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Clinical trials for Dominant gene

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   44334   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 ).  
     
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    29 result(s) found for: Dominant gene. Displaying page 1 of 2.
    1  2  Next»
    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: 2019-001493-28 Sponsor Protocol Number: OPA1 Start Date*: 2020-09-24
    Sponsor Name:Medizinische Universität Graz, Univ.-Augenklinik
    Full Title: Raxone® treatment for patients with dominant optic atrophy due to OPA1 gene mutation
    Medical condition: Autosomal dominant optic atrophy (ADOA)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10019895 Hereditary optic atrophy PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: AT (Completed)
    Trial results: View results
    EudraCT Number: 2019-002827-14 Sponsor Protocol Number: TreatSPG11 Start Date*: 2021-01-19
    Sponsor Name:IRCCS Fondazione Stella Maris
    Full Title: Phase 2 pharmacological experimental study to test the safety of miglustat in subjects with hereditary spastic paraplegia type 11
    Medical condition: Spastic paraparesis type 11 (SPG11), caused by mutations in spatacsin protein, is characterized by the association of peripheral neuropathy, parkinsonism, ataxia, cognitive impairment, hypotrophy o...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10019903 Hereditary spastic paraplegia PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2014-001315-40 Sponsor Protocol Number: RF-2010-2319722 Start Date*: 2014-12-03
    Sponsor Name:IRCCS Istituto Centro San Giovanni di Dio Fatebenefratelli
    Full Title: Amyloid Imaging in the “Italian Network for autosomal dominant Alzheimer’s disease and frontotemporal lobar degeneration (ItalianDIAfN)”
    Medical condition: Autosomal Dominant Alzheimer's Disease
    Disease: Version SOC Term Classification Code Term Level
    17.0 100000004852 10001896 Alzheimer's disease LLT
    Population Age: Adults, Elderly 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: 2011-004308-39 Sponsor Protocol Number: R727-CL-1018 Start Date*: 2012-02-29
    Sponsor Name:Regeneron Pharmaceuticals, Inc.
    Full Title: A Phase 2 Pilot Study with a Randomized Double-Blind Treatment Phase to Evaluate the Pharmacodynamics and Safety of REGN727 in Patients with Autosomal Dominant Hypercholesterolemia and Gain-of-Func...
    Medical condition: Hypercholesterolemia
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2007-004913-32 Sponsor Protocol Number: DG-051-CV-202 Start Date*: 2008-04-15
    Sponsor Name:deCODE genetics ehf.
    Full Title: A randomized, double-blind, placebo-controlled evaluation of the pharmacodynamics, pharmacokinetics, and safety of DG-051 in patients with a history of MI
    Medical condition: Cardiovascular diseases
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IS (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2017-000204-15 Sponsor Protocol Number: 060286 Start Date*: 2017-08-08
    Sponsor Name:Professor, DMSc Tina Vilsbøll
    Full Title: Glimepiride monotherapy vs. combination of glimepiride and linagliptin therapy in patients with HNF1A-diabetes
    Medical condition: "Hepatocyte nuclear factor 1-alfa diabetes", also called "Maturity onset diabetes of the young type 3"'. It is a monogenic form of inherited diabetes.
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004861 10026948 Maturity-onset diabetes of the young LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DK (Completed)
    Trial results: View results
    EudraCT Number: 2022-003853-70 Sponsor Protocol Number: PNRR-MR1-2022-12375914 Start Date*: 2023-05-02
    Sponsor Name:OSPEDALE SAN RAFFAELE
    Full Title: Towards a personalized precision medicine in rare disease: tirzepatide (a dual glucose­ dependent insulinotropic polypeptide and glucagon-like peptide-I receptor agonist) monotherapy in patients wi...
    Medical condition: Wolfram syndrome type 1
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10078338 Wolfram syndrome PT
    20.0 10010331 - Congenital, familial and genetic disorders 10078338 Wolfram syndrome PT
    20.0 10010331 - Congenital, familial and genetic disorders 10078338 Wolfram syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2007-003331-23 Sponsor Protocol Number: LA29-0207 Start Date*: 2008-11-20
    Sponsor Name:ApoPharma Inc.
    Full Title: A six-month double-blind, randomized, placebo-controlled study investigating the safety and tolerability of deferiprone in participants with Friedreich's ataxia
    Medical condition: Friedreich's Ataxia
    Disease: Version SOC Term Classification Code Term Level
    9.1 10017374 Friedreich's ataxia LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed) BE (Completed) FR (Completed) IT (Completed) ES (Completed)
    Trial results: (No results available)
    EudraCT Number: 2015-002378-19 Sponsor Protocol Number: CLN-PXT3003-02 Start Date*: 2015-08-27
    Sponsor Name:PHARNEXT
    Full Title: International, multi-center, randomized, double-blind, placebo-controlled phase III study assessing in parallel groups the efficacy and safety of 2 doses of PXT3003 in patients with Charcot-Marie-T...
    Medical condition: Charcot-Marie-Tooth Disease - Type 1A
    Disease: Version SOC Term Classification Code Term Level
    18.0 100000004850 10008414 Charcot-Marie-Tooth disease LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Completed) DE (Suspended by CA) ES (Completed) BE (Completed) GB (Completed) NL (Completed)
    Trial results: View results
    EudraCT Number: 2012-000592-17 Sponsor Protocol Number: MODY-TREAT Start Date*: 2012-08-03
    Sponsor Name:Signe H. Østoft, Diabetes Research Division
    Full Title: The effects of GLP-1 in Maturity- onset diabetes of the young (MODY)
    Medical condition: 'Maturity onset diabetes of the young', also called 'Hepatocyte nuclear factor 1-alfa daiabetes'. It is a monogenic form of inherited diabetes.
    Disease: Version SOC Term Classification Code Term Level
    14.1 10027433 - Metabolism and nutrition disorders 10026948 Maturity-onset diabetes of the young LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DK (Completed)
    Trial results: View results
    EudraCT Number: 2014-002113-39 Sponsor Protocol Number: BAY88-8223/16298 Start Date*: 2014-10-29
    Sponsor Name:Bayer AG
    Full Title: A phase II randomized, double-blind, placebo-controlled trial of radium-223 dichloride versus placebo when administered to metastatic HER2 negative hormone receptor positive breast cancer subjects ...
    Medical condition: HER2 negative hormone receptor positive metastatic breast cancer with bone metastases treated with standard of care hormonal treatment.
    Disease: Version SOC Term Classification Code Term Level
    20.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10055113 Breast cancer metastatic PT
    Population Age: Adults, Elderly Gender: Female
    Trial protocol: GB (Completed) ES (Completed) AT (Completed) NO (Completed) CZ (Completed) DK (Prematurely Ended) NL (Completed) SE (Completed) PL (Completed) FI (Completed) IE (Completed) FR (Completed) DE (Completed)
    Trial results: View results
    EudraCT Number: 2014-005432-33 Sponsor Protocol Number: UX001-CL301 Start Date*: 2016-06-07
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended-Release Tablets in Patients with GNE Myopathy (GNEM) or Hereditary Inclusion...
    Medical condition: GNE Myopathy, also known as Hereditary Inclusion Body Myopathy (HIBM), Distal Myopathy with Rimmed Vacuoles (DMRV), Nonaka’s disease, or quadriceps sparing myopathy (QSM)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10075048 Hereditary inclusion body myopathy LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: BG (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2009-014728-44 Sponsor Protocol Number: KKSH-071 Start Date*: 2010-01-13
    Sponsor Name:Martin Luther Universität Halle-Wittenberg
    Full Title: A randomized phase II trial of irinotecan drug-eluting beads administered by hepatic chemoembolization with intravenous cetuximab (DEBIRITUX) versus systemic treatment with intravenous cetuximab an...
    Medical condition: Patients with unresectable liver metastases of colorectal cancer who previously failed pretreatment with 5FU/oxaliplatin and/or 5FU/irinotecan (2nd- or 3rd-line).
    Disease: Version SOC Term Classification Code Term Level
    14.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10052362 Metastatic colorectal cancer LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Temporarily Halted)
    Trial results: View results
    EudraCT Number: 2019-002076-13 Sponsor Protocol Number: NS-065/NCNP-01-301 Start Date*: 2020-03-02
    Sponsor Name:NS Pharma, Inc.
    Full Title: A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)
    Medical condition: Duchenne Muscular Dystrophy (DMD)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) SE (Completed) ES (Ongoing) NL (Ongoing) GR (Completed) IT (Completed) NO (Completed)
    Trial results: View results
    EudraCT Number: 2019-001833-14 Sponsor Protocol Number: EAURF2018-02 Start Date*: 2020-12-04
    Sponsor Name:EAU Research Foundation
    Full Title: Open-label, single-arm, Phase II study, evaluating safety and efficacy of INCB054828 (Pemigatinib) as adjuvant therapy for molecularly-selected, high-risk patients with urothelial carcinoma who hav...
    Medical condition: pT3-4 and/or pN1-3 urothelial carcinoma (UC) of the urinary bladder or upper urinary tract after radical cystectomy /radical nephroureterectomy previously treated with at least three cycles of ne...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004864 10046714 Urothelial carcinoma bladder LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2005-002942-20 Sponsor Protocol Number: EFC5826 Start Date*: 2005-12-29
    Sponsor Name:sanofi-aventis recherche et developpement
    Full Title: Randomized, multinational, multicenter, double-blind, placebo-controlled, two-arm parallel group trial of rimonabant 20 mg OD for reducing the risk of major cardiovascular events in abdominally obe...
    Medical condition: Patients with abdominal obesity at increased risk for cardiovascular events (myocardial infarctio, stroke and cardiovascular death)
    Disease: Version SOC Term Classification Code Term Level
    10.1 10051615 LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Prohibited by CA) GB (Prematurely Ended) ES (Completed) DE (Completed) AT (Prematurely Ended) FI (Completed) BE (Completed) DK (Suspended by CA) HU (Completed) CZ (Suspended by CA) IE (Prematurely Ended) PT (Completed) GR (Completed) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2021-003417-19 Sponsor Protocol Number: BN42644 Start Date*: 2022-01-03
    Sponsor Name:F. HOFFMANN - LA ROCHE LTD.
    Full Title: A TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7204239 IN COMBI...
    Medical condition: Spinal Muscular Atrophy (SMA)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10079415 Spinal muscular atrophy type III LLT
    20.1 100000004850 10051203 Spinal muscular atrophy congenital LLT
    20.1 100000004850 10041583 Spinal muscular atrophy, unspecified LLT
    20.0 100000004850 10079413 Spinal muscular atrophy type I LLT
    20.0 100000004850 10079416 Spinal muscular atrophy type II LLT
    20.0 100000004850 10079417 Spinal muscular atrophy infantile onset LLT
    20.0 100000004850 10079419 Spinal muscular atrophy pre-symptomatic LLT
    20.0 100000004850 10079418 Spinal muscular atrophy later onset LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: IT (Trial now transitioned) PL (Trial now transitioned) DE (Trial now transitioned) BE (Trial now transitioned) NL (Trial now transitioned) ES (Ongoing) FR (Trial now transitioned) PT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2010-020061-24 Sponsor Protocol Number: CACZ885D2203 Start Date*: 2010-08-24
    Sponsor Name:Novartis Pharma Services AG
    Full Title: An open-label, multicenter, efficacy and safety study of 4-month canakinumab treatment with 5-month follow-up and long-term treatment period in patients with active recurrent or chronic TNF-recepto...
    Medical condition: TNF-receptor associated periodic syndrome (TRAPS)
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10067783 Tumor necrosis factor receptor-associated periodic syndrome LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IE (Completed) GB (Completed) IT (Completed)
    Trial results: View results
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    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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