Miglustat    (DrugBank: Miglustat)

3 diseases
IDDisease name (Link within this page)Number of trials
19Lysosomal storage disease21
256Muscle glycogenosis23
299Cystic fibrosis6

19. Lysosomal storage disease    [ 784 clinical trials,   673 drugs,   (DrugBank: 101 drugs),   68 drug target genes,   184 drug target pathways]
Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
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.
21 / 784 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03910621
(ClinicalTrials.gov)
April 2, 20208/3/2019Safety and Efficacy of Miglustat in Chinese NPC PatientsA Single Arm Uncontrolled 12 Months Clinical Study to Evaluate the Safety and Efficacy of Miglustat (Zavesca) for the Treatment of Niemann Pick Type C Disease (NPC) in Chinese SubjectsNiemann-Pick Disease, Type CDrug: MiglustatActelionNULLRecruiting4 YearsN/AAll18Phase 4China
2NCT03822013
(ClinicalTrials.gov)
January 14, 201922/1/2019Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD)Survey of Miglustat Therapeutic Effects on Neurological and Systemic Symptoms of Infantile Type of Sandhoff and Taysachs DiseasesGM2 Gangliosidosis;Supportive CareDrug: MiglustatTehran University of Medical SciencesMashhad University of Medical Sciences;Kashan University of Medical SciencesRecruiting6 Months24 MonthsAll30Phase 3Iran, Islamic Republic of
3NCT02030015
(ClinicalTrials.gov)
December 22, 201517/12/2013Synergistic Enteral Regimen for Treatment of the GangliosidosesSynergistic Enteral Regimen for Treatment of the Gangliosidoses (Syner-G)GM1 Gangliosidoses;GM2 Gangliosidoses;Tay-Sachs Disease;Sandhoff DiseaseDrug: miglustat;Other: Ketogenic DietUniversity of MinnesotaRare Diseases Clinical Research Network;National Center for Advancing Translational Science (NCATS);National Institute of Neurological Disorders and Stroke (NINDS);National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK);Lysosomal Disease NetworkRecruitingN/A204 MonthsAll30Phase 4United States
4EUCTR2014-005194-37-DE
(EUCTR)
19/10/201528/04/2015A non-therapeutic study in patients with Niemann-Pick disease type C in order to characterise the individual patient disease profile and historic signo-symptomatology progression patternA prospective non-therapeutic study in patients diagnosed with Niemann-Pick disease type C in order to characterise the individual patient disease profile and historic signo-symptomatology progression pattern - Characterisation of Niemann-Pick disease type C Niemann-Pick disease - type C
MedDRA version: 18.0;Level: PT;Classification code 10029403;Term: Niemann-Pick disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: MIglustat
Product Name: Zavesca
Product Code: A16AX06
INN or Proposed INN: MIGLUSTAT
Orphazyme ApSNULLNot RecruitingFemale: yes
Male: yes
40Phase 1Spain;Germany;Switzerland
5EUCTR2014-005194-37-ES
(EUCTR)
13/07/201508/05/2015A non-therapeutic study in patients with Niemann-Pick disease type C in order to characterise the individual patient disease profile and historic signo-symptomatology progression patternA prospective non-therapeutic study in patients diagnosed with Niemann-Pick disease type C in order to characterise the individual patient disease profile and historic signo-symptomatology progression pattern - Characterisation of Niemann-Pick disease type C Niemann-Pick disease - type C
MedDRA version: 18.0;Level: PT;Classification code 10029403;Term: Niemann-Pick disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: MIglustat
Product Name: Zavesca
Product Code: A16AX06
INN or Proposed INN: MIGLUSTAT
Orphazyme ApSNULLNot RecruitingFemale: yes
Male: yes
40Phase 1Spain;Germany;Switzerland
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT02520934
(ClinicalTrials.gov)
July 20156/8/2015Miglustat on Gaucher Disease Type IIIBEvaluation of Combination Therapy With Miglustat and Enzyme Replacement Therapy on Gaucher Disease Type IIIBGaucher DiseaseDrug: Miglustat;Drug: ERTNational Taiwan University HospitalActelionActive, not recruiting6 YearsN/AAll19N/ATaiwan
7EUCTR2007-005516-61-IT
(EUCTR)
06/02/200923/03/2009Multicenter randomized study to assess the efficacy and the safety of two therapeutic regimens(high dose of imiglucerase versus co-administration of imiglucerase and miglustat) in type I Gaucher disease patients who have not responded to previous treatment with low dose imiglucerase - NDMulticenter randomized study to assess the efficacy and the safety of two therapeutic regimens(high dose of imiglucerase versus co-administration of imiglucerase and miglustat) in type I Gaucher disease patients who have not responded to previous treatment with low dose imiglucerase - ND GAUCHER disease
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Trade Name: CEREZYME
INN or Proposed INN: Imiglucerase
Trade Name: ZAVESCA
INN or Proposed INN: Miglustat
ISTITUTO GIANNINA GASLININULLNot RecruitingFemale: yes
Male: yes
Italy
8EUCTR2007-007164-20-ES
(EUCTR)
20/05/200821/02/2008Uso racional de los tratamientos por inhibición de sustrato y enzimático sustitutivo en pacientes con Enfermedad de Gaucher tipo 1Uso racional de los tratamientos por inhibición de sustrato y enzimático sustitutivo en pacientes con Enfermedad de Gaucher tipo 1 Tratamiento de la enfermedad de Gaucher
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Trade Name: CEREZYME
INN or Proposed INN: IMIGLUCERASA
Trade Name: MIGLUSTAT (ZAVESCA)
INN or Proposed INN: MIGLUSTAT
INSTITUTO ARAGONÉS DE CIENCIAS DE LA SALUDNULLNot RecruitingFemale: yes
Male: yes
Spain
9NCT01760564
(ClinicalTrials.gov)
January 20082/1/2013Application of Miglustat in Patients With Niemann-Pick Type CApplication of Miglustat in Patients With Niemann-Pick Type CNiemann-Pick Disease Type CDrug: MiglustatNational Taiwan University HospitalNULLCompletedN/AN/ABoth5Phase 3Taiwan
10EUCTR2005-001651-37-HU
(EUCTR)
10/12/200704/10/2007Open-label, non-comparative, multi-center study to evaluate the long-term efficacy, safety and tolerability of oral miglustat as a maintenance therapy after a switch from enzyme replacement therapy in adult patients with stable type 1 Gaucher disease.Open-label, non-comparative, multi-center study to evaluate the long-term efficacy, safety and tolerability of oral miglustat as a maintenance therapy after a switch from enzyme replacement therapy in adult patients with stable type 1 Gaucher disease. Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 18 of the protocol:1BACKGROUND AND RATIONALE1.1Disease
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Trade Name: Zavesca
Product Name: Miglustat
Product Code: OGT918
INN or Proposed INN: miglustat
Actelion Pharmaceuticals LtdNULLNot RecruitingFemale: yes
Male: yes
50Hungary;Czech Republic;Germany;United Kingdom;Spain;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2006-004661-34-FR
(EUCTR)
08/12/200618/10/2006Evaluation d’un traitement par miglustat (Zavesca®) chez les patients atteints de mucopolysaccharidose de type III (maladie de Sanfilippo).Essai thérapeutique de phase IIb randomisé en aveugle contre placebo. - ZAV-MPSIII 2006Evaluation d’un traitement par miglustat (Zavesca®) chez les patients atteints de mucopolysaccharidose de type III (maladie de Sanfilippo).Essai thérapeutique de phase IIb randomisé en aveugle contre placebo. - ZAV-MPSIII 2006 Mucopolysaccharidose de type III
MedDRA version: 8.1;Level: LLT;Classification code 10056890;Term: Mucopolysaccharidosis III
Trade Name: ZAVESCA
Product Name: ZAVESCA
INN or Proposed INN: miglustat
Other descriptive name: MIGLUSTAT
HOSPICES CIVILS DE LYONNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
24Phase 2bFrance
12EUCTR2006-005842-35-IT
(EUCTR)
22/11/200628/10/2009Efficacy and safety of treatment with N-butyl-deoxynojirimycin (NB-DNJ-miglustat) in patients with Niemann-Pick disease type C. - NDEfficacy and safety of treatment with N-butyl-deoxynojirimycin (NB-DNJ-miglustat) in patients with Niemann-Pick disease type C. - ND NIEMANN-PICK DISEASE TYPE C
MedDRA version: 9.1;Level: LLT;Classification code 10029403
Trade Name: ZAVESCA
INN or Proposed INN: Miglustat
Dipartimento di Pediatria Universita` di Napoli Federico IINULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Italy
13EUCTR2005-001651-37-CZ
(EUCTR)
02/10/200617/08/2006Open-label, non-comparative, multi-center study to evaluate the long-term efficacy, safety and tolerability of oral miglustat as a maintenance therapy after a switch from enzyme replacement therapy in adult patients with stable type 1 Gaucher disease. - MAINTENANCEOpen-label, non-comparative, multi-center study to evaluate the long-term efficacy, safety and tolerability of oral miglustat as a maintenance therapy after a switch from enzyme replacement therapy in adult patients with stable type 1 Gaucher disease. - MAINTENANCE Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 16 of the protocol:1BACKGROUND AND RATIONALE1.1Disease
MedDRA version: 8.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Trade Name: Zavesca
Product Name: Miglustat
Product Code: OGT918
INN or Proposed INN: miglustat
Actelion Pharmaceuticals LtdNULLNot RecruitingFemale: yes
Male: yes
50Hungary;Germany;United Kingdom;Czech Republic;Spain;Italy
14EUCTR2005-001651-37-ES
(EUCTR)
12/04/200625/01/2006A phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher DiseaseA phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher Disease Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 16 of the protocol:1BACKGROUND AND RATIONALE1.1Disease Trade Name: Zavesca
Product Name: Miglustat
Product Code: OGT918
INN or Proposed INN: miglustat
Actelion Pharmaceuticals LtdNULLNot RecruitingFemale: yes
Male: yes
50Phase 4Hungary;Czech Republic;Germany;United Kingdom;Spain;Italy
15EUCTR2005-001651-37-DE
(EUCTR)
28/02/200616/05/2006Open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance therapy after a switch from Enzyme Replacement Therapy in adult patients with stable Type 1 Gaucher disease - MAINTENANCEOpen-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance therapy after a switch from Enzyme Replacement Therapy in adult patients with stable Type 1 Gaucher disease - MAINTENANCE Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 18 of the protocol:1BACKGROUND AND RATIONALE1.1Disease
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Trade Name: ZAVESCA
Product Name: Miglustat
Product Code: OGT918
INN or Proposed INN: miglustat
Actelion Pharmaceuticals LtdNULLNot RecruitingFemale: yes
Male: yes
50Hungary;Czech Republic;Spain;Germany;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
16EUCTR2005-001651-37-IT
(EUCTR)
06/02/200614/03/2006A phase IV, open label, non comparative, multicenter study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch form enzyme replacement therapy ERT in adult patients with stable type 1 Gaucher disease - NDA phase IV, open label, non comparative, multicenter study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch form enzyme replacement therapy ERT in adult patients with stable type 1 Gaucher disease - ND Gaucher disease is the most common of the glycosphingolipids storage disease and has autosomal recessive inheritance.
MedDRA version: 6.1;Level: PT;Classification code 10018048
Trade Name: ZAVESCA 84CPS 100MG
INN or Proposed INN: Miglustat
Actelion Registration LtdNULLNot RecruitingFemale: yes
Male: yes
50Phase 4Hungary;Czech Republic;Germany;United Kingdom;Spain;Italy
17NCT00319046
(ClinicalTrials.gov)
February 1, 200626/4/2006Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher DiseaseOpen-label, Non Comparative, Multi-center Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Oral Miglustat as a Maintenance Therapy After a Switch From Enzyme Replacement Therapy in Adult Patients With Stable Type 1 Gaucher DiseaseGaucher Disease Type 1Drug: MiglustatActelionNULLCompleted18 YearsN/AAll42Phase 3United States;Australia;Brazil;Canada;Czechia;France;Germany;Hungary;Italy;Netherlands;Spain;Taiwan;United Kingdom;Czech Republic
18EUCTR2005-001651-37-GB
(EUCTR)
27/10/200503/10/2005A phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher DiseaseA phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher Disease Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 16 of the protocol: 1BACKGROUND AND RATIONALE 1.1DiseaseActelion Pharmaceuticals LtdNULLNot Recruiting Female: yes
Male: yes
50Phase 4Hungary;Czech Republic;Spain;Germany;Italy;United Kingdom
19NCT00418847
(ClinicalTrials.gov)
July 20044/1/2007Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 GangliosidosisPharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis: Single and Multiple Oral DosesGangliosidoses GM2Drug: miglustatThe Hospital for Sick ChildrenActelionCompleted6 Years20 YearsBoth5Phase 2Canada
20NCT00672022
(ClinicalTrials.gov)
July 20042/5/2008Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral DosesPharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset GM2 Gangliosidosis: Single and Steady State Oral DosesGM2 Gangliosidoses;Tay-Sachs;Sandhoff DiseaseDrug: Zavesca (Miglustat)Children's Research InstituteActelionCompleted6 Months5 YearsBoth10Phase 3United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21NCT00517153
(ClinicalTrials.gov)
January 200215/8/2007Miglustat in Niemann-Pick Type C DiseaseA Phase II Randomized Controlled Study of Miglustat in Adult and Juvenile Patients With Niemann-Pick Type C DiseaseNiemann-Pick Type C DiseaseDrug: miglustatActelionNULLCompleted4 YearsN/ABoth29Phase 2United States

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.
23 / 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
1EUCTR2019-000954-67-AT
(EUCTR)
19/06/202013/03/2020A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe diseaseA Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 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
Other descriptive name: AT2221 65 mg Formulated Capsules
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
2EUCTR2019-000954-67-SI
(EUCTR)
03/06/202010/03/2020A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe diseaseA Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: cipaglucosidase alfa
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Amicus Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 3United States;Taiwan;Slovenia;Greece;Spain;Austria;United Kingdom;Italy;France;Hungary;Canada;Argentina;Poland;Belgium;Australia;Denmark;Netherlands;Germany;Japan;Sweden;Bosnia and Herzegovina;Korea, Republic of
3EUCTR2019-000954-67-DE
(EUCTR)
17/04/202023/12/2019A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe diseaseA Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: cipaglucosidase alfa
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Amicus Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
4EUCTR2019-000954-67-GB
(EUCTR)
08/04/202013/12/2019A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe diseaseA Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: cipaglucosidase alfa
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Amicus Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
5EUCTR2019-000954-67-GR
(EUCTR)
02/04/202013/02/2020A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe diseaseA Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: cipaglucosidase alfa
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Amicus Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Korea, Republic of;Bosnia and Herzegovina;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2019-000954-67-FR
(EUCTR)
14/03/202020/01/2020A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe diseaseA Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: cipaglucosidase alfa
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Amicus Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 3United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden
7EUCTR2019-000954-67-BE
(EUCTR)
31/01/202006/02/2020A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe diseaseA Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Adult Subjects With Late Onset Pompe Disease (LOPD)
MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ATB200
Product Code: ATB200
INN or Proposed INN: N/A
Product Name: AT2221
Product Code: AT2221
INN or Proposed INN: MIGLUSTAT
Amicus Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 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
8EUCTR2018-000755-40-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.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
9JPRN-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
10EUCTR2018-000755-40-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: 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Taiwan;Slovakia;Greece;Spain;Israel;Italy;France;Australia;Denmark;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Poland;Brazil;Belgium;Romania;Bulgaria;Germany;Japan;New Zealand;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2018-000755-40-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.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
12EUCTR2018-000755-40-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.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
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.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
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.NULLAuthorised-recruitment may be ongoing or finishedFemale: 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
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-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.NULLAuthorised-recruitment may be ongoing or finishedFemale: 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
19EUCTR2018-000755-40-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.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
20EUCTR2015-004798-34-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: 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21EUCTR2015-004798-34-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
22EUCTR2010-024647-32-IT
(EUCTR)
18/03/201109/05/2011Combined enzyme enhancement therapy (EET) and enzyme replacement therapy (ERT) in patients with Pompe disease - NDCombined enzyme enhancement therapy (EET) and enzyme replacement therapy (ERT) in patients with Pompe disease - ND Patients with Pompe Disease (Type II Glycogenosis) on therapy with Enzyme Replacement Therapy (ERT) for at least 6 months
MedDRA version: 13.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders
Trade Name: ZAVESCA
INN or Proposed INN: Miglustat
UNIVERSITA` DEGLI STUDI DI NAPOLI FEDERICO IINULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Italy
23NCT01451879
(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

299. Cystic fibrosis    [ 1,592 clinical trials,   1,539 drugs,   (DrugBank: 255 drugs),   81 drug target genes,   162 drug target pathways]
Searched query = "Cystic fibrosis"
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.
6 / 1,592 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT02325362
(ClinicalTrials.gov)
March 17, 201510/12/2014Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the F508del MutationSingle Center, Double-blind, Randomized, Placebo-controlled, Two-period/Two-treatment Crossover, Proof-of-mechanism Study Investigating the Effect of Miglustat on the Nasal Potential Difference in Adult Patients With Cystic Fibrosis Homozygous for the F508del MutationCystic FibrosisDrug: Miglustat ; placebo;Drug: Placebo ; MiglustatAssistance Publique - Hôpitaux de ParisActelion;CRCM (Centres de Ressources et de Compétences de la Mucoviscidose)Completed18 YearsN/AAll16Phase 2;Phase 3France
2NCT00945347
(ClinicalTrials.gov)
July 200923/7/2009Does a Nasal Instillation of Miglustat Normalize the Nasal Potential Difference in Cystic Fibrosis Patients ?Does a Nasal Instillation of Miglustat Normalize the Nasal Potential Difference in Cystic Fibrosis Patients Homozygous for the F508del Mutation? A Randomized, Double Blind Placebo-controlled Study.Cystic FibrosisDrug: Miglustat;Drug: PlaceboCliniques universitaires Saint-Luc- Université Catholique de LouvainNULLCompleted14 YearsN/ABoth10Phase 2Belgium
3EUCTR2008-002352-20-BE
(EUCTR)
11/08/200817/07/2008Single center, double-blind, randomized, placebo-controlled, two-period/two-treatment crossover study investigating the effect of miglustat on the nasal potential difference in patients with cystic fibrosis homozygous for the F508del mutationSingle center, double-blind, randomized, placebo-controlled, two-period/two-treatment crossover study investigating the effect of miglustat on the nasal potential difference in patients with cystic fibrosis homozygous for the F508del mutation Cystic fibrosis homozygous for the F508del mutation
MedDRA version: 9.1;Level: LLT;Classification code 10011762;Term: Cystic fibrosis
Trade Name: Zavesca
INN or Proposed INN: miglustat
Actelion Pharmaceuticals LtdNULLNot RecruitingFemale: yes
Male: yes
Belgium
4NCT00742092
(ClinicalTrials.gov)
August 200826/8/2008Miglustat in Cystic FibrosisSingle Center, Double-blind, Randomized, Placebo-controlled, Two-period/Two-treatment Crossover Study Investigating the Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the F508del MutationCystic FibrosisDrug: miglustat;Drug: placeboActelionNULLCompleted12 YearsN/ABoth11Phase 2Belgium
5NCT00537602
(ClinicalTrials.gov)
November 200728/9/2007Miglustat / OGT 918 in the Treatment of Cystic FibrosisSingle Center, Double-blind, Randomized, Placebo-controlled, 2-period/2-treatment Crossover Study Investigating the Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the ?F508 MutationCystic FibrosisDrug: miglustat;Drug: placeboActelionNULLTerminated12 YearsN/ABoth6Phase 2Spain
No.TrialIDDate_
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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size
PhaseCountries
6EUCTR2006-002049-35-ES
(EUCTR)
22/07/200728/05/2007Estudio unicéntrico, a doble ciego, aleatorizado y controlado con placebo, cruzado de 2 brazos, para investigar el efecto de miglustat sobre la diferencia de potencial nasal en pacientes con fibrosis quística homocigotos para la mutación ?F508Estudio unicéntrico, a doble ciego, aleatorizado y controlado con placebo, cruzado de 2 brazos, para investigar el efecto de miglustat sobre la diferencia de potencial nasal en pacientes con fibrosis quística homocigotos para la mutación ?F508 fibrosis quística
MedDRA version: 8.1;Level: LLT;Classification code 10011762;Term: Cystic fibrosis
Trade Name: Zavesca
Product Name: miglustat
Product Code: OGT 918
INN or Proposed INN: Miglustat
Other descriptive name: 1,5 (Butylimino)-1,5-dideoxy-D-glucitol
Actelion Pharmaceuticals LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
25Spain