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Clinical trials for Congenital

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   44335   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 ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
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    2,029 result(s) found for: Congenital. Displaying page 1 of 102.
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    EudraCT Number: 2018-003500-40 Sponsor Protocol Number: PQ-110-002 Start Date*: 2019-04-08
    Sponsor Name:ProQR Therapeutics IV B.V.
    Full Title: An Open-Label Extension Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of QR-110 in Subjects with Leber Congenital Amaurosis (LCA) due to the C.2991+1655a>G Mutation (P....
    Medical condition: Leber Congenital Amaurosis (LCA) due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10070667 Leber's congenital amaurosis PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: BE (Completed)
    Trial results: View results
    EudraCT Number: 2011-005239-14 Sponsor Protocol Number: 062011 Start Date*: 2012-01-05
    Sponsor Name:Pertti Suominen [...]
    1. Pertti Suominen
    2. Pertti Suominen
    Full Title:
    Medical condition:
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10010495 Congenital heart disease NOS LLT
    Population Age: Newborns, Under 18 Gender: Male, Female
    Trial protocol: FI (Completed)
    Trial results: (No results available)
    EudraCT Number: 2009-014571-49 Sponsor Protocol Number: CDP0901 Start Date*: 2010-01-15
    Sponsor Name:AZIENDA OSPEDALIERA DI BOLOGNA POLICLINICO S. ORSOLA M. MALPIGHI
    Full Title: The use of systemic propranolol in congenital hemangiomas
    Medical condition: congenital hemangiomas
    Disease: Version SOC Term Classification Code Term Level
    12.1 10018818 Haemangioma congenital LLT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2010-023481-41 Sponsor Protocol Number: salerno1 Start Date*: 2011-02-22
    Sponsor Name:UNIVERSITA` DEGLI STUDI DI NAPOLI FEDERICO II
    Full Title: Evaluation of long term risk-benefit profile of levothyroxin treatment in children with congenital hypothyroidism:influence of initial levothyroxine dose on neurodevelopment, growth, cardiovascular...
    Medical condition: Congenital hypothyroidism
    Disease: Version SOC Term Classification Code Term Level
    9.1 10021115 LLT
    Population Age: Newborns, Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2008-004999-53 Sponsor Protocol Number: LTS10916 Start Date*: 2009-02-06
    Sponsor Name:sanofi-aventis recherche & développement
    Full Title: Long-term safety study in patients included in CLARINET study with cyanotic congenital heart disease palliated with a systemic-to-pulmonary artery shunt and for whom the shunt is still in place at...
    Medical condition: Patients at one year of age who are included in CLARINET, with cyanotic congenital heart disease palliated with a systemic-to-pulmonary artery shunt, and for whom the shunt is still in place.
    Disease: Version SOC Term Classification Code Term Level
    8.1 10019273 Heart disease congenital LLT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: PT (Completed) HU (Completed) ES (Completed) BE (Completed) DE (Completed) FR (Completed) IT (Completed) GB (Completed) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2006-007042-18 Sponsor Protocol Number: CHD Vasovist Start Date*: 2007-03-08
    Sponsor Name:Kings College London [...]
    1. Kings College London
    2. Guy's & St Thomas'NHS Foundation Trust
    Full Title: Improved Diagnosis of Congenital Heart Disease by MRI using Vasovist - CHD Vasovist
    Medical condition: Congenital Heart Disease
    Disease: Version SOC Term Classification Code Term Level
    9.1 10010495 Congenital heart disease NOS LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2019-004764-22 Sponsor Protocol Number: SPR001-203 Start Date*: 2020-10-15
    Sponsor Name:Spruce Biosciences, Inc.
    Full Title: A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Adult Subjects with Classic Congenital Adrenal Hyperplasia
    Medical condition: Classic Congenital Adrenal Hyperplasia
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10010323 Congenital adrenal hyperplasia LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (Trial now transitioned) SE (Trial now transitioned) DE (Ongoing) DK (Trial now transitioned) GB (GB - no longer in EU/EEA) PL (Trial now transitioned) IT (Trial now transitioned) LT (Trial now transitioned) LV (Trial now transitioned) RO (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2009-010088-17 Sponsor Protocol Number: MID/09 Start Date*: 2009-02-16
    Sponsor Name:AZIENDA OSPEDALIERA OSPEDALI GALLIERA
    Full Title: Whole liver iron overload measured by the biosusceptometer Magnetic Iron Detector (MID) in thalassemia and MDS patients treated with deferasirox
    Medical condition: Patients with diagnosis of thalassemia major with iron overload or regularly transfused MDS patients (Low/Intermediate-1 IPSS risk and 1 year life expectancy)
    Disease: Version SOC Term Classification Code Term Level
    9.1 10010329 Congenital anaemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2006-000946-38 Sponsor Protocol Number: EFC5314 Start Date*: 2007-01-24
    Sponsor Name:sanofi-aventis recherche & développement
    Full Title: International randomized double blind clinical study evaluating the efficacy and safety of clopidogrel 0.2mg/kg once daily versus placebo in neonates and infants with cyanotic congenital heart dise...
    Medical condition: Neonates or infants (age less than or equal to 92 days at the time of randomization) with systemic to pulmonary arterial shunt for palliation of cyanotic congenital heart disease.
    Disease: Version SOC Term Classification Code Term Level
    8.1 10019273 Heart disease congenital LLT
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: BE (Completed) GB (Completed) DE (Completed) FI (Completed) SE (Completed) DK (Completed) PT (Completed) FR (Completed) HU (Completed) IT (Completed) NL (Completed) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2009-016528-30 Sponsor Protocol Number: TOSCANE PHRC N 2009 Start Date*: 2010-02-25
    Sponsor Name:CHU DIJON
    Full Title: Etude multicentrique, randomisée de non infériorité de deux stratégies thérapeutiques chez des enfants atteints de toxoplasmose congénitale
    Medical condition: Toxoplasmose congénitale
    Disease: Version SOC Term Classification Code Term Level
    12.0 10010652 Congenital toxoplasmosis LLT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2011-002419-27 Sponsor Protocol Number: FORMA-02 Start Date*: 2014-06-11
    Sponsor Name:Octapharma AG
    Full Title: Prospective, open-label, uncontrolled, Phase III study to assess the efficacy and safety of Octafibrin for on-demand treatment of acute bleeding and to prevent bleeding during and after surgery in ...
    Medical condition: Congenital fibrinogen deficiency.
    Disease: Version SOC Term Classification Code Term Level
    18.1 100000004850 10066357 Congenital hypofibrinogenemia LLT
    18.1 100000004850 10066356 Congenital hypofibrinogenaemia LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) BG (Completed)
    Trial results: View results
    EudraCT Number: 2020-004381-19 Sponsor Protocol Number: NBI-74788-CAH2006 Start Date*: 2021-06-07
    Sponsor Name:Neurocrine Biosciences, Inc.
    Full Title: A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Pediatric Subjects with Classic Congenital Adrenal Hyperplasia, Followed by O...
    Medical condition: Classic Congenital Adrenal Hyperplasia (CAH)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10010323 Congenital adrenal hyperplasia LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GR (Trial now transitioned) DE (Trial now transitioned) FR (Trial now transitioned) PL (Trial now transitioned) BE (Trial now transitioned) ES (Ongoing) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2015-002232-41 Sponsor Protocol Number: P140310 Start Date*: 2015-11-10
    Sponsor Name:ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
    Full Title:
    Medical condition:
    Disease: Version SOC Term Classification Code Term Level
    18.1 100000004850 10010420 Congenital CMV infection LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: FR (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2009-016640-39 Sponsor Protocol Number: PCLD003 Start Date*: 2010-05-31
    Sponsor Name:Radboud University Nijmegen Medical Centre
    Full Title: Everolimus added to long acting octreotide as a volume reducing treatment of polycystic livers.
    Medical condition: Isolated polycystic liver disease
    Disease: Version SOC Term Classification Code Term Level
    12.1 10048834 Polycystic liver disease LLT
    12.1 10048834 Polycystic liver disease PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Completed)
    Trial results: View results
    EudraCT Number: 2010-024604-10 Sponsor Protocol Number: S52898 Start Date*: 2011-03-18
    Sponsor Name:UZ Gasthuisberg Leuven
    Full Title: An open-label, Phase II clinical study to evaluate the efficacy and safety of lanreotide autogel 90mg every 4 weeks in the treatment of symptomatic polycystic liver disease, including a dose escala...
    Medical condition: Polycystic liver disease
    Disease: Version SOC Term Classification Code Term Level
    12.1 10048834 Polycystic liver disease LLT
    12.1 10048834 Polycystic liver disease PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed)
    Trial results: View results
    EudraCT Number: 2007-000390-31 Sponsor Protocol Number: HyperHAES-PÄD-CARDIO-02 Start Date*: 2007-10-30
    Sponsor Name:Universitätsklinikum Erlangen
    Full Title: Application of hypertonic hyperoncotic solutions for improvement of cardiac output after open heart surgery in children - monocentric, randomised, placebo controlled, double-blind clinical trial (p...
    Medical condition: Children after open-heart surgery for congenital cardiac disease
    Disease: Version SOC Term Classification Code Term Level
    9.1 10019273 Heart disease congenital LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: DE (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2012-001784-77 Sponsor Protocol Number: ML6721 Start Date*: 2012-05-07
    Sponsor Name:University Hospitals Leuven
    Full Title: Fontan patients: Comprehensive evaluation of the pulmonary circulation to identify pulmonary vascular disease and its influence on ventricular hemodynamics.
    Medical condition: Fontan patients
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10010495 Congenital heart disease NOS LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: BE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2020-000535-45 Sponsor Protocol Number: PQ-110-005 Start Date*: 2020-11-13
    Sponsor Name:ProQR Therapeutics IV B.V.
    Full Title: An Open-Label, Dose Escalation and Double-Masked, Randomized, Controlled Study to Evaluate the Safety and Tolerability of Sepofarsen in Pediatric Subjects <8 Years of Age with Leber Congenital Amau...
    Medical condition: Leber Congenital Amaurosis 10 (LCA10) due to c.2991+1655A>G mutation (p.Cys998X) in the CEP290 Gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10070667 Leber's congenital amaurosis PT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: BE (Completed) DE (Completed) GB (GB - no longer in EU/EEA) NL (Ongoing) IT (Completed)
    Trial results: View results
    EudraCT Number: 2007-004692-19 Sponsor Protocol Number: 963 Start Date*: 2007-12-19
    Sponsor Name:Biotest AG
    Full Title: Prevention of congenital cytomegalovirus infection in infants of mothers with primary cytomegalovirus infection during pregnancy. A randomised, open, controlled, multicentre and multinational study...
    Medical condition: Congenital CMV infection after primary CMV infection during pregnancy
    Disease: Version SOC Term Classification Code Term Level
    18.0 100000004850 10010420 Congenital CMV infection LLT
    Population Age: In utero, Newborns, Infants and toddlers, Children, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) BE (Completed) HU (Completed) AT (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2016-002109-20 Sponsor Protocol Number: AAV2-hRPE65v2-301 Start Date*: 2016-10-24
    Sponsor Name:Spark Therapeutics, Inc.
    Full Title: A Safety and Efficacy Study in Subjects with Leber Congenital Amaurosis (LCA) Using Adeno-Associated Viral Vector to Deliver the Gene for Human RPE65 to the Retinal Pigment Epithelium (RPE) [AAV2-h...
    Medical condition: Leber Congenital Amaurosis (LCA)
    Disease: Version SOC Term Classification Code Term Level
    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: Outside EU/EEA
    Trial results: View results
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