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Clinical trials for Congenital deafness

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   44365   clinical trials with a EudraCT protocol, of which   7389   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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    19 result(s) found for: Congenital deafness. Displaying page 1 of 1.
    EudraCT Number: 2014-001920-31 Sponsor Protocol Number: DMID11-0069 Start Date*: 2014-12-09
    Sponsor Name:University College London
    Full Title: A PHASE II RANDOMIZED AND CONTROLLED INVESTIGATION OF SIX WEEKS OF ORAL VALGANCICLOVIR THERAPY IN INFANTS AND CHILDREN WITH CONGENITAL CYTOMEGALOVIRUS INFECTION AND HEARING LOSS.
    Medical condition: Children born with congenital CMV infection may develop sequelae on follow-up, particularly sensorineural hearing loss. A recent systematic review reports that 13.5% of babies with congenital CMV ...
    Disease: Version SOC Term Classification Code Term Level
    17.1 100000004854 10040016 Sensorineural hearing loss LLT
    17.1 100000004854 10040017 Sensorineural hearing loss of combined types LLT
    17.1 100000004854 10040018 Sensorineural hearing loss, unspecified LLT
    17.1 100000004854 10040015 Sensorineural deafness LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: GB (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: 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: 2010-024300-10 Sponsor Protocol Number: EARLY_PRO-TECT_ALPORT Start Date*: 2012-02-27
    Sponsor Name:University Medical Center Göttingen
    Full Title: Early prospective therapy trial to delay renal failure in children with Alport syndrome.
    Medical condition: Alport's syndrome
    Disease: Version SOC Term Classification Code Term Level
    19.1 10010331 - Congenital, familial and genetic disorders 10001843 Alport's syndrome PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2020-004594-43 Sponsor Protocol Number: ISTEM02 Start Date*: 2021-05-27
    Sponsor Name:CECS/I-Stem
    Full Title: AUDIOWOLF: A phase II, open-label, efficacy study of daily administration of sodium valproate in patients clinically affected by Wolfram syndrome due to monogenic mutation
    Medical condition: Wolfram syndrome
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10078338 Wolfram syndrome PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2022-001092-14 Sponsor Protocol Number: TIGEM5_USH Start Date*: 2023-10-12
    Sponsor Name:FONDAZIONE TELETHON
    Full Title: A phase I/II open label, dose escalation study of sub-retinal administration of a “mixture of two adeno-associated viral vectors of serotype 8 containing the 5 ´- half sequence of the human MYO7A ...
    Medical condition: Usher syndrome (USH) is characterized by the association of sensorineural hearing loss, Retinitis Pigmentosa (RP), and, in some cases, vestibular dysfunction. It is the most frequent cause of deaf-...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10063396 Usher's syndrome PT
    20.0 10010331 - Congenital, familial and genetic disorders 10063396 Usher's syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2006-005455-15 Sponsor Protocol Number: CACZ885D2304 Start Date*: 2007-06-13
    Sponsor Name:Novartis Pharma Services AG
    Full Title: A three-part multicenter study, with a randomized, doubleblind, placebo controlled, withdrawal design in Part II to assess efficacy, safety, and tolerability of ACZ885 (antiinterleukin-1β monoclona...
    Medical condition: Muckle-Wells Syndrome (Autoinflammatory Disease)
    Disease: Version SOC Term Classification Code Term Level
    9.1 10064569 Muckle-Wells syndrome LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) ES (Completed) FR (Completed) GB (Completed) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2016-004395-22 Sponsor Protocol Number: 402-C-1603 Start Date*: 2017-12-19
    Sponsor Name:Reata Pharmaceuticals, Inc.
    Full Title: A Phase 2/3 Trial of the Efficacy and Safety of Bardoxolone Methyl in Patients with Alport Syndrome
    Medical condition: Alport Syndrome
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10001843 Alport's syndrome PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) GB (GB - no longer in EU/EEA) ES (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2021-004192-13 Sponsor Protocol Number: R3R01-ASFSGS-201 Start Date*: 2022-08-12
    Sponsor Name:River 3 Renal, Corporation
    Full Title: A Phase II, Multi-center, Open-Label Study to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of R3R01 in Alport Syndrome Patients with Uncontrolled Proteinuria on ACE/ARB Inhibition and...
    Medical condition: Alport Syndrome (AS) and Primary Steroid-Resistent Focal Segmental Glomerulosclerosis (FSGS)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10001843 Alport's syndrome PT
    21.1 10038359 - Renal and urinary disorders 10067757 Focal segmental glomerulosclerosis PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Trial now transitioned) FR (Trial now transitioned) NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2007-004367-22 Sponsor Protocol Number: CACZ885D2306 Start Date*: 2008-05-01
    Sponsor Name:Novartis Pharma Services AG
    Full Title: An open-label, long-term safety and efficacy study of ACZ885 (anti-interleukin-1β monoclonal antibody) administered for at least 6 months in patients with the following cryopyrin-associated periodi...
    Medical condition: The following cryopyrin-associated periodic syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease
    Disease: Version SOC Term Classification Code Term Level
    9.1 10064569 Muckle-Wells syndrome LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) DE (Completed) FR (Completed) ES (Completed) IT (Prematurely Ended) BE (Completed) Outside EU/EEA
    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: 2004-002980-26 Sponsor Protocol Number: CACZ885A2102 Start Date*: 2004-12-10
    Sponsor Name:Novartis Pharma Services AG
    Full Title: An open-label, phase II dose titration study of ACZ885 (human anti-IL-1beta monoclonal antibody) to assess the clinical efficacy, safety, pharmacokinetics and pharmacodynamics in patients with NALP...
    Medical condition: Muckle-Wells Syndrome: rare hereditary, autosomal dominant, systemic inflammatory disease, characterized by recurrent episodes of fever, arthralgia, myalgia, urticarial rash, and conjunctivitis. La...
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) DE (Completed) ES (Completed) FR (Completed) 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: 2021-000621-27 Sponsor Protocol Number: RTRX-RE021-201 Start Date*: 2022-10-20
    Sponsor Name:Travere Therapeutics, Inc.
    Full Title: A Phase 2, Open-Label, Single-Arm, Cohort Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Sparsentan Treatment in Pediatric Subjects with Selected Proteinuric Glomerular Diseases (E...
    Medical condition: Proteinuric glomerular diseases including: •Focal segmental glomerulosclerosis (FSGS) •Minimal change disease (MCD) •Immunoglobulin A nephropathy (IgAN) •Immunoglobulin A vasculitis (IgAV) •Al...
    Disease: Version SOC Term Classification Code Term Level
    21.1 10038359 - Renal and urinary disorders 10067757 Focal segmental glomerulosclerosis PT
    20.0 10038359 - Renal and urinary disorders 10021263 IgA nephropathy PT
    22.1 100000004858 10082959 IgA vasculitis LLT
    20.0 10010331 - Congenital, familial and genetic disorders 10001843 Alport's syndrome PT
    21.1 10038359 - Renal and urinary disorders 10058326 Minimal change disease LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed) ES (Temporarily Halted) SE (Trial now transitioned) PL (Temporarily Halted) DE (Restarted) IT (Trial now transitioned) NL (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: 2011-002744-27 Sponsor Protocol Number: CR01849 Start Date*: 2011-11-01
    Sponsor Name:Imperial College
    Full Title: Pharmacodynamic Studies of a Histone Deacetylase Inhibitor in FRDA
    Medical condition: Friedreich’s ataxia
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10017374 Friedreich's ataxia PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2022-000079-38 Sponsor Protocol Number: DB-OTO-001 Start Date*: 2023-05-09
    Sponsor Name:Decibel Therapeutics
    Full Title: A Phase 1/2, Open-Label, Multicenter Trial With a Single Ascending Dose Cohort With Unilateral Intracochlear Injection Followed by a Bilateral Injection Expansion Cohort to Evaluate the Safety, Tol...
    Medical condition: Congenital auditory neuropathy secondary to biallelic mutations of the otoferlin gene (hOTOF)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004854 10011874 Deaf LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2018-001489-41 Sponsor Protocol Number: P00003466 Start Date*: 2018-07-02
    Sponsor Name:Karolinska Universitetssjukhuset
    Full Title: Radiation- and alkylator-free hematopoietic cell transplantation for bone marrow failure due to dyskeratosis congenita / telomere disease
    Medical condition: Dyskeratosis congenita / telomere disease
    Disease: Version SOC Term Classification Code Term Level
    20.0 10042613 - Surgical and medical procedures 10001756 Allogenic bone marrow transplantation therapy PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: SE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2019-000599-40 Sponsor Protocol Number: KH176-202 Start Date*: 2019-10-23
    Sponsor Name:Khondrion B.V.
    Full Title: A Phase IIb double-blind, randomised, placebo-controlled, multi-centre, confirmative three-way cross-over study on cognitive function with two doses of KH176 in subjects with a genetically confirme...
    Medical condition: A genetically confirmed mitochondrial desoxyribonucleic acid (DNA) transfer ribonucleic acid (tRNA)Leu(UUR) m.3243A>G mutation (including but not limited to MELAS, MIDD and mixed compositions).
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Completed) GB (GB - no longer in EU/EEA) DE (Completed) DK (Completed)
    Trial results: View results
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