Flag of the European Union EU Clinical Trials Register Help

Clinical trials for Chromosome disorders

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   44338   clinical trials with a EudraCT protocol, of which   7368   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 ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     
    55 result(s) found for: Chromosome disorders. Displaying page 1 of 3.
    1  2  3  Next»
    EudraCT Number: 2010-021134-66 Sponsor Protocol Number: IJL-AFHT-TH10 Start Date*: Information not available in EudraCT
    Sponsor Name:Institut Jerome Lejeune
    Full Title: Efficacy assessment of systematic treatment with folinic acid and thyroid hormone on psychomotor development of Down Syndrome young children
    Medical condition: Down syndrome
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10044688 Trisomy 21 PT
    14.0 10010331 - Congenital, familial and genetic disorders 10013616 Down's syndrome LLT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2007-001339-69 Sponsor Protocol Number: CML-paed-II-Study Start Date*: 2008-01-25
    Sponsor Name:THE TECHNICAL UNIVERSITY OF DRESDEN
    Full Title: Protocol for standardized diagnostic procedures, registration, and treatment recommendations in children and adolescents with Philadelphia chromosome-positive chronic myeloid leukemia (CML). A mult...
    Medical condition: Newly diagnosed patients with BCR-ABL-positive CML
    Disease: Version SOC Term Classification Code Term Level
    9.1 10005329 Blood and lymphatic system disorders SOC
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2022-001315-44 Sponsor Protocol Number: TAK-935-18-002(OV935) Start Date*: 2022-05-24
    Sponsor Name:Takeda Development Center America, Inc.
    Full Title: A Multicenter, Open-label, Pilot Study of TAK-935 (OV935) in Patients With 15Q Duplication Syndrome or CDKL5 Deficiency Disorder (ARCADE Study)
    Medical condition: Epileptic Encephalopathies: Dravet Syndrome, Lennox Gastaut Syndrome, CDKL5 Deficiency Disorder, and Chromosome 15 Duplication Syndrome
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10073682 Dravet syndrome LLT
    20.1 10029205 - Nervous system disorders 10048816 Lennox-Gastaut syndrome PT
    22.1 10010331 - Congenital, familial and genetic disorders 10083005 CDKL5 deficiency disorder PT
    23.0 10010331 - Congenital, familial and genetic disorders 10083952 Dup15q syndrome LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    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: 2018-002485-39 Sponsor Protocol Number: TAK-935-18-001(OV935)Amendment1 Start Date*: 2019-06-14
    Sponsor Name:Ovid Therapeutics, Inc.
    Full Title: A PHASE 2, PROSPECTIVE, INTERVENTIONAL, OPEN-LABEL, MULTI-SITE, EXTENSION STUDY TO ASSESS THE LONG-TERM SAFETY AND TOLERABILITY OF TAK-935 (OV935) AS ADJUNCTIVE THERAPY IN PATIENTS WITH RARE EPILEP...
    Medical condition: developmental and epileptic encephalopathies
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: ES (Ongoing) PL (Trial now transitioned) PT (Trial now transitioned) Outside EU/EEA
    Trial results: (No results available)
    EudraCT Number: 2008-008342-20 Sponsor Protocol Number: RAA08-015 Start Date*: 2009-02-23
    Sponsor Name:King's College London
    Full Title: Genetically determined brain abnormalities in Down’s Syndrome.- towards a treatment: A randomised, single-blind, placebo-controlled trial of lithium carbonate in Down’s Syndrome (DownsLit).
    Medical condition: Down's Syndrome
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10013616 Down's syndrome LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (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: 2019-001881-14 Sponsor Protocol Number: 20072650 Start Date*: 2021-03-19
    Sponsor Name:Department of Endocrinology and Internal Medicine, Aarhus University Hospital
    Full Title: Endocrine, cardiovascular, pharmacologic and physiologic aspects of sex hormone treatment in Turner syndrome - project part 1
    Medical condition: Ovarian dysgenesis and related hypogonadism caused by Turner syndrome
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10045181 Turner's syndrome PT
    Population Age: Adults Gender: Female
    Trial protocol: DK (Trial now transitioned)
    Trial results: (No results available)
    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: 2009-013667-19 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: 2011-000460-10 Sponsor Protocol Number: EMR200104_10 Start Date*: 2011-08-03
    Sponsor Name:Merck Serono SA
    Full Title: First year growth response associated genetic markers validation Phase IV open-label study in Growth Hormone Deficient and Turner Syndrome pre-pubertal children : the PREDICT Pharmacogenetics V...
    Medical condition: idiopathic growth hormone deficienty Turner Syndrome
    Disease: Version SOC Term Classification Code Term Level
    13.1 10014698 - Endocrine disorders 10056438 Growth hormone deficiency PT
    13.1 10010331 - Congenital, familial and genetic disorders 10045181 Turner's syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed) ES (Completed) CZ (Completed) IT (Completed)
    Trial results: View results
    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: 2019-004934-40 Sponsor Protocol Number: 20072651 Start Date*: 2021-03-19
    Sponsor Name:Department of Endocrinology and Internal Medicine, Aarhus University Hospital
    Full Title: Endocrine, cardiovascular, pharmacologic and physiologic aspects of sex hormone treatment in Turner syndrome - project part B
    Medical condition: Ovarian dysgenesis and related hypogonadism causes by Turner syndrome
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10045181 Turner's syndrome PT
    Population Age: Adults Gender: Female
    Trial protocol: DK (Trial now transitioned)
    Trial results: (No results available)
    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: 2011-004867-65 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
    EudraCT Number: 2016-003967-21 Sponsor Protocol Number: MGT009 Start Date*: 2017-06-09
    Sponsor Name:MeiraGTx UK II Limited
    Full Title: An open label, multi-centre, Phase I/II dose escalation trial of a recombinant adeno-associated virus vector (AAV2/5-hRKp.RPGR) for gene therapy of adults and children with X-linked Retinitis Pigme...
    Medical condition: X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10038914 Retinitis pigmentosa PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2013-001263-23 Sponsor Protocol Number: BP27832 Start Date*: 2014-03-19
    Sponsor Name:F. Hoffmann-La Roche Ltd
    Full Title: A MULTI-CENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED PHASE 2 STUDY OF THE EFFICACY, SAFETY AND TOLERABILITY OF RO5186582 IN ADULTS AND ADOLESCENTS WITH DOWN SYNDROME (CLEMATIS)
    Medical condition: Down Syndrome
    Disease: Version SOC Term Classification Code Term Level
    17.0 10010331 - Congenital, familial and genetic disorders 10044688 Trisomy 21 PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed) ES (Completed) IT (Completed)
    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: 2005-001169-32 Sponsor Protocol Number: CA180-035 Start Date*: 2005-11-16
    Sponsor Name:Bristol-Myers Squibb International Corporation
    Full Title: A Randomized Two-Arm, Multicenter, Open-Label Phase III Study of BMS-354825 Administered Orally at a Dose of 70 mg Twice Daily or 140 mg Once Daily in Subjects with Chronic Myeloid Leukemia in Acce...
    Medical condition: Subjects with Chronic Myeloid Leukemia (CML) in Accelerated Phase (AP) or in Myeloid (My) or Lymphoid (Ly) Blast Phase (BP) or with Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia (Ph...
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) SE (Completed) HU (Completed) DK (Completed) IE (Completed) CZ (Completed) FI (Completed) ES (Completed) IT (Completed) BE (Completed) DE (Completed) AT (Completed)
    Trial results: View results
    Download Options:
    Number of Trials to download:
    Download Content:
    Download Format:
    Note, where multi-state trials are shown in search results, selecting "Full Trial details" will download full information for each of the member states/countries involved in the trial.
    1  2  3  Next»
    Query did not match any studies.
    For support, Contact us.
    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.

    European Medicines Agency © 1995-Tue May 27 20:38:59 CEST 2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA