Miglustat ( DrugBank: Miglustat )
3 diseases
| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 19 | Lysosomal storage disease | 22 |
| 256 | Muscle glycogenosis | 32 |
| 299 | Cystic fibrosis | 6 |
19. Lysosomal storage disease
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03910621 (ClinicalTrials.gov) | April 2, 2020 | 8/3/2019 | Safety and Efficacy of Miglustat in Chinese NPC Patients | A 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 Subjects | Niemann-Pick Disease, Type C | Drug: Miglustat | Actelion | NULL | Completed | 4 Years | N/A | All | 17 | Phase 4 | China |
| 2 | NCT03822013 (ClinicalTrials.gov) | January 14, 2019 | 22/1/2019 | Effects 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 Diseases | GM2 Gangliosidosis;Supportive Care | Drug: Miglustat | Tehran University of Medical Sciences | Mashhad University of Medical Sciences;Kashan University of Medical Sciences | Recruiting | 6 Months | 24 Months | All | 30 | Phase 3 | Iran, Islamic Republic of |
| 3 | NCT02030015 (ClinicalTrials.gov) | December 22, 2015 | 17/12/2013 | Synergistic Enteral Regimen for Treatment of the Gangliosidoses | Synergistic Enteral Regimen for Treatment of the Gangliosidoses (Syner-G) | GM1 Gangliosidoses;GM2 Gangliosidoses;Tay-Sachs Disease;Sandhoff Disease | Drug: miglustat;Other: Ketogenic Diet | University of Minnesota | Rare 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 Network | Recruiting | N/A | 204 Months | All | 30 | Phase 4 | United States |
| 4 | EUCTR2014-005194-37-DE (EUCTR) | 19/10/2015 | 28/04/2015 | A 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 pattern | A 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 ApS | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 1 | Spain;Germany;Switzerland | ||
| 5 | EUCTR2014-005194-37-ES (EUCTR) | 13/07/2015 | 08/05/2015 | A 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 pattern | A 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 ApS | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 1 | Spain;Germany;Switzerland | ||
| 6 | NCT02520934 (ClinicalTrials.gov) | July 2015 | 6/8/2015 | Miglustat on Gaucher Disease Type IIIB | Evaluation of Combination Therapy With Miglustat and Enzyme Replacement Therapy on Gaucher Disease Type IIIB | Gaucher Disease | Drug: Miglustat;Drug: ERT | National Taiwan University Hospital | Actelion | Active, not recruiting | 6 Years | N/A | All | 19 | N/A | Taiwan |
| 7 | EUCTR2007-005516-61-IT (EUCTR) | 06/02/2009 | 23/03/2009 | Multicenter 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 | Multicenter 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 GASLINI | NULL | Not Recruiting | Female: yes Male: yes | Italy | ||||
| 8 | EUCTR2007-007164-20-ES (EUCTR) | 20/05/2008 | 21/02/2008 | Uso racional de los tratamientos por inhibición de sustrato y enzimático sustitutivo en pacientes con Enfermedad de Gaucher tipo 1 | Uso 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 SALUD | NULL | Not Recruiting | Female: yes Male: yes | Spain | ||||
| 9 | NCT01760564 (ClinicalTrials.gov) | January 2008 | 2/1/2013 | Application of Miglustat in Patients With Niemann-Pick Type C | Application of Miglustat in Patients With Niemann-Pick Type C | Niemann-Pick Disease Type C | Drug: Miglustat | National Taiwan University Hospital | NULL | Completed | N/A | N/A | Both | 5 | Phase 3 | Taiwan |
| 10 | EUCTR2005-001651-37-HU (EUCTR) | 10/12/2007 | 04/10/2007 | 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. | 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 Ltd | NULL | Not Recruiting | Female: yes Male: yes | 50 | Hungary;Czech Republic;Germany;United Kingdom;Spain;Italy | |||
| 11 | EUCTR2006-004661-34-FR (EUCTR) | 08/12/2006 | 18/10/2006 | Evaluation 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 | Evaluation 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 LYON | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 2 | France | ||
| 12 | EUCTR2006-005842-35-IT (EUCTR) | 22/11/2006 | 28/10/2009 | Efficacy and safety of treatment with N-butyl-deoxynojirimycin (NB-DNJ-miglustat) in patients with Niemann-Pick disease type C. - ND | Efficacy 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 II | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Italy | ||||
| 13 | EUCTR2005-001651-37-CZ (EUCTR) | 02/10/2006 | 17/08/2006 | 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. - MAINTENANCE | 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. - 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 Ltd | NULL | Not Recruiting | Female: yes Male: yes | 50 | Hungary;Germany;United Kingdom;Czech Republic;Spain;Italy | |||
| 14 | EUCTR2005-001651-37-ES (EUCTR) | 12/04/2006 | 25/01/2006 | A 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 | A 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 Ltd | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 4 | Hungary;Czech Republic;Germany;United Kingdom;Spain;Italy | ||
| 15 | EUCTR2005-001651-37-DE (EUCTR) | 28/02/2006 | 16/05/2006 | Open-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 | Open-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 Ltd | NULL | Not Recruiting | Female: yes Male: yes | 50 | Hungary;Czech Republic;Spain;Germany;Italy;United Kingdom | |||
| 16 | EUCTR2005-001651-37-IT (EUCTR) | 06/02/2006 | 14/03/2006 | A 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 | A 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 Ltd | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 4 | Hungary;Czech Republic;Germany;United Kingdom;Spain;Italy | ||
| 17 | NCT00319046 (ClinicalTrials.gov) | February 1, 2006 | 26/4/2006 | Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in 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 Type 1 | Drug: Miglustat | Actelion | NULL | Completed | 18 Years | N/A | All | 42 | Phase 3 | United States;Australia;Brazil;Canada;Czechia;France;Germany;Hungary;Italy;Netherlands;Spain;Taiwan;United Kingdom;Czech Republic |
| 18 | EUCTR2005-001651-37-GB (EUCTR) | 27/10/2005 | 03/10/2005 | A 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 | A 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 1.1Disease | Trade Name: Zavesca Product Name: Miglustat Product Code: OGT918 INN or Proposed INN: miglustat | Actelion Pharmaceuticals Ltd | NULL | Not Recruiting | Female: yes Male: yes | 50 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): yes | Hungary;Czech Republic;Spain;Germany;Italy;United Kingdom | ||
| 19 | NCT00672022 (ClinicalTrials.gov) | July 2004 | 2/5/2008 | Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses | Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset GM2 Gangliosidosis: Single and Steady State Oral Doses | GM2 Gangliosidoses;Tay-Sachs;Sandhoff Disease | Drug: Zavesca (Miglustat) | Children's Research Institute | Actelion | Completed | 6 Months | 5 Years | Both | 10 | Phase 3 | United States |
| 20 | NCT00418847 (ClinicalTrials.gov) | July 2004 | 4/1/2007 | Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis | Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis: Single and Multiple Oral Doses | Gangliosidoses GM2 | Drug: miglustat | The Hospital for Sick Children | Actelion | Completed | 6 Years | 20 Years | Both | 5 | Phase 2 | Canada |
| 21 | NCT00517153 (ClinicalTrials.gov) | January 2002 | 15/8/2007 | Miglustat in Niemann-Pick Type C Disease | A Phase II Randomized Controlled Study of Miglustat in Adult and Juvenile Patients With Niemann-Pick Type C Disease | Niemann-Pick Type C Disease | Drug: miglustat | Actelion | NULL | Completed | 4 Years | N/A | Both | 29 | Phase 2 | United States |
| 22 | EUCTR2022-002514-16-Outside-EU/EEA (EUCTR) | 29/07/2022 | A Clinical Study to evaluate the Safety and Efficacy of miglustat (Zavesca®) for the treatment of Niemann-Pick Disease Type C (NPC) in Chinese subjects | A single-arm uncontrolled 12-month Clinical Study to evaluate the Safety and Efficacy of miglustat (Zavesca®) for the Treatment of Niemann-Pick Disease Type C (NPC) in Chinese subjects | Niemann-Pick Disease Type C (NPC) MedDRA version: 20.0;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: Zavesca (Miglustat) Product Name: Miglustat INN or Proposed INN: Miglustat Other descriptive name: Zavesca | Actelion Pharmaceuticals Trading (Shanghai) Co., Ltd | NULL | NA | Female: yes Male: yes | Phase 4 | China |
256. Muscle glycogenosis
Clinical trials : 180 / Drugs : 133 - (DrugBank : 29) / Drug target genes : 25 - Drug target pathways : 105
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04808505 (ClinicalTrials.gov) | April 2023 | 10/3/2021 | A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 | An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 Years | Glycogen Storage Disease Type II Infantile Onset | Biological: Cipaglucosidase alfa;Drug: Miglustat | Amicus Therapeutics | NULL | Recruiting | N/A | 17 Years | All | 36 | Phase 3 | United States |
| 2 | EUCTR2019-000954-67-AT (EUCTR) | 19/06/2020 | 13/03/2020 | A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease | A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease | Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 24.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT2221 Product Code: AT2221 INN or Proposed INN: MIGLUSTAT Other descriptive name: AT2221 65 mg Formulated Capsules | Amicus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | Australia;Netherlands;Korea, Republic of;Bosnia and Herzegovina;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden;United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark | ||
| 3 | EUCTR2019-000954-67-SI (EUCTR) | 03/06/2020 | 10/03/2020 | A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease | A 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 3 | United 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 | ||
| 4 | EUCTR2019-000954-67-DE (EUCTR) | 17/04/2020 | 23/12/2019 | A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease | A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease | Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 24.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT2221 Product Code: AT2221 INN or Proposed INN: MIGLUSTAT | Amicus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | Germany;Japan;Sweden;Bosnia and Herzegovina;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Korea, Republic of | ||
| 5 | EUCTR2019-000954-67-GB (EUCTR) | 08/04/2020 | 13/12/2019 | A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease | A 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United 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 | ||
| 6 | EUCTR2019-000954-67-GR (EUCTR) | 02/04/2020 | 13/02/2020 | A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease | A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease | Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 24.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT2221 Product Code: AT2221 INN or Proposed INN: MIGLUSTAT | Amicus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United 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 | ||
| 7 | EUCTR2019-000954-67-IT (EUCTR) | 01/04/2020 | 17/06/2021 | A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease | A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease - AMICUS ATB200-07 | Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 24.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ATB200 Product Code: [ATB200] INN or Proposed INN: cipaglucosidasi alfa Product Name: AT2221 Product Code: [AT2221] INN or Proposed INN: MIGLUSTAT | AMICUS THERAPEUTICS, INC. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 3 | United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Korea, Republic of;Bosnia and Herzegovina;Slovenia;Korea, Democratic People's Republic of;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden | ||
| 8 | EUCTR2019-000954-67-DK (EUCTR) | 16/03/2020 | 20/01/2020 | A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease | A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease | Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 24.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT2221 Product Code: AT2221 INN or Proposed INN: MIGLUSTAT | Amicus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Bosnia and Herzegovina;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden | ||
| 9 | EUCTR2019-000954-67-FR (EUCTR) | 14/03/2020 | 20/01/2020 | A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease | A 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 3 | United 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 | ||
| 10 | NCT03911505 (ClinicalTrials.gov) | February 13, 2020 | 9/4/2019 | ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD | An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe Disease | Pompe Disease (Late-onset) | Biological: Cipaglucosidase Alfa;Drug: Miglustat | Amicus Therapeutics | NULL | Recruiting | 0 Years | 17 Years | All | 22 | Phase 3 | United States;Australia;Canada;Japan;Taiwan |
| 11 | EUCTR2019-000954-67-ES (EUCTR) | 11/02/2020 | 20/12/2019 | A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease | A 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 3 | United States;Greece;Austria;Netherlands;Sweden;Korea, Republic of;Poland;Slovakia;Slovenia;Bulgaria;France;Bosnia and Herzegovina;Argentina;Romania;Hungary;Japan;United Kingdom;Spain;Canada;Belgium;Taiwan;Denmark;Italy;Australia;Germany | ||
| 12 | EUCTR2019-000954-67-BE (EUCTR) | 31/01/2020 | 06/02/2020 | A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease | A 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 3 | United States;Greece;Austria;Netherlands;Sweden;Korea, Republic of;Poland;Slovakia;Slovenia;Bulgaria;France;Bosnia and Herzegovina;Argentina;Romania;Hungary;Japan;United Kingdom;Spain;Canada;Belgium;Taiwan;Denmark;Italy;Australia;Germany | ||
| 13 | EUCTR2018-000755-40-AT (EUCTR) | 25/09/2019 | 04/06/2019 | A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared 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. | NULL | Not Recruiting | Female: yes Male: yes | 110 | Phase 3 | United 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 | ||
| 14 | JPRN-JapicCTI-194887 | 17/9/2019 | 26/07/2019 | ATB200/AT2221phase3 double blind randomized study | 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 | Pompe disease | Intervention 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. | NULL | complete | 18 | BOTH | 110 | Phase 3 | Japan, Asia except Japan, North America, South America, Europe, Oceania | |
| 15 | EUCTR2018-000755-40-GR (EUCTR) | 28/08/2019 | 21/05/2019 | A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared 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. | NULL | Not Recruiting | Female: yes Male: yes | 110 | Phase 3 | United 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 | ||
| 16 | EUCTR2018-000755-40-NL (EUCTR) | 28/08/2019 | 24/01/2019 | A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared 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. | NULL | Not Recruiting | Female: yes Male: yes | 110 | Phase 3 | United 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 | ||
| 17 | EUCTR2018-000755-40-SI (EUCTR) | 22/05/2019 | 03/04/2019 | A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared 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. | NULL | Not Recruiting | Female: yes Male: yes | 110 | Phase 3 | United 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 | ||
| 18 | EUCTR2018-000755-40-IT (EUCTR) | 17/05/2019 | 25/01/2021 | 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 - 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. | NULL | Not Recruiting | Female: yes Male: yes | 110 | Phase 3 | United 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 | ||
| 19 | EUCTR2018-000755-40-BG (EUCTR) | 16/04/2019 | 28/02/2019 | 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. | NULL | Not 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): no | United 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 | ||
| 20 | EUCTR2018-000755-40-DE (EUCTR) | 08/04/2019 | 29/11/2018 | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 110 | Phase 3 | United 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 | ||
| 21 | EUCTR2018-000755-40-GB (EUCTR) | 18/03/2019 | 06/12/2018 | A 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United 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 | ||
| 22 | EUCTR2018-000755-40-BE (EUCTR) | 11/03/2019 | 03/01/2019 | A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared 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. | NULL | Not Recruiting | Female: yes Male: yes | 110 | Phase 3 | United 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 | ||
| 23 | EUCTR2018-000755-40-SK (EUCTR) | 04/03/2019 | 08/01/2019 | A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared 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. | NULL | Not Recruiting | Female: yes Male: yes | 110 | Phase 3 | United States;Greece;Austria;Netherlands;Sweden;Korea, Republic of;Poland;Slovakia;Slovenia;Bulgaria;France;Bosnia and Herzegovina;Argentina;Romania;Hungary;Japan;United Kingdom;Spain;Canada;Belgium;Taiwan;Denmark;Italy;Australia;Germany | ||
| 24 | EUCTR2018-000755-40-HU (EUCTR) | 28/02/2019 | 28/12/2018 | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 110 | Phase 3 | United 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 | ||
| 25 | EUCTR2018-000755-40-ES (EUCTR) | 12/02/2019 | 18/01/2019 | A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared 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. | NULL | Not Recruiting | Female: yes Male: yes | 110 | Phase 3 | United 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 | ||
| 26 | EUCTR2018-000755-40-DK (EUCTR) | 06/02/2019 | 30/11/2018 | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 110 | Phase 3 | United 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 | ||
| 27 | EUCTR2018-000755-40-SE (EUCTR) | 30/01/2019 | 12/12/2018 | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 110 | Phase 3 | United 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 | ||
| 28 | EUCTR2015-004798-34-DE (EUCTR) | 30/08/2016 | 31/03/2016 | This 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 32 | Phase 1;Phase 2 | United States;Australia;Netherlands;Germany;United Kingdom | ||
| 29 | EUCTR2015-004798-34-GB (EUCTR) | 01/08/2016 | 04/01/2016 | This 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 32 | Phase 1;Phase 2 | United States;Australia;Netherlands;Germany;United Kingdom | ||
| 30 | EUCTR2015-004798-34-NL (EUCTR) | 25/07/2016 | 25/04/2016 | This 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. | NULL | Authorised-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): no | United States;Australia;Germany;Netherlands;New Zealand;United Kingdom | ||
| 31 | EUCTR2010-024647-32-IT (EUCTR) | 18/03/2011 | 09/05/2011 | Combined enzyme enhancement therapy (EET) and enzyme replacement therapy (ERT) in patients with Pompe disease - ND | Combined 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 II | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Italy | ||||
| 32 | NCT01451879 (ClinicalTrials.gov) | October 2008 | 15/9/2011 | Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies | Effects of Immunomodulation Therapy on Anti-rhGAA Immune Response in Subjects With Pompe Disease Receiving rhGAA Enzyme Replacement Therapy | Pompe Disease | Drug: Rituximab;Drug: Miglustat | University of Florida | NULL | Completed | N/A | 65 Years | All | 11 | N/A | United States |
299. Cystic fibrosis
Clinical trials : 1,695 / Drugs : 1,527 - (DrugBank : 268) / Drug target genes : 111 - Drug target pathways : 174
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT02325362 (ClinicalTrials.gov) | March 17, 2015 | 10/12/2014 | Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the F508del Mutation | Single 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 Mutation | Cystic Fibrosis | Drug: Miglustat ; placebo;Drug: Placebo ; Miglustat | Assistance Publique - Hôpitaux de Paris | Actelion;CRCM (Centres de Ressources et de Compétences de la Mucoviscidose) | Completed | 18 Years | N/A | All | 16 | Phase 2/Phase 3 | France |
| 2 | NCT00945347 (ClinicalTrials.gov) | July 2009 | 23/7/2009 | Does 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 Fibrosis | Drug: Miglustat;Drug: Placebo | Cliniques universitaires Saint-Luc- Université Catholique de Louvain | NULL | Completed | 14 Years | N/A | Both | 10 | Phase 2 | Belgium |
| 3 | EUCTR2008-002352-20-BE (EUCTR) | 11/08/2008 | 17/07/2008 | Single 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 | Single 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 Ltd | NULL | Not Recruiting | Female: yes Male: yes | Belgium | ||||
| 4 | NCT00742092 (ClinicalTrials.gov) | August 2008 | 26/8/2008 | Miglustat in Cystic Fibrosis | Single 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 | Drug: miglustat;Drug: placebo | Actelion | NULL | Completed | 12 Years | N/A | Both | 11 | Phase 2 | Belgium |
| 5 | NCT00537602 (ClinicalTrials.gov) | November 2007 | 28/9/2007 | Miglustat / OGT 918 in the Treatment of Cystic Fibrosis | Single 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 Mutation | Cystic Fibrosis | Drug: miglustat;Drug: placebo | Actelion | NULL | Terminated | 12 Years | N/A | Both | 6 | Phase 2 | Spain |
| 6 | EUCTR2006-002049-35-ES (EUCTR) | 22/07/2007 | 28/05/2007 | Estudio 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 | Estudio 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 Ltd | NULL | Not Recruiting | Female: yes Male: yes | 25 | Phase 2 | Spain |