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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
  • 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   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 4 of 70.
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    EudraCT Number: 2018-000368-27 Sponsor Protocol Number: MAN-ALG-18-001 Start Date*: 2018-10-22
    Sponsor Name:FONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS UNIVERSITA' CATTOLICA DEL SACRO CUORE
    Full Title: ND
    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
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    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: 2010-023666-46 Sponsor Protocol Number: F8VR-1006 Start Date*: 2011-12-13
    Sponsor Name:LFB BIOTECHNOLOGIES
    Full Title: EFFICACY AND SAFETY STUDY OF FACTANE 200 IU/ml ADMINISTERED BY CONTINUOUS INFUSION IN SEVERE HAEMOPHILIA A PATIENTS DURING MAJOR SURGICAL PROCEDURES
    Medical condition: HAEMOPHILIA A
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10018938 Haemophilia A (Factor VIII) LLT
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: PL (Completed)
    Trial results: View results
    EudraCT Number: 2005-004435-22 Sponsor Protocol Number: AVI-40-3 Start Date*: 2005-12-13
    Sponsor Name:OCTAPHARMA AG
    Full Title: INCIDENCE OF INHIBITORS IN PREVIOUSLY UNTREATED PATIENTS WITH SEVERE HEAMOPHILIA A TREATED WITH OCTANATE
    Medical condition: Previously untreated patients with severe (FVIII:C<2%) haemophilia A, no inhibitor activity prior to admission, no concomitant therapy with Interferon, patients registered for regular treatment at ...
    Disease: Version SOC Term Classification Code Term Level
    8.0 10016080 PT
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: CZ (Completed) FR (Not Authorised)
    Trial results: View results
    EudraCT Number: 2021-003907-16 Sponsor Protocol Number: SPIMD-301 Start Date*: 2022-03-02
    Sponsor Name:Stealth BioTherapeutics Inc.
    Full Title: A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondr...
    Medical condition: Primary Mitochondrial Disease Resulting from Pathogenic Nuclear DNA Mutations (nPMD)
    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 10052635 Cytoplasmic disorders congenital HLGT
    20.0 10010331 - Congenital, familial and genetic disorders 10052637 Genetic mitochondrial abnormalities NEC HLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: HU (Completed) NO (Completed) ES (Completed) IT (Completed) NL (Completed) DE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2009-016869-28 Sponsor Protocol Number: F13CD-3760 (mentor™4) Start Date*: 2010-09-20
    Sponsor Name:Novo Nordisk A/S
    Full Title: A Phase 3b Trial Investigating the Pharmacokinetics and Safety Profile of a Single Intravenous Dose of rFXIII in Paediatric (1 to less than 6 Years Old) Subjects with Congenital FXIII A-subunit Def...
    Medical condition: Congenital FXIII A-subunit Deficiency
    Disease: Version SOC Term Classification Code Term Level
    13.1 10010331 - Congenital, familial and genetic disorders 10016083 Factor XIII deficiency PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2011-003633-33 Sponsor Protocol Number: MAXEPA01 Start Date*: 2011-09-29
    Sponsor Name:Central Manchester University Hospitals NHS Foundation Trust
    Full Title: The effect of purified fish oils on glucose regulation in Congenital Hyperinsulinism of Infancy (CHI)
    Medical condition: Congenital hyperinsulinism
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10062329 Congenital endocrine anomaly PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2014-003764-20 Sponsor Protocol Number: BI71023_3002 Start Date*: 2015-03-11
    Sponsor Name:CSL Behring LLC
    Full Title: A Prospective, Multicenter, Open Enrollment Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
    Medical condition: Congenital Factor XIII deficiency
    Disease: Version SOC Term Classification Code Term Level
    17.1 10010331 - Congenital, familial and genetic disorders 10016083 Factor XIII deficiency PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2015-001096-43 Sponsor Protocol Number: PHRN14-AM/PERFORMUS Start Date*: 2015-07-10
    Sponsor Name:CHRU de Tours
    Full Title: Treatment of superficial voluminous complicated slow-flow vascular malformations with sirolimus: a phase 2 trial in children observational-phase designed
    Medical condition: vascular malformations
    Disease: Version SOC Term Classification Code Term Level
    18.0 10010331 - Congenital, familial and genetic disorders 10074979 Vascular malformation PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2010-020192-23 Sponsor Protocol Number: F13CD-3835 Start Date*: 2010-09-20
    Sponsor Name:Novo Nordisk A/S
    Full Title: A Multi-Centre, Multinational, Open-Label, Single-Arm and Multiple Dosing Trial on Safety and Efficacy of Monthly Replacement Therapy with Recombinant Factor XIII (rFXIII) in Paediatric Subjects wi...
    Medical condition: Congenital Factor XIII A-subunit Deficiency
    Disease: Version SOC Term Classification Code Term Level
    17.0 10010331 - Congenital, familial and genetic disorders 10016083 Factor XIII deficiency PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2013-003240-23 Sponsor Protocol Number: 967908487 Start Date*: 2013-12-09
    Sponsor Name:St. James' Hospital
    Full Title: PERSONALising Factor VIII prophylaxis regimens: Efficacy of standard versus pharmacokinetically based regimens in adult patients with severe Haemophilia A (PERSONAL trial)
    Medical condition: Severe Haemophilia A
    Disease: Version SOC Term Classification Code Term Level
    14.1 100000004850 10018937 Haemophilia A LLT
    Population Age: Adults Gender: Male
    Trial protocol: IE (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2020-005908-18 Sponsor Protocol Number: 1111_1111 Start Date*: 2021-11-03
    Sponsor Name:AOU FEDERICO II
    Full Title: Functional-genetic stratification as a guide to personalized treatment in developmental and epileptic encephalopathies due to potassium channel mutations
    Medical condition: Epileptic Encephalopathy with developmental delay
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10015039 Epilepsy congenital PT
    20.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    20.0 10029205 - Nervous system disorders 10029205 Nervous system disorders SOC
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2017-001631-39 Sponsor Protocol Number: AT342-02 Start Date*: Information not available in EudraCT
    Sponsor Name:Audentes Therapeutics Inc.
    Full Title: VALENS: A Phase 1/2, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT342, an AAV8-Delivered Gene Tra...
    Medical condition: Crigler Najjar syndrome
    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 10011386 Crigler-Najjar syndrome PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2021-004307-40 Sponsor Protocol Number: ATN-106 Start Date*: 2022-01-31
    Sponsor Name:Octapharma AG
    Full Title: A multicentre, prospective, open-label, uncontrolled Phase 3 study to assess the efficacy, safety and pharmacokinetics of Atenativ in patients with congenital antithrombin deficiency undergoing sur...
    Medical condition: Congenital antithrombin deficiency
    Disease: Version SOC Term Classification Code Term Level
    23.0 100000004850 10083881 Antithrombin deficiency LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: HU (Trial now transitioned) AT (Trial now transitioned) FR (Trial now transitioned) IT (Trial now transitioned) DE (Trial now transitioned) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2008-005945-46 Sponsor Protocol Number: NN7008-3568 Start Date*: 2010-01-18
    Sponsor Name:Novo Nordisk A/S
    Full Title: Safety and Efficacy of N8 in Prevention and On-demand Treatment of Bleeding Episodes in Subjects with Haemophilia A
    Medical condition: Haemophilia A
    Disease: Version SOC Term Classification Code Term Level
    16.1 100000004850 10018937 Haemophilia A LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: DE (Completed) ES (Completed) IT (Completed) GB (Completed) PL (Completed) LT (Completed) LV (Completed)
    Trial results: View results
    EudraCT Number: 2009-012150-20 Sponsor Protocol Number: BAY81-8973/14319 Start Date*: 2011-02-02
    Sponsor Name:Bayer HealthCare AG
    Full Title: A phase II/III, randomized, cross-over, open-label trial to demonstrate superiority of prophylaxis over on-demand therapy in previously treated subjects with severe hemophilia A treated with plasma...
    Medical condition: Severe Hemophilia-A (< 1% FVIII:C)
    Disease: Version SOC Term Classification Code Term Level
    13.1 10010331 - Congenital, familial and genetic disorders 10060613 Hemophilia A (Factor VIII) LLT
    13.1 10010331 - Congenital, familial and genetic disorders 10053753 Hemophilia A without inhibitors LLT
    13.1 10010331 - Congenital, familial and genetic disorders 10060612 Hemophilia A LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male
    Trial protocol: CZ (Completed)
    Trial results: View results
    EudraCT Number: 2011-001785-17 Sponsor Protocol Number: GENA-13 Start Date*: 2011-09-27
    Sponsor Name:Octapharma AG
    Full Title: Clinical Study in Previously Treated Children with Severe Haemophilia A to Investigate the Long-Term Immunogenicity, Tolerability and Efficacy of Human-cl rhFVIII
    Medical condition: Severe Haemophilia A
    Disease: Version SOC Term Classification Code Term Level
    17.0 100000004850 10018938 Haemophilia A (Factor VIII) LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (Completed) FR (Completed) CZ (Completed) PL (Completed) RO (Completed)
    Trial results: View results
    EudraCT Number: 2016-001764-11 Sponsor Protocol Number: PTC124-GD-030-DMD Start Date*: 2020-01-21
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A Phase 2 Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Ataluren (PTC124®) in Patients Aged ≥2 to <5 Years Old With Nonsense Mutation Dystrophinopathy
    Medical condition: nonsense mutation dystrophinopathy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    20.0 10010331 - Congenital, familial and genetic disorders 10059117 Becker's muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2017-004130-28 Sponsor Protocol Number: ZMK_201501-ONF Start Date*: 2018-07-25
    Sponsor Name:Universitätsklinikum Bonn
    Full Title: Occurence of Oronasal Fistulas (ONF) using a fibrin sealant patch in primary palatoplasty
    Medical condition: Cleft lip and palate
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10009260 Cleft lip and palate PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2011-001144-30 Sponsor Protocol Number: NN7088-3860 Start Date*: 2012-05-15
    Sponsor Name:Novo Nordisk A/S
    Full Title: Efficacy and Safety of NNC 0129-0000-1003 (N8-GP) during Surgical Procedures in Patients with Haemophilia A
    Medical condition: Haemophilia A
    Disease: Version SOC Term Classification Code Term Level
    16.1 100000004850 10018937 Haemophilia A LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male
    Trial protocol: NL (Completed) DE (Completed) SE (Completed) DK (Completed) GB (Completed) ES (Completed) HU (Completed) IT (Completed) BG (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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