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 256. 筋型糖原病 [臨床試験数:81,薬物数:71(DrugBank:20),標的遺伝子数:32,標的パスウェイ数:97] 

Searched query = "Muscle glycogenosis", "Muscular glycogenosis", "Muscle glycogen storage disease", "Muscular glycogen storage disease", "Glycogen storage disease type 0", "GSD0", "Glycogen synthase deficiency", "Glycogen storage disease type II", "GSDII", "Pompe disease", "Alpha-1,4-glucosidase acid deficiency", "Glycogen storage disease type III", "GSDIII", "Cori disease", "Glycogen debranching enzyme deficiency", "Glycogen storage disease type IV", "GSDIV", "Andersen disease", "Glycogen-branching enzyme deficiency", "GBED", "Glycogen storage disease type V", "GSDV", "McArdle disease", "Muscle phosphorylase deficiency", "Muscular phosphorylase deficiency", "Glycogen storage disease type VII", "GSDVII", "Tarui disease", "Phosphofructokinase deficiency", "PFK deficiency", "Glycogen storage disease type IX", "GSDIX", "Phosphorylase kinase deficiency", "Phosphoglycerate kinase deficiency", "PGK deficiency", "Glycogen storage disease type X", "GSDX", "Phosphoglycerate mutase deficiency", "Glycogen storage diseass type XI", "GSDXI", "Kanno disease", "Lactate dehydrogenase deficiency", "Glycogen storage diseass type XII", "GSDXII", "Aldolase A deficiency", "Glycogen storage diseass type XIII", "GSDXIII", "Beta-enolase deficiency", "Glycogen storage diseass type XIV", "GSDXIV", "Phosphoglucomutase deficiency", "Glycogen storage diseass type XV", "GSDXV", "Glycogenin 1 deficiency"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/21/2019, 11/20/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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Inclusion_
agemin
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PhaseCountries
1NCT04138277December 20194 November 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 TherapeuticsNot recruiting18 YearsN/AAll110Phase 3
2NCT04094948October 1, 201930 September 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 UniversityNot recruiting18 YearsN/AAll40Phase 2United States
3NCT03911505August 20192 September 2019A Study to Evaluate the PK, Safety, Efficacy, and PD With ATB200/AT2221 in LOPD Subjects Aged 12 to <18An Open-label Study of the Pharmacokinetics, Safety, Efficacy, and Pharmacodynamics of ATB200/AT2221 in Pediatric Subjects Aged 12 to < 18 Years With Late-onset Pompe DiseasePompe Disease (Late-onset)Biological: ATB200;Drug: AT2221Amicus TherapeuticsRecruiting12 Years17 YearsAll14Phase 3United States
4NCT03945370May 6, 201920 May 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, DenmarkNot recruiting18 YearsN/AAll10N/A
5NCT03865836March 1, 201918 March 2019Expanded Access for ATB200/AT2221 for the Treatment of LOPDExpanded Access for ATB200/AT2221 for the Treatment of Late Onset Pompe Disease Patients That do Not Qualify for Pivotal Study ATB200-03Pompe DiseaseBiological: ATB200;Drug: AT2221Amicus TherapeuticsNot recruiting1 YearN/AAllN/A
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
Recruitment_
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Inclusion_
agemin
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agemax
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PhaseCountries
6EUCTR2018-000755-40-HU28/02/201930 April 2019A 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/PlaceboAdult 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
Pharmaceutical Form: Powder for concentrate for solution for injection/infusion
INN or Proposed INN: ALGLUCOSIDASE ALFA
CAS Number: 420784-05-0
Current Sponsor code: ATB200
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 15-
Product Name: AT2221
Product Code: AT2221
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: MIGLUSTAT
CAS Number: 72599-27-0
Current Sponsor code: AT2221
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 65-
Pharmaceutical form of the placebo: Capsule, hard
Route of administration of the placebo: Oral use
Trade Name: Myozyme
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: ALGLUCOSIDASE ALFA
CAS Number: 420784-05-0
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 5-
Amicus Therapeutics, Inc.Authorised Female: yes
Male: yes
110Phase 3United States;Taiwan;Slovakia;Greece;Spain;Israel;Italy;France;Australia;Denmark;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Brazil;Belgium;Poland;Romania;Bulgaria;Germany;Japan;New Zealand;Sweden
7NCT03687333December 4, 201818 December 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 CompanyRecruitingN/A12 MonthsAll10Phase 4China
8NCT03729362December 4, 20184 November 2019PROPEL 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 TherapeuticsRecruiting18 YearsN/AAll100Phase 3United States;Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Spain;Taiwan;United Kingdom
9ChiCTR18000185142018-11-0624 September 2018A 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 TreatmentGenetic and MetabolismCase series:Alglucosidase Alfa treatment;Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of MedicineNot Recruiting01BothCase series:10;Phase 4 studyChina
10NCT03533673November 1, 201828 October 2019AAV2/8-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/AAll6Phase 1/Phase 2United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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PhaseCountries
11EUCTR2017-004370-34-HU29/08/201810 October 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 TherapyLate-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]
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: ALGLUCOSIDASE ALFA
CAS Number: 420784-05-0
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 20-
Amicus Therapeutics, Inc.Not 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
12NCT03642860August 15, 20189 October 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 IncRecruiting15 Years85 YearsAll20Phase 2Denmark
13EUCTR2017-004153-17-DK03/05/201814 May 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 GlycogenosesCori Forbe's Disease Also called: glycogen storage disease Type III or debrancher deficiency. Tarui's disease Also 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 Level: PT 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 System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0 Level: LLT 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
Pharmaceutical Form: Oral liquid
INN or Proposed INN: Not available
CAS Number: 210-647-2
Current Sponsor code: UX0007
Other descriptive name: TRIHEPTANOIN
Concentration unit: % percent
Concentration type: equal
Concentration number: 100-
Pharmaceutical form of the placebo: Oral liquid
Route of administration of the placebo: Oral use
Copenhagen Neuromuscular CenterAuthorisedFemale: yes
Male: yes
20Phase 2Denmark
14EUCTR2017-004370-34-BE09/04/20187 January 2019A 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 TherapyLate-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]
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: ALGLUCOSIDASE ALFA
CAS Number: 420784-05-0
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 20-
Amicus Therapeutics, Inc.Not 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
15NCT02240407October 17, 201711 June 2019Re-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 FloridaLacerta Therapeutics, IncRecruiting18 Years50 YearsAll7Phase 1United States
No.TrialIDDate_
enrollement
Last_Refreshed_
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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Inclusion_
agemin
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size
PhaseCountries
16NCT03019406October 12, 201728 October 2019A 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: alglucosidase alfa GZ419829;Drug: avalglucosidase alfa GZ402666Genzyme, a Sanofi CompanyNot recruiting6 Months17 YearsAll22Phase 2United States;France;Japan;Taiwan;United Kingdom
17NCT02898753June 21, 201711 November 2019VAL-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, LLCNot recruiting18 YearsN/AAll12Phase 1/Phase 2United States;United Kingdom
18NCT03045042April 201716 December 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 recruiting2 YearsN/AAll35N/ASpain
19NCT02185651October 201618 December 2018A 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 TherapeuticsNot recruiting18 Years65 YearsAll2Phase 1United States
20NCT02782741October 201611 November 2019Study 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: alglucosidase alfa (GZ419829);Drug: avalglucosidase alfa(GZ402666)SanofiNot 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
No.TrialIDDate_
enrollement
Last_Refreshed_
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
21NCT02919631October 201610 October 2016Triheptanoin in Mc ArdleThe Effect of Triheptanoin in Adults With Mc Ardle Disease (Glycogen Storage Disease Type V)Glycogen Storage Disease Type VDrug: Placebo oil;Drug: TriheptanoinInstitut National de la Santé Et de la Recherche Médicale, FranceRigshospitalet, Denmark;Rigshospitalet, DenmarkNot recruiting18 Years65 YearsBoth12Phase 2
22EUCTR2015-004798-34-GB01/08/201619 November 2018This 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 DISEASEPompe 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
Pharmaceutical Form: Powder for concentrate for solution for injection/infusion
INN or Proposed INN: ALGLUCOSIDASE ALFA
CAS Number: 420784-05-0
Current Sponsor code: ATB200
Concentration unit: mg/l milligram(s)/litre
Concentration type: equal
Concentration number: 15-
Product Code: AT2221
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: MIGLUSTAT
CAS Number: 72599-27-0
Current Sponsor code: AT2221
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 65-
Amicus Therapeutics, Inc.AuthorisedFemale: yes
Male: yes
32Phase 1;Phase 2United States;Australia;Netherlands;Germany;United Kingdom
23EUCTR2015-004798-34-NL25/07/201628 February 2019This 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 DISEASEPompe 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
Pharmaceutical Form: Powder for concentrate for solution for injection/infusion
INN or Proposed INN: ALGLUCOSIDASE ALFA
CAS Number: 420784-05-0
Current Sponsor code: ATB200
Concentration unit: mg/l milligram(s)/litre
Concentration type: equal
Concentration number: 15-
Product Code: AT2221
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: MIGLUSTAT
CAS Number: 72599-27-0
Current Sponsor code: AT2221
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 65-
Amicus Therapeutics, Inc.AuthorisedFemale: yes
Male: yes
32Phase 1;Phase 2United States;Australia;Germany;Netherlands;United Kingdom
24EUCTR2016-000942-77-GB15/07/201616 January 2017Study 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 DiseasePompe 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
Pharmaceutical Form: Powder for concentrate for solution for infusion
CAS Number: 1802558-87-7
Current Sponsor code: GZ402666
Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
Trade Name: MYOZYME®
Product Name: Myozyme®
Pharmaceutical Form: Powder for concentrate for solution for infusion
INN or Proposed INN: ALGLUCOSIDASE ALFA
CAS Number: 420784-05-0
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 50-
Genzyme CorporationAuthorisedFemale: yes
Male: yes
100Phase 3United States;Taiwan;Spain;Turkey;Austria;Russian Federation;Colombia;Italy;United Kingdom;Switzerland;France;Czech Republic;Mexico;Canada;Argentina;Belgium;Brazil;Denmark;Netherlands;Germany;Japan;Korea, Republic of;Sweden
25NCT02824068June 201626 August 2019Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (ATBIG-Pompe-Study)Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 MonthsPompe DiseaseDrug: glucosidase alfaKlinikum der Universitaet MuenchenGenzyme, a Sanofi CompanyRecruiting8 YearsN/AAll100Phase 1Argentina;Brazil;Germany;Italy;Taiwan
No.TrialIDDate_
enrollement
Last_Refreshed_
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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Inclusion_
agemin
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size
PhaseCountries
26NCT02675465January 20166 May 2019First-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 TherapeuticsNot recruiting18 Years75 YearsAll32Phase 1/Phase 2United States;Australia;Germany;Netherlands;New Zealand;United Kingdom
27NCT02357225August 201511 June 2018A 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 FloridaNot recruiting8 Years60 YearsAll2Early Phase 1United States
28NCT02525172August 201525 April 2016Immune 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 HospitalRecruitingN/AN/ABoth8Phase 4Taiwan
29NCT02432768April 201528 October 2019The 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 IncNot recruiting18 Years65 YearsAll22Phase 2Denmark
30NCT01710813March 20, 201528 October 2019Alglucosidase 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 CompanyNot recruitingN/AN/AAll110Phase 1United States;Belgium;Czechia;Germany;Italy;Taiwan;Czech Republic
No.TrialIDDate_
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31NCT03112889January 201512 February 2018Sodium 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, LondonNot recruiting18 Years64 YearsAll8Phase 2Denmark;United Kingdom
32EUCTR2015-000582-31-Outside-EU/EEA10 July 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 RegimenPompe 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
Pharmaceutical Form: Powder for concentrate for solution for infusion
INN or Proposed INN: ALGLUCOSIDASE ALFA
CAS Number: 420784-05-0
Concentration unit: mg/g milligram(s)/gram
Concentration type: equal
Concentration number: 50-
Genzyme Corporation IncNot AvailableFemale: yes
Male: yes
14United States;Australia;Canada
33NCT01410890November 3, 201414 October 2019Pharmacokinetics 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 (Late-Onset);Glycogen Storage Disease Type II (GSD II)Biological: alglucosidase alfaGenzyme, a Sanofi CompanyRecruitingN/AN/AAll20Phase 4United States;Bulgaria;India;Russian Federation;Ukraine;United Kingdom;Germany
34EUCTR2014-001637-88-DK12/08/20142 October 2017Effects 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 diseaseMcArdle 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
Pharmaceutical Form: Prolonged-release tablet
INN or Proposed INN: SODIUM VALPROATE
CAS Number: 1069-66-5
Concentration unit: mg milligram(s)
Concentration type: range
Concentration number: 300-500
John VissingNot RecruitingFemale: yes
Male: yes
15Phase 2Denmark
35EUCTR2013-003321-28-BE12/05/201420 August 2018NeoGAA 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 diseasePompe 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
Pharmaceutical Form: Powder for concentrate for solution for infusion
Current Sponsor code: GZ402666
Other descriptive name: RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
Genzyme CorporationAuthorisedFemale: yes
Male: yes
21Phase 2France;United States;Belgium;Denmark;Netherlands;Germany;Italy;United Kingdom
No.TrialIDDate_
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36EUCTR2012-002933-12-GB07/04/201430 April 2019A 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 2014McArdle 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
Pharmaceutical Form: Prolonged-release tablet
INN or Proposed INN: Sodium valproate
CAS Number: 1069-66-5
Other descriptive name: Valproic acid
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 200-
Trade Name: Epilim Chrono 300 Controlled Released tablets
Product Name: Epilim Chrono 300 Controlled Released tablets
Pharmaceutical Form: Prolonged-release tablet
INN or Proposed INN: Sodium valproate
Current Sponsor code: 11/0090
Other descriptive name: valproic acid
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 300-
Trade Name: Epilim Chrono 500 Controlled Released tablets
Product Name: Epilim Chrono 500 Controlled Released tablets
Pharmaceutical Form: Prolonged-release tablet
INN or Proposed INN: Sodium valproate
Current Sponsor code: 11/0090
Other descriptive name: valproic acid
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 500-
University College London,Not Recruiting Female: yes
Male: yes
8Phase 2United States;Denmark;United Kingdom
37NCT01924845April 201425 June 2018BMN 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 PharmaceuticalNot recruiting18 YearsN/AAll24Phase 3United States;Belgium;France;Germany;Italy;Netherlands;Portugal;United Kingdom
38NCT02032524February 27, 201428 January 2019NeoGAA 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 DiseaseGlycogen Storage Disease Type II Pompe DiseaseDrug: GZ402666Genzyme, a Sanofi CompanyRecruitingN/AN/AAll21Phase 2/Phase 3United States;Belgium;Denmark;France;Germany;Netherlands;United Kingdom
39NCT02054832November 20136 October 2015Sleep 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 MitchellNot recruiting2 Years50 YearsBoth11N/ACanada
40NCT02405598October 201326 September 2016Evaluation of Salbutamol as an Adjuvant Therapy for Pompe DiseaseEvaluation of Salbutamol as an Adjuvant Therapy for Pompe DiseasePompe DiseaseDrug: SalbutamolNational Taiwan University HospitalNot recruiting2 YearsN/ABoth14Phase 4Taiwan
No.TrialIDDate_
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Last_Refreshed_
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41NCT01942590September 201315 July 2019Safety 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.Not recruiting18 YearsN/AAll17Phase 1/Phase 2United States
42NCT01898364July 201323 March 2015Safety 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 CompanyNot recruiting18 YearsN/ABoth24Phase 1United States;Belgium;Denmark;France;Germany;Netherlands;United Kingdom;Italy
43NCT01885936June 201322 July 2019Safety 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 UniversityNot recruiting18 YearsN/AAll16Phase 1/Phase 2United States
44EUCTR2013-002257-30-GB28 February 2019A 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 DISEASEPompe 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
Pharmaceutical Form: Solution for injection
INN or Proposed INN: duvoglustat HCl
CAS Number: 73285-50-4
Current Sponsor code: AT2220 HCl
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 30.6-
Trade Name: Myozyme
Product Name: Myozyme
Pharmaceutical Form: Powder for concentrate for solution for infusion
INN or Proposed INN: ALGLUCOSIDASE ALFA
CAS Number: 420784-05-0
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: range
Concentration number: 0.5-4
Amicus Therapeutics, Inc.Not Recruiting Female: yes
Male: yes
24Phase 2United States;Netherlands;United Kingdom
45NCT01597596August 201219 October 2017A 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 CompanyNot recruitingN/A12 MonthsAll4Phase 4United States;Germany;Taiwan
No.TrialIDDate_
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46NCT01859624June 201215 July 2019Albuterol 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 UniversityNot recruiting18 YearsN/AAll8Phase 1United States
47NCT00566878March 20128 January 2018Pompe 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 CompanyRecruitingN/AN/AFemale5N/AUnited States
48NCT01526785March 201219 October 2017A 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 CompanyNot recruiting1 YearN/AAll113Phase 4United States
49NCT00567073November 201124 June 2019Pompe Pregnancy Sub-RegistryA Sub-Registry to Observe the Effect of Myozyme (Alglucosidase Alfa) Treatment on Pregnancy and Infant Growth in Women With Pompe DiseaseGlycogen Storage Disease Type II (GSD-II);Pompe Disease (Late-onset);Glycogenesis 2 Acid Maltase DeficiencyBiological: alglucosidase alpha;Other: No TreatmentGenzyme, a Sanofi CompanyRecruitingN/AN/AFemale20Phase 2United States;Italy
50NCT01380743October 31, 201120 August 2018Drug-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 TherapeuticsNot recruiting18 Years65 YearsAll25Phase 2United States;Canada;France;United Kingdom
No.TrialIDDate_
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51NCT01435772August 15, 201111 June 2018Extension 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 PharmaceuticalNot recruiting13 YearsN/AAll21Phase 2United States;Australia;France;Germany;New Zealand;United Kingdom
52NCT01288027June 201119 October 2017Exploratory 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 CompanyNot recruiting18 YearsN/AAll16Phase 4United States;Germany;Netherlands;United Kingdom
53EUCTR2010-024647-32-IT18/03/201119 March 2012Combined 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 - NDPatients 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
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Miglustat
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
UNIVERSITA` DEGLI STUDI DI NAPOLI FEDERICO IIAuthorisedFemale: yes
Male: yes
Italy
54NCT01230801December 201025 May 2015Safety/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 PharmaceuticalNot recruiting13 YearsN/ABoth30Phase 1/Phase 2United States;Australia;France;Germany;United Kingdom
55NCT00976352September 201024 September 2018Safety 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)Not recruiting2 Years18 YearsAll9Phase 1/Phase 2United States
No.TrialIDDate_
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56NCT00701129October 200919 October 2017An 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 CompanyNot recruitingN/AN/AAll4Phase 4United States;Israel
57EUCTR2008-002302-18-DE16/02/200924 July 2012An 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
Pharmaceutical Form: Powder for oral solution
CAS Number: 73285-50-4
Current Sponsor code: AT2220
Other descriptive name: 1-deoxynojirimycin hydrochloride
Concentration unit: g gram(s)
Concentration type: equal
Concentration number: 2.5-
Product Code: AT2220
Pharmaceutical Form: Powder for oral solution
CAS Number: 73285-50-4
Current Sponsor code: AT2220
Other descriptive name: 1-deoxynojirimycin hydrochloride
Concentration unit: g gram(s)
Concentration type: equal
Concentration number: 5.0-
Amicus Therapeutics, Inc.Not RecruitingFemale: yes
Male: yes
18United Kingdom;Germany
58EUCTR2008-008293-31-NL10/02/200919 March 2012Early 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 bioreactorGlycogen Storage Disease Type II (Pompe's Disease)
MedDRA version: 9.1 Level: LLT Classification code 10036143 Term: Pompe's disease
Product Name: alglucosidase alfa
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: alglucosidase alfa
CAS Number: n.a.
Current Sponsor code: rhGAA
Other descriptive name: alglucosidase alfa
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 50-
Erasmus MC SophiaAuthorisedFemale: yes
Male: yes
Netherlands
59NCT00701701December 14, 200823 April 2019Immune 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 CompanyNot recruiting1 MonthN/AAll4Phase 4United States;Israel;Canada
60NCT00688597December 8, 200820 August 2018Study 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 TherapeuticsNot recruiting18 Years74 YearsAll3Phase 2United States;Australia;Canada;France;Germany;Netherlands;United Kingdom
No.TrialIDDate_
enrollement
Last_Refreshed_
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61NCT01451879October 200816 December 2017Observational 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 FloridaNot recruitingN/A65 YearsAll11N/AUnited States
62NCT00486889August 200816 December 2017Growth 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 CompanyNot recruitingN/A24 MonthsAll30Phase 4United States
63NCT00520143July 200719 February 2015Alglucosidase 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 CompanyNot recruiting18 YearsN/ABothN/AUnited States
64EUCTR2007-001375-11-NL16/05/200719 March 2012Effects 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 applicablePompe 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®
Pharmaceutical Form: Intravenous infusion
AuthorisedFemale: yes
Male: yes
Netherlands
65NCT00483379May 200719 October 2017High 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 CompanyNot recruiting6 MonthsN/AAll13Phase 4United States;Australia;Canada
No.TrialIDDate_
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66EUCTR2006-003644-31-FR18/01/200719 March 2012An 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 AGLU02704Glycogen Storage Disease type II (Pompe's disease)Trade Name: Myozyme
Product Name: Myozyme
Product Code: rhGAA, acid alpha glucosidase
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: Alglucosidase alfa
CAS Number: n.a.
Current Sponsor code: rhGAA
Other descriptive name: alglucosidase alpha
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 50-
Genzyme Europe BVAuthorisedFemale: yes
Male: yes
90Germany;Netherlands;France
67NCT00268944December 200519 February 2015Safety 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 CompanyNot recruiting18 YearsN/ABoth5Phase 3France
68NCT00158600September 200519 October 2017A 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 CompanyNot recruiting8 YearsN/AAll90Phase 3United States;France;Netherlands
69EUCTR2005-002829-31-GB31/08/200524 April 2012Full 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 LOTSGlycogen Storage Disease type II (Pompe´s disease)Product Name: Myozyme
Product Code: rhGAA, acid alpha glucosidase
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: Alglucosidase alfa
CAS Number: n.a.
Current Sponsor code: rhGAA
Other descriptive name: alglucosidase alpha
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 5-
Genzyme Europe BVAuthorisedFemale: yes
Male: yes
5United Kingdom
70EUCTR2005-001629-27-DE03/08/200519 March 2012A 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 AGLU01602Pompe 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
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: Alglucosidase alfa
CAS Number: n.a.
Current Sponsor code: rhGAA
Other descriptive name: n.a.
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 5-
Genzyme Europe BVNot RecruitingFemale: yes
Male: yes
17Germany;Italy
No.TrialIDDate_
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71NCT00125879June 200519 February 2015Extension 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 CompanyNot recruitingN/AN/ABoth16Phase 2/Phase 3United States;France;Germany;Israel;Italy;Netherlands;Taiwan
72NCT00250939February 200519 February 2015A 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 CompanyNot recruiting5 Years18 YearsBoth5Phase 2Netherlands
73NCT00074932November 200419 February 2015Expanded 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 CompanyNot recruitingN/AN/ABoth9N/AUnited States
74EUCTR2004-002168-59-IT14/10/200419 March 2012An 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-Onset Pompe 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-Onset Pompe 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
Pharmaceutical Form: Powder for infusion*
INN or Proposed INN: alglucosidase alfa
CAS Number: 420784-05-0
Current Sponsor code: myozyme
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 52.5-
Product Name: Recombinant human acid alfa-glucosidase (rhGAA)
Product Code: NA
Pharmaceutical Form: Powder for infusion*
INN or Proposed INN: alglucosidase alfa
CAS Number: 420784-05-0
Current Sponsor code: myozyme
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 52.5-
GENZYMENot RecruitingFemale: yes
Male: yes
20Italy
75NCT00074919December 200319 February 2015Expanded 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 CompanyNot recruitingN/AN/ABothN/AUnited States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
76NCT00059280April 200319 February 2015A 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 CompanyNot recruitingN/A26 WeeksBoth16Phase 2/Phase 3United States;France;Israel;Taiwan;United Kingdom
77NCT00763932April 200319 February 2015Extension 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 CompanyNot recruiting18 YearsN/ABoth7Phase 2United States;France;South Africa
78NCT00765414April 200319 February 2015Extension 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 CompanyNot recruiting16 YearsN/AFemale1Phase 2United States
79NCT00053573February 200319 February 2015rhGAA 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 CompanyNot recruiting6 Months36 MonthsBoth20Phase 1/Phase 2United States;France;Israel;United Kingdom
80NCT00051935January 200319 February 2015A 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 CompanyNot recruitingN/AN/ABoth2Phase 2United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
81NCT00025896May 200119 February 2015Safety 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 CompanyNot recruitingN/AN/ABoth8Phase 2United States

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