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Clinical trials for Chromosomal 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   43881   clinical trials with a EudraCT protocol, of which   7295   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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    21 result(s) found for: Chromosomal Disorder. Displaying page 1 of 2.
    1  2  Next»
    EudraCT Number: 2015-004444-19 Sponsor Protocol Number: CRIT124D2201 Start Date*: 2016-04-05
    Sponsor Name:Novartis Pharmaceutical corporation
    Full Title: An open-label, behavioral-treatment-controlled evaluation of the effects of extended release methylphenidate (Ritalin® LA) on the frequency of cytogenetic abnormalities in children 6 - 12 years of ...
    Medical condition: To determine whether the administration of extended-release methylphenidate in treatment-naïve children with ADHD affects the frequency of chromosomal abnormalities.
    Disease: Version SOC Term Classification Code Term Level
    18.1 10037175 - Psychiatric disorders 10003736 Attention deficit/hyperactivity disorder PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2007-004370-55 Sponsor Protocol Number: 2007-004370-55 Start Date*: 2007-12-14
    Sponsor Name:AZIENDA OSPEDALIERA OSPEDALE NIGUARDA CA' GRANDA (A.O. DI RILIEVO NAZIONALE)
    Full Title: Effects of tetrahydrobiopterin (6R-BH4) on flow-mediated dilation in CADASIL patients: a randomised controlled trial
    Medical condition: CADASIL (Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy), is a rare autosomal dominant disorder characterized by recurrent strokes starting in mid-adulth...
    Disease: Version SOC Term Classification Code Term Level
    9.1 10008804 Chromosomal abnormalities and abnormal gene carriers HLGT
    Population Age: Adults Gender: Male, Female
    Trial protocol: IT (Completed)
    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: 2014-000406-35 Sponsor Protocol Number: UX023-CL201 Start Date*: Information not available in EudraCT
    Sponsor Name:Ultragenyx Pharmaceuticals Inc.
    Full Title: A Randomized, open Label, Dose Finding, Phase 2 Study to Assess the Pharmacodynamics and Safety of the anti-FGF23 antibody, KRN23, in Pediatric Patients with X-linked Hypophosphatemia (XLH)
    Medical condition: X-linked hypophosphatemia (XLH) is a disorder of renal phosphate wasting, and the most common heritable form of rickets. In XLH patients, high circulating levels of fibroblast growth factor 23 (FG...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10016206 Familial hypophosphataemic rickets LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed) NL (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2018-000202-37 Sponsor Protocol Number: BUR02 Start Date*: 2018-10-09
    Sponsor Name:Kyowa Kirin Pharmaceutical Development Ltd
    Full Title: A Phase 3b Open-label Study of the Anti-FGF23 Antibody, Burosumab (KRN23) in Adult Patients with X-linked Hypophosphatemia (XLH)
    Medical condition: XLH is a rare, genetic disorder that is serious, chronically debilitating and represents an unmet medical need. This genetic deficiency is estimated to occur in about 1:20,000 live births (Burn...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10016206 Familial hypophosphataemic rickets LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Completed) GB (GB - no longer in EU/EEA) IT (Ongoing)
    Trial results: View results
    EudraCT Number: 2015-001775-41 Sponsor Protocol Number: UX023-CL304 Start Date*: 2015-12-16
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: An Open-Label, Single-Arm, Phase 3 Study to Evaluate the Effects of KRN23 on Osteomalacia in Adults with X-linked Hypophosphatemia (XLH)
    Medical condition: XLH is a disorder of hypophosphatemia, renal phosphate wasting, and the most common inheritable form of rickets. In XLH patients, excess circulating fibroblast growth factor (FGF23) impair phospha...
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004850 10016206 Familial hypophosphataemic rickets LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DK (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2014-005529-11 Sponsor Protocol Number: UX023-CL303 Start Date*: 2016-02-01
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study with Open-Label Extension to Assess the Efficacy and Safety of KRN23 in Adults with X-linked Hypophosphatemia (XLH)
    Medical condition: XLH is a disorder of hypophosphatemia, renal phosphate wasting, and the most common inheritable form of rickets. In XLH patients, excess circulating fibroblast growth factor (FGF23) impair phospha...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10016206 Familial hypophosphataemic rickets LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Completed) IE (Completed) FR (Completed) DK (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2019-000469-19 Sponsor Protocol Number: BUR-CL207 Start Date*: 2020-11-03
    Sponsor Name:Kyowa Kirin Pharmaceutical Development Ltd.
    Full Title: A Phase 1/2, Open-label, Multicenter, Non-randomized Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Pediatric Patients from Birth to Less than 1 Year of Ag...
    Medical condition: XLH is a rare, genetic disorder that is serious, chronically debilitating and represents an unmet medical need. This genetic deficiency is estimated to occur in about 1:20,000 live births (Burnett ...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10016206 Familial hypophosphataemic rickets LLT
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Ongoing) DE (Prematurely Ended) SE (Completed) AT (Completed) IT (Ongoing)
    Trial results: View results
    EudraCT Number: 2014-005658-20 Sponsor Protocol Number: FGCL-3019-067 Start Date*: 2016-02-23
    Sponsor Name:FibroGen, Inc.
    Full Title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of FG-3019 in Patients with Idiopathic Pulmonary Fibrosis
    Medical condition: Idiopathic Pulmonary Fibrosis
    Disease: Version SOC Term Classification Code Term Level
    19.1 10038738 - Respiratory, thoracic and mediastinal disorders 10021240 Idiopathic pulmonary fibrosis PT
    19.1 10038738 - Respiratory, thoracic and mediastinal disorders 10038738 Respiratory, thoracic and mediastinal disorders SOC
    19.1 10038738 - Respiratory, thoracic and mediastinal disorders 10033979 Parenchymal lung disorders NEC HLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BG (Completed)
    Trial results: View results
    EudraCT Number: 2006-004025-28 Sponsor Protocol Number: HTA 05-14-02 Start Date*: 2007-06-26
    Sponsor Name:Royal Liverpool Children’s NHS Trust
    Full Title: MENDS: The use of MElatonin in children with Neuro-developmental Disorders and impaired Sleep; a randomised, double-blind, placebo-controlled, parallel study
    Medical condition: children with neuro-developmental disorders and impaired sleep
    Disease: Version SOC Term Classification Code Term Level
    9.1 10064062 Neurodevelopmental disorder PT
    9.1 10040984 Sleep disorder PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: (No results available)
    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 (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-004946-27 Sponsor Protocol Number: E2020-A001-219 Start Date*: 2017-05-18
    Sponsor Name:Eisai Inc.
    Full Title: A 10-Week, Double-Blind, Placebo-Controlled Study To Evaluate The Efficacy And Safety Of Donepezil Hydrochloride (Aricept) In The Treatment Of The Cognitive Dysfunction Exhibited By Children With D...
    Medical condition: Down Syndrome, Cognitive Dysfunction
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    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: 2014-002388-13 Sponsor Protocol Number: AZA-JMML-001 Start Date*: 2015-02-20
    Sponsor Name:Celgene Corporation
    Full Title: A Phase 2, multicenter, open-label study to evaluate the pharmacokinetics, pharmacodynamics, safety and activity of azacitidine and to compare azacitidine to historical controls in pediatric subjec...
    Medical condition: newly diagnosed advanced myelodysplastic syndrome (MDS) or juvenile myelomonocytic leukemia (JMML) prior to hematopoietic stem cell transplantation (HSCT)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10054439 Juvenile chronic myelomonocytic leukemia LLT
    20.0 100000004851 10028536 Myelodysplastic syndromes HLT
    20.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10023249 Juvenile chronic myelomonocytic leukaemia PT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) ES (Completed) GB (Completed) IE (Completed) AT (Completed) CZ (Completed) BE (Completed) IT (Completed) SE (Completed) DK (Completed) NL (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2005-000454-73 Sponsor Protocol Number: CC-5013-MDS-004 Start Date*: 2005-08-02
    Sponsor Name:Celgene Corporation
    Full Title: A MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBOCONTROLLED, 3-ARM STUDY OF THE EFFICACY AND SAFETY OF 2 DOSES OF LENALIDOMIDE VERSUS PLACEBO IN RED BLOOD CELL (RBC) TRANSFUSION-DEPENDENT SUBJECTS W...
    Medical condition: Transfusion-dependant, low-or intermediate-1-risk myelodysplastic syndromes associated with a deletion 5q cytogenetic abnormality
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) SE (Completed) DE (Completed) ES (Completed) IT (Completed) BE (Completed)
    Trial results: View results
    EudraCT Number: 2015-002805-13 Sponsor Protocol Number: LTF-304 Start Date*: 2015-11-27
    Sponsor Name:bluebird bio, Inc
    Full Title: Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
    Medical condition: Cerebral Adrenoleukodystrophy (CALD)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10051260 Adrenoleukodystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Ongoing) NL (Ongoing) IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2021-003955-40 Sponsor Protocol Number: TMC114FD1HTX1004 Start Date*: 2022-05-10
    Sponsor Name:Janssen-Cilag International NV
    Full Title: A Single-dose, Open-label, Randomized, Crossover Pivotal Bioequivalence Study in Healthy Participants to Assess the Bioequivalence of Darunavir 600 mg in the Presence of Cobicistat 90 mg When Admin...
    Medical condition: Human Immunodeficiency Virus-1 (Healthy participants)
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004862 10020443 Human immunodeficiency virus syndrome LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: BE (Completed)
    Trial results: View results
    EudraCT Number: 2018-003287-31 Sponsor Protocol Number: M16-104 Start Date*: 2019-06-05
    Sponsor Name:AbbVie Deutschland GmbH & Co. KG
    Full Title: A Phase 2, Multicenter, Single Arm, Open Label Study of Venetoclax Plus Lenalidomide and Dexamethasone for the Treatment of Newly Diagnosed t(11;14)-Positive Multiple Myeloma in Subjects Who Are In...
    Medical condition: Multiple Myeloma
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004864 10028228 Multiple myeloma LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2017-000783-14 Sponsor Protocol Number: SY-1425-201 Start Date*: 2017-10-27
    Sponsor Name:Syros Pharmaceuticals Inc.
    Full Title: A Biomarker-Directed Phase 2 Trial of SY-1425, a Selective Retinoic Acid Receptor Alpha Agonist, in Adult Patients with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)
    Medical condition: Acute Myeloid Leukemia (AML)
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: FR (Ongoing)
    Trial results: View results
    EudraCT Number: 2020-003397-43 Sponsor Protocol Number: TMC114IFD1004 Start Date*: 2021-01-19
    Sponsor Name:Janssen-Cilag International NV
    Full Title: A Single-dose, Open-label, Randomized, Crossover Pivotal Bioequivalence Study in Healthy Participants to Assess the Bioequivalence of Darunavir 675 mg in the Presence of 150 mg Cobicistat When Admi...
    Medical condition: Human immunodeficiency virus type 1 (HIV-1) infection (Healthy participants)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10021881 - Infections and infestations 10020161 HIV infection PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: BE (Completed)
    Trial results: View results
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