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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 1 of 70.
    1  2  3  4  5  6  7  8  9  Next» Last»»
    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: 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: 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: 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: 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: 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: 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: 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: 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: 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: 2008-007413-76 Sponsor Protocol Number: steroiditutkimus Start Date*: 2009-02-27
    Sponsor Name:Pertti Suominen
    Full Title: Metyyliprednisolonin farmakokinetiikka ja -dynamiikka lasten avosydänleikkausten yhteydessä
    Medical condition: Lasten ja nuorten sairaalassa (HUS) hoidettavat 1-12 kk ikäiset avosydänleikkaukseen tulevat potilaat, joilla on sydänvikanan TOF, AVSD tai VSD. Lasten ja nuorten sairaalassa (HUS) hoidettavat ko...
    Disease: Version SOC Term Classification Code Term Level
    9.1 10010495 Congenital heart disease NOS LLT
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: FI (Ongoing)
    Trial results: (No results available)
    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
    EudraCT Number: 2017-000813-22 Sponsor Protocol Number: PQ-110-001 Start Date*: 2017-06-19
    Sponsor Name:ProQR Therapeutics
    Full Title: An Open-Label, Single Arm, Multiple Dose, Dose Escalation Study to Evaluate the Safety and Tolerability of QR-110 in Subjects with Leber’s Congenital Amaurosis (LCA) due to c.2991+1655A>G Mutation ...
    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
    19.1 10010331 - Congenital, familial and genetic disorders 10070667 Leber's congenital amaurosis PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed)
    Trial results: View results
    EudraCT Number: 2015-002396-18 Sponsor Protocol Number: 2015-602295-02 Start Date*: 2015-11-30
    Sponsor Name:Ethicare GmbH
    Full Title: ORODISPERSIBLE MINITABLETS OF ENALAPRIL IN YOUNG CHILDREN WITH HEART FAILURE DUE TO CONGENITAL HEART DISEASE
    Medical condition: Heart Failure due to Congenital Heart Disease
    Disease: Version SOC Term Classification Code Term Level
    18.1 100000004850 10010495 Congenital heart disease NOS LLT
    Population Age: Newborns, Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: HU (Completed) GB (Prematurely Ended) NL (Ongoing) AT (Completed) DE (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2015-003418-25 Sponsor Protocol Number: CTU/2014/120 Start Date*: 2016-03-29
    Sponsor Name:MeiraGTx UK II Ltd
    Full Title: An Open-label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno-Associated Virus Vector (AAV2/5-OPTIRPE65) for Gene Therapy of Adults and Children with Retinal Dystrophy associated with D...
    Medical condition: Leber Congenital Amaurosis (LCA) caused by mutations in RPE65
    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: GB (Completed)
    Trial results: View results
    EudraCT Number: 2019-004981-16 Sponsor Protocol Number: 1991-201-008 Start Date*: 2022-06-17
    Sponsor Name:Editas Medicine, Inc.
    Full Title: Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Efficacy of EDIT-101 in Adult and Pediatric Participants with Leber Congenital Amaurosis Type 10 (LCA10), with Cen...
    Medical condition: Leber Congenital Amaurosis Type 10 (LCA10), with Centrosomal Protein 290 (CEP290)-Related Retinal Degeneration Caused by a Compound Heterozygous or Homozygous Mutation Involving c.2991+1655A>G in I...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10015920 Eye disorders congenital HLGT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: NL (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2019-004873-17 Sponsor Protocol Number: NBI-74788-CAH3003 Start Date*: 2020-10-15
    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 Adult Subjects with Classic Congenital Adrenal Hyperplasia, Followed by Open-...
    Medical condition: Classic Congenital Adrenal Hyperplasia (CAH)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10010323 Congenital adrenal hyperplasia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Trial now transitioned) CZ (Trial now transitioned) FR (Trial now transitioned) PT (Trial now transitioned) BG (Trial now transitioned) PL (Trial now transitioned) GR (Trial now transitioned) AT (Trial now transitioned) NL (Ongoing) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2011-000989-35 Sponsor Protocol Number: P101001 Start Date*: Information not available in EudraCT
    Sponsor Name:ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
    Full Title:
    Medical condition:
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10066131 Congenital central hypoventilation syndrome PT
    Population Age: Adolescents, Under 18, Adults Gender: Female
    Trial protocol: FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2011-005378-44 Sponsor Protocol Number: CMV-MM-1 Start Date*: 2012-05-03
    Sponsor Name:LUMC
    Full Title: CONgenital Cmv: Efficacy of antiviral treatment in a Randomized controlled Trial
    Medical condition: Congenital cytomegalovirus infection. Sensorineural Hearingloss.
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10010420 Congenital CMV infection LLT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: NL (Prematurely Ended)
    Trial results: (No results available)
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