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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 2 of 70.
    « Previous 1  2  3  4  5  6  7  8  9  Next» Last»»
    EudraCT Number: 2008-005508-14 Sponsor Protocol Number: 08/0171 Start Date*: 2010-02-09
    Sponsor Name:University College london
    Full Title: A phase 3 randomized, placebo-controlled blinded investigation of six weeks vs. six months of oral valgancoclovir therapy in infants with symtomatic congenital cytomegalovirus infection. DMID # 06-...
    Medical condition: Symptomatic Congenital Cytomegalovirus.
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10010430 Congenital cytomegalovirus infection PT
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2013-003532-68 Sponsor Protocol Number: FIBCON Start Date*: 2014-03-11
    Sponsor Name:Guy's and St Thomas NHS Foundation Trust
    Full Title: Fibrinogen concentrate supplementation in the management of bleeding during paediatric cardiopulmonary bypass: a phase 1B/2A, open label dose escalation study (Version 1.0, Jan 28, 2014)
    Medical condition: Neonates and infants who are at risk of mediastinal bleeding following cardiopulmonary bypass surgery for congenital heart disease
    Disease: Version SOC Term Classification Code Term Level
    16.1 100000004849 10055817 Haemorrhage intrapericardial LLT
    16.1 100000004850 10010495 Congenital heart disease NOS LLT
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2011-004154-25 Sponsor Protocol Number: 984 Start Date*: 2013-06-25
    Sponsor Name:Biotest AG
    Full Title: A prospective, open-label, phase I/II study investigating pharmacokinetic properties of BT524 and efficacy and safety of BT524 in the treatment and prophylaxis of bleeding in patients with congenit...
    Medical condition: Patients with congenital afibrinogenemia or severe congenital hypofibrinogenemia.
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10016075 Factor I deficiency PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Prematurely Ended) DE (Completed) BG (Completed)
    Trial results: View results
    EudraCT Number: 2017-004596-31 Sponsor Protocol Number: NL62772 Start Date*: 2019-03-25
    Sponsor Name:UMC Utrecht
    Full Title: CeRebrUm and CardIac protection with Allopurinol in neonates with critical congenital heart disease requiring cardiac surgery with cardiopulmonary bypass
    Medical condition: Brain injury in neonates with critical congenital heart disease requiring cardiac surgery with cardiopulmonary bypass.
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10010495 Congenital heart disease NOS LLT
    20.0 10029205 - Nervous system disorders 10067967 Brain injury PT
    Population Age: Newborns, Under 18 Gender: Male, Female
    Trial protocol: NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2020-004400-34 Sponsor Protocol Number: LTS15823 Start Date*: 2021-03-23
    Sponsor Name:Sanofi-Aventis Recherche & Développement
    Full Title: Multicenter, open-label, extension study to characterize the long-term efficacy and safety of early versus delayed treatment with venglustat (GZ/SAR402671) in patients at risk of rapidly progressiv...
    Medical condition: Congenital cystic kidney disease
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10010428 Congenital cystic kidney disease PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: FR (Prematurely Ended) NL (Completed) BE (Prematurely Ended) DE (Completed) PT (Prematurely Ended) IT (Prematurely Ended) RO (Completed)
    Trial results: View results
    EudraCT Number: 2015-003996-32 Sponsor Protocol Number: 69HCL14-0447 Start Date*: 2015-11-03
    Sponsor Name:Hospices Civils de Lyon
    Full Title: Multicentric evaluation of in utero dexamethasone (DEX) on the cognitive development of children at risk of Congenital Adrenal Hyperplasia - PRENATAL DEX Study
    Medical condition: Patient with Congenital Adrenal Hyperplasia (CAH) or sibling of a CAH patient . Patient no connection with Congenital Adrenal Hyperplasia
    Disease: Version SOC Term Classification Code Term Level
    18.1 10010331 - Congenital, familial and genetic disorders 10061630 Adrenogenital syndrome PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2006-003148-51 Sponsor Protocol Number: F13CD-1725 Start Date*: 2008-08-15
    Sponsor Name:Novo Nordisk A/S
    Full Title: A Multi-Centre, Open-Label, Single-Arm and Multiple Dosing Trial on Efficacy and Safety of Monthly Replacement Therapy with Recombinant Factor XIII (rFXIII) in Subjects with Congenital Factor XIII ...
    Medical condition: Congenital Factor XIII Deficiency
    Disease: Version SOC Term Classification Code Term Level
    9.1 10010432 Congenital deficiency of other clotting factors LLT
    9.1 10061992 Haemophilia LLT
    9.1 10009735 Coagulation disorders congenital HLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) FR (Completed) DE (Completed) AT (Completed) ES (Completed) FI (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2008-007883-41 Sponsor Protocol Number: F13CD-3720(Mentor™2) Start Date*: 2009-08-13
    Sponsor Name:Novo Nordisk A/S
    Full Title: A Multi-Centre, Open-Label, Single-Arm, and Multiple Dosing Trial on Safety of Monthly Replacement Therapy with Recombinant Factor XIII (rFXIII) in Subjects with Congenital Factor XIII Deficiency
    Medical condition: Congenital Factor XIII Deficiency
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10061992 Haemophilia PT
    14.1 10010331 - Congenital, familial and genetic disorders 10010432 Congenital deficiency of other clotting factors LLT
    14.1 10010331 - Congenital, familial and genetic disorders 10009735 Coagulation disorders congenital HLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: FI (Completed) DE (Completed) GB (Completed) FR (Completed) ES (Completed) IT (Completed) AT (Completed)
    Trial results: View results
    EudraCT Number: 2019-004765-40 Sponsor Protocol Number: SPR001-204 Start Date*: 2020-11-17
    Sponsor Name:Spruce Biosciences, Inc.
    Full Title: A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Reducing Supraphysiologic Glucocorticoid Use in Adult Subjects with Classic Co...
    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: DK (Trial now transitioned) NL (Trial now transitioned) DE (Trial now transitioned) SE (Trial now transitioned) PL (Trial now transitioned) LT (Trial now transitioned) LV (Trial now transitioned) IT (Trial now transitioned) EE (Trial now transitioned) RO (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2015-002397-21 Sponsor Protocol Number: 2015-602295-03 Start Date*: 2015-11-30
    Sponsor Name:Ethicare GmbH
    Full Title: FOLLOW-UP SAFETY TRIAL IN CHILDREN WITH CHRONIC HEART FAILURE THERAPY RECEIVING ORODISPERSIBLE MINITABLETS OF ENALAPRIL
    Medical condition: Heart Failure due to Dilated Cardiomyopathy and Congenital Heart Disease
    Disease: Version SOC Term Classification Code Term Level
    18.1 100000004850 10010495 Congenital heart disease NOS LLT
    18.1 100000004849 10056419 Dilated cardiomyopathy LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: HU (Completed) GB (GB - no longer in EU/EEA) NL (Ongoing) AT (Completed) DE (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2005-004008-36 Sponsor Protocol Number: ANAK-01 Start Date*: 2005-10-27
    Sponsor Name:ISTITUTO GIANNINA GASLINI
    Full Title: Efficacy and safety of treatment with Anakinra in patients with CINCA/MUCKLE-WELLS syndrome and systemic juvenile idiopathic arthritis
    Medical condition: CINCA/MUCKLE-WELLS syndrome and systemic juvenile idiopathic arthritis
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2013-001189-40 Sponsor Protocol Number: 3 Start Date*: Information not available in EudraCT
    Sponsor Name:University of Tartu
    Full Title: PHARMACOKINETICS OF ORAL SPIRONOLACTONE IN CHILDREN UP TO 2 YEARS OF AGE
    Medical condition: cardiac failure, ascites and/or oedema
    Disease: Version SOC Term Classification Code Term Level
    17.0 10007541 - Cardiac disorders 10010394 Congenital cardiac disorders HLGT
    17.0 10018065 - General disorders and administration site conditions 10049630 Oedema due to renal disease PT
    17.0 10018065 - General disorders and administration site conditions 10030103 Oedema generalized LLT
    17.0 10017947 - Gastrointestinal disorders 10003445 Ascites PT
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: EE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2011-004214-42 Sponsor Protocol Number: RETIRD02 Start Date*: 2012-09-03
    Sponsor Name:QLT Inc.
    Full Title: An Open-Label Study to Evaluate the Effects of Repeated Treatments of Oral QLT091001 on Safety and Vision Outcome in Subjects with Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due ...
    Medical condition: Leber Congenital Amaurosis Retinitis pigmentosa
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10070667 Leber's congenital amaurosis PT
    20.0 10010331 - Congenital, familial and genetic disorders 10038914 Retinitis pigmentosa PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed) DE (Completed) NL (Completed)
    Trial results: (No results available)
    EudraCT Number: 2020-002383-32 Sponsor Protocol Number: 997 Start Date*: 2021-07-02
    Sponsor Name:Biotest AG
    Full Title: Prevention of maternal-fetal Cytomegalovirus transmission after primary maternal infection with gestational age ≤ 14 weeks – an open-label, single-arm, prospective trial investigating efficacy and ...
    Medical condition: Prevention of maternal-fetal cytomegalovirus transmission after primary maternal infection with gestational age ≤ 14 weeks
    Disease: Version SOC Term Classification Code Term Level
    21.0 10010331 - Congenital, familial and genetic disorders 10010430 Congenital cytomegalovirus infection PT
    20.0 100000004865 10036654 Prevention LLT
    Population Age: Adults Gender: Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2021-004858-30 Sponsor Protocol Number: BAY94-9027/21824 Start Date*: 2022-09-05
    Sponsor Name:Bayer Consumer Care AG
    Full Title: A phase 3, single group treatment, open-label, study to evaluate the safety of BAY 94-9027 infusions for prophylaxis and treatment of bleeding in previously treated children aged 7 to <12 years wit...
    Medical condition: Severe hemophilia A (<1% FVIII:C)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10060612 Hemophilia A LLT
    20.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: NO (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2020-005032-30 Sponsor Protocol Number: FLT201-01 Start Date*: 2023-03-13
    Sponsor Name:Freeline Therapeutics Limited
    Full Title: A Phase 1/2, open-label, safety, tolerability and efficacy study of FLT201 in adult patients with Gaucher disease Type 1 (Galileo-1)
    Medical condition: Type 1 Gaucher 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 10075697 Gaucher's disease type I PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2018-003501-25 Sponsor Protocol Number: PQ-110-003 Start Date*: 2019-02-28
    Sponsor Name:ProQR Therapeutics IV B.V.
    Full Title: A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety, Tolerability and Systemic Exposure of QR-110 in Subjects with Leber’s Congenital Amaurosis (LCA) due t...
    Medical condition: Leber’s 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: DE (Completed) NL (Ongoing) BE (Completed) FR (Ongoing) GB (GB - no longer in EU/EEA) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2011-001118-32 Sponsor Protocol Number: EP06-401 Start Date*: 2011-05-25
    Sponsor Name:Sandoz GmbH
    Full Title: Twelve-month study on the immunogenicity, safety, and efficacy of Zarzio®/Filgrastim HEXAL® in patients with severe chronic neutropenia
    Medical condition: Severe chronic neutropenia (i.e. congenital neutropenia, cyclic neutropenia and chronic idiopathic neutropenia)
    Disease: Version SOC Term Classification Code Term Level
    14.1 10005329 - Blood and lymphatic system disorders 10051645 Idiopathic neutropenia PT
    14.1 10010331 - Congenital, familial and genetic disorders 10069819 Congenital neutropenia LLT
    14.1 10005329 - Blood and lymphatic system disorders 10053176 Cyclic neutropenia PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) SE (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2016-001247-39 Sponsor Protocol Number: CV185-362 Start Date*: 2017-04-24
    Sponsor Name:Bristol-Myers Squibb International Corporation
    Full Title: A Prospective, Randomized, Open Label, Multi-center Study of the Safety and Pharmacokinetics of Apixaban versus Vitamin K Antagonist or LMWH in Pediatric Subjects with Congenital or Acquired Heart ...
    Medical condition: Congenital or Acquired Heart Disease Requiring Chronic Anticoagulation for Thromboembolism Prevention
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10019273 Heart disease congenital PT
    20.0 10007541 - Cardiac disorders 10007636 Cardiomyopathy PT
    20.0 10007541 - Cardiac disorders 10019276 Heart disease, unspecified LLT
    20.0 10038738 - Respiratory, thoracic and mediastinal disorders 10037400 Pulmonary hypertension PT
    20.0 10047065 - Vascular disorders 10023320 Kawasaki's disease PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) GB (GB - no longer in EU/EEA) IT (Completed) FI (Completed) AT (Completed) ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2016-002157-38 Sponsor Protocol Number: 57911 Start Date*: 2017-09-14
    Sponsor Name:Radboud University Medical Center
    Full Title: Treatment of congenital vascular malformations using Sirolimus: improving quality of Life
    Medical condition: congenital vascular malformation : Vascular malformations can involve lymphatic vessels, capillaries, veins and arteries or even combinations. These vascular malformations are present at birth an...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000005125 10047091 Vascular malformations and acquired anomalies HLT
    20.0 100000158692 10047090 Vascular malformation peripheral LLT
    20.0 10017947 - Gastrointestinal disorders 10070181 Gastrointestinal vascular malformation PT
    20.0 10010331 - Congenital, familial and genetic disorders 10074979 Vascular malformation PT
    Population Age: Infants and toddlers, Children, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: NL (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
    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.

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