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Clinical trials for Hereditary Diseases

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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    905 result(s) found for: Hereditary Diseases. Displaying page 1 of 46.
    1  2  3  4  5  6  7  8  9  Next» Last»»
    EudraCT Number: 2011-004104-38 Sponsor Protocol Number: ARPA-IGF1-2011 Start Date*: 2012-02-17
    Sponsor Name:FIBHULP
    Full Title:
    Medical condition:
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10017374 Friedreich's ataxia PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2015-002432-40 Sponsor Protocol Number: 042/15 Start Date*: 2016-03-03
    Sponsor Name:ASSOCIAZIONE "LA NOSTRA FAMIGLIA" - SEZIONE SCIENTIFICA I.R.C.C.S. "E.MEDEA"
    Full Title: Safety and Efficacy of yIFN treatment in Friedreich ataxia
    Medical condition: FRDA is a progressive neurodegenerative disease inherited as recessive trait. It manifests itself usually in adolescence and affects various systems including the central and peripheral nervous sys...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10017374 Friedreich's ataxia PT
    20.0 10010331 - Congenital, familial and genetic disorders 10017374 Friedreich's ataxia PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: View results
    EudraCT Number: 2014-001054-42 Sponsor Protocol Number: CSL830_3002 Start Date*: 2015-03-04
    Sponsor Name:CSL Behring GmbH
    Full Title: An open-label, randomized study to evaluate the long-term clinical safety and efficacy of subcutaneous administration of human plasma-derived C1-esterase inhibitor in the prophylactic treatment of ...
    Medical condition: Hereditary Angioedema Types I and II
    Disease: Version SOC Term Classification Code Term Level
    18.0 10010331 - Congenital, familial and genetic disorders 10019860 Hereditary angioedema PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) GB (Completed) HU (Completed) CZ (Completed) ES (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2021-003603-18 Sponsor Protocol Number: 331-201 Start Date*: 2022-10-06
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 1/2, Open-Label, Dose-Escalation Study to Determine the Safety, Tolerability, and Efficacy of BMN 331, an Adeno-Associated Virus (AAV) Vector-Mediated Gene Transfer of Human SERPING1, in Su...
    Medical condition: Hereditary Angioedema (HAE)
    Disease: Version SOC Term Classification Code Term Level
    23.1 10010331 - Congenital, familial and genetic disorders 10019860 Hereditary angioedema PT
    21.0 10010331 - Congenital, familial and genetic disorders 10080957 Hereditary angioedema C1 inhibitor deficiency LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2018-000605-24 Sponsor Protocol Number: CSL312_2001 Start Date*: 2018-12-20
    Sponsor Name:CSL Behring LLC
    Full Title: A multicenter, randomized, placebo-controlled, parallel-arm study to investigate the efficacy, pharmacokinetics, and safety of CSL312 in subjects with hereditary angioedema
    Medical condition: Hereditary angioedema
    Disease: Version SOC Term Classification Code Term Level
    23.1 10010331 - Congenital, familial and genetic disorders 10019860 Hereditary angioedema PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2010-023388-16 Sponsor Protocol Number: SNT-III-004 Start Date*: 2011-06-07
    Sponsor Name:Santhera Pharmaceuticals (Switzerland) Limited
    Full Title: A Phase IIIb Double-Blind, Randomised, Placebo-Controlled Study of Patient Reported Outcomes in Friedreich’s Ataxia Patients after withdrawal from Treatment with Idebenone (PROTI Study)
    Medical condition: Friedreich`s Ataxia
    Disease: Version SOC Term Classification Code Term Level
    12.1 10017374 Friedreich's ataxia LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed) FR (Ongoing) DE (Completed) AT (Completed) NL (Completed)
    Trial results: View results
    EudraCT Number: 2013-000916-10 Sponsor Protocol Number: CSL830_3001 Start Date*: 2013-12-10
    Sponsor Name:CSL Behring GmbH
    Full Title: A double-blind, randomized, placebo-controlled, cross-over study to evaluate the clinical efficacy and safety of subcutaneous administration of human plasma-derived C1-esterase inhibitor in the pro...
    Medical condition: Hereditary Angioedema (HAE) type I and II
    Disease: Version SOC Term Classification Code Term Level
    17.0 10010331 - Congenital, familial and genetic disorders 10019860 Hereditary angioedema PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) HU (Completed) IT (Completed) ES (Completed) CZ (Completed)
    Trial results: View results
    EudraCT Number: 2020-003918-12 Sponsor Protocol Number: CSL312_3002 Start Date*: 2021-06-08
    Sponsor Name:CSL Behring LLC
    Full Title: An Open-label Study to Evaluate the Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema
    Medical condition: Hereditary angioedema
    Disease: Version SOC Term Classification Code Term Level
    23.1 10010331 - Congenital, familial and genetic disorders 10019860 Hereditary angioedema PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Ongoing) CZ (Ongoing) HU (Ongoing) NL (Ongoing) IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-004230-37 Sponsor Protocol Number: BCX7353-312 Start Date*: Information not available in EudraCT
    Sponsor Name:BioCryst Pharmaceuticals Inc
    Full Title: An open-label study to provide berotralstat access to subjects with type 1 and 2 hereditary angioedema who were previously enrolled in berotralstat studies
    Medical condition: Hereditary Angioedema
    Disease: Version SOC Term Classification Code Term Level
    23.1 10010331 - Congenital, familial and genetic disorders 10019860 Hereditary angioedema PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: FR (Ongoing) CZ (Ongoing) SK (Ongoing) PL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2010-019670-32 Sponsor Protocol Number: B1 Start Date*: 2013-01-31
    Sponsor Name:AZIENDA OSPEDALIERA L. SACCO (A.O. DI RILIEVO NAZIONALE)
    Full Title: Pharmacokinetics and Safety of Human Pasteurised C1-Inhibitor Concentrate (Berinert/CE1145) in Subjects with Congenital C1-INH Deficiency and Frequent Hereditary Angioedema (HAE) Attacks.
    Medical condition: Hereditary Angioedema (HAE)
    Disease: Version SOC Term Classification Code Term Level
    14.1 100000004850 10056912 C1 esterase inhibitor deficiency LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2005-003139-38 Sponsor Protocol Number: CE1145_3003 Start Date*: 2015-02-12
    Sponsor Name:ZLB Behring GmbH
    Full Title: Open-label extension study of CE1145 (Human pasteurized C1 esterase inhibitor concentrate) in subjects with congenital C1-INH deficiency and acute HAE attacks
    Medical condition: Congenital C1-INH deficiency
    Disease: Version SOC Term Classification Code Term Level
    17.1 10010331 - Congenital, familial and genetic disorders 10019860 Hereditary angioedema PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2017-003281-27 Sponsor Protocol Number: BCX7353-204 Start Date*: 2017-12-27
    Sponsor Name:BioCryst Pharmaceuticals Inc
    Full Title: An Open-Label study to Evaluate the Long-Term Safety of Daily Oral BCX7353 in subjects with Type I and II Hereditary Angioedema
    Medical condition: Hereditary Angioedema
    Disease: Version SOC Term Classification Code Term Level
    23.1 10010331 - Congenital, familial and genetic disorders 10019860 Hereditary angioedema PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Completed) HU (Completed) DK (Completed) AT (Completed) ES (Completed) SK (Completed) PL (Completed) BE (Completed) NL (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2011-000987-92 Sponsor Protocol Number: C11209 Start Date*: 2011-09-26
    Sponsor Name:Pharming Technologies B.V.
    Full Title: Open-label, phase II, single arm study to evaluate the safety, immunogenicity, pharmacokinetics and efficacy of recombinant human C1 inhibitor for the treatment of acute attacks in pediatric patien...
    Medical condition: Attacks associated with hereditary angioedema (HAE) in pediatric population 2-13 years of age.
    Disease: Version SOC Term Classification Code Term Level
    17.1 10010331 - Congenital, familial and genetic disorders 10019860 Hereditary angioedema PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) IT (Completed) CZ (Completed) SK (Completed) HU (Completed)
    Trial results: View results
    EudraCT Number: 2020-000570-25 Sponsor Protocol Number: CSL312_3001 Start Date*: 2021-02-09
    Sponsor Name:CSL Behring LLC
    Full Title: A multicenter, double-blind, randomized, placebo-controlled, parallel-arm study to investigate the efficacy and safety of subcutaneous administration of CSL312 (garadacimab) in the prophylactic tr...
    Medical condition: Hereditary angioedema
    Disease: Version SOC Term Classification Code Term Level
    23.1 10010331 - Congenital, familial and genetic disorders 10019860 Hereditary angioedema PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) HU (Completed) NL (Completed) IT (Ongoing)
    Trial results: View results
    EudraCT Number: 2021-000171-36 Sponsor Protocol Number: C20-54 Start Date*: 2021-03-23
    Sponsor Name:INSERM
    Full Title: PHASE I-II EFFICACY-TOXICITY OF ARTESUNATE IN FRIEDREICH ATAXIA
    Medical condition: Friedreich Ataxia
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10017374 Friedreich's ataxia PT
    Population Age: Adolescents, Under 18, Adults Gender: Male
    Trial protocol: FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2014-002655-26 Sponsor Protocol Number: BCX4161-301 Start Date*: 2015-02-09
    Sponsor Name:BioCryst Pharmaceuticals Inc
    Full Title: OPuS-2 A multicentre, randomised, double blind, placebo controlled, parallel group study to evaluate the efficacy and safety of two dose levels of BCX4161 for 12 weeks as an oral prophylaxis treatm...
    Medical condition: Hereditary Angioedema
    Disease: Version SOC Term Classification Code Term Level
    17.1 10010331 - Congenital, familial and genetic disorders 10019860 Hereditary angioedema PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) DE (Completed) HU (Completed) BE (Completed) FR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2022-002621-98 Sponsor Protocol Number: SHP643-302 Start Date*: 2022-08-19
    Sponsor Name:Takeda
    Full Title: A Phase 3 Multi-center, Open-label Study to Evaluate the Efficacy and Safety of Lanadelumab (SHP643) in Japanese Subjects with Hereditary Angioedema
    Medical condition: Hereditary Angioedema (HAE)
    Disease: Version SOC Term Classification Code Term Level
    23.1 10010331 - Congenital, familial and genetic disorders 10075280 Hereditary angioedema attack LLT
    23.1 10010331 - Congenital, familial and genetic disorders 10019860 Hereditary angioedema PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2011-002744-27 Sponsor Protocol Number: CR01849 Start Date*: 2011-11-01
    Sponsor Name:Imperial College
    Full Title: Pharmacodynamic Studies of a Histone Deacetylase Inhibitor in FRDA
    Medical condition: Friedreich’s ataxia
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10017374 Friedreich's ataxia PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2019-002618-38 Sponsor Protocol Number: 721 Start Date*: 2020-06-25
    Sponsor Name:ASSOCIAZIONE "LA NOSTRA FAMIGLIA" - SEZIONE SCIENTIFICA I.R.C.C.S. "E.MEDEA"
    Full Title: Clinical trial - phase II - to test safety and efficay of Etravirine's treatment in Friedreich Ataxia's patients
    Medical condition: Friedreich's ataxia
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10017374 Friedreich's ataxia PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2013-002319-82 Sponsor Protocol Number: BCX4161-203 Start Date*: 2013-09-30
    Sponsor Name:BioCryst Pharmaceuticals Inc.
    Full Title: A Phase 2a double-blind placebo-controlled 2-period crossover study to evaluate the safety and efficacy of BCX4161 as a prophylactic treatment to reduce the frequency of attacks in subjects with he...
    Medical condition: Hereditary Angioedema
    Disease: Version SOC Term Classification Code Term Level
    16.1 10010331 - Congenital, familial and genetic disorders 10019860 Hereditary angioedema PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DE (Completed) GB (Completed)
    Trial results: View results
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