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Clinical trials for Hepatosplenomegaly

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   44335   clinical trials with a EudraCT protocol, of which   7366   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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    33 result(s) found for: Hepatosplenomegaly. Displaying page 1 of 2.
    1  2  Next»
    EudraCT Number: 2015-000104-26 Sponsor Protocol Number: UX003-CL203 Start Date*: 2015-07-23
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: An Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less than 5 years old
    Medical condition: Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome)
    Disease:
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA ES (Prematurely Ended) PT (Completed)
    Trial results: View results
    EudraCT Number: 2015-004604-37 Sponsor Protocol Number: NL55891.078.15 Start Date*: 2017-02-17
    Sponsor Name:Erasmus University Medical Center
    Full Title: Aspirin provocation of patients with Systemic Mastocytosis
    Medical condition: Systemic mastocytosis is a myeloproliferative disorder in which there is an accumulation of aberrant mast cells. It has various subtypes, ranging from indolent to aggressive mastocytosis. Symptoms ...
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-001109-22 Sponsor Protocol Number: PBGM01-001 Start Date*: Information not available in EudraCT
    Sponsor Name:Passage Bio, Inc.
    Full Title: A Phase 1/2 Open-Label, Multicenter, Dose Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of PBGM01 Delivered into the Cisterna Magna of Pediatric Su...
    Medical condition: GM1 gangliosidosis (GM1) is an autosomal recessive disorder that results from mutations in the human galactosidase beta 1 gene (GLB1), which encodes beta-galactosidase (β-gal).
    Disease:
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2014-002421-35 Sponsor Protocol Number: ISIS304801-CS6 Start Date*: 2014-10-27
    Sponsor Name:Ionis Pharmaceuticals, Inc.
    Full Title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of ISIS 304801 Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS)
    Medical condition: Familial Chylomicronemia Syndrome (FCS)
    Disease: Version SOC Term Classification Code Term Level
    18.1 100000004850 10017339 Fredrickson Type I lipidaemia LLT
    18.1 100000004850 10060593 Fredrickson Type I lipidemia LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Completed) DE (Completed) IT (Completed) HU (Completed) ES (Completed)
    Trial results: View results
    EudraCT Number: 2006-005216-27 Sponsor Protocol Number: 78993095 Start Date*: 2007-03-26
    Sponsor Name:HUS, Hospital for Children and Adolescents
    Full Title: Selkäytimeen annettu entsyymikorvaus mukopolysakkaridoosi I:n selkäytimen puristuman hoidossa. Kansainvälinen monikeskustutkimus. A study of intrathecal enzyme replacement therapy for spinal cord c...
    Medical condition: Mucopolysaccharidosis I H/S, lysosomal storage disease with clinical manifestations such as progressive joint stiffness, growth retardation, corneal clouding, hepatosplenomegaly, cardiac and respir...
    Disease: Version SOC Term Classification Code Term Level
    9.1 10056887 Mucopolysaccharidosis IH/S LLT
    Population Age: Gender: Male, Female
    Trial protocol: FI (Completed)
    Trial results: (No results available)
    EudraCT Number: 2010-020063-21 Sponsor Protocol Number: ILUVIT1 Start Date*: 2010-06-15
    Sponsor Name:Charité-Universitätsmedizin
    Full Title: “A 20-week open-label study to assess the efficacy and safety of single doses of Ilaris® (Canakinumab, ACZ885) in patients with active, refractory urticarial vasculitis”
    Medical condition: Urticarial vasculitis
    Disease: Version SOC Term Classification Code Term Level
    12.1 10048820 Urticarial vasculitis LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2018-003199-10 Sponsor Protocol Number: OLE-NLRC4/XIAP.2016.001 Start Date*: Information not available in EudraCT
    Sponsor Name:AB2 Bio Ltd.
    Full Title: Open-label extension study with Tadekinig alfa (r-hIL-18BP) to monitor safety and tolerability in patients with IL-18 driven monogenic autoinflammatory conditions: NLRC4 mutation and XIAP deficiency
    Medical condition: NLRC4 mutation XIAP deficiency
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2021-000678-27 Sponsor Protocol Number: CABL001J12301 Start Date*: 2021-08-10
    Sponsor Name:Novartis Pharma AG
    Full Title: A phase III, multi-center, open-label, randomized study of oral asciminib versus Investigator selected TKI in patients with newly diagnosed Philadelphia Chromosome Positive Chronic Myelogenous Leuk...
    Medical condition: Chronic Myelogenous Leukemia in chronic phase (CML-CP) in newly diagnosed patients
    Disease: Version SOC Term Classification Code Term Level
    21.0 100000004864 10009012 Chronic myelogenous leukemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) HU (Trial now transitioned) DE (Trial now transitioned) CZ (Trial now transitioned) IT (Trial now transitioned) ES (Ongoing) SK (Trial now transitioned) SE (Trial now transitioned) AT (Trial now transitioned) NO (Trial now transitioned) BE (Trial now transitioned) BG (Trial now transitioned) FI (Trial now transitioned) NL (Completed) DK (Trial now transitioned) PT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2007-000208-34 Sponsor Protocol Number: CAMN107A2303 Start Date*: 2007-06-11
    Sponsor Name:Novartis Pharma Services AG
    Full Title: A phase III multi-center, open label, randomised study of imatinib versus nilotinib in adult patients with newly diagnosed Philadelphia chromosome positive (Ph+) chronic myelogenous leukemia in chr...
    Medical condition: Adult patients with newly diagnosed Philadelphia chromosome positive (Ph+) chronic myelogenous leukemia in chronic phase (CML-CP)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10009015 Chronic myeloid leukemia LLT
    20.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10009013 Chronic myeloid leukaemia PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Completed) CZ (Completed) BE (Completed) IT (Completed) ES (Completed) SK (Completed) AT (Completed) FR (Completed) NL (Completed) FI (Completed) DK (Completed) DE (Completed) PT (Prematurely Ended) HU (Completed) GB (Completed)
    Trial results: View results
    EudraCT Number: 2013-005101-31 Sponsor Protocol Number: AV001 Start Date*: 2014-06-17
    Sponsor Name:Pfizer Inc
    Full Title: A Multicenter Phase 3 Randomized, Open-Label Study of Bosutinib versus Imatinib in Adult Patients with Newly Diagnosed Chronic Phase Chronic Myelogenous Leukemia
    Medical condition: Chronic Myeloid Leukemia (CML)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004864 10054352 Chronic phase chronic myeloid leukemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) GB (Completed) IT (Completed) SE (Completed) CZ (Completed) HU (Completed) SK (Completed) FI (Completed) NO (Completed) ES (Completed) DK (Completed) NL (Completed) PL (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2014-001617-12 Sponsor Protocol Number: AP24534-14-203 Start Date*: 2017-10-09
    Sponsor Name:Takeda Development Center Americas, Inc.
    Full Title: A Randomized, Open-label, Phase 2 Trial of Ponatinib in Patients with Resistant Chronic Phase Chronic Myeloid Leukemia to Characterize the Efficacy and Safety of a Range of Doses
    Medical condition: Chronic Phase Chronic Myeloid Leukemia
    Disease: Version SOC Term Classification Code Term Level
    21.0 100000004864 10054352 Chronic phase chronic myeloid leukemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Completed) CZ (Completed) DE (Completed) FI (Prematurely Ended) ES (Completed) DK (Completed) NO (Completed) BE (Completed) FR (Trial now transitioned) NL (Completed) PT (Completed) PL (Trial now transitioned) IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2006-005712-27 Sponsor Protocol Number: CA180056 Start Date*: 2007-08-01
    Sponsor Name:Bristol Myers Squibb International Corporation
    Full Title: An Open-Label, Randomized, Multicenter Phase III Trial of Dasatinib (SPRYCEL®) vs. Standard Dose Imatinib (400 mg) in the Treatment of Subjects with Newly Diagnosed Chronic Phase Philadelphia Chrom...
    Medical condition: Newly Diagnosed Chronic Phase Philadelphia Chromosome Positive Chronic Myeloid Leukemia
    Disease: Version SOC Term Classification Code Term Level
    9.1 10054352 Chronic phase chronic myeloid leukemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Completed) CZ (Completed) GR (Completed) BE (Completed) NL (Completed) DE (Completed) AT (Completed) HU (Completed) ES (Completed) IT (Completed) DK (Completed) PL (Completed)
    Trial results: View results
    EudraCT Number: 2015-001318-92 Sponsor Protocol Number: AP24534-15-303 Start Date*: 2015-11-03
    Sponsor Name:ARIAD Pharmaceuticals, Inc.
    Full Title: A Randomized, Open-label Study of Ponatinib Versus Nilotinib in Patients with Chronic Myeloid Leukemia in Chronic Phase Following Resistance to Imatinib
    Medical condition: Chronic Phase Chronic Myeloid Leukemia
    Disease: Version SOC Term Classification Code Term Level
    18.0 100000004864 10054352 Chronic phase chronic myeloid leukemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) HU (Completed) DE (Completed) GB (GB - no longer in EU/EEA) NL (Completed) ES (Completed) DK (Prematurely Ended) CZ (Completed) PT (Completed) FR (Completed) AT (Completed) PL (Completed)
    Trial results: View results
    EudraCT Number: 2005-005047-26 Sponsor Protocol Number: CAMN107A2302 Start Date*: 2007-10-15
    Sponsor Name:Novartis Pharma Services
    Full Title: A phase III multi-center, open-label, randomized study of the efficacy of nilotinib versus imatinib in adult patients with Philadelphia chromosome positive (Ph+ ) chronic myelogenous leukemia in ...
    Medical condition: Nilotinib will be evaluated in patients having showed a suboptimal cytogenetic response to imatinib
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) CZ (Prematurely Ended) ES (Completed) DE (Completed) IT (Prematurely Ended) NL (Prematurely Ended) HU (Prematurely Ended) FR (Completed) GR (Completed)
    Trial results: View results
    EudraCT Number: 2016-002461-66 Sponsor Protocol Number: CABL001A2301 Start Date*: 2017-10-09
    Sponsor Name:Novartis Pharma AG
    Full Title: A phase 3, multi-center, open-label, randomized study of oral ABL001 versus bosutinib in patients with Chronic Myelogenous Leukemia in chronic phase (CML-CP), previously treated with 2 or more tyro...
    Medical condition: Chronic Myelogenous Leukemia in chronic phase (CML-CP), previously treated with 2 or more tyrosine kinase inhibitors
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004864 10009012 Chronic myelogenous leukemia LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: HU (Completed) CZ (Completed) ES (Ongoing) BG (Completed) DE (Completed) GB (GB - no longer in EU/EEA) FR (Completed) NL (Completed) BE (Completed) IT (Completed) RO (Completed)
    Trial results: (No results available)
    EudraCT Number: 2007-006185-15 Sponsor Protocol Number: SPIRIT 2 Start Date*: 2008-04-22
    Sponsor Name:Newcastle-upon-Tyne Hospitals NHS Trust
    Full Title: STI571 Prospective International RandomIsed Trial 2 - A phase III, prospective randomised comparison of imatinib (STI571, Glivec/Gleevec) 400mg daily versus dasatinib (Sprycel) 100mg daily in pati...
    Medical condition: newly-diagnosed chronic-phase Chronic myeloid leukaemia (CML)
    Disease: Version SOC Term Classification Code Term Level
    9.1 10009013 Chronic myeloid leukaemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2018-001594-24 Sponsor Protocol Number: CABL001E2201 Start Date*: 2018-10-17
    Sponsor Name:Novartis Pharma AG
    Full Title: A phase 2, multi-center, open-label, randomized study of oral asciminib added to imatinib versus continued imatinib versus switch to nilotinib in patients with CML-CP who have been previously treat...
    Medical condition: Chronic Myelogenous Leukemia in chronic phase (CML-CP), previously treated with imatinib and have not achieved deep molecular response
    Disease: Version SOC Term Classification Code Term Level
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10009012 Chronic myelogenous leukemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: AT (Trial now transitioned) CZ (Trial now transitioned) ES (Ongoing) PT (Completed) GB (GB - no longer in EU/EEA) DE (Completed) PL (Completed) DK (Completed) FR (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2013-004867-29 Sponsor Protocol Number: CACZ885G2306 Start Date*: 2014-09-10
    Sponsor Name:Novartis Pharma Services AG
    Full Title: ß-SPECIFIC 4 Patients: Study of Pediatric EffiCacy and Safety wIth FIrst-line use of Canakinumab An open-label canakinumab (ACZ885) dose reduction or dose interval prolongation efficacy and saf...
    Medical condition: Active systemic manifestations of Systemic Juvenile Idiopathic Arthritis (SJIA)
    Disease: Version SOC Term Classification Code Term Level
    19.0 10028395 - Musculoskeletal and connective tissue disorders 10059176 Juvenile idiopathic arthritis PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: SE (Completed) AT (Completed) ES (Completed) HU (Completed) DE (Completed) IT (Completed) NL (Completed) BE (Completed)
    Trial results: View results
    EudraCT Number: 2008-007054-35 Sponsor Protocol Number: CAMN107A2404 Start Date*: 2009-08-17
    Sponsor Name:Novartis Pharma Services AG
    Full Title: A RANDOMIZED PHASE III STUDY OF IMATINIB DOSE OPTIMIZATION COMPARED WITH NILOTINIB IN PATIENTS WITH CHRONIC MYELOGENOUS LEUKEMIA AND SUBOPTIMAL RESPONSE TO STANDARD-DOSE IMATINIB
    Medical condition: IMATINIB DOSE OPTIMIZATION COMPARED WITH NILOTINIB IN PATIENTS WITH CML AND SUBOPTIMAL RESPONSE TO STANDARD-DOSE IMATINIB
    Disease: Version SOC Term Classification Code Term Level
    14.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10009012 Chronic myelogenous leukemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) FI (Completed)
    Trial results: View results
    EudraCT Number: 2018-000192-33 Sponsor Protocol Number: SB-913-1602 Start Date*: 2018-08-17
    Sponsor Name:Sangamo Therapeutics, Inc.
    Full Title: A Phase 1/2, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Tolerability of SB-913, a rAAV2/6-based Gene Transfer in Subjects with Mucopolysaccharidosis II (MPS II)
    Medical condition: Mucopolysaccharidosis type II (MPS II)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056889 Mucopolysaccharidosis II PT
    Population Age: Adolescents, Under 18, Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
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