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Clinical trials for 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   43895   clinical trials with a EudraCT protocol, of which   7301   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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    34 result(s) found for: Pick Disease. Displaying page 1 of 2.
    1  2  Next»
    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: 2014-000422-38 Sponsor Protocol Number: PET_imaging_of_Tau Start Date*: 2014-09-19
    Sponsor Name:Skåne University Hospital, Region Skåne
    Full Title: An open-label study evaluating the diagnostic accuracy of [18F]-AV-1451 PET to detect and distinguish neurodegenerative disorders characterized by cerebral accumulation of tau
    Medical condition: Neurodegenerative disorders with Tau-pathology; including Alzheimer's disease, progressive supranuclear palsy, frontotemporal dementia, corticobasal degeneration and important differential diagnost...
    Disease: Version SOC Term Classification Code Term Level
    17.0 10029205 - Nervous system disorders 10001897 Alzheimer's disease (incl subtypes) HLT
    17.0 10029205 - Nervous system disorders 10053643 Neurodegenerative disorder PT
    17.0 10029205 - Nervous system disorders 10009846 Cognitive impairment LLT
    17.0 10029205 - Nervous system disorders 10001896 Alzheimer's disease LLT
    17.0 10029205 - Nervous system disorders 10012285 Dementia due to Pick's disease LLT
    17.0 10029205 - Nervous system disorders 10012267 Dementia PT
    17.0 10029205 - Nervous system disorders 10067889 Dementia with Lewy bodies PT
    17.0 10029205 - Nervous system disorders 10036813 Progressive supranuclear palsy PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2017-000094-36 Sponsor Protocol Number: BioFINDER_2 Start Date*: 2017-03-31
    Sponsor Name:Skåne University Hospital
    Full Title: The BioFINDER 2 study - improved diagnostics and increased understanding of the pathophysiology of cognitive disorders
    Medical condition: Neurodegenerative disorders with Tau-pathology; including, but not limited to, Alzheimer's disease, progressive supranuclear palsy, frontotemporal dementia, corticobasal degeneration and mild cogni...
    Disease: Version SOC Term Classification Code Term Level
    19.1 10029205 - Nervous system disorders 10053643 Neurodegenerative disorder PT
    19.1 10029205 - Nervous system disorders 10012267 Dementia PT
    19.1 10029205 - Nervous system disorders 10067889 Dementia with Lewy bodies PT
    19.1 10029205 - Nervous system disorders 10028037 Movement disorders (incl parkinsonism) HLGT
    19.1 10029205 - Nervous system disorders 10012271 Dementia Alzheimer's type PT
    19.1 10029205 - Nervous system disorders 10001897 Alzheimer's disease (incl subtypes) HLT
    19.1 10029205 - Nervous system disorders 10009846 Cognitive impairment LLT
    19.1 10029205 - Nervous system disorders 10048598 Cognitive disorders LLT
    19.1 10029205 - Nervous system disorders 10001896 Alzheimer's disease LLT
    19.1 10029205 - Nervous system disorders 10012285 Dementia due to Pick's disease LLT
    19.1 10029205 - Nervous system disorders 10036813 Progressive supranuclear palsy PT
    19.1 10029205 - Nervous system disorders 10074616 Prodromal Alzheimer's disease PT
    19.1 10029205 - Nervous system disorders 10066571 Progression of Alzheimer's disease LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Ongoing)
    Trial results: (No results available)
    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
    EudraCT Number: 2017-003180-35 Sponsor Protocol Number: R04725 Start Date*: 2018-06-15
    Sponsor Name:Manchester University NHS Foundation Trust
    Full Title: Feasibility study on the effects of postnatal enalapril on maternal cardiovascular function following preterm pre-eclampsia
    Medical condition: Postnatal (PN) cardiovascular dysfunction following preterm pre-eclampsia (pPE)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10036585 - Pregnancy, puerperium and perinatal conditions 10036485 Pre-eclampsia PT
    20.0 10036585 - Pregnancy, puerperium and perinatal conditions 10036485 Pre-eclampsia PT
    20.0 10036585 - Pregnancy, puerperium and perinatal conditions 10040444 Severe pre-eclampsia LLT
    20.1 100000004849 10007648 Cardiovascular disease, unspecified LLT
    Population Age: Adults Gender: Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2005-004087-24 Sponsor Protocol Number: FEN-PAI-3002 Start Date*: 2008-01-29
    Sponsor Name:JANSSEN-CILAG GmbH
    Full Title: Global assessment of treatment with IONSYS and its handling by patients, doctors and nursing staff in the management of acute moderate to severe post-surgery pain in hospitalised patients.
    Medical condition: Acute, moderate to severe postoperative pain after elective surgery in a hospital setting in patients needing at least 24 hours strong opioids for pain therapy.
    Disease: Version SOC Term Classification Code Term Level
    9.1 10054711 Postoperative pain LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2015-005682-24 Sponsor Protocol Number: RIOTA2015 Start Date*: 2016-03-16
    Sponsor Name:Sven Olaf Skouby, Professor, MD, DMSc. Unit of Reproductive Medicine, Herlev/Gentofte Hospital
    Full Title: REDUCING THE IMPACT OF OVARIAN STIMULATION - THE RIOT PROJECT STUDY RIOT A: THE ROLE OF AROMATASE INHIBITOR IN REDUCING THE DETRIMENTAL EFFECTS OF OVARIAN STIMULATION TO OPTIMIZE OUTCOMES IN FRESH...
    Medical condition: Infertility
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004872 10016401 Female infertility of other specified origin LLT
    20.0 100000004872 10025511 Male infertility, unspecified LLT
    20.0 100000004872 10016403 Female infertility of tubal origin LLT
    Population Age: Adults Gender: Female
    Trial protocol: DK (Completed)
    Trial results: (No results available)
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