Flag of the European Union EU Clinical Trials Register Help

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 ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     
    1,385 result(s) found for: Congenital disorders. Displaying page 3 of 70.
    « Previous 1  2  3  4  5  6  7  8  9  Next» Last»»
    EudraCT Number: 2007-004543-30 Sponsor Protocol Number: A6111137 Start Date*: 2008-11-24
    Sponsor Name:Pfizer Inc. 235 East 42nd Street, NEW YORK, NY 10017. US
    Full Title: A PHASE 3 PROSPECTIVE, RANDOMIZED, DOUBLE MASKED, 12 WEEK, PARALLEL GROUP STUDY EVALUATING THE EFFICACY AND SAFETY OF LATANOPROST AND TIMOLOL IN PAEDIATRIC SUBJECTS WITH GLAUCOMA
    Medical condition:
    Disease: Version SOC Term Classification Code Term Level
    9.1 10010486 Congenital glaucoma LLT
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed) DE (Completed) ES (Completed) IT (Completed) SI (Completed) BE (Completed) PT (Completed) FR (Completed) CZ (Completed) SK (Completed) DK (Prematurely Ended) GR (Completed) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2013-005393-22 Sponsor Protocol Number: RETIRD04 Start Date*: 2016-11-03
    Sponsor Name:QLT Inc.
    Full Title: A Study of the Efficacy and Safety of QLT091001 in Subjects with Inherited Retinal Disease (IRD) Caused by Mutation in Retinal Pigment Epithelium Protein 65 (RPE65) or Lecithin:Retinol Acyltransfer...
    Medical condition: Inherited retinal disease (IRD) phenotypically diagnosed as Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP) caused by mutations in the retinal pigment epithelium protein 65 (RPE65) or...
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10038914 Retinitis pigmentosa PT
    19.0 10010331 - Congenital, familial and genetic disorders 10070667 Leber's congenital amaurosis PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DK (Prematurely Ended) NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2013-002856-33 Sponsor Protocol Number: ALN-TTRSC-002 Start Date*: 2013-11-15
    Sponsor Name:Alnylam Pharmaceuticals, Inc.
    Full Title: A Phase 2, Open-Label Trial to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics and Exploratory Clinical Activity of ALN-TTRSC in Patients with Transthyretin (TTR) Cardiac Amyloidosis
    Medical condition: Transthyretin (TTR) Cardiac Amyloidosis
    Disease: Version SOC Term Classification Code Term Level
    16.1 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2017-000672-28 Sponsor Protocol Number: VX10-770-107 Start Date*: 2017-03-27
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Phase 2, Single-Blind, Placebo-Controlled Study to Evaluate the Effect of VX-770 on Hyperpolarized Helium-3 Magnetic Resonance Imaging in Subjects With Cystic Fibrosis, the G551D Mutation, and FE...
    Medical condition: cystic fibrosis
    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: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2016-001004-33 Sponsor Protocol Number: VX15-809-115 Start Date*: 2018-01-10
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Phase 3, 2-Part, Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor Combination Therapy in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for t...
    Medical condition: Cystic Fibrosis
    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: Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2014-001229-34 Sponsor Protocol Number: ALN-TTRSC-003 Start Date*: 2014-08-01
    Sponsor Name:Alnylam Pharmaceuticals, Inc.
    Full Title: A Phase 2, Open-label Extension Study to Evaluate the Long-Term Safety, Clinical Activity, and Pharmacokinetics of ALN-TTRSC in Patients with Transthyretin (TTR) Cardiac Amyloidosis Who Have Previo...
    Medical condition: Transthyretin (TTR) Cardiac Amyloidosis
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2017-001309-34 Sponsor Protocol Number: VX14-809-106 Start Date*: 2017-03-29
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Phase 3b, Open-Label Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Cystic Fibrosis and Advanced Lung Disease, Homozygous for the F508del-CFTR ...
    Medical condition: Cystic Fibrosis
    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: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2017-000673-37 Sponsor Protocol Number: VX12-770-113 Start Date*: 2017-03-27
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis, Residual CFTR Function, and FEV1 ≥40% Predicted
    Medical condition: cystic fibrosis
    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: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2015-002209-12 Sponsor Protocol Number: ABR59689 Start Date*: 2017-06-21
    Sponsor Name:Erasmus MC-Sophia Children's Hospital
    Full Title: Less opioids after major abdominal surgery in young infants using wound catheter infusion with local anesthetics: a randomized controlled trial.
    Medical condition: Children < 1 year of age undergoing major open abdominal surgery.
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10021298 Ileal atresia PT
    20.1 10010331 - Congenital, familial and genetic disorders 10010526 Congenital large intestinal atresia PT
    20.1 10010331 - Congenital, familial and genetic disorders 10002120 Anal atresia PT
    20.1 10010331 - Congenital, familial and genetic disorders 10010626 Congenital small intestinal atresia PT
    20.1 10010331 - Congenital, familial and genetic disorders 10062022 Intestinal atresia PT
    20.1 10010331 - Congenital, familial and genetic disorders 10013812 Duodenal atresia PT
    20.1 10010331 - Congenital, familial and genetic disorders 10028210 Multiple gastrointestinal atresias PT
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: NL (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2021-003250-23 Sponsor Protocol Number: 78162.018.21 Start Date*: 2025-01-24
    Sponsor Name:Amsterdam UMC
    Full Title: Cannabidiol (Epidyolex) for behavioural problems in patients with Tuberous Sclerosis Complex, Sanfilippo and Fragile X Syndrome: an N-of-1 series
    Medical condition: Tuberous Sclerosis Complex (TSC), Sanfilippo syndrome, Fragile X syndrome (FXS)
    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 10027424 Metabolic and nutritional disorders congenital HLGT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2004-005275-42 Sponsor Protocol Number: 1140 Start Date*: 2005-03-21
    Sponsor Name:AZIENDA OSPEDALIERA ISTITUTI OSPITALIERI DI VERONA
    Full Title: Study on the effect of r-hGH in combination with bisphosphonate treatment on bone metabolism in Osteogenesis Imperfecta
    Medical condition: Osteogenesis Imperfecta
    Disease: Version SOC Term Classification Code Term Level
    6.1 10010331 SOC
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2009-010404-28 Sponsor Protocol Number: G83627057 Start Date*: 2009-06-12
    Sponsor Name:Fundación de Investigación Biomédica del Hospital Universitario La Paz, Madrid
    Full Title: PREVENCIÓN DEL SÍNDROME DE BAJO GASTO POSTOPERATORIO EN RECIÉN NACIDOS SOMETIDOS A CIRUGÍA CARDIOVASCULAR: ESTUDIO PILOTO SOBRE LA FARMACOCINÉTICA Y FARMACODINAMIA DOSIS-DEPENDIENTE DE DOS INODILA...
    Medical condition: Este estudio evaluará la eficacia de dos inodilatadores (IND) en el tratamiento del síndrome de bajo gasto postoperatorio (SBGP) en recién nacidos sometidos a cirugía cardiovascular (CCV)
    Disease: Version SOC Term Classification Code Term Level
    9 10024899 Low cardiac output syndrome LLT
    9 10010495 Congenital heart disease NOS LLT
    Population Age: Newborns, Under 18 Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2015-005732-18 Sponsor Protocol Number: DU176b-A-U157 Start Date*: 2016-06-29
    Sponsor Name:Daiichi Sankyo , Inc.
    Full Title: A Phase 1, Open-Label, Single-dose, Non-randomized Study to Evaluate Pharmacokinetics and Pharmacodynamics of Edoxaban in Pediatric Patients
    Medical condition: Edoxaban is being investigated for use in pediatric patients who may require anticoagulation therapy.
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004850 10010495 Congenital heart disease NOS LLT
    19.0 100000004851 10057396 Thrombophilia LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: ES (Completed) FR (Ongoing) IT (Completed)
    Trial results: View results
    EudraCT Number: 2011-003325-10 Sponsor Protocol Number: 1.01 Start Date*: 2012-08-10
    Sponsor Name:VU University Medical Center
    Full Title: A randomized controlled trail on the effects of periconceptional and prenatal folic acid supplementation on congenital anomalies and preterm birth
    Medical condition: congenital anomalies
    Disease: Version SOC Term Classification Code Term Level
    14.1 10036585 - Pregnancy, puerperium and perinatal conditions 10004953 Birth premature LLT
    14.1 10010331 - Congenital, familial and genetic disorders 10007607 Cardiac septal defects congenital HLT
    14.1 10010331 - Congenital, familial and genetic disorders 10041524 Spina bifida PT
    Population Age: Adults Gender: Female
    Trial protocol: NL (Completed)
    Trial results: (No results available)
    EudraCT Number: 2019-000602-30 Sponsor Protocol Number: Kamada-AATInhaled-008 Start Date*: 2019-09-30
    Sponsor Name:Kamada Ltd.
    Full Title: A Prospective Phase III Multi-center, Placebo Controlled, Double Blind Study to Evaluate the Efficacy and Safety of “Kamada-AAT for Inhalation” 80 mg per day in Adult Patients with Congenital Alpha...
    Medical condition: Adult Patients with Congenital Alpha-1 Antitrypsin Deficiency with Moderate and Severe Airflow Limitation (40% ≤ FEV1 ≤ 80% of predicted; FEV1/SVC ≤ 70%), and with no history of two or more moderat...
    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 Gender: Male, Female
    Trial protocol: NL (Trial now transitioned) GB (GB - no longer in EU/EEA) IE (Trial now transitioned) SE (Trial now transitioned) FI (Trial now transitioned) BE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2010-023242-69 Sponsor Protocol Number: GENA-11 Start Date*: 2011-05-31
    Sponsor Name:Octapharma AG
    Full Title: Clinical Study to Investigate the Long-Term Efficacy, Safety, and Immunogenicity of human-cl rhFVIII in Previously Treated Patients with Severe Haemophilia A – Extension Study to GENA-01
    Medical condition: Severe haemophilia A (FVIII:C <1%)
    Disease: Version SOC Term Classification Code Term Level
    12.1 10018937 Haemophilia A LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male
    Trial protocol: DE (Completed) BG (Completed)
    Trial results: View results
    EudraCT Number: 2012-004434-42 Sponsor Protocol Number: BAY94-9027/15912 Start Date*: 2013-04-15
    Sponsor Name:Bayer AG
    Full Title: A multi-center, phase III, non-controlled, open-label trial to evaluate the pharmacokinetics, safety, and efficacy of BAY 94-9027 for prophylaxis and treatment of bleeding in previously treated chi...
    Medical condition: severe hemophilia A (<1% FVIII:C)
    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 10060612 Hemophilia A LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: GB (Completed) BE (Completed) IT (Completed) NL (Completed) LT (Completed) BG (Completed) Outside EU/EEA PL (Completed) AT (Completed) NO (Ongoing) ES (Completed) GR (Completed)
    Trial results: View results
    EudraCT Number: 2017-001678-40 Sponsor Protocol Number: CACZ885N2301E2 Start Date*: 2017-05-15
    Sponsor Name:Novartis pharma AG
    Full Title: An extension study of safety of canakinumab in Japanese patients with periodic fever syndromes
    Medical condition: Three disease of periodic fever syndromes, TNF receptor Associated Periodic Syndrome (TRAPS), Hyper IgDSyndrome (HIDS)/ Mevalonate Kinase Deficiency (MKD) and Familial Mediterranean Fever (FMF
    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: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2011-001142-15 Sponsor Protocol Number: NN7088-3859 Start Date*: 2012-05-14
    Sponsor Name:Novo Nordisk A/S
    Full Title: A Multi-national Trial Evaluating Safety and Efficacy, including Pharmacokinetics, of NNC 0129-0000-1003 (N8-GP) when Administered for Treatment and Prophylaxis of Bleeding in Patients with Haemoph...
    Medical condition: Haemophilia A
    Disease: Version SOC Term Classification Code Term Level
    19.1 100000004850 10018937 Haemophilia A LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male
    Trial protocol: NL (Completed) DE (Completed) SE (Completed) NO (Completed) DK (Completed) ES (Completed) GB (Completed) HU (Completed) IT (Completed) BG (Completed)
    Trial results: View results
    EudraCT Number: 2021-004039-10 Sponsor Protocol Number: NL81112.041.22 Start Date*: 2022-06-24
    Sponsor Name:UMC Utrecht - Van Creveldkliniek
    Full Title: Pharmacokinetic-guided dosing of emicizumab in congenital haemophilia A patients – The DosEmi study
    Medical condition: Congenital hemophilia A
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10060612 Hemophilia A LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male
    Trial protocol: NL (Trial now transitioned)
    Trial results: (No results available)
    Download Options:
    Number of Trials to download:
    Download Content:
    Download Format:
    Note, where multi-state trials are shown in search results, selecting "Full Trial details" will download full information for each of the member states/countries involved in the trial.
    « Previous 1  2  3  4  5  6  7  8  9  Next» Last»»
    Query did not match any studies.
    For support, Contact us.
    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
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Fri May 30 15:55:22 CEST 2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA