19. Lysosomal storage disease Clinical trials / Disease details
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 |