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Clinical trials for FMR1

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   44335   clinical trials with a EudraCT protocol, of which   7366   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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    13 result(s) found for: FMR1. Displaying page 1 of 1.
    EudraCT Number: 2021-002542-33 Sponsor Protocol Number: ZYN2-CL-033 Start Date*: 2022-02-22
    Sponsor Name:Zynerba Pharmaceuticals, Inc.
    Full Title: A Randomized, Double-Blind, Placebo-Controlled Multiple-Center, Efficacy and Safety Study of ZYN002 Administered as a Transdermal Gel to Children, Adolescents and Young Adults with Fragile X Syndro...
    Medical condition: Fragile X syndrome (FXS) is a rare genetic disorder caused by the deficiency or absence of Fragile X Messenger Ribonucleoprotein 1 (FMRP), an RNA-binding protein and the gene product of the FMR1 ge...
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10017324 Fragile X syndrome PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: IE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2011-004349-42 Sponsor Protocol Number: NP27936 Start Date*: 2012-07-16
    Sponsor Name:F. Hoffmann-La Roche Ltd.
    Full Title: A randomized, double-blind, 12- week, parallel group, placebo-controlled study of the efficacy and safety of RO4917523 in patients with Fragile X Syndrome.
    Medical condition: Fragile X Syndrome (FXS)
    Disease: Version SOC Term Classification Code Term Level
    14.1 100000004873 10025463 Major depressive disorder, single episode LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed) SE (Completed) ES (Completed)
    Trial results: Removed from public view
    EudraCT Number: 2008-003287-18 Sponsor Protocol Number: PET-FRAX-001 Start Date*: 2008-09-17
    Sponsor Name:University Antwerp [...]
    1. University Antwerp
    2. University Hospital Leuven gasthuisberg
    Full Title: Functional consequences of decreased RNA expression of certain GABA(A) receptor subunits in fragile X patients using Positron Emission Tomography and [11C]flumazenil.
    Medical condition: Fragile X syndrome is the most common form of inherited mental retardation with a prevalence of 1/4000 males and 1/6000 females. Patients are characterized by a mild to severe form of cognitive imp...
    Disease:
    Population Age: Adolescents, Under 18, Adults Gender: Male
    Trial protocol: BE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2009-013667-19 Sponsor Protocol Number: CAFQ056A2212 Start Date*: 2010-11-16
    Sponsor Name:Novartis Pharma Services AG
    Full Title: A randomized, double-blind, placebo-controlled, parallel group study to evaluate AFQ056 in adult patients with Fragile X Syndrome
    Medical condition: Fragile X Syndrome
    Disease: Version SOC Term Classification Code Term Level
    15.1 10010331 - Congenital, familial and genetic disorders 10017324 Fragile X syndrome PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DK (Completed) GB (Completed) DE (Completed) IT (Completed) ES (Completed)
    Trial results: View results
    EudraCT Number: 2010-019353-18 Sponsor Protocol Number: CAFQ056B2131 Start Date*: 2011-10-31
    Sponsor Name:Novartis Pharma Services AG
    Full Title: A sequential, open-label, two-period study to assess the pharmacokinetics, safety and tolerability of two dose levels of AFQ056 in male, adolescent patients with Fragile X Syndrome (12 to 18 years ...
    Medical condition: Fragile X Syndrome
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10017324 Fragile X syndrome PT
    Population Age: Adolescents, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2014-000251-89 Sponsor Protocol Number: 1042-0800 Start Date*: 2014-10-22
    Sponsor Name:Antwerp University Hospital
    Full Title: A controlled double-blind crossover trial of ganaxolone in children with fragile X syndrome
    Medical condition: behavioral problems in children with fragile X syndrome
    Disease: Version SOC Term Classification Code Term Level
    17.0 10010331 - Congenital, familial and genetic disorders 10017324 Fragile X syndrome PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: BE (Completed)
    Trial results: View results
    EudraCT Number: 2010-022638-96 Sponsor Protocol Number: CAFQ056B2214 Start Date*: 2011-06-15
    Sponsor Name:Novartis Pharma Services AG
    Full Title: A randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy and safety of AFQ056 in adolescent patients with Fragile X Syndrome
    Medical condition: Fragile X Syndrome
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10017324 Fragile X syndrome PT
    Population Age: Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed) SE (Completed) FR (Completed) DK (Completed) DE (Completed) ES (Completed) Outside EU/EEA IT (Completed) BE (Completed) NL (Completed)
    Trial results: View results
    EudraCT Number: 2009-017837-23 Sponsor Protocol Number: SXF-TRA-152 Start Date*: 2010-09-27
    Sponsor Name:Yolanda de Diego Otero
    Full Title: Ensayo piloto fase II de tratamiento doble ciego, aleatorizado, de una vía cruzada, para investigar la efectividad y seguridad de la combinación de Ácido Ascórbico (vitamina C) y Tocoferol (vitamin...
    Medical condition: Sindrome X frágil. CIE12 : 759.83. enfermedad rara. ORPHA 908. OMIM NUMBER 300264
    Disease: Version SOC Term Classification Code Term Level
    11 10003453 Ácido ascórbico disminuido LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: ES (Completed)
    Trial results: (No results available)
    EudraCT Number: 2013-002885-38 Sponsor Protocol Number: HGT-HIT-094 Start Date*: 2013-10-22
    Sponsor Name:Shire HGT Inc
    Full Title: A Controlled,Randomized,Two-arm,Open-label,Assessor-blinded,Multicenter Study of Intrathecal Idursulfase-IT Administered in Conjunction with Elaprase® in Pediatric Patients with Hunter Syndrome and...
    Medical condition: Long-term treatment of Hunter syndrome and cognitive impairment
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10056889 Mucopolysaccharidosis II PT
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (Completed) ES (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2015-005460-42 Sponsor Protocol Number: SXF2-8 Start Date*: 2016-07-07
    Sponsor Name:Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud
    Full Title: Phase III clinical trial, double-blind, cross-way, to evaluate the safety and efficacy ascorbic acid (vitamin C) and tocopherol (vitamin E) combination versus placebo for the treatment of cognitive...
    Medical condition: Fragile x syndrome
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10017324 Fragile X syndrome PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: ES (Completed)
    Trial results: (No results available)
    EudraCT Number: 2011-001952-12 Sponsor Protocol Number: CAFQ056B2279 Start Date*: 2011-11-03
    Sponsor Name:Novartis Pharma Services AG
    Full Title: An open-label study to evaluate the long-term safety, tolerability and efficacy of AFQ056 in adult patients with Fragile X Syndrome
    Medical condition: Fragile X Syndrome
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10017324 Fragile X syndrome PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DE (Completed) FR (Completed) GB (Prematurely Ended) DK (Prematurely Ended) ES (Completed) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2007-005088-82 Sponsor Protocol Number: CAFQ056A2204 Start Date*: 2008-05-15
    Sponsor Name:Novartis Pharma Services AG
    Full Title: A multi-centre, randomized, double-blind, placebo-controlled, two-period, crossover proof-of-concept study in patients with Fragile X Syndrome to assess the efficacy, safety and tolerability of mul...
    Medical condition: Fragile X Syndrome
    Disease: Version SOC Term Classification Code Term Level
    9.1 10017324 Fragile X syndrome PT
    Population Age: Adults Gender: Male
    Trial protocol: FR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2011-004867-65 Sponsor Protocol Number: CAFQ056B2154 Start Date*: 2012-02-29
    Sponsor Name:Novartis Pharma Services AG
    Full Title: Sequential, two-period study to assess the pharmacokinetics, safety & tolerability of single and multiple oral doses of AFQ056 in patients with FXS (Fragile X syndrome) aged 5-11 years (Cohort 1) a...
    Medical condition: Fragile X Syndrome
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10017324 Fragile X syndrome PT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA ES (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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