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Clinical trials for Disease X

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   44347   clinical trials with a EudraCT protocol, of which   7375   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.
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    7,466 result(s) found for: Disease X. Displaying page 1 of 374.
    1  2  3  4  5  6  7  8  9  Next» Last»»
    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: 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: 2010-019353-18 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: 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: 2009-011145-18 Sponsor Protocol Number: TEN01 Start Date*: 2009-11-02
    Sponsor Name:Bio Products Laboratory Ltd
    Full Title: A Phase III Open, Multicentre Study to Investigate the Pharmacokinetics, Safety and Efficacy of BPL's High Purity Factor X in the Treatment of Severe and Moderate Factor X Deficiency
    Medical condition: Factor X deficiency
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10052474 Factor X deficiency PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) ES (Completed) DE (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: 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: 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: 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: 2014-004490-17 Sponsor Protocol Number: 72 Start Date*: 2014-12-17
    Sponsor Name:Bipebjerg University Hospital
    Full Title: Effect of ACE-Inhibition on Microvascular Function in Women with Assessed Microvascular Dysfunction and No Obstructive Coronary Artery Disease.
    Medical condition: Microvascular dysfunction/microvascular angina
    Disease: Version SOC Term Classification Code Term Level
    18.0 100000004849 10065566 Microvascular angina LLT
    Population Age: Adults, Elderly Gender: Female
    Trial protocol: DK (Completed)
    Trial results: View results
    EudraCT Number: 2015-002153-35 Sponsor Protocol Number: GAP Start Date*: 2015-09-10
    Sponsor Name:Bispebjerg University Hospital
    Full Title: Effect of Glucagon-like peptide-1 stimulation on coronary microvascular dysfunction in women with angina pectoris and no obstructive stenosis of major coronary vessels
    Medical condition: Patients with angina pectoris and coronary microvascular dysfunction without significant stenosis og major coronary vessels
    Disease: Version SOC Term Classification Code Term Level
    18.0 100000004849 10065566 Microvascular angina LLT
    Population Age: Adults, Elderly Gender: Female
    Trial protocol: DK (Completed)
    Trial results: View results
    EudraCT Number: 2012-005013-38 Sponsor Protocol Number: mz01 Start Date*: 2013-02-21
    Sponsor Name:
    Full Title: Effect of GLP-1 on microvascular function in patients with type 2 diabetes
    Medical condition: Microvessel angina is a result of myocardial ischemia from insufficient myocardial perfusion secondary to microvessel dysfunction. Diseased arteries may appear angiographically normal but are dysfu...
    Disease: Version SOC Term Classification Code Term Level
    14.1 100000004849 10065566 Microvascular angina LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DK (Completed)
    Trial results: (No results available)
    EudraCT Number: 2019-002093-32 Sponsor Protocol Number: LUM-002 Start Date*: 2019-10-24
    Sponsor Name:Lument AB
    Full Title: Randomised, open, non-inferiority within patient-controlled trial evaluating the diagnostic usefulness of Lumentin® 44 when used as contrast agent in CT-enterography as compared to MRI- enterograph...
    Medical condition: None. Lumentin 44 is a contrast agent. The diagnostic usefulness of CT with Lumentin 44 as contrast agent as compared to MRE will be investigated in this trial. Patients with confirmed small bowel...
    Disease: Version SOC Term Classification Code Term Level
    22.1 100000004848 10011603 CT scan LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Completed)
    Trial results: View results
    EudraCT Number: 2015-004846-25 Sponsor Protocol Number: Pfizer-Prevenar13-2015 Start Date*: 2016-07-15
    Sponsor Name:Maastricht University
    Full Title: Immunization against oxLDL in patients with lysosomal lipid diseases and associated metabolic disorders
    Medical condition: Familial hypercholesterolemia Niemann-Pick disease type B (NPB) Niemann-Pick disease type C (NPC) Partial lipodystrophy in children and adults
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: BE (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 (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 (Trial now transitioned) DE (Trial now transitioned) NL (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2007-001840-33 Sponsor Protocol Number: 205.389 Start Date*: 2007-11-13
    Sponsor Name:Boehringer Ingelheim Pharma GmbH & Co. KG
    Full Title: Effect of inhalation of tiotropium once daily 18 mcg versus salmeterol twice daily 50 mcg on time to first exacerbation in COPD patients (a randomised, double-blind, double-dummy, parallel group, o...
    Medical condition: chronic obstructive pulmonary disease (COPD)
    Disease: Version SOC Term Classification Code Term Level
    9.1 10010952 COPD LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: LT (Completed) HU (Completed) FI (Completed) GB (Completed) AT (Completed) CZ (Completed) DK (Completed) PT (Completed) LV (Completed) ES (Completed) NL (Completed) SI (Completed) FR (Completed) BE (Completed) DE (Completed) IT (Completed) SK (Completed) BG (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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