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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   44381   clinical trials with a EudraCT protocol, of which   7393   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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    1,387 result(s) found for: Congenital disorders. Displaying page 5 of 70.
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    EudraCT Number: 2011-001033-16 Sponsor Protocol Number: NN7008-3809 Start Date*: Information not available in EudraCT
    Sponsor Name:Novo Nordisk A/S
    Full Title: Safety and Efficacy of NNC 0155-0000-0004 in Prevention and Treatment of Bleeds in Paediatric Previously Untreated Patients with Haemophilia A
    Medical condition: Haemophilia A
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10018937 Haemophilia A LLT
    Population Age: Newborns, Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: GB (Prematurely Ended) AT (Completed) ES (Completed) GR (Completed) DK (Completed) HU (Completed) LT (Completed) PL (Completed) PT (Completed)
    Trial results: View results
    EudraCT Number: 2005-001290-10 Sponsor Protocol Number: CICL670A2203 including amendment 1 Start Date*: 2006-01-30
    Sponsor Name:Novartis Pharma Services AG
    Full Title: A study to provide expanded access of EXJADE® (deferasirox) to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot adequately be treat...
    Medical condition: Chronic iron overload from blood transfusions in patients with congenital disorders of red blood cells
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) BE (Completed) IE (Prematurely Ended) ES (Completed) DE (Completed) GR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2020-003371-18 Sponsor Protocol Number: MAA-202 Start Date*: 2021-03-30
    Sponsor Name:Catalyst Biosciences, Inc.
    Full Title: Phase 1/2 Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Efficacy of Marzeptacog alfa (activated) in Treatment of Episodic Bleeding in Subjects with Inherited Bleeding Disorders
    Medical condition: Factor VII deficiency, Glanzmann thrombasthenia (GT) and Hemophilia A with inhibitors on emicizumab prophylaxis (HAwI-E)
    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 10016079 Factor VII deficiency PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2023-001026-34 Sponsor Protocol Number: CSL627_3003 Start Date*: 2024-12-19
    Sponsor Name:CSL Behring
    Full Title: A Phase 3, Open-label, Multicenter, Pharmacokinetics, Efficacy, and Safety Study of a Recombinant Single-chain Factor VIII (rVIII-SingleChain) in Chinese Previously Treated Patients (PTPs) with Hem...
    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 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: (No results available)
    EudraCT Number: 2021-006191-16 Sponsor Protocol Number: BAY94-9027/21924 Start Date*: 2024-10-29
    Sponsor Name:Bayer HealthCare Pharmaceuticals Inc.
    Full Title: A multicenter, prospective, open-label, clinical study to assess the effect of using a new risk score approach to select the most appropriate prophylaxis regimen for reaching a favorable outcome, w...
    Medical condition: Hemophilia A Prophylaxis of bleeding
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10060613 Hemophilia A (Factor VIII) LLT
    20.0 10042613 - Surgical and medical procedures 10071818 Bleeding prophylaxis LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2010-019249-25 Sponsor Protocol Number: 981 Start Date*: 2010-08-12
    Sponsor Name:Biotest AG
    Full Title: An open, prospective trial investigating pharmacokinetics and safety (Part A) of the human normal immunoglobulin for intravenous infusion (IVIG) BT090 and tolerability and safety of escalating infu...
    Medical condition: Primary immunodeficiency disease (PID)
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10010509 Congenital hypogammaglobulinemia LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) HU (Completed)
    Trial results: View results
    EudraCT Number: 2009-011055-43 Sponsor Protocol Number: GENA-08 Start Date*: 2009-10-22
    Sponsor Name:Octapharma AG
    Full Title: Clinical Study to Investigate the Efficacy, Safety, and Immunogenicity of human-cl rhFVIII in Previously Treated Patients With Severe Haemophilia A
    Medical condition: Severe haemophilia A (FVIII:C <=1%)
    Disease: Version SOC Term Classification Code Term Level
    13.1 10010331 - Congenital, familial and genetic disorders 10018937 Haemophilia A LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male
    Trial protocol: DE (Completed) AT (Completed) GB (Completed)
    Trial results: View results
    EudraCT Number: 2014-002701-38 Sponsor Protocol Number: AT1001-042 Start Date*: 2014-12-17
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: An Open-Label, Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in subjects with Fabry Disease
    Medical condition: Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme a-galactosidase A.
    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: Adults, Elderly Gender: Male, Female
    Trial protocol: AT (Completed) BE (Completed) GB (Completed) ES (Completed) DK (Completed)
    Trial results: View results
    EudraCT Number: 2006-004458-26 Sponsor Protocol Number: 12332 Start Date*: 2007-02-14
    Sponsor Name:Bayer Healthcare LLC
    Full Title: Randomized, comparative, open label treatment with double-blind placebo-controlled periods within treatment study to evaluate the efficacy and safety of a once-a-week prophylaxis treatment with BAY...
    Medical condition: Haemophilia A
    Disease: Version SOC Term Classification Code Term Level
    8.1 10018937 Haemophilia A LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male
    Trial protocol: NL (Completed) FR (Completed) AT (Prematurely Ended) CZ (Completed) BE (Completed) SK (Prematurely Ended) GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2009-012149-43 Sponsor Protocol Number: BAY81-8973/12954 Start Date*: 2010-09-06
    Sponsor Name:Bayer HealthCare AG
    Full Title: Ensayo aleatorizado, cruzado, abierto, en dos partes, para evaluar la farmacocinética, la eficacia y el perfil de seguridad de FVIII recombinante libre de proteínas plasmáticas formulado con sacaro...
    Medical condition: Hemofilia A severa (< 1% FVIII:C)
    Disease: Version SOC Term Classification Code Term Level
    12.1 10053753 Hemophilia A without inhibitors LLT
    12.1 10060612 Hemophilia A LLT
    12.1 10060613 Hemophilia A (Factor VIII) LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male
    Trial protocol: ES (Completed) SE (Completed) GB (Completed) NO (Completed) DE (Completed) IT (Completed) AT (Completed) DK (Completed)
    Trial results: View results
    EudraCT Number: 2007-006024-35 Sponsor Protocol Number: cysteamine gel formulation/07/cysta Start Date*: 2008-02-04
    Sponsor Name:Orphan Europe SARL
    Full Title: Adaptive dose regimen of Cystadrops for cOrneal Crystal deposiTs and ocular manifestations in nephropathic cystinosis : an open label, dose-response pilot study
    Medical condition: Nephropathic cystinotic patients with cystine crystals corneal deposits
    Disease: Version SOC Term Classification Code Term Level
    9.1 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2021-002094-26 Sponsor Protocol Number: 8100 Start Date*: 2022-11-22
    Sponsor Name:Les Hôpitaux Universitaires de Strasbourg
    Full Title: Optimization of the management of drepanocytosis patients treated with hydroxyurea: Interest of the pharmacological therapeutic follow-up
    Medical condition: Drepanocytosis
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    21.0 10010331 - Congenital, familial and genetic disorders 10051835 Drepanocytosis LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2010-023803-92 Sponsor Protocol Number: NN1731-3562 Start Date*: 2011-06-01
    Sponsor Name:Novo Nordisk A/S
    Full Title: Efficacy and Safety of NNC 0078-0000-0007 in Treatment of Acute Bleeding Episodes in Patients with Congenital Haemophilia and Inhibitors
    Medical condition: Congenital Haemophilia and Inhibitors
    Disease: Version SOC Term Classification Code Term Level
    13.1 10010331 - Congenital, familial and genetic disorders 10056492 Haemophilia A with anti factor VIII PT
    13.1 10010331 - Congenital, familial and genetic disorders 10056494 Haemophilia B with anti factor IX PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male
    Trial protocol: GB (Completed) AT (Completed) GR (Completed) PL (Completed) ES (Prematurely Ended) IT (Completed) HU (Completed)
    Trial results: View results
    EudraCT Number: 2020-005949-16 Sponsor Protocol Number: 202000894 Start Date*: 2021-10-25
    Sponsor Name:University Medical Center Groningen
    Full Title: A GnRH Agonist IN pre-menopausal women STudy to treat severe Polycystic Liver Disease
    Medical condition: Severe polycystic liver disease in female patients
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10048834 Polycystic liver disease PT
    20.0 10010331 - Congenital, familial and genetic disorders 10036045 Polycystic kidney LLT
    20.0 10010331 - Congenital, familial and genetic disorders 10036046 Polycystic kidney, autosomal dominant LLT
    23.0 10010331 - Congenital, familial and genetic disorders 10083939 Autosomal dominant polycystic liver disease LLT
    Population Age: Adults Gender: Female
    Trial protocol: NL (Trial now transitioned) DE (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2005-005711-17 Sponsor Protocol Number: AGT4HB Start Date*: 2009-09-25
    Sponsor Name:St. Jude Children’s Research Hospital
    Full Title: AN OPEN LABEL DOSE-ESCALATION STUDY OF A SELF COMPLEMENTARY ADENO-ASSOCIATED VIRAL VECTOR (scAAV2/8-LP1-hFIXco) FOR GENE TRANSFER IN SUBJECTS WITH HEMOPHILIA B
    Medical condition: Gene therapy for Haemophilia B
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004865 10001140 Adeno-associated in vivo gene therapy LLT
    19.0 10010331 - Congenital, familial and genetic disorders 10061992 Haemophilia PT
    Population Age: Adults Gender: Male
    Trial protocol: GB (Temporarily Halted)
    Trial results: (No results available)
    EudraCT Number: 2009-015112-18 Sponsor Protocol Number: CSLCT-BIO-08-53 Start Date*: 2011-11-30
    Sponsor Name:CSL Behring GmbH
    Full Title: A Phase III, Open-Label, Multicentre Study to Evaluate Efficacy, Pharmacokinetics, and Safety of Biostate® in Paediatric Subjects with Haemophilia A.
    Medical condition: Haemophilia A
    Disease: Version SOC Term Classification Code Term Level
    15.0 10010331 - Congenital, familial and genetic disorders 10018937 Haemophilia A LLT
    Population Age: Newborns, Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: BG (Completed)
    Trial results: View results
    EudraCT Number: 2019-001147-51 Sponsor Protocol Number: COMPIS Start Date*: 2020-03-31
    Sponsor Name:Västra götalandsregionen
    Full Title: Congenital myopathy intervention study
    Medical condition: Congenital myopathy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10062547 Congenital myopathy PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: SE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2017-001078-41 Sponsor Protocol Number: VX13-809-011 Start Date*: 2017-03-27
    Sponsor Name: Vertex Pharmaceuticals Incorporated
    Full Title: A Phase 3, Open-label Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Lumacaftor in Combination With Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis, Homozy...
    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: 2019-004949-32 Sponsor Protocol Number: P1-GM-101 Start Date*: 2021-03-30
    Sponsor Name:Lysogene
    Full Title: An open-label adaptive-design study of intracisternal administration of adeno-associated viral vector serotype rh.10 carrying the human β-galactosidase cDNA for the treatment of GM1 gangliosidosis
    Medical condition: GM1 gangliosidosis
    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: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: FR (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2020-005736-30 Sponsor Protocol Number: CDAII-LUSPA_FA02-2020 Start Date*: 2023-07-17
    Sponsor Name:FOndazione per la Ricerca sulle ANemie ed EMoglobinopatie in ItaliA - For Anemia
    Full Title: A phase II, multicenter, open label study to evaluate the efficacy and safety of Luspatercept (ACE-536) in adult patients with Congenital Dyserythropoietic Anemia type Il (CDA ll).
    Medical condition: Congenital Dyserythropoietic Anemia type II (CDAII)
    Disease: Version SOC Term Classification Code Term Level
    21.1 10010331 - Congenital, familial and genetic disorders 10081457 Congenital dyserythropoietic anaemia PT
    21.1 10010331 - Congenital, familial and genetic disorders 10081457 Congenital dyserythropoietic anaemia PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: (No results available)
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