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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 9 of 70.
    «« First « Previous 5  6  7  8  9  10  11  12  13  Next» Last»»
    EudraCT Number: 2017-004547-21 Sponsor Protocol Number: ZP4207-17109 Start Date*: 2018-08-24
    Sponsor Name:Zealand Pharma A/S
    Full Title: A Two-Period, Open-label Trial Evaluating the Efficacy and Safety of Dasiglucagon for the Treatment of Children with Congenital Hyperinsulinism
    Medical condition: Children with congenital hyperinsulinism
    Disease: Version SOC Term Classification Code Term Level
    20.0 10027433 - Metabolism and nutrition disorders 10061211 Hyperinsulinism PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Completed)
    Trial results: View results
    EudraCT Number: 2012-004343-80 Sponsor Protocol Number: 545.12 Start Date*: 2012-09-20
    Sponsor Name:OSPEDALE PEDIATRICO BAMBINO GESU' DI ROMA
    Full Title: STUDY PHASE III, RANDOMIZED, DOUBLE-BLIND CONTROLLED TO EVALUATE THE EFFECTIVENESS OF FUROSEMIDE IN CONTINUOUS INFUSION VS ethacrynic acid IN CONTINUOUS INFUSION IN CHILDREN SUBJECT TO A heart sur...
    Medical condition: ALL CHILDREN AGE 'LESS THAN 10 YEARS OF AGE' WITH CONGENITAL HEART PROCEDURE SUBJECT TO cardiac surgery in whom the diuretic ARE GIVEN IN ACCORDANCE WITH THE FOLLOWING CONTINUOUS INFUSION PROTOCOL ...
    Disease: Version SOC Term Classification Code Term Level
    14.1 10007541 - Cardiac disorders 10007541 Cardiac disorders SOC
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2005-005697-71 Sponsor Protocol Number: 060402 Start Date*: 2006-01-26
    Sponsor Name:Baxalta Innovations GmbH
    Full Title: Antihemophilic Factor (Recombinant) Plasma/Albumin-Free Method (rAHF PFM): A Phase 3/4, Prospective, Controlled, Randomized, Multi-Center Study to Compare the Efficacy and Safety of Continuous Infu...
    Medical condition: Subjects with Severe or Moderately Severe Hemophilia A (baseline factor VIII (FVIII) level <= 2% of normal) hemophilia A undergoing major orthopedic surgery
    Disease: Version SOC Term Classification Code Term Level
    18.0 100000004850 10060612 Hemophilia A LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: AT (Completed) SE (Completed) BE (Completed) PT (Completed) ES (Completed) HU (Completed) NL (Completed) GB (Completed) IT (Completed) PL (Completed)
    Trial results: View results
    EudraCT Number: 2013-000597-29 Sponsor Protocol Number: LTS13619 Start Date*: 2015-09-30
    Sponsor Name:sanofi-aventis recherche & développement
    Full Title: An Open-Label Study to Determine the Long-Term Safety, Tolerability and Biological Activity of UshStat® in Patients with Usher Syndrome type 1B
    Medical condition: Usher syndrome type 1B
    Disease: Version SOC Term Classification Code Term Level
    18.1 10010331 - Congenital, familial and genetic disorders 10063396 Usher's syndrome PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2017-005103-27 Sponsor Protocol Number: BETASBMA Start Date*: 2021-11-18
    Sponsor Name:AZIENDA OSPEDALIERA DI PADOVA
    Full Title: A PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF CLENBUTEROL IN PATIENTS WITH SPINAL AND BULBAR MUSCULAR ATROPHY (SBMA)
    Medical condition: Motor neuron disease characterized by atrophy and muscle weakness in the spinal and bulbar region
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10068597 Bulbospinal muscular atrophy congenital PT
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2020-000250-94 Sponsor Protocol Number: MAST2 Start Date*: 2020-07-17
    Sponsor Name:ISTITUTO AUXOLOGICO ITALIANO
    Full Title: Mutation-specific therapy for the long QT syndrome
    Medical condition: Long QT syndrome
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10057926 Long QT syndrome congenital PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2010-021781-29 Sponsor Protocol Number: BAY81-8973/13400 Start Date*: 2011-07-07
    Sponsor Name:Bayer HealthCare AG
    Full Title: A multicenter Phase III uncontrolled open-label trial to evaluate safety and efficacy of BAY 81-8973 in children with severe haemophilia A under prophylaxis therapy
    Medical condition: Severe Hemophilia-A (< 1% FVIII:C)
    Disease: Version SOC Term Classification Code Term Level
    13.1 10010331 - Congenital, familial and genetic disorders 10060612 Hemophilia A LLT
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: HU (Completed) LT (Completed) SE (Completed) DK (Completed) IE (Completed) LV (Completed) BG (Completed) IT (Completed) PL (Completed) AT (Completed) ES (Completed) GB (Completed) NO (Completed)
    Trial results: View results
    EudraCT Number: 2019-004394-10 Sponsor Protocol Number: ACT16248 Start Date*: 2020-06-30
    Sponsor Name:Genzyme Corporation
    Full Title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Efficacy, Pharmacodynamics, and Pharmacokinetics of Lademirsen (SAR339375) for Subcutaneous Injection Administe...
    Medical condition: Congenital, hereditary and neonatal diseases
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10001843 Alport's syndrome PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2019-005039-94 Sponsor Protocol Number: IC2019-05 Start Date*: 2020-12-08
    Sponsor Name:Institut Curie
    Full Title: RETINO 2018 : Ocular conservative treatment for retinoblastoma: efficacy of the new management strategies and visual outcome.
    Medical condition: Retinoblastoma
    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: Newborns, Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2010-024242-30 Sponsor Protocol Number: CE1145_4001 Start Date*: 2011-10-06
    Sponsor Name:CSL Behring GmbH
    Full Title: Prospective open-label uncontrolled multi-center post-marketing study to assess inhibitory antibody formation in subjects with congenital C1-INH deficiency and acute hereditary angioedema (HAE) att...
    Medical condition: congenital C1-INH deficiency and acute hereditary angioedema (HAE) attacks
    Disease: Version SOC Term Classification Code Term Level
    16.0 10010331 - Congenital, familial and genetic disorders 10019860 Hereditary angioedema PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: PL (Completed) HU (Completed) BG (Completed)
    Trial results: View results
    EudraCT Number: 2011-002880-42 Sponsor Protocol Number: LAL-CL05 Start Date*: 2011-12-05
    Sponsor Name:Synageva Biopharma Corp.
    Full Title: An Open Label Multicenter Extension Study to Evaluate the Long-Term Efficacy and Safety of SBC 102 in Children with Lysosomal Acid Lipase Deficiency Who Previously Received Treatment with SBC-102
    Medical condition: Growth failure in children due to lysosomal acid lipase deficiency (Wolman disease).
    Disease: Version SOC Term Classification Code Term Level
    14.0 10027433 - Metabolism and nutrition disorders 10027433 Metabolism and nutrition disorders SOC
    14.0 10027433 - Metabolism and nutrition disorders 10021605 Inborn errors of metabolism HLGT
    14.0 10010331 - Congenital, familial and genetic disorders 10024579 Lysosomal storage disorders HLT
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Prematurely Ended) FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2011-002393-23 Sponsor Protocol Number: CSL627_1001 Start Date*: 2012-01-10
    Sponsor Name:CSL Behring GmbH
    Full Title: A Phase I/III Open-label, Multicenter, Crossover Safety, Efficacy and Pharmacokinetic Study of Recombinant Coagulation Factor VIII (rFVIII) Compared to Recombinant Factor VIII (rFVIII; INN: octocog...
    Medical condition: Hemophilia A.
    Disease: Version SOC Term Classification Code Term Level
    17.1 100000004850 10060612 Hemophilia A LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male
    Trial protocol: DE (Completed) SE (Completed) AT (Completed) IT (Completed) PL (Completed) GB (Completed) ES (Completed) HU (Completed) NL (Completed) CZ (Completed)
    Trial results: View results
    EudraCT Number: 2012-005753-23 Sponsor Protocol Number: NI-0501-05 Start Date*: 2013-06-19
    Sponsor Name:NovImmune SA
    Full Title: A multicentre study for the long-term follow-up of HLH patients who received treatment with NI-0501, an anti-interferon gamma monoclonal antibody.
    Medical condition: haemophagocytic lymphohistiocytosis
    Disease: Version SOC Term Classification Code Term Level
    17.1 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) CZ (Completed) DE (Completed) ES (Completed) AT (Completed) SE (Completed) GB (GB - no longer in EU/EEA) FR (Completed) NL (Completed)
    Trial results: View results
    EudraCT Number: 2010-021134-66 Sponsor Protocol Number: IJL-AFHT-TH10 Start Date*: Information not available in EudraCT
    Sponsor Name:Institut Jerome Lejeune
    Full Title: Efficacy assessment of systematic treatment with folinic acid and thyroid hormone on psychomotor development of Down Syndrome young children
    Medical condition: Down syndrome
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10044688 Trisomy 21 PT
    14.0 10010331 - Congenital, familial and genetic disorders 10013616 Down's syndrome LLT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2018-000358-23 Sponsor Protocol Number: CMS-001 Start Date*: 2019-02-06
    Sponsor Name:CATALYST PHARMACEUTICALS INC.
    Full Title: A phase 3, multicenter, double-blind, placebo-controlled, randomized, outpatient two-period two-treatment crossover study to evaluate the efficacy and safety of amifampridine phosphate (3,4 diamino...
    Medical condition: Congenital myasthenic syndromes
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029205 - Nervous system disorders 10028424 Myasthenic syndrome PT
    20.0 10029205 - Nervous system disorders 10028425 Myasthenic syndromes in diseases classified elsewhere LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2020-003485-39 Sponsor Protocol Number: NN7533-4470 Start Date*: 2022-07-19
    Sponsor Name:Novo Nordisk A/S
    Full Title: A multicentre trial evaluating the efficacy and safety of oral decitabine-tetrahydrouridine (NDec) in patients with sickle cell disease
    Medical condition: Sickle cell disease
    Disease: Version SOC Term Classification Code Term Level
    21.0 10010331 - Congenital, familial and genetic disorders 10040644 Sickle cell disease PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GR (Trial now transitioned) IT (Trial now transitioned) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-003320-23 Sponsor Protocol Number: AC-055H301 Start Date*: 2017-10-11
    Sponsor Name:Actelion Pharmaceuticals Ltd
    Full Title: Prospective, multi-center, double-blind, randomized, placebo-controlled, parallel-group study assessing the efficacy and safety of macitentan in Fontan-palliated adult and adolescent subjects.
    Medical condition: Congenital Heart Failure
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10045545 Univentricular heart PT
    21.1 10042613 - Surgical and medical procedures 10065950 Cavopulmonary anastomosis PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Completed) DK (Completed) FR (Completed) PL (Completed) IE (Completed) CZ (Completed)
    Trial results: View results
    EudraCT Number: 2017-002155-28 Sponsor Protocol Number: P160902J Start Date*: 2018-08-30
    Sponsor Name:ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
    Full Title: Evaluation of allopurinol treatment for autistic disorders and epilepsy in adenylosuccinate lyase deficiency (ADSL)
    Medical condition: patient with adenulosuccinate lyase deficiency
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10062018 Inborn error of metabolism PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2020-003445-11 Sponsor Protocol Number: PHA022121-C201 Start Date*: 2020-12-18
    Sponsor Name:Pharvaris Netherlands BV
    Full Title: A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C...
    Medical condition: Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor Deficiency
    Disease: Version SOC Term Classification Code Term Level
    23.1 10010331 - Congenital, familial and genetic disorders 10019860 Hereditary angioedema PT
    21.0 10010331 - Congenital, familial and genetic disorders 10080956 Hereditary angioedema type I LLT
    21.0 10010331 - Congenital, familial and genetic disorders 10080957 Hereditary angioedema C1 inhibitor deficiency LLT
    21.0 10010331 - Congenital, familial and genetic disorders 10080960 Hereditary angioedema type II LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Completed) ES (Completed) HU (Completed) NL (Completed) PL (Completed) DE (Completed) CZ (Completed) BG (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2015-000958-38 Sponsor Protocol Number: LFB-FVIIa-007-14 Start Date*: 2016-01-06
    Sponsor Name:LFB USA, Inc.
    Full Title: A Phase III Study on the Safety, Pharmacokinetics, and Efficacy of Coagulation Factor VIIa (Recombinant) in Congenital Hemophilia A or B Pediatric Patients from birth to <12 years old with Inhibit...
    Medical condition: Congenital Hemophilia A or B patients with inhibitors to Factor VIII or Factor IX
    Disease: Version SOC Term Classification Code Term Level
    19.1 10010331 - Congenital, familial and genetic disorders 10056492 Haemophilia A with anti factor VIII PT
    19.1 10010331 - Congenital, familial and genetic disorders 10056494 Haemophilia B with anti factor IX PT
    Population Age: Newborns, Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: CZ (Completed) BG (Completed) IT (Completed)
    Trial results: View results
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