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Clinical trials for Genetic structure

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   44358   clinical trials with a EudraCT protocol, of which   7384   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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    48 result(s) found for: Genetic structure. Displaying page 1 of 3.
    1  2  3  Next»
    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: 2012-001427-12 Sponsor Protocol Number: CACZ885M2201 Start Date*: 2013-06-21
    Sponsor Name:Novartis Pharma Services AG
    Full Title: A multicenter, randomized, double-blind, placebo-controlled study of the safety, tolerability and effects on arterial structure and function of ACZ885 in patients with intermittent claudication
    Medical condition: Peripheral Artery Disease
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004866 10003611 Atherosclerosis of arteries of the extremities, unspecified LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2008-003059-56 Sponsor Protocol Number: ICAND Start Date*: 2008-07-29
    Sponsor Name:ISTITUTO NEUROLOGICO "CARLO BESTA"
    Full Title: Phase II trial to assess safety and efficacy of Iron chelating agent Deferiprone in patients with Pantothenate Kinase-Associated Neurodegeneration – ICAND – Trial
    Medical condition: PKAN
    Disease: Version SOC Term Classification Code Term Level
    9.1 10042259 Structural brain disorders NEC HLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2017-003083-13 Sponsor Protocol Number: 250-202 Start Date*: Information not available in EudraCT
    Sponsor Name:Allievex Corporation
    Full Title: A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Safety and Efficacy of Intracerebroventricular AX 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo ...
    Medical condition: Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPS IIIB)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    20.0 10010331 - Congenital, familial and genetic disorders 10056918 Sanfilippo's syndrome LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) ES (Prematurely Ended) GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2018-004740-36 Sponsor Protocol Number: PTCEMF-GD-004 Start Date*: 2019-10-14
    Sponsor Name:PTC Therapeutics, Inc.
    Full Title: A MULTICENTER RANDOMIZED PLACEBO-CONTROLLED PHASE 3 STUDY ON THE SAFETY AND EFFICACY OF DEFLAZACORT (EMFLAZA®) IN SUBJECTS WITH LIMB-GIRDLE MUSCULAR DYSTROPHY 2I (LGMD2I
    Medical condition: Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10028356 Muscular dystrophy PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) FR (Completed) DK (Completed)
    Trial results: View results
    EudraCT Number: 2015-001985-25 Sponsor Protocol Number: 250-201 Start Date*: 2015-11-06
    Sponsor Name:Allievex Corporation
    Full Title: A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular AX 250 in Patients with Mucopolysaccharidosis Type IIIB ...
    Medical condition: Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B, MPS IIIB)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10056918 Sanfilippo's syndrome LLT
    20.1 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed) DE (Completed) ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2015-004143-39 Sponsor Protocol Number: SHIP002 Start Date*: 2016-08-09
    Sponsor Name:Erasmus MC
    Full Title: A Phase 3 randomised, double-blind, controlled trial of inhaled 7% hypertonic saline versus 0.9% isotonic saline for 48 weeks in patients with Cystic Fibrosis at 3-6 years of age in parallel with t...
    Medical condition: Cystic Fibrosis
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10011763 Cystic fibrosis lung PT
    20.0 10010331 - Congenital, familial and genetic disorders 10011762 Cystic fibrosis PT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: NL (Completed) BE (Completed) DK (Completed) FR (Completed) ES (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2015-001382-10 Sponsor Protocol Number: CS/2015/4775 Start Date*: 2019-01-21
    Sponsor Name:University Hospitals Bristol NHS Foundation Trust
    Full Title: Treatment of Barth Syndrome by CARDIOlipin MANipulation (CARDIOMAN): A randomised placebo controlled pilot trial conducted by the nationally commissioned Barth Syndrome Service
    Medical condition: Barth Syndrome is a rare, life threatening, genetic disease which affects young males. It is caused by abnormal fats (lipids) in the powerhouses of cells (mitochondria) and those who suffer with it...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2015-001383-18 Sponsor Protocol Number: Start Date*: 2015-11-30
    Sponsor Name:University of Oxford
    Full Title: An open label Phase 2 clinical trial of retinal gene therapy for choroideremia using an adeno-associated viral vector (AAV2) encoding Rab-escort protein 1 (REP1)
    Medical condition: Choroideremia - a currently incurable retinal degeneration caused by null mutations in the CHM gene encoding REP1 protein located on the X chromosome. The condition causes blindness in males by the...
    Disease: Version SOC Term Classification Code Term Level
    18.1 100000004853 10008791 Choroideremia LLT
    Population Age: Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2015-003503-39 Sponsor Protocol Number: ABR54654 Start Date*: 2016-07-27
    Sponsor Name:University Medical Center Utrecht
    Full Title: Placebo-controlled Trial in Subjects at Ultra-high Risk for Psychosis With Omega-3 Fatty Acids in Europe
    Medical condition: Individuals at Ultra High Risk for Psychosis
    Disease:
    Population Age: Adolescents, Under 18 Gender: Male, Female
    Trial protocol: NL (Completed) AT (Completed) IT (Completed) ES (Ongoing) DE (Completed) GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2018-004689-32 Sponsor Protocol Number: SHP675-301 Start Date*: 2021-09-24
    Sponsor Name:Shire Human Genetic Therapies, Inc.
    Full Title: A Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease
    Medical condition: Fabry disease
    Disease: Version SOC Term Classification Code Term Level
    24.1 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: PT (Prematurely Ended) FI (Prematurely Ended) GR (Prematurely Ended) PL (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2013-004664-58 Sponsor Protocol Number: BESTCILIA-WP5 Start Date*: 2014-08-26
    Sponsor Name:Paediatric Pulmonary Service, Department of Paediatrics and Adolescent Medicine, Rigshospitalet, Copenhagen, Denmark
    Full Title: RANDOMIZED CONTROLLED TRIAL (RCT) TO DETERMINE THE EFFICACY AND SAFETY OF AZITHROMYCIN (AZN) MAINTENANCE THERAPY FOR 6 MONTHS IN SUBJECTS WITH PCD - A DOUBLE-BLIND, PARALLEL GROUP STUDY
    Medical condition: Primary ciliary dyskinesia (PCD)
    Disease: Version SOC Term Classification Code Term Level
    18.0 10010331 - Congenital, familial and genetic disorders 10069713 Primary ciliary dyskinesia PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) DK (Prematurely Ended) GB (Prematurely Ended) NL (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2022-003787-24 Sponsor Protocol Number: PM-CARE-PNRR-MAD-2022-12375905 Start Date*: 2023-04-20
    Sponsor Name:OSPEDALE SAN RAFFAELE
    Full Title: Precision Medicine in patients with unresectable CholAngiocarcinoma; RadioEmbolization and combined biological therapy (Single arm, multicenter phase II study investigating the efficacy and safety...
    Medical condition: Unresectable intrahepatic cholangiocarcinoma
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10073077 Intrahepatic cholangiocarcinoma LLT
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10008593 Cholangiocarcinoma PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2006-005734-20 Sponsor Protocol Number: R076477-SCA-3002 Start Date*: 2008-04-24
    Sponsor Name:Janssen L.P.
    Full Title: A Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Flexible Dose Paliperidone ER in the Treatment of Subjects with Schizoaffective Disorder
    Medical condition: Schizoaffective disorder
    Disease: Version SOC Term Classification Code Term Level
    8.1 10039621 Schizoaffective disorder LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: BG (Completed)
    Trial results: View results
    EudraCT Number: 2014-003572-23 Sponsor Protocol Number: GCP#01.01.030 Start Date*: 2017-12-27
    Sponsor Name:Gamida Cell Ltd
    Full Title: Allogeneic Stem Cell Transplantation of CordIn™, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients with Hemoglobinopathies
    Medical condition: Hemoglobinopathies
    Disease: Version SOC Term Classification Code Term Level
    18.0 100000004850 10054658 Thalassemia LLT
    18.0 100000004850 10040645 Sickle cell disease NOS LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2017-002530-23 Sponsor Protocol Number: MYK-461-005 Start Date*: 2018-10-04
    Sponsor Name:MyoKardia, Inc.
    Full Title: A Randomized, Double blind, Placebo controlled Clinical Study to Evaluate Mavacamten (MYK-461) in Adults with Symptomatic Obstructive Hypertrophic Cardiomyopathy
    Medical condition: Hypertrophic Cardiomyopathy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10020871 Hypertrophic cardiomyopathy PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) GB (Completed) PT (Completed) ES (Completed) CZ (Completed) BE (Completed) NL (Completed) PL (Completed) DK (Completed) FR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2019-003374-91 Sponsor Protocol Number: SRP-9001-301 Start Date*: 2022-09-12
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBA...
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10052655 Duchenne muscular dystrophy gene carrier PT
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: FR (Completed) BE (Completed) ES (Completed) DE (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2005-003517-33 Sponsor Protocol Number: CRFB002A2302 Start Date*: 2005-11-11
    Sponsor Name:Novartis Pharma Services AG
    Full Title: A randomized, double-masked, active-controlled, multicenter study comparing the efficacy and safety of ranibizumab (0.3 mg and 0.5 mg) administered as two dosing regimens in patients with subfoveal...
    Medical condition: Male and female patients ≥50 years of age with either with predominantly classic, minimally classic, or occult lesions with no classic component, all with primary or recurrent subfoveal CNV seconda...
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: HU (Completed) AT (Completed) DE (Completed) ES (Completed) PT (Completed) GB (Completed) CZ (Completed) IT (Completed) BE (Completed) FI (Completed) SE (Completed) GR (Completed)
    Trial results: View results
    EudraCT Number: 2006-006395-37 Sponsor Protocol Number: BRD/06/055 Start Date*: 2007-09-06
    Sponsor Name:Joint UCLH and UCL Biomedical Research Unit
    Full Title: A randomised, multi-centre feasibility trial in AL Amyloidosis, comparing CTD with SCT in patients with low risk of Treatment Related Mortality and CTD with Mel-Dex in patients in whom SCT would no...
    Medical condition: The medical condition to be investigated is systemic AL Amyloidosis, a disorder of protein folding in which normally soluble proteins are deposited as abnormal, insoluble fibrils that progressively...
    Disease: Version SOC Term Classification Code Term Level
    9.1 10002022 Amyloidosis LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2015-002283-16 Sponsor Protocol Number: 1P50HL112349 Start Date*: 2016-03-08
    Sponsor Name:National Heart, Lung, and Blood Institute / National Institutes of Health
    Full Title: Phase II randomized, placebo-controlled, double blind clinical trial of valsartan for attenuating disease evolution in early sarcomeric HCM
    Medical condition: Hypertrophic cardiomyopathy
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004850 10020204 HOCM Hypertrophic obstructive cardiomyopathy LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DK (Completed)
    Trial results: View results
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