Rhgaa    (DrugBank: -)

1 disease
IDDisease name (Link within this page)Number of trials
256Muscle glycogenosis38

256. Muscle glycogenosis    [ 161 clinical trials,   114 drugs,   (DrugBank: 23 drugs),   26 drug target genes,   106 drug target pathways]
Searched query = "Muscle glycogenosis", "Muscular glycogenosis", "Muscle glycogen storage disease", "Muscular glycogen storage disease", "Glycogen storage disease type 0", "GSD0", "Glycogen synthase deficiency", "Glycogen storage disease type II", "GSDII", "Pompe disease", "Alpha-1,4-glucosidase acid deficiency", "Glycogen storage disease type III", "GSDIII", "Cori disease", "Glycogen debranching enzyme deficiency", "Glycogen storage disease type IV", "GSDIV", "Andersen disease", "Glycogen-branching enzyme deficiency", "GBED", "Glycogen storage disease type V", "GSDV", "McArdle disease", "Muscle phosphorylase deficiency", "Muscular phosphorylase deficiency", "Glycogen storage disease type VII", "GSDVII", "Tarui disease", "Phosphofructokinase deficiency", "PFK deficiency", "Glycogen storage disease type IX", "GSDIX", "Phosphorylase kinase deficiency", "Phosphoglycerate kinase deficiency", "PGK deficiency", "Glycogen storage disease type X", "GSDX", "Phosphoglycerate mutase deficiency", "Glycogen storage diseass type XI", "GSDXI", "Kanno disease", "Lactate dehydrogenase deficiency", "Glycogen storage diseass type XII", "GSDXII", "Aldolase A deficiency", "Glycogen storage diseass type XIII", "GSDXIII", "Beta-enolase deficiency", "Glycogen storage diseass type XIV", "GSDXIV", "Phosphoglucomutase deficiency", "Glycogen storage diseass type XV", "GSDXV", "Glycogenin 1 deficiency"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
38 / 161 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT02898753
(ClinicalTrials.gov)
June 21, 20178/9/2016VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe DiseaseA Three-Month, Open-Label, Randomized, Dose-escalation Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 Versus Myozyme®/Lumizyme® in Patients With Late-Onset GSD-II (Pompe Disease) Followed by Open-Label Treatment With VAL-1221 in All PatientsPompe DiseaseDrug: VAL-1221;Drug: RhGAAValerion Therapeutics, LLCNULLTerminated18 YearsN/AAll12Phase 1;Phase 2United States;United Kingdom
2EUCTR2016-004578-16-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
3NCT02185651
(ClinicalTrials.gov)
October 20161/7/2014A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated ReactionA Pilot Study of the Effects of Oral Administration of Zavesca® on Anti-rhGAA Immune Response in Subjects With Pompe Disease Receiving rhGAA Enzyme Replacement TherapyPompe Disease;Hypersensitivity ReactionDrug: Zavesca® PrescriptionUniversity of FloridaAmicus TherapeuticsTerminated18 Years65 YearsAll2Phase 1United States
4EUCTR2013-001768-48-AT
(EUCTR)
21/02/201612/01/2016A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe DiseaseA Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease Pompe disease
MedDRA version: 18.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: reveglucosidase alpha
Other descriptive name: GILT-rhGAA
Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: reveglucosidase alpha
Other descriptive name: GILT-rhGAA
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
70Phase 3Portugal;France;United States;Belgium;Austria;Netherlands;Germany;Japan;Italy;United Kingdom;Switzerland
5EUCTR2013-001768-48-PT
(EUCTR)
30/01/201502/07/2014A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe DiseaseA Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease Pompe disease
MedDRA version: 18.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: GILT-rhGAA
Other descriptive name: GILT-rhGAA
Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: GILT-rhGAA
Other descriptive name: GILT-rhGAA
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
50Phase 3France;United States;Portugal;Argentina;Belgium;Austria;Netherlands;Germany;Colombia;Japan;Italy;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2013-001768-48-DE
(EUCTR)
23/09/201422/05/2014A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe DiseaseA Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease Pompe disease
MedDRA version: 18.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: reveglucosidase alpha
Other descriptive name: GILT-rhGAA
Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: reveglucosidase alpha
Other descriptive name: GILT-rhGAA
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
50Phase 3Portugal;France;United States;Argentina;Belgium;Austria;Netherlands;Colombia;Germany;Japan;Italy;United Kingdom
7EUCTR2013-001768-48-IT
(EUCTR)
22/09/201401/07/2014A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe DiseaseA Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease Pompe disease
MedDRA version: 17.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: GILT-rhGAA
Other descriptive name: GILT-rhGAA
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
50Phase 3Portugal;France;United States;Argentina;Belgium;Austria;Netherlands;Germany;Colombia;Japan;United Kingdom;Italy
8EUCTR2013-001768-48-BE
(EUCTR)
08/09/201412/05/2014A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe DiseaseA Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease Pompe disease
MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: reveglucosidase alpha
Other descriptive name: GILT-rhGAA
Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: reveglucosidase alpha
Other descriptive name: GILT-rhGAA
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
50Phase 3Portugal;France;United States;Argentina;Belgium;Austria;Netherlands;Germany;Colombia;Japan;Italy;United Kingdom
9NCT01924845
(ClinicalTrials.gov)
April 201413/8/2013BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects With Late-onset Pompe DiseaseLate-onset Pompe DiseaseDrug: BMN 701BioMarin PharmaceuticalNULLTerminated18 YearsN/AAll24Phase 3United States;Belgium;France;Germany;Italy;Netherlands;Portugal;United Kingdom
10EUCTR2013-001768-48-GB
(EUCTR)
27/11/201322/10/2013A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe DiseaseA Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease Pompe disease
MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 701
Product Code: BMN 701
INN or Proposed INN: reveglucosidase alfa
Other descriptive name: GILT-rhGAA
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
70Phase 3Portugal;France;United States;Belgium;Austria;Denmark;Netherlands;Germany;Colombia;Japan;Italy;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2011-005595-42-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;Phase 4United States;Taiwan;Canada;Turkey;Russian Federation;Germany;India
12EUCTR2011-001805-28-DE
(EUCTR)
14/02/201328/06/2012A long term study of the safety of BMN 701 in patients with POMPE diseaseA Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 Study POMPE disease
MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 701
Product Code: BMN 701
Other descriptive name: GILT-rhGAA
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
21Phase 2United States;Australia;Germany;New Zealand;United Kingdom
13EUCTR2010-022231-11-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;China;India
14EUCTR2010-023561-22-DE
(EUCTR)
04/07/201207/02/2012A Study of the Safety and Tolerability of BMN 701 in Patients with Late-onset Pompe DiseaseA Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant human GAA) in Patients with Late-onset Pompe Disease - A Phase 1/2 Study of BMN 701 in Patients with Late-onset Pompe Disease Pompe disease
MedDRA version: 16.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 701
Product Code: BMN 701
Other descriptive name: GILT-rhGAA
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
30Phase 1;Phase 2France;United States;Netherlands;Germany;United Kingdom
15EUCTR2011-001805-28-GB
(EUCTR)
03/07/201226/04/2012A Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 StudyA Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 Study Pompe disease
MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 701
Product Code: BMN 701
Other descriptive name: GILT-rhGAA
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
30Phase 2United States;France;Australia;Germany;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16EUCTR2010-020611-36-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
17NCT01380743
(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
18EUCTR2010-020611-36-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
19EUCTR2010-020611-36-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
20EUCTR2010-023561-22-GB
(EUCTR)
15/08/201111/04/2011A Study of the Safety and Tolerability of BMN 701 in Patients with Late-onset Pompe DiseaseA Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant human GAA) in Patients with Late-onset Pompe Disease - A Phase 1/2 Study of BMN 701 in Patients with Late-onset Pompe Disease Pompe disease
MedDRA version: 13.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 701
Product Code: BMN 701
Other descriptive name: GILT-rhGAA
BioMarin Pharmaceutical IncNULLNot RecruitingFemale: yes
Male: yes
28Phase 1;Phase 2United States;Germany;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21NCT01451879
(ClinicalTrials.gov)
October 200815/9/2011Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation TherapiesEffects of Immunomodulation Therapy on Anti-rhGAA Immune Response in Subjects With Pompe Disease Receiving rhGAA Enzyme Replacement TherapyPompe DiseaseDrug: Rituximab;Drug: MiglustatUniversity of FloridaNULLCompletedN/A65 YearsAll11N/AUnited States
22EUCTR2006-003644-31-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
23EUCTR2006-003644-31-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
24EUCTR2005-002759-42-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
25NCT00520143
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26EUCTR2006-003644-31-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 BVNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
90Germany;Netherlands;France
27NCT00268944
(ClinicalTrials.gov)
December 200522/12/2005Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory SupportProspective, Open-label, Single-arm, Exploratory Study of the Effect and Safety of rhGAA in Patients With Advanced Late-onset Pompe Disease Who Are Receiving Respiratory SupportPompe Disease (Late-onset);Glycogen Storage Disease Type II (GSD-II);Acid Maltase Deficiency Disease;Glycogenosis 2Biological: MyozymeGenzyme, a Sanofi CompanyNULLCompleted18 YearsN/ABoth5Phase 3France
28EUCTR2005-002829-31-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)Genzyme Europe BVNULLNot Recruiting Female: yes
Male: yes
5Phase 2Belgium;United Kingdom
29NCT00250939
(ClinicalTrials.gov)
February 20058/11/2005A Study of rhGAA in Patients With Late-Onset Pompe DiseaseSingle-center, Open-label Study of Safety, Pharmacokinetics and Efficacy of rhGAA in Patients With Late-Onset Pompe DiseasePompe Disease (Late-onset);Glycogen Storage Disease Type II (GSD-II);Acid Maltase Deficiency Disease;Glycogenosis 2Biological: MyozymeGenzyme, a Sanofi CompanyNULLCompleted5 Years18 YearsBoth5Phase 2Netherlands
30EUCTR2004-002168-59-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
31NCT00059280
(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
32NCT00765414
(ClinicalTrials.gov)
April 20032/10/2008Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.An Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid a-Glucosidase (rhGAA) Given as Enyzme Replacement Therapy to a Single Patient With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy StudiesPompe Disease Late-Onset;Glycogen Storage Disease Type II GSD IIBiological: MyozymeGenzyme, a Sanofi CompanyNULLCompleted16 YearsN/AFemale1Phase 2United States
33NCT00763932
(ClinicalTrials.gov)
April 200330/9/2008Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) StudiesA Multicenter, Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid a-Glucosidase (rhGAA) in Patients With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy StudiesPompe Disease Infantile-Onset;Glycogen Storage Disease Type IIBiological: MyozymeGenzyme, a Sanofi CompanyNULLCompleted18 YearsN/ABoth7Phase 2United States;France;South Africa
34NCT00053573
(ClinicalTrials.gov)
February 200331/1/2003rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of rhGAA Treatment in Patients Greater Than 6 Months and Less Than or Equal to 36 Months Old With Infantile-Onset GSD-IIGlycogen Storage Disease Type II;Pompe Disease;Acid Maltase Deficiency Disease;Glycogenosis 2Biological: MyozymeGenzyme, a Sanofi CompanyNULLCompleted6 Months36 MonthsBoth20Phase 1;Phase 2United States;France;Israel;United Kingdom
35NCT00051935
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
36NCT00025896
(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
37EUCTR2005-002829-31-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
38EUCTR2015-000512-18-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