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Clinical trials for Congenital and hereditary disorders NEC

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   44412   clinical trials with a EudraCT protocol, of which   7421   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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    10 result(s) found for: Congenital and hereditary disorders NEC. Displaying page 1 of 1.
    EudraCT Number: 2014-002839-33 Sponsor Protocol Number: C1-3201 Start Date*: 2015-07-09
    Sponsor Name:Pharming Group NV
    Full Title: A Phase 2 Multicenter, Randomized, Double-Blind, Placebo-Controlled, 3-Period Crossover Study to Evaluate the Efficacy and Safety of Recombinant Human C1 Inhibitor in the Prophylaxis of Angioedema ...
    Medical condition: Prophylaxis of Angioedema Attacks on Patients with Hereditary Angioedema (HAE)
    Disease: Version SOC Term Classification Code Term Level
    17.1 10010331 - Congenital, familial and genetic disorders 10027664 Congenital and hereditary disorders NEC HLGT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: CZ (Completed) IT (Completed)
    Trial results: View results
    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: 2014-000584-41 Sponsor Protocol Number: BP-004 Start Date*: 2014-10-23
    Sponsor Name:Bellicum Pharmaceuticals, Inc.
    Full Title: Phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR αβ+ T cells in pediatric patients affected by hematological disorders
    Medical condition: Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplastic syndromes;Congenital immune deficiencies;Severe aplastic anemia;Fanconi anemia; Osteopetrosis;Selected cases of hemoglobinopath...
    Disease: Version SOC Term Classification Code Term Level
    16.1 10005329 - Blood and lymphatic system disorders 10018849 Haematological disorders NEC HLGT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Completed) GB (GB - no longer in EU/EEA) ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2013-005508-33 Sponsor Protocol Number: MC-FludT.16/NM Start Date*: 2014-09-29
    Sponsor Name:medac Gesellschaft für klinische Spezialpräparate mbH
    Full Title: Clinical phase II trial to compare Treosulfan-based conditioning therapy with Busulfan-based conditioning prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients ...
    Medical condition: Male and female children with non-malignant diseases requiring myeloablative conditioning treatment with following allogeneic haematopoietic stem cell transplantation (allo-HSCT) – i.e. primary imm...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10021606 Inborn errors of metabolism NEC HLT
    20.0 100000004870 10036700 Primary immunodeficiency syndromes HLT
    20.0 100000004850 10018903 Haemoglobinopathies congenital HLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) AT (Completed) CZ (Completed) PL (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2020-004245-37 Sponsor Protocol Number: UCDCRC/20/05 Start Date*: 2020-12-18
    Sponsor Name:University College Dublin
    Full Title: Randomised Placebo-Controlled Trial of Early Targeted Treatment of Patent Ductus Arteriosus with Paracetamol in Extremely Low Birth Weight Infants (ETAPA)
    Medical condition: Patent Ductus Arteriosus
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10034130 Patent ductus arteriosus PT
    Population Age: Preterm newborn infants, Under 18 Gender: Male, Female
    Trial protocol: IE (Trial now transitioned) CZ (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2018-000453-41 Sponsor Protocol Number: BMBF-Fz01KG1602 Start Date*: 2018-05-29
    Sponsor Name:University Hospital Tuebingen
    Full Title: Effects of closed-loop automatic control of the inspiratory fraction of oxygen (FiO2-C) on outcome of extremely preterm infants – a randomized controlled parallel group multicenter trial for safety...
    Medical condition: Extremely low gestational age neonates (ELGANs), i.e. those who are born at <28 weeks gestation and who uniformly suffer from intermittent hypoxemic episodes.
    Disease: Version SOC Term Classification Code Term Level
    21.1 10038738 - Respiratory, thoracic and mediastinal disorders 10006475 Bronchopulmonary dysplasia PT
    20.1 10015919 - Eye disorders 10038933 Retinopathy of prematurity PT
    20.1 10017947 - Gastrointestinal disorders 10055667 Necrotising enterocolitis neonatal PT
    20.1 10018065 - General disorders and administration site conditions 10011912 Death neonatal PT
    21.1 10038738 - Respiratory, thoracic and mediastinal disorders 10071101 Primary apnoea of premature newborns LLT
    Population Age: Preterm newborn infants, Under 18 Gender: Male, Female
    Trial protocol: DE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2016-002974-11 Sponsor Protocol Number: IBU24h-EchoG Start Date*: 2016-11-10
    Sponsor Name:María Carmen Bravo Laguna
    Full Title: Phase III, randomized, multicenter, double-blind clinical trial to evaluate two echo-guided administration regimens of ibuprofen in the treatment of patent ductus arteriosus: impact on intestinal p...
    Medical condition: patent ductus arteriosus
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10034130 Patent ductus arteriosus PT
    Population Age: Preterm newborn infants, Under 18 Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2019-003666-41 Sponsor Protocol Number: MEC2020-0078 Start Date*: 2020-04-21
    Sponsor Name:Erasmus MC University Medical Center
    Full Title: Doxapram versus placebo in preterm newborns: an international double blinded multicenter randomized controlled trial.
    Medical condition: Preterm infants are at high risk of respiratory failure due to immaturity of the respiratory system. Respiratory failure is caused by a comprised lung function and impaired control of breathing. Co...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10038738 - Respiratory, thoracic and mediastinal disorders 10077323 Infantile mixed apnea LLT
    Population Age: Preterm newborn infants, Under 18 Gender: Male, Female
    Trial protocol: NL (Trial now transitioned) BE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2019-004297-26 Sponsor Protocol Number: C19-29 Start Date*: 2020-04-17
    Sponsor Name:Institut National de la Santé et de la Recherche Médicale (INSERM)
    Full Title: Prophylactic treatment of the ductus arteriosus in preterm infants by acetaminophen
    Medical condition: Patent ductus arteriosus
    Disease:
    Population Age: Preterm newborn infants, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed) EE (Completed) IE (Completed) FI (Completed) BE (Completed) PT (Completed) SE (Completed) HU (Completed) DK (Completed) GB (Not Authorised) NO (Completed) AT (Completed) GR (Completed) PL (Ongoing) IT (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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