Flag of the European Union EU Clinical Trials Register Help

Clinical trials for Chromosome disorder

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 ).  
     
    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
     
    33 result(s) found for: Chromosome disorder. Displaying page 1 of 2.
    1  2  Next»
    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: 2004-002601-69 Sponsor Protocol Number: CA180-013 Start Date*: 2005-01-25
    Sponsor Name:Bristol Myers Squibb International Corporation
    Full Title: A Phase II Study to Determine the Activity of BMS-354825 in Subjects with Chronic Phase Philadelphia Chromosome-Positive Chronic Myeloid Leukemia Who Have Disease that is Resistant to High Dose Ima...
    Medical condition: Chronic phase Philadelphia chromosome-positive chronic myeloid leukemia
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: AT (Completed) DE (Completed) SE (Completed) FI (Completed) GB (Completed) IE (Completed) IT (Completed) BE (Completed) ES (Completed) DK (Completed)
    Trial results: View results
    EudraCT Number: 2010-022946-25 Sponsor Protocol Number: AALL0622 Start Date*: 2011-12-19
    Sponsor Name:Children's Oncology Group
    Full Title: Intensified Tyrosine Kinase Inhibitor Therapy (Dasatinib: IND# 73969, NSC# 732517) in Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia (ALL)
    Medical condition: Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia (ALL)
    Disease: Version SOC Term Classification Code Term Level
    14.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10000844 Acute lymphoblastic leukaemia LLT
    14.1 10022891 - Investigations 10034877 Philadelphia chromosome positive LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    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: 2004-004450-96 Sponsor Protocol Number: CA180-017 Start Date*: 2005-01-25
    Sponsor Name:Bristol Myers Squibb International Corporation
    Full Title: A Randomized Multi-center Open Label Study of BMS-354825 vs. Imatinib Mesylate (Gleevec) 800 mg/d in Subjects with Chronic Phase Philadelphia Chromosome-Positive Chronic Myeloid Leukemia Who Have D...
    Medical condition: Chronic phase Philadelphia-Chromosome Positive (Ph+) Chronic Myeloid leukemia (CML)
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: AT (Completed) SE (Completed) DE (Completed) HU (Completed) FI (Completed) GB (Completed) IE (Completed) DK (Completed) EE (Completed) ES (Completed) BE (Completed)
    Trial results: View results
    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: 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: 2005-001294-99 Sponsor Protocol Number: CA180-034 Start Date*: 2005-07-18
    Sponsor Name:Bristol-Myers Squibb International Corporation
    Full Title: A Randomized Two-by-Two, Multicenter, Open-Label Phase III Study of BMS-354825 Administered Orally at a Dose of 50 mg or 70 mg Twice Daily or 100 mg or 140 mg Once Daily in Subjects with Chronic Ph...
    Medical condition: Subjects with Chronic Phase Philadelphia Chromosome or BCR-ABL Positive Chronic Myelogenous Leukemia who are resistant or intolerant to Imatinib Mesylate
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: AT (Completed) SE (Completed) GB (Completed) HU (Completed) DK (Completed) IE (Completed) CZ (Completed) FI (Completed) IT (Completed) EE (Completed) ES (Completed) DE (Completed) BE (Completed)
    Trial results: View results
    EudraCT Number: 2009-010280-17 Sponsor Protocol Number: TSHYPE-2601-2009 Start Date*: 2009-04-17
    Sponsor Name:Organisation name was not entered
    Full Title: TS HYPE - Turner syndrome and Hypertension; a double-blinded randomised interventional trial.
    Medical condition: Turner syndrome, hypertension and aortopathy
    Disease: Version SOC Term Classification Code Term Level
    9.1 10045181 Turner's syndrome LLT
    9.1 10020772 Hypertension LLT
    Population Age: Adults, Elderly Gender: Female
    Trial protocol: DK (Completed)
    Trial results: (No results available)
    EudraCT Number: 2006-003075-12 Sponsor Protocol Number: 290478 Start Date*: Information not available in EudraCT
    Sponsor Name:Department Of Paediatric Cardiology
    Full Title: Effect of carvedilol on left ventricular systolic and diastolic function and the neurohormonal axis in patients with Duchenne muscular dystrophy and left ventricular dysfunction.
    Medical condition: Duchenne muscular dystrophy is an X-linked inherited disorder which primarily affects skeletal muscle through a mutation in dystrophin which maps to the long arm of the X chromosome (Xp21). Patient...
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: IE (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2006-001279-39 Sponsor Protocol Number: CA180-083 Start Date*: 2006-09-15
    Sponsor Name:Bristol-Myers Squibb International Corporation
    Full Title: A Phase II Study of Dasatinib (BMS-354825) in Subjects with Chronic or Advanced Phase Chronic Myelogenous Leukemia or Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia who are Resistant...
    Medical condition: Subjects with Chronic or Advanced Phase Chronic Myelogenous Leukemia (CML) or Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia (ALL) who are Resistant or Intolerant to Imatinib.
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) CZ (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2021-006220-40 Sponsor Protocol Number: 2022-0101-01 Start Date*: 2022-06-30
    Sponsor Name:Hvidovre Hospital, Department of Gynecology
    Full Title: Double-blinded randomized placebo-controlled trial on tacrolimus in women with an euploid pregnancy loss and PCOS and/or thyroid autoimmunity
    Medical condition: Polycystic ovarian syndrome and/or thyroid autoimmunity in combination with an euploid pregnancy loss
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004872 10065161 Polycystic ovarian syndrome LLT
    21.0 100000004848 10069094 Thyroid peroxidase antibody positive LLT
    20.1 100000004848 10060326 Thyroglobulin antibody positive LLT
    22.1 100000004868 10072314 Pregnancy loss LLT
    Population Age: Adults Gender: Female
    Trial protocol: DK (Prematurely Ended)
    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: 2021-003791-13 Sponsor Protocol Number: BP42992 Start Date*: 2022-01-11
    Sponsor Name:F. HOFFMANN - LA ROCHE LTD.
    Full Title: A PHASE II, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL GROUP STUDY TO EVALUATE THE SAFETY, EFFICACY, AND PHARMACODYNAMICS OF 52 WEEKS OF TREATMENT WITH BASMISANIL IN CHILDREN WITH DUP15...
    Medical condition: Dup15q Syndrome
    Disease: Version SOC Term Classification Code Term Level
    23.0 100000004850 10083952 Dup15q syndrome LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: IT (Prematurely Ended) ES (Prematurely Ended) PT (Prematurely Ended) PL (Completed) NL (Ongoing)
    Trial results: View results
    EudraCT Number: 2010-021790-37 Sponsor Protocol Number: UK_S-H_08_0006 Start Date*: Information not available in EudraCT
    Sponsor Name:Universitätsklinikum Schleswig-Holstein, Campus Lübeck
    Full Title: Comparison of clinical and metabolic effects of testosterone and estradiol in adult gonadectomized patients with 46,XY DSD due to complete androgen insensitivity syndrome (CAIS)
    Medical condition: The medical condition under investigation is complete androgen insensitivity syndrome (CAIS) in gonadectomized patients with 46,XY karyotype due to mutations of the androgen receptor, which lead to...
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10056292 Androgen insensitivity syndrome PT
    Population Age: Adults Gender: Female
    Trial protocol: DE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2011-003075-11 Sponsor Protocol Number: 9HB01EXT Start Date*: 2011-12-05
    Sponsor Name:Biogen Idec Research Ltd
    Full Title: An Open-Label, Multicenter Evaluation of the Long-Term Safety and Efficacy of Recombinant, Human Coagulation Factor IX Fusion Protein (rFIXFc) in the Prevention and Treatment of Bleeding Episodes i...
    Medical condition: The medical condition to be investigated is Hemophilia B, or Christmas disease. Hemophilia B is a deficiency in the clotting FIX and is a recessively inherited coagulation disorder due to an X-chro...
    Disease: Version SOC Term Classification Code Term Level
    19.1 100000004850 10053754 Hemophilia B without inhibitors LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male
    Trial protocol: GB (Completed) DE (Completed) BE (Completed) SE (Completed) PL (Completed) IT (Completed) IE (Completed) NL (Completed)
    Trial results: View results
    EudraCT Number: 2019-003787-48 Sponsor Protocol Number: BP41674 Start Date*: 2020-06-11
    Sponsor Name:F. Hoffmann-La Roche
    Full Title: AN OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND PHARMACODYNAMICS OF RO7248824 IN PARTICIPANTS WITH ANGELMAN SYNDROME
    Medical condition: Angelman Syndrome
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10049004 Angelman's syndrome PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) NL (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2021-003009-23 Sponsor Protocol Number: ION582-CS1 Start Date*: 2022-07-19
    Sponsor Name:Ionis Pharmaceuticals, Inc.
    Full Title: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION582 in Patients with Angelman Syndrome
    Medical condition: Angelman syndrome is a neurogenetic disorder caused by a loss of function of the maternally inherited UBE3A gene (chromosome 15) which codes for UBE3A ubiquitin protein ligase. AS presents early in...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10049004 Angelman's syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) IT (Trial now transitioned) NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-000600-29 Sponsor Protocol Number: UX023-CL301 Start Date*: 2016-09-15
    Sponsor Name:Ultragenyx Pharmaceuticals Inc
    Full Title: A Randomized, Open-Label, Phase 3 Study to Assess the Efficacy and Safety of KRN23 Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients with X-linked Hypophosphatemia (XLH)
    Medical condition: X-linked hypophosphatemia (XLH) is a disorder of renal phosphate wasting, defective bone mineralisation, and impaired growth plate or endochondral ossification caused by inactivating mutations in t...
    Disease: Version SOC Term Classification Code Term Level
    19.1 100000004850 10016206 Familial hypophosphataemic rickets LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: DK (Prematurely Ended) IE (Completed) DE (Completed) ES (Prematurely Ended) SE (Completed) GB (Completed) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2011-003076-36 Sponsor Protocol Number: 9HB02PED Start Date*: 2012-05-29
    Sponsor Name:Biogen Idec Research Ltd
    Full Title: Open-label, Multicenter Evaluation of Safety, Pharmacokinetics, and Efficacy of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in the Prevention and Treatment of Bleeding Episodes in...
    Medical condition: The medical condition to be investigated is Hemophilia B, or Christmas disease. Hemophilia B is a deficiency in the clotting FIX and is a recessively inherited coagulation disorder due to an X-chro...
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10053754 Hemophilia B without inhibitors LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: GB (Completed) IE (Completed) NL (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  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-Sun May 11 23:15:28 CEST 2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA