;Italy;United Kingdom
110NCT02054832
(ClinicalTrials.gov)
November 201331/1/2014Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard Versus Modified Uncooked CornstarchA Comparison of Quality of Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard and Modified Uncooked CornstarchGlycogen Storage Disease Type IA;Glycogen Storage Disease Type IB;Glycogen Storage Disease Type III;Glycogen Storage Disease Type 0Dietary Supplement: GlycosadeJohn MitchellNULLCompleted2 Years50 YearsBoth11N/ACanada
111NCT02405598
(ClinicalTrials.gov)
October 201322/3/2015Evaluation of Salbutamol as an Adjuvant Therapy for Pompe DiseaseEvaluation of Salbutamol as an Adjuvant Therapy for Pompe DiseasePompe DiseaseDrug: SalbutamolNational Taiwan University HospitalNULLCompleted2 YearsN/ABoth14Phase 4Taiwan
112NCT01942590
(ClinicalTrials.gov)
September 201311/9/2013Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement TherapyA Clinical Investigation of the Safety and Efficacy of Clenbuterol on Motor Function in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement TherapyPompe DiseaseDrug: Clenbuterol;Drug: PlaceboDwight Koeberl, M.D., Ph.D.NULLCompleted18 YearsN/AAll17Phase 1/Phase 2United States
113NCT01898364
(ClinicalTrials.gov)
July 20132/7/2013Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.An Open-label, Multicenter, Multinational, Ascending Dose Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Repeated Biweekly Infusions of neoGAA in naïve and Alglucosidase Alfa Treated Late-onset Pompe Disease Patients.Pompe Disease;Glycogen Storage Disease Type II (GSD II);Acid Maltase DeficiencyDrug: GZ402666Genzyme, a Sanofi CompanyNULLCompleted18 YearsN/ABoth24Phase 1United States;Belgium;Denmark;France;Germany;Netherlands;United Kingdom;Italy
114NCT01885936
(ClinicalTrials.gov)
June 201321/6/2013Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe DiseaseA Phase 1/2 Double-Blind Study of the Safety and Efficacy of Albuterol on Motor Function in Individuals With Late-onset Pompe Disease Receiving Enzyme Replacement TherapyPompe DiseaseDrug: Albuterol;Drug: PlaceboDuke UniversityNULLCompleted18 YearsN/AAll16Phase 1/Phase 2United States
115EUCTR2011-005595-42-DE
(EUCTR)
17/04/201330/11/2012A study to compare alglucosidase alfa manufactured at the 160 L scale and the 4000 L scale in patients with infantile-onset Pompe disease who have never been treated.A Phase 3/4, Prospective, Multinational, Open-label, Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve 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]
Trade Name: Myozyme
Product Name: Myozyme
INN or Proposed INN: ALGLUCOSIDASE ALFA
Other descriptive name: rhGAA
Genzyme Corporation, a Sanofi companyNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
24Phase 3/4United States;Taiwan;Canada;Turkey;Russian Federation;Germany;India
116EUCTR2011-001805-28-DE
(EUCTR)
14/02/201328/06/2012A long term study of the safety of BMN 701 in patients with POMPE diseaseA Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 Study POMPE disease
MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 701
Product Code: BMN 701
Other descriptive name: GILT-rhGAA
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
21Phase 2United States;Australia;Germany;New Zealand;United Kingdom
117EUCTR2010-022231-11-DE
(EUCTR)
13/12/201201/08/2012Pharmacokinetics of Alglucosidase Alfa in Patients Aged 8 Years of Age andOlderA Phase 3/4 Prospective Study to Characterize the Pharmacokinetics ofAlglucosidase Alfa in Patients Aged 8 Years of Age and Older - PAPAYA Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 18.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Myozyme
Product Name: Myozyme
INN or Proposed INN: ALGLUCOSIDASE ALFA
Other descriptive name: rhGAA
Genzyme CorporationNULLNot RecruitingFemale: yes
Male: yes
20Phase 1;Phase 4United States;Poland;Ukraine;Russian Federation;Bulgaria;Germany;United Kingdom;China;India
118NCT01597596
(ClinicalTrials.gov)
August 201210/5/2012A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe DiseaseA Phase 3/4, Prospective, Multinational, Open-label, Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe DiseasePompe Disease (Infantile-Onset);Glycogen Storage Disease Type II (GSD II);Glycogenosis 2;Acid Maltase DeficiencyBiological: alglucosidase alfaGenzyme, a Sanofi CompanyNULLTerminatedN/A12 MonthsAll4Phase 4United States;Germany;Taiwan
119EUCTR2010-023561-22-DE
(EUCTR)
04/07/201207/02/2012A Study of the Safety and Tolerability of BMN 701 in Patients with Late-onset Pompe DiseaseA Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant human GAA) in Patients with Late-onset Pompe Disease - A Phase 1/2 Study of BMN 701 in Patients with Late-onset Pompe Disease Pompe disease
MedDRA version: 16.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 701
Product Code: BMN 701
Other descriptive name: GILT-rhGAA
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
30Phase 1/2France;United States;Netherlands;Germany;United Kingdom
120EUCTR2011-001805-28-GB
(EUCTR)
03/07/201226/04/2012A Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 StudyA Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 Study Pompe disease
MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 701
Product Code: BMN 701
Other descriptive name: GILT-rhGAA
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
30Phase 2United States;France;Australia;Germany;United Kingdom
121NCT01859624
(ClinicalTrials.gov)
June 20128/5/2013Albuterol in Individuals With Late Onset Pompe Disease (LOPD)A Clinical Investigation of the Safety and Efficacy of Albuterol on Motor Function in Individuals With Late-onset Pompe Disease, Whether or Not Receiving Enzyme Replacement TherapyPompe DiseaseDrug: AlbuterolDuke UniversityNULLCompleted18 YearsN/AAll8Phase 1United States
122NCT01526785
(ClinicalTrials.gov)
March 20122/2/2012A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe DiseaseA Phase 4 Open Label, Prospective Study in Patients With Pompe Disease to Evaluate The Efficacy and Safety of Alglucosidase Alfa Produced at the 4000L ScalePompe DiseaseDrug: Alglucosidase alfaGenzyme, a Sanofi CompanyNULLTerminated1 YearN/AAll113Phase 4United States
123NCT00566878
(ClinicalTrials.gov)
March 20121/12/2007Pompe Lactation Sub-RegistryA Sub-Registry to Determine the Presence of Alglucosidase Alfa in Breast Milk From Women With Pompe Disease Treated With Alglucosidase Alfa.Glycogen Storage Disease;Pompe DiseaseBiological: alglucosidase alfaGenzyme, a Sanofi CompanyNULLRecruitingN/AN/AFemale5N/AUnited States
124EUCTR2010-020611-36-NL
(EUCTR)
13/01/201223/08/2011A study to evaluate muscle tissue, blood, urine and imaging assessments in patients with Late-Onset Pompe Disease treated with alglucosidase alfa.A Phase 4 Prospective Exploratory Muscle Biopsy, Biomarker, and Imaging Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa - Exploratory Muscle Biopsy Assessment Study (EMBASSY) Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 14.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Myozyme
Product Name: Myozyme
Product Code: not applicable
INN or Proposed INN: ALGLUCOSIDASE ALFA
Other descriptive name: rhGAA
Genzyme Europe B.V.NULLNot RecruitingFemale: yes
Male: yes
15Phase 4United States;Denmark;Germany;Netherlands;United Kingdom
125EUCTR2011-002154-32-GB
(EUCTR)
07/12/201107/10/2011Drug-drug interaction study between AT2220 and ERTAN OPEN-LABEL MULTI-CENTER, INTERNATIONAL STUDY TO INVESTIGATE DRUG-DRUG INTERACTIONS BETWEEN AT2220 AND ALGLUCOSIDASE ALFA IN PATIENTS WITH POMPE DISEASE Pompe Disease
MedDRA version: 14.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Duvoglustat hydrochloride
Product Code: AT2220
INN or Proposed INN: Duvoglustat hydrochloride
Other descriptive name: 1-deoxynojirimycin hydrochloride
Product Name: Duvoglustat hydrochloride
Product Code: AT2220
INN or Proposed INN: Duvoglustat hydrochloride
Other descriptive name: 1-deoxynojirimycin hydrochloride
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
22United States;France;Canada;United Kingdom
126NCT01380743
(ClinicalTrials.gov)
October 31, 201123/6/2011Drug-drug Interaction StudyAn Open-Label, Multi-Center, International Study to Investigate Drug-Drug Interactions Between AT2220 and Alglucosidase Alfa in Patients With Pompe DiseasePompe DiseaseDrug: duvoglustat;Drug: rhGAAAmicus TherapeuticsNULLCompleted18 Years65 YearsAll25Phase 2United States;Canada;France;United Kingdom
127EUCTR2010-020611-36-GB
(EUCTR)
12/10/201126/05/2011A study to evaluate muscle tissue, blood, urine and imaging assessments in patients with Late-Onset Pompe Disease treated with alglucosidase alfa.A Phase 4 Prospective Exploratory Muscle Biopsy, Biomarker, and Imaging Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa - Exploratory Muscle Biopsy Assessment Study (EMBASSY) Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 14.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Myozyme
Product Name: Myozyme
Product Code: not applicable
INN or Proposed INN: ALGLUCOSIDASE ALFA
Other descriptive name: rhGAA
Genzyme Europe B.V.NULLNot RecruitingFemale: yes
Male: yes
15Phase 4United States;Netherlands;Germany;United Kingdom
128EUCTR2010-020611-36-DE
(EUCTR)
28/09/201130/06/2011A study to evaluate muscle tissue, blood, urine and imaging assessments in patients with Late-Onset Pompe Disease treated with alglucosidase alfa.A Phase 4 Prospective Exploratory Muscle Biopsy, Biomarker, and Imaging Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa - Exploratory Muscle Biopsy Assessment Study (EMBASSY) Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 14.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Myozyme
Product Name: Myozyme
Product Code: not applicable
INN or Proposed INN: ALGLUCOSIDASE ALFA
Other descriptive name: rhGAA
Genzyme Europe B.V.NULLNot RecruitingFemale: yes
Male: yes
16Phase 4United States;Netherlands;Germany;United Kingdom
129NCT01435772
(ClinicalTrials.gov)
August 15, 20118/9/2011Extension Study for Patients Who Have Participated in a BMN 701 StudyA Long-Term Study for Extended BMN 701 Treatment of Patients With Pompe Disease Who Have Participated in a BMN 701 StudyPompe DiseaseBiological: BMN 701BioMarin PharmaceuticalNULLTerminated13 YearsN/AAll21Phase 2United States;Australia;France;Germany;New Zealand;United Kingdom
130EUCTR2010-023561-22-GB
(EUCTR)
15/08/201111/04/2011A Study of the Safety and Tolerability of BMN 701 in Patients with Late-onset Pompe DiseaseA Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant human GAA) in Patients with Late-onset Pompe Disease - A Phase 1/2 Study of BMN 701 in Patients with Late-onset Pompe Disease Pompe disease
MedDRA version: 13.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 701
Product Code: BMN 701
Other descriptive name: GILT-rhGAA
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
28Phase 1;Phase 2United States;Germany;United Kingdom
131NCT01288027
(ClinicalTrials.gov)
June 201127/1/2011Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase AlfaA Phase 4 Prospective Exploratory Muscle Biopsy, Biomarker, and Imaging Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase AlfaPompe Disease (Late-Onset);Glycogen Storage Disease Type II (GSD II);Glycogenesis 2 Acid Maltase DeficiencyBiological: Alglucosidase AlfaGenzyme, a Sanofi CompanyNULLCompleted18 YearsN/AAll16Phase 4United States;Germany;Netherlands;United Kingdom
132EUCTR2010-024647-32-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
133NCT01230801
(ClinicalTrials.gov)
January 17, 201127/10/2010Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe DiseaseA Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant Human GAA) in Patients With Late-onset Pompe DiseasePompe DiseaseBiological: BMN 701BioMarin PharmaceuticalNULLCompleted13 YearsN/AAll22Phase 1/Phase 2United States;Australia;France;Germany;United Kingdom
134NCT00976352
(ClinicalTrials.gov)
September 201013/7/2009Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe DiseasePhase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV-GAA) Gene Vector in Patients With Pompe DiseasePompe DiseaseDrug: rAAV1-CMV-GAA (study agent) Administration;Other: RMSTUniversity of FloridaNational Heart, Lung, and Blood Institute (NHLBI)Completed2 Years18 YearsAll9Phase 1/Phase 2United States
135NCT00701129
(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
136EUCTR2008-002302-18-DE
(EUCTR)
16/02/200926/09/2008An open-label, multicenter, study to evaluate the safety, tolerability, pharmacodynamics, and pharmacokinetics of three dosing regimens of oral AT2220 in patients with Pompe disease - Pompe Disease
MedDRA version: 9.1;Level: LLT;Classification code 10036143;Term: Pompe's disease
Product Code: AT2220
Other descriptive name: 1-deoxynojirimycin hydrochloride
Product Code: AT2220
Other descriptive name: 1-deoxynojirimycin hydrochloride
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
18United Kingdom;Germany
137EUCTR2008-008293-31-NL
(EUCTR)
10/02/200916/12/2008Early introduction in the Netherlands of alglucosidase alfa manufactured in a 4000 liter bioreactorEarly introduction in the Netherlands of alglucosidase alfa manufactured in a 4000 liter bioreactor Glycogen Storage Disease Type II (Pompe's Disease)
MedDRA version: 9.1;Level: LLT;Classification code 10036143;Term: Pompe's disease
Product Name: alglucosidase alfa
INN or Proposed INN: alglucosidase alfa
Other descriptive name: alglucosidase alfa
Erasmus MC SophiaNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Netherlands
138EUCTR2008-002302-18-GB
(EUCTR)
18/12/200807/01/2009An open-label, multicenter, study to evaluate the safety, tolerability, pharmacodynamics, and pharmacokinetics of three dosing regimens of oral AT2220 in patients with Pompe disease - Pompe Disease
MedDRA version: 9.1;Level: LLT;Classification code 10036143;Term: Pompe's disease
Product Code: AT2220
Other descriptive name: 1-deoxynojirimycin hydrochloride
Product Code: AT2220
Other descriptive name: 1-deoxynojirimycin hydrochloride
Amicus Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
18Germany;United Kingdom
139NCT00701701
(ClinicalTrials.gov)
December 14, 200817/6/2008Immune Tolerance Induction StudyAn Exploratory Study of the Safety and Efficacy of Immune Tolerance Induction (ITI) in Patients With Pompe Disease Who Have Previously Received MyozymePompe Disease;Glycogen Storage Disease Type II (GSD-II);Glycogenesis 2 Acid Maltase DeficiencyBiological: Myozyme® (alglucosidase alfa)Genzyme, a Sanofi CompanyNULLTerminated1 MonthN/AAll4Phase 4United States;Israel;Canada
140NCT00688597
(ClinicalTrials.gov)
December 8, 200830/5/2008Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe DiseaseAn Open-Label, Multicenter Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Three Dosing Regimens of Oral AT2220 in Patients With Pompe DiseasePompe DiseaseDrug: DuvoglustatAmicus TherapeuticsNULLTerminated18 Years74 YearsAll3Phase 2United States;Australia;Canada;France;Germany;Netherlands;United Kingdom
141NCT01451879
(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
142NCT00486889
(ClinicalTrials.gov)
August 26, 200813/6/2007Growth and Development Study of Alglucosidase Alfa.A Long-term Study to Evaluate Growth and Development Outcomes in Patients With Infantile-Onset Pompe Disease Who Are Receiving Alglucosidase Alfa.Pompe Disease;Glycogen Storage Disease Type II (GSD-II);Acid Maltase Deficiency DiseaseBiological: alglucosidase alfaGenzyme, a Sanofi CompanyNULLActive, not recruitingN/A24 MonthsAll30Phase 4United States
143EUCTR2006-003644-31-DE
(EUCTR)
03/08/200711/07/2007An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704 Glycogen Storage Disease type II (Pompe's disease)
MedDRA version: 9.1;Level: LLT;Classification code 10036143;Term: Pompe's disease
Trade Name: Myozyme
Product Name: Myozyme
Product Code: rhGAA, acid alpha glucosidase
INN or Proposed INN: Alglucosidase alfa
Other descriptive name: alglucosidase alpha
Genzyme Europe B.V.NULLNot RecruitingFemale: yes
Male: yes
82Germany;Netherlands;France
144EUCTR2006-003644-31-NL
(EUCTR)
01/08/200704/06/2007An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704 Glycogen Storage Disease type II (Pompe's disease)
MedDRA version: 9.1;Level: LLT;Classification code 10036143;Term: Pompe's disease
Trade Name: Myozyme
Product Name: Myozyme
Product Code: rhGAA, acid alpha glucosidase
INN or Proposed INN: Alglucosidase alfa
Other descriptive name: alglucosidase alpha
Genzyme Europe BVNULLNot RecruitingFemale: yes
Male: yes
82Germany;Netherlands;France
145EUCTR2005-002759-42-DE
(EUCTR)
18/07/200724/07/2009A Randomized, Double-Blind, Multicenter, Multinational, Placebo-Controlled Study of the Safety, Efficacy, and Pharmacokinetics of Myozyme, Recombinant Human Acid alpha-Glucosidase (rhGAA), Treatment in Patients with Late-Onset Pompe Disease - LOTSA Randomized, Double-Blind, Multicenter, Multinational, Placebo-Controlled Study of the Safety, Efficacy, and Pharmacokinetics of Myozyme, Recombinant Human Acid alpha-Glucosidase (rhGAA), Treatment in Patients with Late-Onset Pompe Disease - LOTS Glycogen Storage Disease type II (Pompe's disease)
MedDRA version: 9.1;Level: LLT;Classification code 10036143;Term: Pompe's disease
Trade Name: Myozyme
Product Name: Myozyme
INN or Proposed INN: Alglucosidase alfa
Other descriptive name: alglucosidase alpha
Genzyme Europe BVNULLNot RecruitingFemale: yes
Male: yes
90Germany
146NCT00520143
(ClinicalTrials.gov)
July 200721/8/2007Alglucosidase Alfa Temporary Access ProgramAlglucosidase Alfa Temporary Access ProgramGlycogen Storage Disease Type II (GSD-II);Pompe Disease (Late-Onset);Acid Maltase Deficiency Disease;Glycogenosis 2Biological: alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])Genzyme, a Sanofi CompanyNULLApproved for marketing18 YearsN/ABothN/AUnited States
147EUCTR2007-001375-11-NL
(EUCTR)
16/05/200724/05/2007Effects and health economic aspects of enzyme therapy in children and adults with Pompe disease Long-term follow-up of patients receiving commercially available Myozyme - not applicableEffects and health economic aspects of enzyme therapy in children and adults with Pompe disease Long-term follow-up of patients receiving commercially available Myozyme - not applicable Pompe disease (glycogen storage disease type II) is a genetic, lysosomal storage disorder with a frequency of 1 in 40.000 newborns. The disease is caused by deficiency of alpha-glucosidase, a lysosomal hydrolase involved in the degradation of glycogen.Trade Name: Myozyme
Product Name: Myozyme
Product Code: MYOZYME®
NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Netherlands
148NCT00483379
(ClinicalTrials.gov)
May 20076/6/2007High Dose or High Dose Frequency Study of Alglucosidase AlfaAn Exploratory, Open-Label Study of the Safety and Efficacy of High Dose or High Dosing Frequency Alglucosidase Alfa Treatment in Patients With Pompe Disease Who Do Not Have an Optimal Response to the Standard Dose RegimenPompe Disease;Glycogen Storage Disease Type II (GSD-II);Glycogenesis 2 Acid Maltase DeficiencyBiological: alglucosidase alfaGenzyme, a Sanofi CompanyNULLCompleted6 MonthsN/AAll13Phase 4United States;Australia;Canada
149NCT00455195
(ClinicalTrials.gov)
March 200730/3/2007Late-Onset Treatment Study Extension ProtocolAn Open-Label Extension Study of Patients With Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704Pompe Disease (Late-Onset);Glycogen Storage Disease Type II (GSD-II);Glycogenesis Type II;Acid Maltase Deficiency (AMD)Biological: alglucosidase alfaGenzyme, a Sanofi CompanyNULLCompleted8 YearsN/AAll81Phase 4United States;Australia;Canada;France;Netherlands
150EUCTR2006-003644-31-FR
(EUCTR)
18/01/200711/12/2006An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704 Glycogen Storage Disease type II (Pompe's disease)Trade Name: Myozyme
Product Name: Myozyme
Product Code: rhGAA, acid alpha glucosidase
INN or Proposed INN: Alglucosidase alfa
Other descriptive name: alglucosidase alpha
Genzyme Europe BVNULLNot RecruitingFemale: yes
Male: yes
90Phase 4France;Germany;Netherlands
151NCT00268944
(ClinicalTrials.gov)
December 200522/12/2005Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory SupportProspective, Open-label, Single-arm, Exploratory Study of the Effect and Safety of rhGAA in Patients With Advanced Late-onset Pompe Disease Who Are Receiving Respiratory SupportPompe Disease (Late-onset);Glycogen Storage Disease Type II (GSD-II);Acid Maltase Deficiency Disease;Glycogenosis 2Biological: MyozymeGenzyme, a Sanofi CompanyNULLCompleted18 YearsN/ABoth5Phase 3France
152NCT00158600
(ClinicalTrials.gov)
September 20058/9/2005A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe DiseaseRandomized, Double-Blind, Placebo-Controlled Study of the Safety, Efficacy and Pharmacokinetics of Myozyme in Patients With Late-Onset Pompe Disease.Pompe Disease (Late-onset);Glycogen Storage Disease Type II (GSD-II);Acid Maltase Deficiency Disease;Glycogenosis 2Biological: alglucosidase alfa;Drug: PlaceboGenzyme, a Sanofi CompanyNULLCompleted8 YearsN/AAll90Phase 3United States;France;Netherlands
153EUCTR2005-002829-31-GB
(EUCTR)
31/08/200507/07/2005Full title of the trial : A Single Centre, Open-Label, Bridging Study of the Safety, Pharmacokinetics and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) Treatment in Patients with Late-Onset Pompe Disease (Glycogen Storage Disease Type II) - mini LOTSFull title of the trial : A Single Centre, Open-Label, Bridging Study of the Safety, Pharmacokinetics and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) Treatment in Patients with Late-Onset Pompe Disease (Glycogen Storage Disease Type II) - mini LOTS Glycogen Storage Disease type II (Pompe´s disease)Product Name: Myozyme
Product Code: rhGAA, acid alpha glucosidase
INN or Proposed INN: Alglucosidase alfa
Other descriptive name: alglucosidase alpha
Genzyme Europe BVNULLNot Recruiting Female: yes
Male: yes
5 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noBelgium;United Kingdom
154EUCTR2005-001629-27-DE
(EUCTR)
03/08/200509/06/2005A Long-term Continuation Study of Patients with Infantile-Onset Pompe Disease who were previously enrolled in Protocol AGLU01602A Long-term Continuation Study of Patients with Infantile-Onset Pompe Disease who were previously enrolled in Protocol AGLU01602 Pompe disease is a rare metabolic muscle disease inherited in an autosomal recessive fashion. Pompe disease is caused by a deficiency of GAA, which is needed for the degradation of lysosomal glycogen. Pompe disease is characterized by organelle bound (lysosomal) accumulation of glycogen in many body tissues. In general, there is an inverse correlation between the amount of residual GAA activity in patients with Pompe disease and the severity of the disease.Product Name: Myozyme
INN or Proposed INN: Alglucosidase alfa
Other descriptive name: n.a.
Genzyme Europe BVNULLNot RecruitingFemale: yes
Male: yes
17Germany;Italy
155EUCTR2005-001629-27-IT
(EUCTR)
21/06/200521/06/2005A Long-term Continuation Study of Patients With Infantile-Onset Pompe Disease WhoWere Previously Enrolled in Protocol AGLU01602A Long-term Continuation Study of Patients With Infantile-Onset Pompe Disease WhoWere Previously Enrolled in Protocol AGLU01602 Pompe disease or glycogenosis type II
MedDRA version: 6.1;Level: HLT;Classification code 10024579
Product Name: Myozyme
Product Code: NA
Other descriptive name: NA
GENZYMENULLNot RecruitingFemale: yes
Male: yes
Germany;Italy
156NCT00125879
(ClinicalTrials.gov)
June 20051/8/2005Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602A Long-Term Continuation Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602Glycogen Storage Disease Type IIBiological: MyozymeGenzyme, a Sanofi CompanyNULLCompletedN/AN/ABoth16Phase 2/Phase 3United States;France;Germany;Israel;Italy;Netherlands;Taiwan
157NCT00250939
(ClinicalTrials.gov)
February 20058/11/2005A Study of rhGAA in Patients With Late-Onset Pompe DiseaseSingle-center, Open-label Study of Safety, Pharmacokinetics and Efficacy of rhGAA in Patients With Late-Onset Pompe DiseasePompe Disease (Late-onset);Glycogen Storage Disease Type II (GSD-II);Acid Maltase Deficiency Disease;Glycogenosis 2Biological: MyozymeGenzyme, a Sanofi CompanyNULLCompleted5 Years18 YearsBoth5Phase 2Netherlands
158NCT00074932
(ClinicalTrials.gov)
November 200423/12/2003Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe DiseaseExpanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe DiseaseGlycogen Storage Disease Type II;Glycogenosis 2Biological: MyozymeGenzyme, a Sanofi CompanyNULLCompletedN/AN/ABoth9N/AUnited States
159EUCTR2004-002168-59-IT
(EUCTR)
14/10/200403/01/2005An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy,Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid alpha-Glucosidase (rhGAA) Treatment in Patients > 6 and <= 36 Months Old with Infantile-OnsetPompe Disease (Glycogen Storage Disease Type II)An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy,Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid alpha-Glucosidase (rhGAA) Treatment in Patients > 6 and <= 36 Months Old with Infantile-OnsetPompe Disease (Glycogen Storage Disease Type II) Treatment for Type II Glycogenosis
MedDRA version: 6.1;Level: PT;Classification code 10053185
Product Name: Recombinant human acid alfa-glucosidase (rhGAA)
Product Code: NA
INN or Proposed INN: alglucosidase alfa
Product Name: Recombinant human acid alfa-glucosidase (rhGAA)
Product Code: NA
INN or Proposed INN: alglucosidase alfa
GENZYMENULLNot RecruitingFemale: yes
Male: yes
20Italy
160NCT00074919
(ClinicalTrials.gov)
December 200323/12/2003Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe DiseaseExpanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe DiseaseGlycogen Storage Disease Type II;Glycogenosis 2Biological: alglucosidase alfaGenzyme, a Sanofi CompanyNULLApproved for marketingN/AN/ABothN/AUnited States
161NCT00765414
(ClinicalTrials.gov)
April 20032/10/2008Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.An Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid a-Glucosidase (rhGAA) Given as Enyzme Replacement Therapy to a Single Patient With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy StudiesPompe Disease Late-Onset;Glycogen Storage Disease Type II GSD IIBiological: MyozymeGenzyme, a Sanofi CompanyNULLCompleted16 YearsN/AFemale1Phase 2United States
162NCT00763932
(ClinicalTrials.gov)
April 200330/9/2008Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) StudiesA Multicenter, Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid a-Glucosidase (rhGAA) in Patients With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy StudiesPompe Disease Infantile-Onset;Glycogen Storage Disease Type IIBiological: MyozymeGenzyme, a Sanofi CompanyNULLCompleted18 YearsN/ABoth7Phase 2United States;France;South Africa
163NCT00059280
(ClinicalTrials.gov)
April 200322/4/2003A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe DiseaseAn Open-label, Multicenter, Multinational Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid Alpha-glucosidase Treatment in Patients Less Than 6 Months Old With Infantile-onset Pompe DiseaseGlycogen Storage Disease Type IIBiological: MyozymeGenzyme, a Sanofi CompanyNULLCompletedN/A26 WeeksBoth16Phase 2/Phase 3United States;France;Israel;Taiwan;United Kingdom
164NCT00053573
(ClinicalTrials.gov)
February 200331/1/2003rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of rhGAA Treatment in Patients Greater Than 6 Months and Less Than or Equal to 36 Months Old With Infantile-Onset GSD-IIGlycogen Storage Disease Type II;Pompe Disease;Acid Maltase Deficiency Disease;Glycogenosis 2Biological: MyozymeGenzyme, a Sanofi CompanyNULLCompleted6 Months36 MonthsBoth20Phase 1/Phase 2United States;France;Israel;United Kingdom
165NCT00051935
(ClinicalTrials.gov)
January 200317/1/2003A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type IIOpen-Label, Pilot Study of the Safety, Pharmacokinetics and Pharmacodynamics of Recombinant Human Acid Alpha-Glucosidase (rhGAA) as Enzyme Replacement Therapy in Siblings With Glycogen Storage Disease Type II (GSD-II).Glycogen Storage Disease Type II;Pompe Disease;Acid Maltase Deficiency Disease;Glycogenosis 2Drug: Alglucosidase alfaGenzyme, a Sanofi CompanyNULLCompletedN/AN/ABoth2Phase 2United States
166NCT00025896
(ClinicalTrials.gov)
May 200131/10/2001Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe DiseaseA Prospective Multinational, Multicenter, Clinical Trial of the Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) in Cross-Reacting Immunologic Material-Positive Patients With Classical Infantile Pompe DiseasePompe Disease;Glycogen Storage Disease Type II;Acid Maltase Deficiency Disease;Glycogenosis 2Drug: recombinant human acid alpha-glucosidase (rhGAA)Genzyme, a Sanofi CompanyNULLCompletedN/AN/ABoth8Phase 2United States
167EUCTR2013-002257-30-GB
(EUCTR)
21/10/2013A dose finding study with intravenous administration of duvoglustat hydrochloride (AT2220) and Myozyme in Pompe patientsAN OPEN-LABEL SAFETY AND DOSE-FINDING STUDY OF INTRAVENOUS DUVOGLUSTAT CO-ADMINISTERED WITH RECOMBINANT HUMAN ACID a-GLUCOSIDASE IN SUBJECTS WITH POMPE DISEASE Pompe disease
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: duvoglustat hydrochloride
Product Code: AT2220
INN or Proposed INN: duvoglustat HCl
Trade Name: Myozyme
Product Name: Myozyme
INN or Proposed INN: ALGLUCOSIDASE ALFA
Amicus Therapeutics, Inc.NULLNot Recruiting Female: yes
Male: yes
24 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Netherlands;United Kingdom
168EUCTR2015-000583-34-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
169EUCTR2021-004047-25-Outside-EU/EEA
(EUCTR)
27/07/2021Evaluate Efficacy and Safety in Chinese Patients with Infantile-Onset Pompe Disease with One Year Alglucosidase Alfa TreatmentA Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients with Infantile-Onset Pompe Disease with One Year Alglucosidase Alfa Treatment - APOLLO-IOPD Infantile-Onset Pompe Disease
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: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: MYOZYME®
Product Name: Alglucosidase Alfa
Product Code: GZ419829
INN or Proposed INN: ALGLUCOSIDASE ALFA
Sanofi(China) Investment Co Ltd, Shanghai BranchNULLNAFemale: yes
Male: yes
Phase 4China
170EUCTR2016-000942-77-PL
(EUCTR)
23/11/2017Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe DiseaseA phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and safety of repeated biweekly infusions of Avalglucosidase Alfa (neoGAA ,GZ402666) and alglucosidase alfa in treatment-naïve patients with late onset Pompe disease Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA or avalglucosidase alfa
Product Code: GZ402666
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLNAFemale: yes
Male: yes
100Phase 3United States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Belgium;Poland;Bulgaria;Germany;Japan;Sweden
171EUCTR2021-002590-26-FR
(EUCTR)
16/08/2021Avalglucosidase alfa French post-trial access for participants with Pompe diseaseA French multicenter Phase 4 open label extension study of long term safety and efficacy in patients with Pompe disease who previously participated in avalglucosidase development studies in France - PTA Avalglucosidase 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: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Avalglucosidase alfa
Product Code: GZ402666
INN or Proposed INN: Avalglucosidase Alfa
Other descriptive name: RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN
Sanofi-Aventis Recherche & DéveloppementNULLNAFemale: yes
Male: yes
18Phase 4France
172EUCTR2015-000512-18-Outside-EU/EEA
(EUCTR)
02/03/2015A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe DiseaseA Phase 4 Open Label, Prospective Study in Patients With Pompe Disease to Evaluate The Efficacy and Safety of Alglucosidase Alfa Produced at the 4000L Scale 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: Lumizyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Other descriptive name: rhGAA
Genzyme Corporation IncNULLNAFemale: yes
Male: yes
114Phase 4United States
173EUCTR2015-000582-31-Outside-EU/EEA
(EUCTR)
15/04/2015An Exploratory, Open-Label Study of the Safety and Efficacy of High Dose or High Dosing Frequency Myozyme® (alglucosidase alfa) Treatment in Patients with Pompe Disease Who Do Not Have an Optimal Response to the Standard Dose RegimenAn Exploratory, Open-Label Study of the Safety and Efficacy of High Dose or High Dosing Frequency Myozyme® (alglucosidase alfa) Treatment in Patients with Pompe Disease Who Do Not Have an Optimal Response to the Standard Dose Regimen 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]
Trade Name: Myozyme 50 mg powder for concentrate for solution for infusion
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme Corporation IncNULLNAFemale: yes
Male: yes
14United States;Australia;Canada
174EUCTR2005-002829-31-BE
(EUCTR)
09/02/2006Full title of the trial : A Single Centre, Open-Label, Bridging Study of the Safety, Pharmacokinetics and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) Treatment in Patients with Late-Onset Pompe Disease (Glycogen Storage Disease Type II) - mini LOTSFull title of the trial : A Single Centre, Open-Label, Bridging Study of the Safety, Pharmacokinetics and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) Treatment in Patients with Late-Onset Pompe Disease (Glycogen Storage Disease Type II) - mini LOTS Glycogen Storage Disease type II (Pompe´s disease)Product Name: Myozyme
Product Code: rhGAA, acid alpha glucosidase
INN or Proposed INN: Alglucosidase alfa
Other descriptive name: alglucosidase alpha
Genzyme Europe BVNULLNot RecruitingFemale: yes
Male: yes
5Phase 2Belgium;United Kingdom
175EUCTR2016-003475-21-DE
(EUCTR)
09/03/2017A Study to Assess Safety and Efficacy of NeoGAA Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated with Alglucosidase AlfaAn Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of NeoGAA (GZ402666) in Patients with Infantile-onset Pompe Disease Treated with Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA
Product Code: GZ402666
INN or Proposed INN: -
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLNot Recruiting Female: yes
Male: yes
34 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;United States;Taiwan;Germany;Japan;United Kingdom
176EUCTR2016-003475-21-FR
(EUCTR)
02/06/2017A Study to Assess Safety and Efficacy of NeoGAA Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated with Alglucosidase AlfaAn Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of NeoGAA (GZ402666) in Patients with Infantile-onset Pompe Disease Treated with Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response Pompe disease (acid alpha-glucosidase deficiency)
MedDRA version: 20.0;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: neoGAA
Product Code: GZ402666
INN or Proposed INN: -
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Trade Name: MYOZYME®
Product Name: Myozyme®
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLNAFemale: yes
Male: yes
34Phase 2United States;France;Taiwan;Germany;Japan;United Kingdom
177EUCTR2015-000584-14-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
178EUCTR2020-004686-39-BE
(EUCTR)
02/04/2021Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase AlfaAn Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of avalglucosidase alfa in Treatment naïve Pediatric Participants with Infantile-Onset Pompe Disease (IOPD) - Baby-COMET 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: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Avalglucosidase Alfa
Product Code: GZ402666
INN or Proposed INN: avalglucosidase alfa
Other descriptive name: RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN
Sanofi-Aventis Recherche & DéveloppementNULLNAFemale: yes
Male: yes
18Phase 3United States;France;Taiwan;Belgium;Netherlands;Germany;United Kingdom;Italy;China
179EUCTR2021-005552-11-Outside-EU/EEA
(EUCTR)
07/03/2022Growth and Development Study of Alglucosidase Alfa.A Long-term Study to Evaluate Growth and Development Outcomes in Patients With Infantile-Onset Pompe Disease Who Are Receiving Alglucosidase Alfa. Pompe disease
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: Diseases [C] - Nervous System Diseases [C10]
Trade Name: Myozyme
INN or Proposed INN: ALGLUCOSIDASE ALFA
Genzyme CorporationNULLNAFemale: yes
Male: yes
Phase 4United States
180EUCTR2020-004686-39-DE
(EUCTR)
21/01/2021Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of enzyme replacement therapy (ERT) With Avalglucosidase AlfaAn Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of avalglucosidase alfa in Treatment naïve Pediatric Participants withInfantile-Onset Pompe Disease (IOPD) - Baby-COMET 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: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Avalglucosidase Alfa
Product Code: GZ402666
INN or Proposed INN: avalglucosidase alfa
Other descriptive name: RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN
Sanofi-Aventis Recherche & DéveloppementNULLNAFemale: yes
Male: yes
18Phase 3United States;France;Taiwan;Belgium;Netherlands;Germany;United Kingdom;Italy;China