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

  
99 trials found
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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agemax
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PhaseCountries
1EUCTR2020-004686-39-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
2NCT05017402
(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
3NCT04848779
(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
4NCT04676373
(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
5EUCTR2018-000755-40-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
6JPRN-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
7EUCTR2018-000755-40-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
8EUCTR2018-000755-40-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
9NCT04327973
(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
10EUCTR2018-000755-40-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
11EUCTR2018-000755-40-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
12EUCTR2018-000755-40-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
13EUCTR2018-000755-40-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
14EUCTR2018-000755-40-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
15EUCTR2018-000755-40-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
16EUCTR2018-000755-40-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
17EUCTR2018-000755-40-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
18EUCTR2018-000755-40-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
19EUCTR2018-000755-40-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
20EUCTR2018-000755-40-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
21EUCTR2016-000942-77-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
22NCT03687333
(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
23NCT03729362
(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
24ChiCTR1800018514
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
25EUCTR2016-000942-77-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
26EUCTR2017-004370-34-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
27EUCTR2016-000942-77-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
28EUCTR2017-004370-34-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
29EUCTR2010-022231-11-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
30NCT02240407
(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
31NCT03019406
(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
32EUCTR2016-004578-16-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
33EUCTR2016-003475-21-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
34EUCTR2016-000942-77-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
35EUCTR2016-000942-77-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
36EUCTR2016-000942-77-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
37EUCTR2016-000942-77-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
38EUCTR2016-000942-77-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
39EUCTR2016-000942-77-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
40NCT02782741
(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
41EUCTR2015-004798-34-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
42EUCTR2016-000942-77-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
43EUCTR2016-000942-77-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
44EUCTR2016-000942-77-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
45EUCTR2015-004798-34-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
46EUCTR2015-004798-34-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
47EUCTR2016-000942-77-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
48NCT01710813
(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
49EUCTR2013-003321-28-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
50NCT01410890
(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
51EUCTR2013-003321-28-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
52EUCTR2013-003321-28-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
53EUCTR2013-003321-28-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
54EUCTR2013-003321-28-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
55NCT01898364
(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
56EUCTR2011-005595-42-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
57EUCTR2010-022231-11-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
58NCT01597596
(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
59NCT00566878
(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
60NCT01526785
(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
61EUCTR2010-020611-36-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
62EUCTR2011-002154-32-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
63NCT01380743
(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
64EUCTR2010-020611-36-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
65EUCTR2010-020611-36-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
66NCT01288027
(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
67NCT00976352
(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
68NCT00701129
(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
69EUCTR2008-008293-31-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
70NCT00701701
(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
71NCT00486889
(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
72EUCTR2006-003644-31-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
73EUCTR2006-003644-31-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
74EUCTR2005-002759-42-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
75NCT00520143
(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
76EUCTR2007-001375-11-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
77NCT00483379
(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
78NCT00455195
(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
79EUCTR2006-003644-31-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
80NCT00158600
(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
81EUCTR2005-002829-31-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
82EUCTR2005-001629-27-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
83NCT00074932
(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
84EUCTR2004-002168-59-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
85NCT00074919
(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
86NCT00059280
(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
87NCT00051935
(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
88NCT00025896
(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
89EUCTR2013-002257-30-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
90EUCTR2015-000583-34-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
91EUCTR2015-000582-31-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
92EUCTR2005-002829-31-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
93EUCTR2021-004047-25-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
94EUCTR2016-003475-21-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
95EUCTR2016-003475-21-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
96EUCTR2015-000584-14-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
97EUCTR2016-000942-77-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
98EUCTR2021-005552-11-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
99EUCTR2015-000512-18-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