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Clinical trials for Neurological disorders congenital

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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    114 result(s) found for: Neurological disorders congenital. Displaying page 1 of 6.
    1  2  3  4  5  6  Next»
    EudraCT Number: 2020-005908-18 Sponsor Protocol Number: 1111_1111 Start Date*: 2021-11-03
    Sponsor Name:AOU FEDERICO II
    Full Title: Functional-genetic stratification as a guide to personalized treatment in developmental and epileptic encephalopathies due to potassium channel mutations
    Medical condition: Epileptic Encephalopathy with developmental delay
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10015039 Epilepsy congenital PT
    20.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    20.0 10029205 - Nervous system disorders 10029205 Nervous system disorders SOC
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2011-004410-42 Sponsor Protocol Number: C11-09 Start Date*: 2012-11-26
    Sponsor Name:Inserm
    Full Title: A phase I/II, open labeled, monocentric study of direct intracranial administration of a replication deficient adeno-associated virus gene transfer vector serotype rh.10 expressing the human ARSA c...
    Medical condition: Early onset forms of MLD
    Disease: Version SOC Term Classification Code Term Level
    14.1 10029205 - Nervous system disorders 10029205 Nervous system disorders SOC
    14.1 10010331 - Congenital, familial and genetic disorders 10067609 Metachromatic leukodystrophy PT
    14.1 10027433 - Metabolism and nutrition disorders 10027433 Metabolism and nutrition disorders SOC
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2011-001455-37 Sponsor Protocol Number: 1331TMF Start Date*: 2011-12-21
    Sponsor Name:Department of Radiology, Oslo University Hospital, Norway
    Full Title: Prospective randomized interventional study. Compare the effect of bleomycin and Tetradecyl Sodium Sulphate (Fibrovein) in the treatment of venous malformations. Clinical neurological and neurophys...
    Medical condition: Venous malformation
    Disease: Version SOC Term Classification Code Term Level
    13.1 10010331 - Congenital, familial and genetic disorders 10047229 Venous malformation NOS LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: NO (Completed)
    Trial results: (No results available)
    EudraCT Number: 2017-003595-30 Sponsor Protocol Number: IRFMN-7358 Start Date*: 2018-02-21
    Sponsor Name:IRCCS- ISTITUTO DI RICERCHE FARMACOLOGICHE MARIO NEGRI
    Full Title: Treat_CCM Clinical Trial. A multicenter randomized clinical trial on Propranolol in Cerebral Cavernous Malformation
    Medical condition: Cerebral Cavernous Malformation (CCM)
    Disease: Version SOC Term Classification Code Term Level
    23.0 10010331 - Congenital, familial and genetic disorders 10071747 Cerebral cavernous malformation PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: View results
    EudraCT Number: 2015-003490-15 Sponsor Protocol Number: CACZ885D2308 Start Date*: 2016-04-14
    Sponsor Name:Novartis Pharmaceuticals
    Full Title: An Open-label, Efficacy and Safety Study of Canakinumab (Anti-interleukin-1β Monoclonal Antibody) Administered for 6 Months (24 Weeks) in Japanese Patients With the Following Cryopyrin-associated P...
    Medical condition: Cryopyrin-associated Periodic Syndromes Familial Cold Autoinflammatory Syndrome Muckle-Wells Syndrome Neonatal Onset Multisystem Inflammatory Disease
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10064570 Familial cold autoinflammatory syndrome PT
    19.0 10010331 - Congenital, familial and genetic disorders 10064574 NOMID LLT
    19.0 10010331 - Congenital, familial and genetic disorders 10068850 Cryopyrin associated periodic syndrome PT
    19.0 10010331 - Congenital, familial and genetic disorders 10064569 Muckle-Wells syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2022-003853-70 Sponsor Protocol Number: PNRR-MR1-2022-12375914 Start Date*: 2023-05-02
    Sponsor Name:OSPEDALE SAN RAFFAELE
    Full Title: Towards a personalized precision medicine in rare disease: tirzepatide (a dual glucose­ dependent insulinotropic polypeptide and glucagon-like peptide-I receptor agonist) monotherapy in patients wi...
    Medical condition: Wolfram syndrome type 1
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10078338 Wolfram syndrome PT
    20.0 10010331 - Congenital, familial and genetic disorders 10078338 Wolfram syndrome PT
    20.0 10010331 - Congenital, familial and genetic disorders 10078338 Wolfram syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2011-000776-34 Sponsor Protocol Number: DFNS01 Start Date*: 2011-06-07
    Sponsor Name:Umea University
    Full Title: THE EFFECT OF DIFLUNISAL ON FAMILIAL TRANSTHYRETIN AMYLOIDOSIS: An open label extension study of ”the diflunisal trial” (IND 68092), and an open label observational study on previously untreated p...
    Medical condition: Familial transthyretin amyloidosis
    Disease: Version SOC Term Classification Code Term Level
    13.1 10010331 - Congenital, familial and genetic disorders 10019893 Hereditary neuropathic amyloidosis, Swedish type LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Completed)
    Trial results: View results
    EudraCT Number: 2019-003028-19 Sponsor Protocol Number: PS_KSS_001_2019 Start Date*: 2019-10-30
    Sponsor Name:IRCCS, OSPEDALE PEDIATRICO BAMBINO GESÙ DI ROMA
    Full Title: Clinical-instrumental definition of the phenotypic spectrum, response to treatment and natural history in Pearson and Kearns-Sayre syndrome
    Medical condition: Pearson and Kearns-Sayre syndrome.
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10058799 Mitochondrial encephalomyopathy PT
    20.1 10010331 - Congenital, familial and genetic disorders 10058799 Mitochondrial encephalomyopathy PT
    20.1 10010331 - Congenital, familial and genetic disorders 10051403 Mitochondrial DNA deletion PT
    20.1 10010331 - Congenital, familial and genetic disorders 10051403 Mitochondrial DNA deletion PT
    20.0 10010331 - Congenital, familial and genetic disorders 10074070 Mitochondrial encephalopathy LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: (No results available)
    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: 2014-002550-39 Sponsor Protocol Number: PDY13949 Start Date*: 2016-10-20
    Sponsor Name:Genzyme Corporation
    Full Title: 4-part, open-label, multicenter, multinational study of the safety, tolerability, pharmacokinetics, pharmacodynamic, and exploratory efficacy of venglustat in combination with Cerezyme in adult pat...
    Medical condition: Gaucher disease
    Disease: Version SOC Term Classification Code Term Level
    24.1 10010331 - Congenital, familial and genetic disorders 10075699 Gaucher's disease type III PT
    24.1 10010331 - Congenital, familial and genetic disorders 10075697 Gaucher's disease type I PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2014-001393-34 Sponsor Protocol Number: MNILCAPS001 Start Date*: 2014-04-14
    Sponsor Name:UZ Leuven
    Full Title: Ilaris® (canakinumab) efficacy and safety in CAPS patients without confirmed mutation on exon 3 of gene CIAS1 and young (<2yrs) CAPS patients with severe neurological (CINCA) phenotype.
    Medical condition: Cryopyrin-Associated Periodic Syndromes
    Disease: Version SOC Term Classification Code Term Level
    17.1 10010331 - Congenital, familial and genetic disorders 10068850 Cryopyrin associated periodic syndrome PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: BE (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2012-003427-38 Sponsor Protocol Number: HGT-GCB-068 Start Date*: 2016-04-07
    Sponsor Name:Shire Human Genetic Therapies
    Full Title: A Multi-center, Open-label, Efficacy and Safety Study of Velaglucerase Alfa Enzyme Replacement Therapy in Children and Adolescents With Type 3 Gaucher Disease
    Medical condition: Type 3 Gaucher disease
    Disease: Version SOC Term Classification Code Term Level
    18.1 10010331 - Congenital, familial and genetic disorders 10075697 Gaucher's disease type I PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2019-004498-18 Sponsor Protocol Number: HUB-NEU-2019-01 Start Date*: 2020-06-30
    Sponsor Name:Dr. Jordi Gascón / Dra. Lola Ledesma
    Full Title: EFFICACY AND SAFETY CLINICAL TRIAL WITH EFAVIRENZ IN PATIENTS DIAGNOSED WITH ADULT NIEMANN-PICK TYPE C WITH COGNITIVE IMPAIRMENT
    Medical condition: Niemann Pick tipo C (NPC)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10029403 Niemann-Pick disease PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2015-005241-31 Sponsor Protocol Number: IEDAT-02-2015 Start Date*: 2016-09-14
    Sponsor Name:EryDel S.p.A.
    Full Title: A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients with Ataxia Telan...
    Medical condition: Patient with neurological symptoms of Ataxia Telangiectasia
    Disease: Version SOC Term Classification Code Term Level
    21.0 10010331 - Congenital, familial and genetic disorders 10003594 Ataxia telangiectasia PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) BE (Completed) NO (Completed) ES (Completed) PL (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2019-000654-59 Sponsor Protocol Number: MT-2-02 Start Date*: 2019-06-25
    Sponsor Name:Minoryx Therapeutics S.L.
    Full Title: An exploratory, open-label, multicenter study in male pediatric patients with cerebral X-linked Adrenoleukodystrophie (cALD) to assess the effect of MIN-102 treatment on the progression of cerebral...
    Medical condition: Cerebral X-linked Adrenoleukodystrophy (cALD)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10051260 Adrenoleukodystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: ES (Ongoing) DE (Trial now transitioned) FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2013-003564-31 Sponsor Protocol Number: UNV-TRI-002 Start Date*: 2014-02-27
    Sponsor Name:Univar B.V.
    Full Title: Multicentre, Retrospective and Prospective Study to Assess Long Term Outcomes of Chelator-Based Treatment With Trientine in Wilson Disease Patients Withdrawn from Therapy With d-Penicillamine
    Medical condition: Wilson disease, also known as hepatolenticular degeneration, a rare automsomal, recessively inherited disorder which results in chronic copper intoxication.
    Disease: Version SOC Term Classification Code Term Level
    19.1 100000004850 10047988 Wilson's disease LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) IT (Prematurely Ended)
    Trial results: (No results available)
    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: 2017-002875-25 Sponsor Protocol Number: NEO-CIRC-003 Start Date*: 2018-07-24
    Sponsor Name:SERMAS-FIBHULP
    Full Title: An international multicentre randomized placebo-controlled, double blind three arm trial to investigate the efficacy of dobutamine with two different starting doses in the treatment of haemodynamic...
    Medical condition: Haemodynamic insufficiency (Haemodynamic insufficiency after birth is commonly seen in babies born prematurely. The condition has a significant clinical impact. A final common pathway is seen but ...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10047065 - Vascular disorders 10009196 Circulatory failure neonatal PT
    Population Age: Preterm newborn infants, Under 18 Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-005229-95 Sponsor Protocol Number: PBKR03-001 Start Date*: 2021-08-23
    Sponsor Name:Passage Bio, Inc.
    Full Title: A Phase 1/2 Open-Label, Multicenter, Dose Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of PBKR03 Administered to Pediatric Subjects with Early Infantile Krabbe Dis...
    Medical condition: Krabbe disease (globoid cell leukodystrophy) is an autosomal recessive lysosomal storage disease (LSD) caused by mutations in the gene encoding the hydrolytic enzyme galactosylceramidase (galactoce...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10023492 Krabbe's disease PT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: NL (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2017-000421-13 Sponsor Protocol Number: 1913 Start Date*: 2017-07-27
    Sponsor Name:Erasmus MC
    Full Title: Newborns with Congenital Diaphragmatic hernia: inhaled Nitric Oxide versus intravenous Sildenafil: an international randomized controlled trial
    Medical condition: Congenital diaphragmatic hernia with pulmonary hypertension
    Disease:
    Population Age: Preterm newborn infants, Newborns, Under 18 Gender: Male, Female
    Trial protocol: NL (Completed) NO (Prematurely Ended) AT (Prematurely Ended) SE (Ongoing) BE (Completed) IT (Prematurely Ended)
    Trial results: (No results available)
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