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Clinical trials for Usher syndrome

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   44339   clinical trials with a EudraCT protocol, of which   7369   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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    8 result(s) found for: Usher syndrome. Displaying page 1 of 1.
    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: 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: 2012-002574-31 Sponsor Protocol Number: US1/001/10(TDU13600) Start Date*: 2013-10-25
    Sponsor Name:sanofi-aventis recherche & développement
    Full Title: A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected SAR421869, Administered to Patients with Retinitis Pigmentosa Associated with Usher Syndrome Type 1B
    Medical condition: Retinitis Pigmentosa, associated with Ushers Syndrome Type 1B.
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10038914 Retinitis pigmentosa 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: FR (Completed)
    Trial results: View results
    EudraCT Number: 2018-002433-38 Sponsor Protocol Number: PQ-421a-001 Start Date*: 2019-02-15
    Sponsor Name:ProQR Therapeutics
    Full Title: A First-in-Human Study to Evaluate the Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene
    Medical condition: Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10038914 Retinitis pigmentosa PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2021-002728-19 Sponsor Protocol Number: PQ-421a-004 Start Date*: Information not available in EudraCT
    Sponsor Name:ProQR Therapeutics IV B.V.
    Full Title: A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of t...
    Medical condition: Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10038914 Retinitis pigmentosa PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Completed) DE (Completed) NO (Prematurely Ended) DK (Completed) NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2021-002729-74 Sponsor Protocol Number: PQ-421a-003 Start Date*: Information not available in EudraCT
    Sponsor Name:ProQR Therapeutics IV B.V.
    Full Title: A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of t...
    Medical condition: Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10038914 Retinitis pigmentosa PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) NO (Completed) DK (Completed) NL (Ongoing) IT (Prematurely Ended) ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2014-000385-22 Sponsor Protocol Number: RF-2010-2318561 Start Date*: 2014-10-07
    Sponsor Name:Ospedale San Raffaele di Milano
    Full Title: Nerve Growth Factor eye drops as a novel treatment for vision loss in patients with Retinitis Pigmentosa: from preclinical to clinical Phase II trial.
    Medical condition: Retinitis pigmentosa with cystoid macular edema
    Disease: Version SOC Term Classification Code Term Level
    17.0 100000004853 10054467 Macular edema LLT
    17.0 10010331 - Congenital, familial and genetic disorders 10038914 Retinitis pigmentosa PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2016-003228-22 Sponsor Protocol Number: AC16092 Start Date*: 2016-12-14
    Sponsor Name:University of Edinburgh [...]
    1. University of Edinburgh
    2. NHS Lothian
    Full Title: Treatment of Osteogenesis Imperfecta with Parathyroid hormone and Zoledronic acid
    Medical condition: Osteogenesis imperfecta
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10031243 Osteogenesis imperfecta PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) IE (Trial now transitioned) DK (Trial now transitioned) FR (Trial now transitioned) NL (Trial now transitioned)
    Trial results: (No results available)
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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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