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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 ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
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    1,387 result(s) found for: Congenital disorders. Displaying page 70 of 70.
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    EudraCT Number: 2011-001255-36 Sponsor Protocol Number: GS-US-205-0162 Start Date*: 2012-01-16
    Sponsor Name:Gilead Sciences, Inc.
    Full Title: Open-Label Phase 2 Trial to Evaluate the Safety and Efficacy of Aztreonam 75 mg Powder and Solvent for Nebuliser Solution/Aztreonam for Inhalation Solution (AZLI) in Pediatric Patients with Cyst...
    Medical condition: Cystic fibrosis and new onset lower respiratory tract culture positive for Pseudomonas aeruginosa
    Disease: Version SOC Term Classification Code Term Level
    14.1 10021881 - Infections and infestations 10070608 Infective pulmonary exacerbation of cystic fibrosis PT
    14.1 10021881 - Infections and infestations 10068288 Cystic fibrosis pulmonary exacerbation LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) BE (Completed) FR (Ongoing) AT (Completed) ES (Completed) NL (Completed) IE (Completed) IT (Completed) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2008-003924-52 Sponsor Protocol Number: PTC124-GD-009-CF Start Date*: 2009-04-08
    Sponsor Name:PTC Therapeutics, Inc
    Full Title: A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis
    Medical condition: Nonsense-Mutation-Mediated Cystic Fibrosis
    Disease: Version SOC Term Classification Code Term Level
    9.1 10011762 Cystic fibrosis LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Completed) NL (Completed) BE (Completed) ES (Completed) FR (Completed) GB (Completed) IT (Completed) DE (Completed)
    Trial results: View results
    EudraCT Number: 2020-004805-30 Sponsor Protocol Number: CLN-PXT3003-06 Start Date*: Information not available in EudraCT
    Sponsor Name:Pharnext SCA
    Full Title: A Multi-Center, Randomized, Double-Blind, Placebo-Controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth type 1A (CMT1A)
    Medical condition: Charcot Marie Tooth Type 1A
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10008414 Charcot-Marie-Tooth disease LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) BE (Completed) NL (Prematurely Ended) DK (Prematurely Ended) ES (Prematurely Ended) IT (Prematurely Ended) FR (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2015-004984-35 Sponsor Protocol Number: EZH-501 Start Date*: 2016-08-03
    Sponsor Name:Epizyme, Inc.
    Full Title: Tazemetostat Rollover Study (TRuST): An Open-Label, Rollover Study
    Medical condition: Subjects will receive tazemetostat as dictated in their antecedent study.
    Disease: Version SOC Term Classification Code Term Level
    19.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10027414 Mesotheliomas malignant and unspecified HLT
    19.1 100000004850 10027404 Mesomelia LLT
    19.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10007284 Carcinoma LLT
    19.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10027406 Mesothelioma PT
    19.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10027408 Mesothelioma malignant advanced LLT
    19.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10026667 Malignant peripheral nerve sheath tumor LLT
    19.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10073335 Rhabdoid tumor LLT
    19.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10042863 Synovial sarcoma PT
    19.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10064886 Renal medullary carcinoma LLT
    19.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10073134 Extraskeletal myxoid chondrosarcoma PT
    19.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10062474 Mesothelioma malignant localized LLT
    19.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10074121 Rhabdoid tumor of the kidney LLT
    19.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10027411 Mesothelioma malignant recurrent PT
    19.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10015100 Epithelioid sarcomas HLT
    19.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10027412 Mesotheliomas HLGT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) PL (Trial now transitioned) BE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2018-003385-14 Sponsor Protocol Number: AROAAT2001 Start Date*: 2019-08-06
    Sponsor Name:Arrowhead Pharmaceuticals, Inc.
    Full Title: A Placebo-Controlled, Multi-dose, Phase 2/3 Study to Determine the Safety, Tolerability and Effect on Liver Histologic Parameters in Response to ARO-AAT in Patients with Alpha-1 Antitrypsin Deficie...
    Medical condition: alpha-1 antitrypsin deficiency (AATD)-associated liver disease
    Disease: Version SOC Term Classification Code Term Level
    23.0 10001806 - Alpha-1 anti-trypsin deficiency 10001806 Alpha-1 anti-trypsin deficiency LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IE (Completed) SE (Completed) ES (Restarted) GB (GB - no longer in EU/EEA) PT (Completed) NL (Ongoing) DE (Completed) AT (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2016-000942-77 Sponsor Protocol Number: EFC14028 Start Date*: 2016-07-15
    Sponsor Name:Genzyme Corporation
    Full Title: A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase ...
    Medical condition: Pompe disease (acid alpha-glucosidase deficiency)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10036143 Pompe's disease LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) DK (Prematurely Ended) SE (Prematurely Ended) NL (Completed) ES (Completed) CZ (Completed) DE (Completed) BE (Completed) AT (Completed) PL (Completed) IT (Completed) PT (Completed) BG (Completed) HU (Completed) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2021-002480-22 Sponsor Protocol Number: TAK-935-3001 Start Date*: 2021-07-22
    Sponsor Name:Takeda Development Center Americas, Inc
    Full Title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy, Safety, and Tolerability of Soticlestat as Adjunctive Therapy in Pediatric and Young Adul...
    Medical condition: Dravet Syndrome (DS)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10073682 Dravet syndrome LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA IT (Completed) FR (Completed) ES (Completed) LV (Completed) GR (Completed) NL (Completed) BE (Completed) PL (Completed) HU (Completed) DE (Completed)
    Trial results: View results
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    Query did not match any studies.
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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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