MIGLUSTAT ( DrugBank: Miglustat )
3 diseases
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 19 | ライソゾーム病 | 22 |
| 256 | 筋型糖原病 | 32 |
| 299 | 嚢胞性線維症 | 6 |
19. ライソゾーム病
臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182
Showing 1 to 10 of 22 diseases
| 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 A Single Arm Uncontrolled 12 Months Clinical Study to Evaluate the Safety and Efficacy of Miglustat( ... | 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 Survey of MiglustatTherapeutic Effects on Neurological and Systemic Symptoms of Infantile Type of Sa ... | 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 Rare Diseases Clinical Research Network;National Center for Advancing Translational Science (NCATS); ... | 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 non-therapeutic study in patients with Niemann-Pick disease type C in order to characterise the in ... | 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 A prospective non-therapeutic study in patients diagnosed with Niemann-Pick disease type C in order ... | 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] Niemann-Pick disease - type C MedDRA version: 18.0;Level: PT;Classification code 10029403;Term: Niem ... | 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 non-therapeutic study in patients with Niemann-Pick disease type C in order to characterise the in ... | 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 A prospective non-therapeutic study in patients diagnosed with Niemann-Pick disease type C in order ... | 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] Niemann-Pick disease - type C MedDRA version: 18.0;Level: PT;Classification code 10029403;Term: Niem ... | 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 Evaluation of Combination Therapy With Miglustatand Enzyme Replacement Therapy on Gaucher Disease Ty ... | 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 ... | 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 ... | 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 Trade Name: CEREZYME INN or Proposed INN: Imiglucerase Trade Name: ZAVESCA INN or Proposed INN: Miglust ... | 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 co ... | 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 co ... | Tratamiento de la enfermedad de Gaucher MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease Tratamiento de la enfermedad de Gaucher MedDRA version: 9.1;Level: LLT;Classification code 10018048; ... | Trade Name: CEREZYME INN or Proposed INN: IMIGLUCERASA Trade Name: MIGLUSTAT (ZAVESCA) INN or Proposed INN: MIGLUSTAT Trade Name: CEREZYME INN or Proposed INN: IMIGLUCERASA Trade Name: MIGLUSTAT(ZAVESCA) INN or Proposed I ... | 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 toler ... | 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 toler ... | 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 Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal reces ... | 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 |
256. 筋型糖原病
臨床試験数 : 180 / 薬物数 : 133 - (DrugBank : 29) / 標的遺伝子数 : 25 - 標的パスウェイ数 : 105
Showing 1 to 10 of 32 diseases
| 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 A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglusta ... | 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 An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immuno ... | 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 study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administr ... | 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 A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB2 ... | 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] Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 24.0;Level: LLT;Classification c ... | 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 Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT222 ... | 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 Australia;Netherlands;Korea, Republic of;Bosnia and Herzegovina;Slovenia;Austria;United Kingdom;Hung ... | ||
| 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 study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administr ... | 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 A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB2 ... | 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] Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0;Level: LLT;Classification c ... | Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT2221 Product Code: AT2221 INN or Proposed INN: MIGLUSTAT Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT222 ... | 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 United States;Taiwan;Slovenia;Greece;Spain;Austria;United Kingdom;Italy;France;Hungary;Canada;Argent ... | ||
| 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 study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administr ... | 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 A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB2 ... | 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] Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 24.0;Level: LLT;Classification c ... | Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT2221 Product Code: AT2221 INN or Proposed INN: MIGLUSTAT Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT222 ... | 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 Germany;Japan;Sweden;Bosnia and Herzegovina;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina ... | ||
| 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 study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administr ... | 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 A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB2 ... | 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] Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0;Level: LLT;Classification c ... | Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT2221 Product Code: AT2221 INN or Proposed INN: MIGLUSTAT Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT222 ... | 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 United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Her ... | ||
| 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 study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administr ... | 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 A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB2 ... | 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] Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 24.0;Level: LLT;Classification c ... | Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT2221 Product Code: AT2221 INN or Proposed INN: MIGLUSTAT Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT222 ... | 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 United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Korea, Republi ... | ||
| 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 study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administr ... | 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 A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB2 ... | 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] Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 24.0;Level: LLT;Classification c ... | Product Name: ATB200 Product Code: [ATB200] INN or Proposed INN: cipaglucosidasi alfa Product Name: AT2221 Product Code: [AT2221] INN or Proposed INN: MIGLUSTAT Product Name: ATB200 Product Code: [ATB200] INN or Proposed INN: cipaglucosidasi alfa Product Name: AT2 ... | 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 United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Korea, Republi ... | ||
| 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 study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administr ... | 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 A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB2 ... | 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] Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 24.0;Level: LLT;Classification c ... | Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT2221 Product Code: AT2221 INN or Proposed INN: MIGLUSTAT Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT222 ... | 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 United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Australia;Denmark;Netherlands;Korea, Republi ... | ||
| 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 study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administr ... | 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 A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB2 ... | 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] Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0;Level: LLT;Classification c ... | Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT2221 Product Code: AT2221 INN or Proposed INN: MIGLUSTAT Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT222 ... | 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 United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Her ... | ||
| 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 ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 ... | 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 An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity ... | 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 |
299. 嚢胞性線維症
臨床試験数 : 1,695 / 薬物数 : 1,527 - (DrugBank : 268) / 標的遺伝子数 : 111 - 標的パスウェイ数 : 174
Showing 1 to 6 of 6 diseases
| 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 Effect of Miglustaton the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for ... | 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 Single Center, Double-blind, Randomized, Placebo-controlled, Two-period/Two-treatment Crossover, Pro ... | 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 MiglustatNormalize the Nasal Potential Difference in Cystic Fibrosis Pa ... | 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. Does a Nasal Instillation of MiglustatNormalize the Nasal Potential Difference in Cystic Fibrosis Pa ... | 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 stud ... | 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 stud ... | Cystic fibrosis homozygous for the F508del mutation MedDRA version: 9.1;Level: LLT;Classification code 10011762;Term: Cystic fibrosis Cystic fibrosis homozygous for the F508del mutation MedDRA version: 9.1;Level: LLT;Classification co ... | 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 Single Center, Double-blind, Randomized, Placebo-controlled, Two-period/Two-treatment Crossover Stud ... | 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 Single Center, Double-blind, Randomized, Placebo-controlled, 2-period/2-treatment Crossover Study In ... | 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 ... | 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 ... | 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 Trade Name: Zavesca Product Name: miglustat Product Code: OGT 918 INN or Proposed INN: Miglustat Other d ... | Actelion Pharmaceuticals Ltd | NULL | Not Recruiting | Female: yes Male: yes | 25 | Phase 2 | Spain |