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Clinical trials for Autosomal dominant disorders

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
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    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).

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    32 result(s) found for: Autosomal dominant disorders. Displaying page 1 of 2.
    1  2  Next»
    EudraCT Number: 2011-000138-12 Sponsor Protocol Number: ALADIN2 Start Date*: 2011-04-04
    Sponsor Name:IST. DI RICERCHE FARMACOLOG. M. NEGRI
    Full Title: A prospective, randomized, double-blind, placebo controlled clinical trial to assess the effects of long-acting somatostatin (Octreotide LAR)therapy on disease progression in patients with Autosoma...
    Medical condition: Autosomal Dominant Polycystic Kidney Disease
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10036046 Polycystic kidney, autosomal dominant LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: View results
    EudraCT Number: 2010-023017-65 Sponsor Protocol Number: B1871019 Start Date*: 2011-03-17
    Sponsor Name:Pfizer Inc., 235 East 42nd Street, New York, NY 10017
    Full Title: A PHASE 2, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY OF THE SAFETY, CLINICAL ACTIVITY AND PHARMACOKINETICS OF BOSUTINIB (PF-05208763) VERSUS PLACEBO IN SUBJECTS WITH AUTOSOMAL...
    Medical condition: Autosomal Dominant Polycystic Kidney Disease (ADPKD)
    Disease: Version SOC Term Classification Code Term Level
    14.1 100000004850 10036046 Polycystic kidney, autosomal dominant LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: SE (Completed) ES (Completed) CZ (Completed) HU (Completed) GB (Completed) PL (Completed) SK (Completed) LT (Completed) IT (Completed) BG (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2021-002551-11 Sponsor Protocol Number: HP-CKDPKD Start Date*: 2022-01-18
    Sponsor Name:Aarhus University
    Full Title: Chronic kidney disease – imaging the metabolic derangements with ultra-sensitive MRI
    Medical condition: Chronic kidney disease and polycystic kidney disease
    Disease: Version SOC Term Classification Code Term Level
    23.1 10038359 - Renal and urinary disorders 10064848 Chronic kidney disease PT
    20.0 10010331 - Congenital, familial and genetic disorders 10036046 Polycystic kidney, autosomal dominant LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DK (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2006-002768-24 Sponsor Protocol Number: 156-04-251 Start Date*: 2007-01-26
    Sponsor Name:Otsuka Pharmaceutical Development & Commercialization, Inc.
    Full Title: A Phase 3, Multi-center, Double-blind, Placebo-controlled, Parallel-arm Trial to Determine Long-term Safety and Efficacy of Oral Tolvaptan Tablet Regimens in Adult Subjects with Autosomal Dominant ...
    Medical condition: Autosomal Dominant Polycystic Kidney Disease (ADPKD)
    Disease: Version SOC Term Classification Code Term Level
    8.1 10036046 Polycystic kidney, autosomal dominant LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DK (Completed) BE (Completed) GB (Completed) DE (Completed) FR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2020-005949-16 Sponsor Protocol Number: 202000894 Start Date*: 2021-10-25
    Sponsor Name:University Medical Center Groningen
    Full Title: A GnRH Agonist IN pre-menopausal women STudy to treat severe Polycystic Liver Disease
    Medical condition: Severe polycystic liver disease in female patients
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10048834 Polycystic liver disease PT
    20.0 10010331 - Congenital, familial and genetic disorders 10036045 Polycystic kidney LLT
    20.0 10010331 - Congenital, familial and genetic disorders 10036046 Polycystic kidney, autosomal dominant LLT
    23.0 10010331 - Congenital, familial and genetic disorders 10083939 Autosomal dominant polycystic liver disease LLT
    Population Age: Adults Gender: Female
    Trial protocol: NL (Trial now transitioned) DE (Ongoing)
    Trial results: (No results available)
    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: 2016-000500-29 Sponsor Protocol Number: P150911 Start Date*: 2016-06-03
    Sponsor Name:ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
    Full Title: A randomized Crossover TrIal to Compare recombinant human rhPTH(1-34) to the ASsociation alfacalcidol/hydrochlorothiazide in the treatment of Autosomal Dominant Hypocalcemia
    Medical condition: Autosomal dominant hypocalcemia (ADH)
    Disease: Version SOC Term Classification Code Term Level
    19.0 10014698 - Endocrine disorders 10021041 Hypoparathyroidism PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (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: 2022-003299-17 Sponsor Protocol Number: 22-1005-CALCIFADE Start Date*: 2023-03-20
    Sponsor Name:University Medical Center Utrecht
    Full Title: A randomized, placebo-controlled, double blind trial to study the effects of Etidronate on ectopic CALCIfication in FAhr’s Disease.
    Medical condition: Fahr’s disease or sondrome is a neurodegenerative disease in which all patients present with bilateral vessel associated calcifications in the basal ganglia in the absence of other secondary causes...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10059626 Fahr's disease LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2020-004400-34 Sponsor Protocol Number: LTS15823 Start Date*: 2021-03-23
    Sponsor Name:Sanofi-Aventis Recherche & Développement
    Full Title: Multicenter, open-label, extension study to characterize the long-term efficacy and safety of early versus delayed treatment with venglustat (GZ/SAR402671) in patients at risk of rapidly progressiv...
    Medical condition: Congenital cystic kidney disease
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10010428 Congenital cystic kidney disease PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: FR (Prematurely Ended) NL (Completed) BE (Prematurely Ended) DE (Completed) PT (Prematurely Ended) IT (Prematurely Ended) RO (Completed)
    Trial results: View results
    EudraCT Number: 2005-005552-41 Sponsor Protocol Number: ALADIN Start Date*: 2006-02-20
    Sponsor Name:IST. DI RICERCHE FARMACOLOG. M. NEGRI
    Full Title: Effect of long-acting somatostatin on disease progression in nephropathy due to autosomal dominant polycystic disease a long-term three year follow-up study
    Medical condition: Autosomal Dominant Polycystic Kidney Disease ADPKD
    Disease: Version SOC Term Classification Code Term Level
    6.1 10038359 SOC
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: View results
    EudraCT Number: 2011-005433-39 Sponsor Protocol Number: ADFNLE Start Date*: 2012-03-22
    Sponsor Name:AZIENDA OSPEDALIERO UNIVERSITARIA DI CAGLIARI
    Full Title: PHASE II OPEN STUDY ABOUT THE EFFECTIVENESS OF FENOFIBRATE AS ADD ON THERAPY FOR DRUG RESISTANT NOCTURNAL FRONTAL LOBE EPILEPSY (FNLE) .
    Medical condition: Drug resistant nocturnal frontal lobe epilepsy
    Disease: Version SOC Term Classification Code Term Level
    14.1 10029205 - Nervous system disorders 10049424 Frontal lobe epilepsy PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    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: 2007-006557-25 Sponsor Protocol Number: RAPYD-STUDY Start Date*: 2007-12-20
    Sponsor Name:AZIENDA OSPEDALIERA OSPEDALE POLICLINICO CONSORZIALE
    Full Title: RAPAMYCIN FOR TREATMENT OF AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE
    Medical condition: ADPKD type I
    Disease: Version SOC Term Classification Code Term Level
    9.1 10038359 Renal and urinary disorders SOC
    Population Age: Adults Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2017-004972-74 Sponsor Protocol Number: 20160227 Start Date*: 2020-09-15
    Sponsor Name:Amgen Inc.
    Full Title: An Open-label, Ascending Multiple dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Romosozumab in Children and Adolescents With Osteogenesis Imperfecta
    Medical condition: Osteogenesis imperfecta (OI) is a group of genetic skeletal disorders characterized by increased bone fragility, low bone mass , and increased bone turnover contributing to osteoporosis, fracture...
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10031243 Osteogenesis imperfecta PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: HU (Completed) DE (Completed) GR (Completed) IT (Completed) AT (Completed)
    Trial results: View results
    EudraCT Number: 2013-004291-35 Sponsor Protocol Number: CACZ885N2301 Start Date*: 2014-05-16
    Sponsor Name:Novartis Farma SpA
    Full Title: A randomized, double-blind, placebo controlled study of canakinumab in patients with Hereditary Periodic Fevers (TRAPS, HIDS, or crFMF), with subsequent randomized withdrawal/ dosing frequency redu...
    Medical condition: Hereditary Periodic Fevers (TRAPS, HIDS, or crFMF)
    Disease: Version SOC Term Classification Code Term Level
    16.1 10010331 - Congenital, familial and genetic disorders 10067783 Tumor necrosis factor receptor-associated periodic syndrome LLT
    16.1 10010331 - Congenital, familial and genetic disorders 10016207 Familial mediterranean fever PT
    16.1 10010331 - Congenital, familial and genetic disorders 10072010 Hyper IgD syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Completed) ES (Completed) IE (Completed) DE (Completed) BE (Completed) HU (Completed) NL (Completed) GR (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: 2021-000621-27 Sponsor Protocol Number: RTRX-RE021-201 Start Date*: 2022-10-20
    Sponsor Name:Travere Therapeutics, Inc.
    Full Title: A Phase 2, Open-Label, Single-Arm, Cohort Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Sparsentan Treatment in Pediatric Subjects with Selected Proteinuric Glomerular Diseases (E...
    Medical condition: Proteinuric glomerular diseases including: •Focal segmental glomerulosclerosis (FSGS) •Minimal change disease (MCD) •Immunoglobulin A nephropathy (IgAN) •Immunoglobulin A vasculitis (IgAV) •Al...
    Disease: Version SOC Term Classification Code Term Level
    21.1 10038359 - Renal and urinary disorders 10067757 Focal segmental glomerulosclerosis PT
    20.0 10038359 - Renal and urinary disorders 10021263 IgA nephropathy PT
    22.1 100000004858 10082959 IgA vasculitis LLT
    20.0 10010331 - Congenital, familial and genetic disorders 10001843 Alport's syndrome PT
    21.1 10038359 - Renal and urinary disorders 10058326 Minimal change disease LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed) ES (Temporarily Halted) SE (Trial now transitioned) PL (Temporarily Halted) DE (Restarted) IT (Trial now transitioned) NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2014-000184-40 Sponsor Protocol Number: 20130173 Start Date*: 2014-09-11
    Sponsor Name:Amgen, Inc.
    Full Title: Prospective, Multicenter, Single-arm Study to Evaluate Efficacy, Safety, and Pharmacokinetics of Denosumab in Children With Osteogenesis Imperfecta
    Medical condition: Osteogenesis Imperfecta
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10031243 Osteogenesis imperfecta PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: CZ (Completed) DE (Completed) GB (GB - no longer in EU/EEA) HU (Completed) ES (Completed) Outside EU/EEA BG (Completed) PL (Prematurely Ended) BE (Completed) FR (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2013-000107-17 Sponsor Protocol Number: EAT2TREAT Start Date*: 2013-05-22
    Sponsor Name:Hospital of the University of Munich, Grosshadern
    Full Title: Pharmacological therapy of episodic ataxia type 2: a placebo-controlled comparison of the efficacy of 4-aminopyridine sustained-release (Fampyra TM) and acetazolamide (Acemit TM)
    Medical condition: Familial episodic ataxia (EA) represents a genetically and phenotypically diverse group of rare autosomal dominant hereditary disorders characterized by episodes of imbalance and incoordination tha...
    Disease: Version SOC Term Classification Code Term Level
    18.1 100000004850 10067447 Episodic ataxia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
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