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Clinical trials for Miglustat

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
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    The EU Clinical Trials Register currently displays   44339   clinical trials with a EudraCT protocol, of which   7369   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    22 result(s) found for: Miglustat. Displaying page 1 of 2.
    1  2  Next»
    EudraCT Number: 2008-007653-12 Sponsor Protocol Number: AC-SWE-001 Start Date*: 2009-07-15
    Sponsor Name:Actelion Pharmaceuticals Sverige AB
    Full Title: Determination of ( 11 C) miglustat uptake in bone tissue and brain using Positron Emission Tomography (PET).
    Medical condition: Gaucher`s diseases type 1 and bone manifestations (patients)
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Completed)
    Trial results: View results
    EudraCT Number: 2019-002827-14 Sponsor Protocol Number: TreatSPG11 Start Date*: 2021-01-19
    Sponsor Name:IRCCS Fondazione Stella Maris
    Full Title: Phase 2 pharmacological experimental study to test the safety of miglustat in subjects with hereditary spastic paraplegia type 11
    Medical condition: Spastic paraparesis type 11 (SPG11), caused by mutations in spatacsin protein, is characterized by the association of peripheral neuropathy, parkinsonism, ataxia, cognitive impairment, hypotrophy o...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10019903 Hereditary spastic paraplegia PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2022-002514-16 Sponsor Protocol Number: AC-056C405 Start Date*: 2022-07-29
    Sponsor Name:Actelion Pharmaceuticals Trading (Shanghai) Co., Ltd
    Full Title: 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
    Medical condition: Niemann-Pick Disease Type C (NPC)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10029403 Niemann-Pick disease PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2006-004661-34 Sponsor Protocol Number: 2006.426/19 Start Date*: 2006-12-08
    Sponsor Name:HOSPICES CIVILS DE LYON
    Full Title: 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 con...
    Medical condition: Mucopolysaccharidose de type III
    Disease: Version SOC Term Classification Code Term Level
    8.1 10056890 Mucopolysaccharidosis III LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2006-002049-35 Sponsor Protocol Number: AC-056-201 Start Date*: 2007-07-22
    Sponsor Name:Actelion Pharmaceuticals Ltd
    Full Title: "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 fibr...
    Medical condition: fibrosis quística
    Disease: Version SOC Term Classification Code Term Level
    8.1 10011762 Cystic fibrosis LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Completed)
    Trial results: View results
    EudraCT Number: 2006-005842-35 Sponsor Protocol Number: FARM59T23W Start Date*: 2006-11-22
    Sponsor Name:Dipartimento di Pediatria Universita` di Napoli Federico II
    Full Title: Efficacy and safety of treatment with N-butyl-deoxynojirimycin (NB-DNJ-miglustat) in patients with Niemann-Pick disease type C.
    Medical condition: NIEMANN-PICK DISEASE TYPE C
    Disease: Version SOC Term Classification Code Term Level
    9.1 10029403 LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2005-001651-37 Sponsor Protocol Number: OGT 918-011 Start Date*: 2005-10-27
    Sponsor Name:Actelion Pharmaceuticals Ltd
    Full Title: 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 ...
    Medical condition: 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: 1 BA...
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) ES (Completed) IT (Completed) DE (Completed) CZ (Completed) HU (Completed)
    Trial results: View results
    EudraCT Number: 2008-002352-20 Sponsor Protocol Number: AC-056A202 Start Date*: 2008-08-11
    Sponsor Name:Actelion Pharmaceuticals Ltd
    Full Title: 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...
    Medical condition: Cystic fibrosis homozygous for the F508del mutation
    Disease: Version SOC Term Classification Code Term Level
    9.1 10011762 Cystic fibrosis LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: BE (Completed)
    Trial results: View results
    EudraCT Number: 2010-024647-32 Sponsor Protocol Number: MIGLU-3 Start Date*: 2011-03-18
    Sponsor Name:UNIVERSITA` DEGLI STUDI DI NAPOLI FEDERICO II
    Full Title: Combined enzyme enhancement therapy (EET) and enzyme replacement therapy (ERT) in patients with Pompe disease
    Medical condition: Patients with Pompe Disease (Type II Glycogenosis) on therapy with Enzyme Replacement Therapy (ERT) for at least 6 months
    Disease: Version SOC Term Classification Code Term Level
    13.1 10010331 - Congenital, familial and genetic disorders 10036143 Pompe's disease LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2007-005516-61 Sponsor Protocol Number: FARM6JBLZ2 Start Date*: 2009-02-06
    Sponsor Name:ISTITUTO GIANNINA GASLINI
    Full Title: 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 di...
    Medical condition: GAUCHER disease
    Disease: Version SOC Term Classification Code Term Level
    9.1 10018048 Gaucher's disease LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2014-005194-37 Sponsor Protocol Number: CT-ORZY-NPC-001 Start Date*: 2015-10-19
    Sponsor Name:Orphazyme ApS
    Full Title: 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 progress...
    Medical condition: Niemann-Pick disease - type C
    Disease: Version SOC Term Classification Code Term Level
    18.0 10010331 - Congenital, familial and genetic disorders 10029403 Niemann-Pick disease PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) ES (Completed)
    Trial results: View results
    EudraCT Number: 2007-007164-20 Sponsor Protocol Number: EC07-90737 Start Date*: 2008-05-20
    Sponsor Name:INSTITUTO ARAGONÉS DE CIENCIAS DE LA SALUD
    Full Title: Uso racional de los tratamientos por inhibición de sustrato y enzimático sustitutivo en pacientes con Enfermedad de Gaucher tipo 1
    Medical condition: Tratamiento de la enfermedad de Gaucher
    Disease: Version SOC Term Classification Code Term Level
    9.1 10018048 Gaucher's disease LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: ES (Completed)
    Trial results: (No results available)
    EudraCT Number: 2015-004438-93 Sponsor Protocol Number: CT-ORZY-NPC-002 Start Date*: 2016-05-09
    Sponsor Name:Orphazyme A/S
    Full Title: Arimoclomol prospective double blind, randomised, placebo-controlled study in patients diagnosed with Niemann Pick disease type C
    Medical condition: Niemann Pick disease type C
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10029403 Niemann-Pick disease PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DK (Completed) DE (Completed) IT (Completed) PL (Completed) ES (Ongoing) GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2020-003136-25 Sponsor Protocol Number: CTD-TCNPC-301 Start Date*: 2021-10-25
    Sponsor Name:Cyclo Therapeutics, Inc.
    Full Title: A Phase 3, Double blind, Randomized, Placebo controlled, Parallel group, Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of 2000 mg/kg of Trappsol® Cyclo™ (Hydroxypropyl β cycl...
    Medical condition: Niemann Pick Disease Type C1
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10029403 Niemann-Pick disease PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Ongoing) DE (Trial now transitioned) PL (Trial now transitioned) IT (Trial now transitioned) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2018-000755-40 Sponsor Protocol Number: ATB200-03 Start Date*: 2019-04-08
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: 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 Algluc...
    Medical condition: Adult Subjects With Late Onset Pompe Disease (LOPD)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10075702 Pompe's disease late onset LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) DK (Completed) GB (GB - no longer in EU/EEA) SE (Completed) HU (Completed) BE (Completed) SK (Completed) ES (Completed) NL (Completed) BG (Prematurely Ended) SI (Completed) GR (Completed) AT (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2019-004498-18 Sponsor Protocol Number: HUB-NEU-2019-01 Start Date*: 2020-06-30
    Sponsor Name:Dr. Jordi Gascón / Dra. Lola Ledesma
    Full Title: EFFICACY AND SAFETY CLINICAL TRIAL WITH EFAVIRENZ IN PATIENTS DIAGNOSED WITH ADULT NIEMANN-PICK TYPE C WITH COGNITIVE IMPAIRMENT
    Medical condition: Niemann Pick tipo C (NPC)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10029403 Niemann-Pick disease PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2019-000954-67 Sponsor Protocol Number: ATB200-07 Start Date*: 2020-04-08
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: 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
    Medical condition: Adult Subjects With Late Onset Pompe Disease (LOPD)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10075702 Pompe's disease late onset LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) ES (Ongoing) DE (Completed) DK (Trial now transitioned) FR (Trial now transitioned) BE (Trial now transitioned) GR (Trial now transitioned) SI (Trial now transitioned) AT (Completed) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2021-005356-10 Sponsor Protocol Number: IB1001-301 Start Date*: 2022-03-15
    Sponsor Name:IntraBio Ltd
    Full Title: Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study
    Medical condition: Niemann-Pick Disease type C (NPC)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: SK (Trial now transitioned) DE (Trial now transitioned) NL (Trial now transitioned) CZ (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2007-002840-21 Sponsor Protocol Number: HGT-GCB-039 Start Date*: 2007-10-30
    Sponsor Name:Shire Human Genetic Therapies Inc
    Full Title: A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gauc...
    Medical condition: Type I Gaucher disease Enfermedad de Gaucher tipo 1
    Disease: Version SOC Term Classification Code Term Level
    9.1 10018048 Gaucher's disease LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Completed) GB (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2015-002548-15 Sponsor Protocol Number: VTS301 Start Date*: 2015-12-01
    Sponsor Name:Vtesse LLC, a Mallinckrodt Pharmaceutical Company
    Full Title: A Phase 2b/3 Prospective, Randomized, Double-blind, Sham-controlled Trial of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Subjects with Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease
    Medical condition: Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) GB (GB - no longer in EU/EEA) ES (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
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