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Clinical trials for Niemann Pick Disease

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
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43881   clinical trials with a EudraCT protocol, of which   7295   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).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
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    15 result(s) found for: Niemann Pick Disease. Displaying page 1 of 1.
    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: 2010-023953-12 Sponsor Protocol Number: DFI12712 Start Date*: 2013-12-16
    Sponsor Name:Genzyme Corporation
    Full Title: A Phase 2, Multi-Center, Randomized, Open-Label, Repeat Dose, Dose-Comparison Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Human Acid Sphingomyelinase in Patients Wit...
    Medical condition: Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease)
    Disease: Version SOC Term Classification Code Term Level
    17.1 100000004850 10041515 Sphingomyelin lipidosis LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) IT (Completed) FR (Ongoing)
    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: 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: 2013-000051-40 Sponsor Protocol Number: LTS13632 Start Date*: 2013-12-16
    Sponsor Name:Genzyme Corporation
    Full Title: A Long-Term Study to Assess the Ongoing Safety and Efficacy of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
    Medical condition: Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease)
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10041515 Sphingomyelin lipidosis LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) IT (Ongoing) DE (Completed) BE (Completed)
    Trial results: View results
    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 (Ongoing) IT (Ongoing) PL (Completed) ES (Ongoing) GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2015-000371-26 Sponsor Protocol Number: DFI12712 Start Date*: 2015-08-14
    Sponsor Name:Genzyme Corporation
    Full Title: A Phase 2/3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Repeat Dose Study to Evaluate the Efficacy, Safety, Pharmacodynamics and Pharmacokinetics of olipudase alfa in Patients With...
    Medical condition: Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease)
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10041515 Sphingomyelin lipidosis LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) GB (GB - no longer in EU/EEA) NL (Completed) FR (Completed) PT (Completed) ES (Ongoing) IT (Completed) BE (Completed)
    Trial results: View results
    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 (Ongoing) PL (Ongoing) IT (Ongoing) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2014-003198-40 Sponsor Protocol Number: DFI13803 Start Date*: 2015-02-19
    Sponsor Name:Genzyme Corporation
    Full Title: A phase 1/2, multi-center, open-label, ascending dose study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and exploratory efficacy of olipudase alfa in pediatric patients...
    Medical condition: Patients with acid sphingomyelinase deficiency (Niemann-Pick disease)
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10041515 Sphingomyelin lipidosis LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed) IT (Completed) FR (Completed) DE (Completed) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2015-005761-23 Sponsor Protocol Number: CTD-TCNPC-201 Start Date*: 2016-09-26
    Sponsor Name:Cyclo Therapeutics, Inc.
    Full Title: A Phase I/II study to evaluate the safety and pharmacokinetics of intravenous Trappsol Cyclo (HP-β-CD) in patients with Niemann-Pick disease type C (NPC-1) and the pharmacodynamic effects of treatm...
    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: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) SE (Completed) IT (Ongoing)
    Trial results: View results
    EudraCT Number: 2018-004331-71 Sponsor Protocol Number: IB1001-201 Start Date*: 2019-05-08
    Sponsor Name:IntraBio Ltd
    Full Title: Effects of N-Acetyl-L-Leucine on Niemann-Pick type C Disease (NPC): A multinational, multi-center, open-label, rater-blinded Phase II 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 Gender: Male, Female
    Trial protocol: DE (Completed) GB (GB - no longer in EU/EEA) ES (Ongoing) SK (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: 2015-004846-25 Sponsor Protocol Number: Pfizer-Prevenar13-2015 Start Date*: 2016-07-15
    Sponsor Name:Maastricht University
    Full Title: Immunization against oxLDL in patients with lysosomal lipid diseases and associated metabolic disorders
    Medical condition: Familial hypercholesterolemia Niemann-Pick disease type B (NPB) Niemann-Pick disease type C (NPC) Partial lipodystrophy in children and adults
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: BE (Ongoing)
    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: 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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    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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