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Clinical trials for Congenital disorders

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
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    • 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   44339   clinical trials with a EudraCT protocol, of which   7369   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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    1,385 result(s) found for: Congenital disorders. Displaying page 7 of 70.
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    EudraCT Number: 2011-001187-21 Sponsor Protocol Number: SCRM-001 Start Date*: 2011-11-28
    Sponsor Name:University of Zurich
    Full Title: A Phase I/II, Open Monocentric Study To Evaluate The Safety And Efficacy Of An Autologous Tissue-Engineered Vascular Graft In Peadiatric Patients Requiring Reconstruction Of Right Ventricle Outflow...
    Medical condition: functional single ventricle physiology functional single left ventricle functional single right ventricle functional single biventricle
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10040729 Single ventricle LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2015-001919-13 Sponsor Protocol Number: NN1731-4214 Start Date*: 2016-03-17
    Sponsor Name:Novo Nordisk A/S
    Full Title: Pharmacogenetic testing of saliva samples from patients with ≥5 exposure days to rFVIIa analogue in the adept™2 trial. Bio-specimen research study
    Medical condition: - Haemophilia A with inhibitors - Haemophilia B with inhibitors
    Disease: Version SOC Term Classification Code Term Level
    18.1 10010331 - Congenital, familial and genetic disorders 10056492 Haemophilia A with anti factor VIII PT
    18.1 10010331 - Congenital, familial and genetic disorders 10056494 Haemophilia B with anti factor IX PT
    Population Age: Adolescents, Under 18, Adults Gender: Male
    Trial protocol: RO (Completed)
    Trial results: View results
    EudraCT Number: 2022-000241-32 Sponsor Protocol Number: NL80290.018.22 Start Date*: 2022-08-08
    Sponsor Name:Amsterdam UMC
    Full Title: Effectiveness of L-serine dietary supplementation in children with a GRIN2B loss-of-function mutation: n-of-1 series
    Medical condition: GRIN2B deficiency
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10027424 Metabolic and nutritional disorders congenital HLGT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: NL (Completed)
    Trial results: (No results available)
    EudraCT Number: 2022-003853-70 Sponsor Protocol Number: PNRR-MR1-2022-12375914 Start Date*: 2023-05-02
    Sponsor Name:OSPEDALE SAN RAFFAELE
    Full Title: Towards a personalized precision medicine in rare disease: tirzepatide (a dual glucose­ dependent insulinotropic polypeptide and glucagon-like peptide-I receptor agonist) monotherapy in patients wi...
    Medical condition: Wolfram syndrome type 1
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10078338 Wolfram syndrome PT
    20.0 10010331 - Congenital, familial and genetic disorders 10078338 Wolfram syndrome PT
    20.0 10010331 - Congenital, familial and genetic disorders 10078338 Wolfram syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2021-002550-82 Sponsor Protocol Number: NL76160.018.21 Start Date*: 2022-12-22
    Sponsor Name:Amsterdam UMC
    Full Title: Effectiveness of ambroxol in children and adults with Gaucher disease 3: n-of-1 series
    Medical condition: Gaucher disease type 3
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10027424 Metabolic and nutritional disorders congenital HLGT
    Population Age: Children, Under 18, Adults Gender: Male, Female
    Trial protocol: NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2016-000067-16 Sponsor Protocol Number: AMO-02-MD-2-001 Start Date*: 2016-04-20
    Sponsor Name:AMO Pharma Ltd.
    Full Title: A Single-Blind, Phase 2 Study To Evaluate The Safety And Efficacy Of Tideglusib 400 mg Or 1000 mg For The Treatment Of Adolescent And Adult Congenital And Juvenile-Onset Myotonic Dystrophy
    Medical condition: Treatment of adolescent and adult congenital and juvenile onset myotonic dystrophy
    Disease: Version SOC Term Classification Code Term Level
    19.1 10010331 - Congenital, familial and genetic disorders 10068871 Myotonic dystrophy PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2020-000996-19 Sponsor Protocol Number: MAA-304 Start Date*: Information not available in EudraCT
    Sponsor Name:Catalyst Biosciences, Inc.
    Full Title: Phase 3 Study to Evaluate the Efficacy and Safety of Subcutaneous Marzeptacog Alfa (Activated) For On-Demand Treatment and Control of Bleeding Episodes in Subjects with Haemophilia A or Haemophilia...
    Medical condition: Marzeptacog alfa (activated) (MarzAA), a novel activated recombinant Factor VII (rFVIIa) variant, is being developed by Catalyst Biosciences to address the unmet need in haemophilia and other bleed...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10066439 Hemophilia LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) HU (Prematurely Ended) PL (Prematurely Ended) DK (Prematurely Ended) BG (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2018-003076-13 Sponsor Protocol Number: UIC1212 Start Date*: 2019-10-14
    Sponsor Name:Instituto Português de Oncologia de Lisboa Francisco Gentil
    Full Title: Effect of topical mesalazine on the formation of adenomas in rectal stumps or ileal pouches of familial adenomatous polyposis or MUTYH associated polyposis patients after colectomy
    Medical condition: Familial adenomatous polyposis(FAP) and MUTYH-associated polyposis (MAP)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056981 Adenomatous polyposis coli PT
    20.1 10010331 - Congenital, familial and genetic disorders 10059327 Familial adenomatous polyposis LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: PT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2015-005524-26 Sponsor Protocol Number: GBI1406 Start Date*: 2016-07-29
    Sponsor Name:Grifols Biologicals Inc.
    Full Title: A Multicenter Phase 2 Open-Label, Single-Arm, Prospective, Interventional Study of Plasma-Derived Factor VIIIIVWF Alphanate® in Immune Tolerance Induction Therapy in Subjects with Congenital Hemoph...
    Medical condition: Inhibitors in patients with severe Congenital Haemophilia A
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004851 10018847 Haematological disorders HLT
    Population Age: Newborns, Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: ES (Prematurely Ended) IT (Completed)
    Trial results: View results
    EudraCT Number: 2020-001157-48 Sponsor Protocol Number: 20HH5896 Start Date*: 2020-07-28
    Sponsor Name:Imperial College London
    Full Title: Identifying a safe and tolerated dose of Imatinib for patients with Pulmonary Arterial Hypertension (PAH)
    Medical condition: Pulmonary Arterial Hypertension (Idiopathic, heritable, or assosicated with anorexigens)
    Disease: Version SOC Term Classification Code Term Level
    21.1 10038738 - Respiratory, thoracic and mediastinal disorders 10036727 Primary pulmonary hypertension LLT
    21.1 10038738 - Respiratory, thoracic and mediastinal disorders 10037400 Pulmonary hypertension PT
    21.1 10038738 - Respiratory, thoracic and mediastinal disorders 10037400 Pulmonary hypertension PT
    21.1 10038738 - Respiratory, thoracic and mediastinal disorders 10068739 Chronic thromboembolic pulmonary hypertension LLT
    20.0 10010331 - Congenital, familial and genetic disorders 10050701 Congenital pulmonary hypertension PT
    20.0 10010331 - Congenital, familial and genetic disorders 10050701 Congenital pulmonary hypertension PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2010-020558-33 Sponsor Protocol Number: 997HA301 Start Date*: 2011-04-14
    Sponsor Name:Biogen Idec Hemophilia, Inc.
    Full Title: A-LONG: An Open-label, Multicenter Evaluation of the Safety, Pharmacokinetics, and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in the Prevention and Treatment of Bleeding in ...
    Medical condition: Severe hemophilia A - defined as <1 IU/dL (<1%) endogenous FVIII
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10056493 Haemophilia A without inhibitors PT
    14.1 10010331 - Congenital, familial and genetic disorders 10053753 Hemophilia A without inhibitors LLT
    14.1 10010331 - Congenital, familial and genetic disorders 10018937 Haemophilia A LLT
    14.1 10010331 - Congenital, familial and genetic disorders 10060612 Hemophilia A LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male
    Trial protocol: SE (Completed) GB (Completed) FR (Ongoing) DE (Completed) BE (Completed) ES (Completed) AT (Completed) IT (Completed) PT (Completed)
    Trial results: View results
    EudraCT Number: 2020-003807-32 Sponsor Protocol Number: BT200-02 Start Date*: 2020-12-16
    Sponsor Name:Medical University of Vienna
    Full Title: A Phase 2a Multiple Dose “Basket Design” Study Of The Safety, Tolerability, And Pharmacologic Activity Of BT200 In Patients With Hereditary Bleeding Disorders
    Medical condition: - Patients with severe congenital hemophilia A - Patients with mild-moderate hemophilia A - Heterozygous carriers of hemophilia A - Patients with VWD Type 1, e.g. “Vicenza” type - Acquired Von Will...
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: AT (Completed)
    Trial results: View results
    EudraCT Number: 2008-001563-11 Sponsor Protocol Number: GENA-01 Start Date*: 2009-10-22
    Sponsor Name:Octapharma AG
    Full Title: Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety and Immunogenicity of human-cl rhFVIII, a Newly Developed Human Cell-Line Derived Recombinant FVIII Concentrate in Previously Tr...
    Medical condition: Severe hemophilia A (FVIII:C <1%)
    Disease:
    Population Age: Adolescents, Under 18, Adults Gender: Male
    Trial protocol: DE (Completed) BG (Completed)
    Trial results: View results
    EudraCT Number: 2021-003537-11 Sponsor Protocol Number: 19855 Start Date*: 2024-03-14
    Sponsor Name:Bayer Healthcare Company Limited
    Full Title: A Post Approval Commitment study to evaluate the efficacy, safety, and pharmacokinetics of KOVALTRY in Chinese children, adolescents/adults with severe hemophilia A.
    Medical condition: KOVALTRY, indicated for use in adults and children with hemophilia A (congenital Factor VIII deficiency) for: - Routine prophylaxis to reduce the frequency of bleeding episodes - On-demand treatmen...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10060612 Hemophilia A LLT
    20.0 10042613 - Surgical and medical procedures 10071818 Bleeding prophylaxis LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2019-001693-28 Sponsor Protocol Number: CONE-01 Start Date*: 2020-02-06
    Sponsor Name:Octapharma Pharmazeutika Produktionsges.m.b.H.
    Full Title: Prospective, open-label, single arm, multicenter, pharmacokinetic, and safety study of a single dose intravenous human plasma-derived C1 Esterase Inhibitor (C1-INH) concentrate in patients with con...
    Medical condition: Hereditary angioedema type I and type II
    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 10080956 Hereditary angioedema type I LLT
    21.0 10010331 - Congenital, familial and genetic disorders 10080960 Hereditary angioedema type II LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2020-003596-17 Sponsor Protocol Number: CMAS825D12201 Start Date*: 2021-03-09
    Sponsor Name:Novartis Pharma AG
    Full Title: A three-period multicenter study, with a randomized withdrawal, double-blinded, placebo-controlled design in Period 2 to evaluate the clinical efficacy, safety and tolerability of MAS825 in NLRC4-G...
    Medical condition: NLRC4-GOF, AIFEC (autoinflammation with infantile enterocolitis)
    Disease: Version SOC Term Classification Code Term Level
    23.1 10010331 - Congenital, familial and genetic disorders 10084306 Autoinflammation with infantile enterocolitis PT
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) CZ (Trial now transitioned) IT (Trial now transitioned) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2018-002210-12 Sponsor Protocol Number: ID-069A302 Start Date*: 2019-03-18
    Sponsor Name:Idorsia Pharmaceuticals Ltd
    Full Title: A multi-center, open-label, uncontrolled, single-arm, extension study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease
    Medical condition: Fabry disease
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    20.0 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Trial now transitioned) FR (Trial now transitioned) AT (Trial now transitioned) NL (Completed) BE (Trial now transitioned) NO (Trial now transitioned) IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2014-002839-33 Sponsor Protocol Number: C1-3201 Start Date*: 2015-07-09
    Sponsor Name:Pharming Group NV
    Full Title: A Phase 2 Multicenter, Randomized, Double-Blind, Placebo-Controlled, 3-Period Crossover Study to Evaluate the Efficacy and Safety of Recombinant Human C1 Inhibitor in the Prophylaxis of Angioedema ...
    Medical condition: Prophylaxis of Angioedema Attacks on Patients with Hereditary Angioedema (HAE)
    Disease: Version SOC Term Classification Code Term Level
    17.1 10010331 - Congenital, familial and genetic disorders 10027664 Congenital and hereditary disorders NEC HLGT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: CZ (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2008-003960-20 Sponsor Protocol Number: NN7008-3543 Start Date*: 2009-06-26
    Sponsor Name:Novo Nordisk A/S
    Full Title: A Multi-Centre, Open-Label, Non-Controlled Trial on Safety and Efficacy of N8 in Prevention and Treatment of Bleeds in Previously Treated Subjects with Haemophilia A Sub-Trial: Safety and Efficacy ...
    Medical condition: Haemophilia A
    Disease: Version SOC Term Classification Code Term Level
    13.1 10010331 - Congenital, familial and genetic disorders 10018937 Haemophilia A LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male
    Trial protocol: DE (Completed) GB (Completed) ES (Completed) IT (Completed) DK (Completed)
    Trial results: View results
    EudraCT Number: 2015-002692-18 Sponsor Protocol Number: 20150616 Start Date*: 2015-10-29
    Sponsor Name:European Commission
    Full Title: The physiology of glucagon-like peptide-1 receptor expression in patients with endogenous hyperinsulinism: correlation with histopathology
    Medical condition: Congenital hyperinsulinism
    Disease: Version SOC Term Classification Code Term Level
    18.0 10027433 - Metabolism and nutrition disorders 10061211 Hyperinsulinism PT
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: NL (Not Authorised)
    Trial results: (No results available)
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