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Clinical trials for Homozygous

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   44334   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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    194 result(s) found for: Homozygous. Displaying page 1 of 10.
    1  2  3  4  5  6  7  8  9  Next» Last»»
    EudraCT Number: 2020-002755-38 Sponsor Protocol Number: CKJX839C12302 Start Date*: 2020-12-30
    Sponsor Name:Novartis Pharma AG
    Full Title: Two part (double-blind) inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2] randomized multicentre study to evaluate safety, tolerability, and efficacy of inclisiran in ad...
    Medical condition: Homozygous Familial Hypercholesterolemia
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10057080 Homozygous familial hypercholesterolemia LLT
    Population Age: Adolescents, Under 18 Gender: Male, Female
    Trial protocol: SI (Completed) GR (Completed) NL (Completed) FR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2023-000946-41 Sponsor Protocol Number: VX19-809-124 Start Date*: 2023-10-17
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Phase 3, Open-label, and Rollover Study to Evaluate the Long-term Safety and Tolerability of Lumacaftor/Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Homozygous for F508del and 12 ...
    Medical condition: Cystic Fibrosis
    Disease:
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2020-005111-51 Sponsor Protocol Number: 20170199 Start Date*: 2020-11-05
    Sponsor Name:Amgen Inc.
    Full Title: A Multicenter, Open-label, Single-arm, Study to Evaluate Safety and Tolerability of Repatha in Patients with Homozygous Familial Hypercholesterolemia (HoFH) in India
    Medical condition: Homozygous Familial Hypercholesterolemia (HoFH)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10057080 Homozygous familial hypercholesterolemia LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2010-023742-79 Sponsor Protocol Number: AEG 733-012 Start Date*: 2011-03-25
    Sponsor Name:AEGERION PHARMACEUTICALS
    Full Title: A Phase III, Long Term, Open Label, Follow on Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor ‘lomitapide’ (AEGR-733) in Patients with Homozygous Familial Hypercholesterolemia
    Medical condition: Homozygous Familial Hypercholesterolemia
    Disease: Version SOC Term Classification Code Term Level
    9.1 10057080 LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: View results
    EudraCT Number: 2016-000411-32 Sponsor Protocol Number: R1500-CL-1331 Start Date*: 2016-07-15
    Sponsor Name:Regeneron Pharmaceuticals, Inc.
    Full Title: AN OPEN-LABEL, SINGLE-ARM, PROOF-OF-CONCEPT STUDY TO EVALUATE THE SAFETY AND EFFICACY OF SINGLE AND MULTIPLE DOSES OF REGN1500 IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA
    Medical condition: Homozygous Familial Hypercholesterolemia
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004850 10057080 Homozygous familial hypercholesterolemia LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (Completed)
    Trial results: View results
    EudraCT Number: 2016-003376-49 Sponsor Protocol Number: MDCO-PCS-16-02 Start Date*: 2017-06-20
    Sponsor Name:The Medicines Company
    Full Title: An Open-Label, Single-Arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of ALN-PCSSC in Subjects with Homozygous Familial Hypercholesterolemia
    Medical condition: Homozygous Familial Hypercholesterolemia
    Disease: Version SOC Term Classification Code Term Level
    19.1 100000004850 10057080 Homozygous familial hypercholesterolemia LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: NL (Completed)
    Trial results: View results
    EudraCT Number: 2011-005399-40 Sponsor Protocol Number: 20110233 Start Date*: 2012-03-09
    Sponsor Name:Amgen Inc
    Full Title: A two part, Phase 2/3 Study to Assess the Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia Part A - Open-label, Single-arm, Multicenter Pilot...
    Medical condition: Homozygous familial hypercholesterolaemia
    Disease: Version SOC Term Classification Code Term Level
    14.1 100000004850 10057100 Homozygous familial hypercholesterolaemia LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) CZ (Completed) ES (Completed) IT (Completed) NL (Completed) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2005-003449-15 Sponsor Protocol Number: 301012-CS5 Start Date*: 2007-07-12
    Sponsor Name:Genzyme Europe B.V.
    Full Title: A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of ISIS 301012 as Add-on Therapy in Homozygous Familial Hypercholesterolemia Subjects
    Medical condition: Reduction of low density lipoprotein C (LDL-C) in Homozygous Familial Hypercholesterolemia (HoFH)
    Disease: Version SOC Term Classification Code Term Level
    9.1 10057080 Homozygous familial hypercholesterolemia LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2015-002276-25 Sponsor Protocol Number: 20120124 Start Date*: 2016-10-21
    Sponsor Name:Amgen Inc
    Full Title: Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects Fr...
    Medical condition: Hypercholesterolemia
    Disease: Version SOC Term Classification Code Term Level
    18.1 100000004850 10057100 Homozygous familial hypercholesterolaemia LLT
    18.1 100000004850 10057099 Heterozygous familial hypercholesterolaemia LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) GB (GB - no longer in EU/EEA) NO (Completed) HU (Completed) AT (Completed) BE (Completed) CZ (Completed) GR (Completed) ES (Completed) NL (Completed) PT (Completed) SI (Completed) PL (Completed) IT (Completed) RO (Completed)
    Trial results: View results
    EudraCT Number: 2017-000351-95 Sponsor Protocol Number: R727-CL-1628 Start Date*: 2017-07-18
    Sponsor Name:Regeneron Pharmaceuticals, Inc.
    Full Title: A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP STUDY TO EVALUATE THE EFFICACY AND SAFETY OF ALIROCUMAB IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA
    Medical condition: HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10057080 Homozygous familial hypercholesterolemia LLT
    Population Age: Adolescents, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed) AT (Completed) GR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2011-005400-15 Sponsor Protocol Number: 20110271 Start Date*: 2012-03-22
    Sponsor Name:Amgen Inc
    Full Title: A Multicenter, Open-label Study to Assess the Long-term Safety, Tolerability, and Efficacy of AMG 145 on LDL-C in Subjects With Severe Familial Hypercholesterolemia
    Medical condition: Familial Hypercholesterolemia
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000012386 10057100 Homozygous familial hypercholesterolaemia LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) GR (Completed) CZ (Completed) GB (Completed) ES (Completed) IT (Completed) NL (Completed)
    Trial results: View results
    EudraCT Number: 2008-007058-36 Sponsor Protocol Number: 733-005 Start Date*: 2009-06-25
    Sponsor Name:Aegerion Pharmaceuticals
    Full Title: A Phase III Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor AEGR-733 in Patients with Homozygous Familial Hypercholesterolemia on Current Lipid-lowering Therapy
    Medical condition: Homozygous Familial Hypercholesterolemia
    Disease: Version SOC Term Classification Code Term Level
    9.1 10048373 Hypercholesterolaemia aggravated LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: View results
    EudraCT Number: 2016-004996-33 Sponsor Protocol Number: VX15-809-114 Start Date*: 2017-05-03
    Sponsor Name:Vertex Pharmaceuticals Incoporated
    Full Title: A Phase 4, Open-label Treatment, Randomized, Multicenter, 2-arm, Parallelgroup, Pilot Study of Adherence to Lumacaftor/Ivacaftor in CF Subjects Homozygous for the F508del-CFTR Mutation
    Medical condition: Cystic fibrosis subjects Homozygous for the F508del-CFTR Mutation
    Disease: Version SOC Term Classification Code Term Level
    19.1 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2017-004794-13 Sponsor Protocol Number: VX16-809-122 Start Date*: 2020-05-01
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Phase 3, 2-part, Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor in Subjects 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del
    Medical condition:
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10011762 Cystic fibrosis PT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2019-002189-11 Sponsor Protocol Number: VX15-661-112 Start Date*: 2019-05-27
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Phase 2, Randomized, Placebo-Controlled, Double-blind Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects Aged 12 Years and Older With Cyst...
    Medical condition: Cystic Fibrosis
    Disease:
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2022-001629-65 Sponsor Protocol Number: D7830C00004 Start Date*: 2023-03-13
    Sponsor Name:ASTRAZENECA AB
    Full Title: A Randomised, Double-blind, Placebo-Controlled, Multi-Center Phase 2b Study to Evaluate the Efficacy, Safety and Tolerability of AZD2693 in Participants with Non-Cirrhotic Non-Alcoholic Steatohepat...
    Medical condition: Non-cirrhotic non-alcoholic steatohepatitis with fibrosis
    Disease: Version SOC Term Classification Code Term Level
    24.1 10019805 - Hepatobiliary disorders 10053219 Non-alcoholic steatohepatitis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2016-001446-25 Sponsor Protocol Number: FHGT002 Start Date*: 2017-09-20
    Sponsor Name:University of Pennsylvania
    Full Title: AAV8-mediated Low Density Lipoprotein Receptor (LDLR) Gene Replacement in Subjects with Homozygous Familial Hypercholesterolemia (HoFH)
    Medical condition: Adults with homozygous familial hypercholesterolemia
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (Completed) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2012-001638-33 Sponsor Protocol Number: IKEM-CEM-01 Start Date*: 2012-10-10
    Sponsor Name:Institute for Clinical and Experimental Medicine
    Full Title: Cholesterol 7alpha-hydroxylase polymorphism as a predictor cholesterolemia responsiveness
    Medical condition: hypercholesterolemia
    Disease: Version SOC Term Classification Code Term Level
    14.1 10027433 - Metabolism and nutrition disorders 10020604 Hypercholesterolemia LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: CZ (Completed)
    Trial results: (No results available)
    EudraCT Number: 2017-003761-99 Sponsor Protocol Number: VX16-809-121 Start Date*: 2018-06-27
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: An Exploratory Phase 2, 2-part, Randomized, Double blind, Placebo controlled Study With a Long term, Open label Period to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjec...
    Medical condition: Cystic Fibrosis
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10011762 Cystic fibrosis PT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2017-003170-13 Sponsor Protocol Number: R1500-CL-1719 Start Date*: 2018-03-15
    Sponsor Name:Regeneron Pharmaceuticals, Inc.
    Full Title: An open-label study to evaluate the long-term safety and efficacy of evinacumab in patients with homozygous familial hypercholesterolemia
    Medical condition: Homozygous familial hypercholesterolemia
    Disease: Version SOC Term Classification Code Term Level
    21.0 100000004861 10020604 Hypercholesterolemia LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: NL (Completed) NO (Prematurely Ended) CZ (Completed) FR (Completed) GR (Completed) GB (GB - no longer in EU/EEA) AT (Completed) IT (Completed)
    Trial results: View results
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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