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Clinical trials for Genetic Disease

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   44346   clinical trials with a EudraCT protocol, of which   7374   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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    2,640 result(s) found for: Genetic Disease. Displaying page 63 of 132.
    «« First « Previous 59  60  61  62  63  64  65  66  67  Next» Last»»
    EudraCT Number: 2016-004366-25 Sponsor Protocol Number: MO39129 Start Date*: 2017-10-09
    Sponsor Name:F. Hoffmann-La Roche Ltd
    Full Title: A SINGLE-ARM, MULTICENTER PHASE IIIB CLINICAL TRIAL TO EVALUATE THE SAFETY AND TOLERABILITY OF PROPHYLACTIC EMICIZUMAB IN HEMOPHILIA A PATIENTS WITH INHIBITORS
    Medical condition: Hemophilia A
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10060612 Hemophilia A LLT
    20.0 100000004850 10053751 Hemophilia A with anti factor VIII LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: PT (Completed) GB (GB - no longer in EU/EEA) DE (Completed) DK (Prematurely Ended) HU (Completed) ES (Completed) PL (Completed) FI (Completed) BE (Completed) SE (Completed) NL (Completed) IT (Completed) RO (Completed)
    Trial results: View results
    EudraCT Number: 2020-000138-16 Sponsor Protocol Number: D601HC00002 Start Date*: 2020-11-23
    Sponsor Name:AstraZeneca AB
    Full Title: A Phase 2b, Open-label, Single-arm, Multi-centre Study Assessing the Efficacy and Safety of Adavosertib as Treatment for Recurrent or Persistent Uterine Serous Carcinoma (ADAGIO)
    Medical condition: Recurrent or persistent Uterine Serous Carcinoma (USC) in patients who have previously received at least 1 prior platinum-based chemotherapy regimen for the treatment of USC
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10029104 Neoplasms benign, malignant and unspecified (incl cysts and polyps) SOC
    20.0 10038604 - Reproductive system and breast disorders 10038604 Reproductive system and breast disorders SOC
    Population Age: Adults, Elderly Gender: Female
    Trial protocol: FR (Completed) DE (Completed) IT (Completed) ES (Temporarily Halted)
    Trial results: View results
    EudraCT Number: 2012-000204-15 Sponsor Protocol Number: VX11-770-108 Start Date*: 2013-06-28
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Phase 3, 2 Part, Open-Label Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who are 2 Through 5 Years of Age and Have a CFTR Gat...
    Medical condition: cystic fibrosis
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10011762 Cystic fibrosis PT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2012-000386-20 Sponsor Protocol Number: VX11-770-109 Start Date*: 2013-07-31
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Phase 3, 2-Arm, Roll-Over Study to Evaluate the Long-term Safety and Pharmacodynamics of Ivacaftor Treatment in Pediatric Subjects With Cystic Fibrosis and a CFTR Gating Mutation
    Medical condition: cystic fibrosis
    Disease: Version SOC Term Classification Code Term Level
    18.0 10010331 - Congenital, familial and genetic disorders 10011762 Cystic fibrosis PT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2011-001034-42 Sponsor Protocol Number: SNT-II-003-OFU Start Date*: 2011-08-25
    Sponsor Name:Santhera Pharmaceuticals (Switzerland) Limited
    Full Title: A single visit, observational, follow-up study of Patients with Leber’s Hereditary Optic Neuropathy following participation in SNT-II-003 trial
    Medical condition: Leber’s Hereditary Optic Neuropathy
    Disease: Version SOC Term Classification Code Term Level
    13.1 10010331 - Congenital, familial and genetic disorders 10062951 Leber's hereditary optic atrophy neuropathy LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2015-003135-35 Sponsor Protocol Number: ECU-aHUS-403 Start Date*: 2016-06-20
    Sponsor Name:Alexion Pharmaceuticals, Inc.
    Full Title: EVALUATION OF POTENTIAL PREDICTORS OF DISEASE PROGRESSION IN PATIENTS WITH aHUS, INCLUDING GENETICS, BIOMARKERS, AND TREATMENT
    Medical condition: atypical Hemolytic-Uremic Syndrome (aHUS)
    Disease: Version SOC Term Classification Code Term Level
    19.1 100000004851 10019515 Hemolytic uremic syndrome LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) BE (Completed) GB (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2016-000066-34 Sponsor Protocol Number: VX15-809-112 Start Date*: 2016-06-24
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Phase 4, Randomized, Double-Blind, Placebo-Controlled, Parallel-Design Study of the Effect of Lumacaftor/Ivacaftor Combination Therapy on Exercise Tolerance in Subjects Aged 12 Years and Older Wi...
    Medical condition: Cystic fibrosis in patients homozygous for the F508del-CFTR Mutation
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10011762 Cystic fibrosis PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2008-002322-11 Sponsor Protocol Number: ARTemis Start Date*: 2009-04-29
    Sponsor Name:Cambridge University Hospitals NHS Foundation Trust [...]
    1. Cambridge University Hospitals NHS Foundation Trust
    2. University of Cambridge
    Full Title: ARTemis : Avastin® Randomised Trial with nEo-adjuvant cheMotherapy for patients wIth early breaSt cancer
    Medical condition: HER2 negative invasive breast cancer
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2015-001297-18 Sponsor Protocol Number: ALN-AAT-001 Start Date*: 2015-06-29
    Sponsor Name:Alnylam Pharmaceuticals, Inc.
    Full Title: A Phase 1/2, Randomized, Single-Blind, Placebo-Controlled, Single-Ascending, and Multiple-Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN...
    Medical condition: ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease
    Disease: Version SOC Term Classification Code Term Level
    19.1 10010331 - Congenital, familial and genetic disorders 10001806 Alpha-1 anti-trypsin deficiency PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2016-001585-29 Sponsor Protocol Number: VX15-809-111 Start Date*: 2016-12-21
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy of Lumacaftor/Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis Who Have an A455E-CFTR...
    Medical condition: Cystic fibrosis
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10011762 Cystic fibrosis PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: NL (Completed)
    Trial results: View results
    EudraCT Number: 2015-001211-13 Sponsor Protocol Number: PRODROMALAD Start Date*: 2015-11-03
    Sponsor Name:OSPEDALE SAN RAFFAELE
    Full Title: IMAGING OF NEUROINFLAMMATION AND NEURODEGENERATION IN ALZHEIMER’S DISEASE
    Medical condition: mild cognitive impairment and familiar Alzheimer disease
    Disease: Version SOC Term Classification Code Term Level
    22.1 100000004848 10050727 RI scan LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: View results
    EudraCT Number: 2019-001899-11 Sponsor Protocol Number: C1701-202 Start Date*: 2019-11-07
    Sponsor Name:Cyclerion Therapeutics, Inc.
    Full Title: A Randomized, Placebo-controlled, Phase 2 Study to Evaluate the Safety and Pharmacodynamics of Once-daily Oral IW-1701 in Patients with Stable Sickle Cell Disease
    Medical condition: Stable Sickle Cell Disease (SCD)
    Disease: Version SOC Term Classification Code Term Level
    21.0 10010331 - Congenital, familial and genetic disorders 10040644 Sickle cell disease PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2021-005402-10 Sponsor Protocol Number: EFC17215 Start Date*: 2022-03-23
    Sponsor Name:Sanofi-aventis recherche & développement
    Full Title: A phase 3, multicenter, multinational, randomized, double-blind, double-dummy, active-comparator study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher...
    Medical condition: Gaucher's disease type III
    Disease: Version SOC Term Classification Code Term Level
    24.1 10010331 - Congenital, familial and genetic disorders 10075699 Gaucher's disease type III PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) FR (Trial now transitioned) HU (Completed) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2008-002119-42 Sponsor Protocol Number: 0822-029 Start Date*: 2008-09-23
    Sponsor Name:Merck& Co., Inc.
    Full Title: A Phase III Study to Assess the Tolerability and Efficacy of MK-0822 (Odanacatib) in Reducing the Risk of Bone Metastases and Prolonging Disease-Free Survival in Women with Breast Cancer Spanish t...
    Medical condition: Prevention of disease recurrence in patients with breast cancer. Spanish indication: Prevención de recurrencia de la enfermedad en pacientes con cáncer de mama
    Disease: Version SOC Term Classification Code Term Level
    9.1 10005993 Bone metastases LLT
    Population Age: Adults, Elderly Gender: Female
    Trial protocol: ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2016-001643-39 Sponsor Protocol Number: 2215-CL-0302 Start Date*: 2017-02-09
    Sponsor Name:Astellas Pharma Global Development, Inc. (APGD)
    Full Title: A Phase 2 Multicenter, Randomized, Double-Blind, Placebo- Controlled Trial of the FLT3 Inhibitor Gilteritinib (ASP2215) Administered as Maintenance Therapy Following Induction/Consolidation Therapy...
    Medical condition: Subjects diagnosed with FLT3/ITD acute myeloid leukemia (AML) in CR1, including CRp and CRi, for whom a decision not to proceed with transplantation has been made, or a suitable donor could not be ...
    Disease: Version SOC Term Classification Code Term Level
    21.0 100000004864 10000886 Acute myeloid leukemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: HU (Completed) ES (Completed) CZ (Completed) PL (Completed) PT (Completed) SE (Completed) GR (Completed) DK (Completed) HR (Completed) FR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2004-001162-40 Sponsor Protocol Number: E2020-A001-233 Start Date*: 2005-03-08
    Sponsor Name:Eisai Ltd
    Full Title: An 18-week, multi-center, randomized, double-blind, placebo-controlled, parallel-group study of the efficacy, safety, and tolerability of donepezil HCl (E2020) in patients with CADASIL who have cog...
    Medical condition: Cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy (CADASIL). CADASIL is a genetic disorder representing a minority of the patients who develop vascular de...
    Disease: Version SOC Term Classification Code Term Level
    7.0 10057678 PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FI (Completed) DE (Completed) SE (Completed) GB (Completed) IT (Completed) ES (Completed)
    Trial results: View results
    EudraCT Number: 2011-003825-81 Sponsor Protocol Number: HGT-FIR-086 Start Date*: 2012-02-28
    Sponsor Name:Shire Orphan Therapies, Inc
    Full Title: A Multicenter, Open-Label, Non-Randomized Study to Assess the Pharmacokinetics, Tolerability, and Safety of a Single Subcutaneous Administration of Icatibant in Children and Adolescents with Heredi...
    Medical condition: Hereditary Angioedema
    Disease: Version SOC Term Classification Code Term Level
    16.0 10010331 - Congenital, familial and genetic disorders 10019860 Hereditary angioedema PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) HU (Prematurely Ended) ES (Prematurely Ended) IT (Completed) AT (Completed)
    Trial results: View results
    EudraCT Number: 2007-003702-95 Sponsor Protocol Number: EPICURE SUB05T02 Start Date*: 2007-07-03
    Sponsor Name:ISTITUTO C. MONDINO
    Full Title: A MULTICENTRE RANDOMIZED CONTROLLED TRIAL COMPARING TOPIRAMATE, STIRIPENTOL AND CLOBAZAM AS ADJUNCTIVE THERAPY TO VALPROATE AND CLOBAZAM IN PAEDIATRIC PATIENTS WITH DRAVET'S SYNDROME (SMEI) NOT ADE...
    Medical condition: Dravet Syndrome
    Disease: Version SOC Term Classification Code Term Level
    10054859 PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2009-017852-28 Sponsor Protocol Number: H8Y-MC-HBBM(a) Start Date*: 2010-05-04
    Sponsor Name:Eli Lilly and Company
    Full Title: A Phase 2, Multicenter, Double-Blind, Placebo-Controlled Comparator Study of 2 Doses of LY2140023 versus Placebo in Patients with DSM-IVTR Schizophrenia
    Medical condition: Schizophrenia
    Disease: Version SOC Term Classification Code Term Level
    12.1 10039626 Schizophrenia LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2009-010276-16 Sponsor Protocol Number: F1D-MC-HGMX Start Date*: 2009-11-19
    Sponsor Name:Eli Lilly
    Full Title: A Long-Term, Open-Label, Safety Study of Oral Olanzapine in Adolescents with Bipolar I Disorder (Manic or Mixed Episodes) or Schizophrenia
    Medical condition: Bipolar I Disorder (Manic or Mixed Episodes) or Schizophrenia
    Disease: Version SOC Term Classification Code Term Level
    13.1 10037175 - Psychiatric disorders 10039628 Schizophrenia and other psychotic disorders HLGT
    13.1 10037175 - Psychiatric disorders 10057667 Bipolar disorder PT
    Population Age: Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
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