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Clinical trials for Hereditary Disease Foundation

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   44335   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 ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
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    42 result(s) found for: Hereditary Disease Foundation. Displaying page 1 of 3.
    1  2  3  Next»
    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: 2015-002762-23 Sponsor Protocol Number: 408-C-1402 Start Date*: 2016-01-25
    Sponsor Name:Reata Pharmaceuticals, Inc.
    Full Title: A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich’s Ataxia
    Medical condition: Friedreich's Ataxia
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10017374 Friedreich's ataxia PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) AT (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2009-018119-14 Sponsor Protocol Number: A091758 Start Date*: 2010-07-23
    Sponsor Name:Cambridge University Hospitals NHS Foundation Trust and University of Cambridge
    Full Title: Huntington's Disease Rilmenidine Safety Trial
    Medical condition: Huntington's Disease
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10070668 Huntington's disease PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2020-004995-17 Sponsor Protocol Number: NL7507301820 Start Date*: 2021-03-11
    Sponsor Name:AMsterdam UMC-AMC
    Full Title: Low dose iron chelation as TReatment of Oxidative stress in Sickle cell disease; TROS study
    Medical condition: Sickle cell disease (an hereditary hemoglobinopathy)
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (Completed)
    Trial results: (No results available)
    EudraCT Number: 2014-004900-31 Sponsor Protocol Number: A8241022 Start Date*: 2015-05-27
    Sponsor Name:Pfizer Inc, 235 East 42nd Street, New York, NY 10017
    Full Title: AN OPEN LABEL EXTENSION STUDY TO INVESTIGATE THE LONG TERM SAFETY, TOLERABILITY AND EFFICACY OF PF-02545920 IN SUBJECTS WITH HUNTINGTON’S DISEASE WHO PREVIOUSLY COMPLETED STUDY A8241021
    Medical condition: HUNTINGTON'S DISEASE
    Disease: Version SOC Term Classification Code Term Level
    17.1 10010331 - Congenital, familial and genetic disorders 10070668 Huntington's disease PT
    17.1 10010331 - Congenital, familial and genetic disorders 10020469 Huntington's chorea LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2014-001291-56 Sponsor Protocol Number: A8241021 Start Date*: 2014-10-27
    Sponsor Name:Pfizer Inc, 235 East 42nd Street, New York, NY 10017
    Full Title: A PHASE 2, RANDOMIZED, PLACEBO CONTROLLED, DOUBLE BLIND PROOF-OF-CONCEPT STUDY OF THE EFFICACY AND SAFETY OF PF-02545920 IN SUBJECTS WITH HUNTINGTON’S DISEASE
    Medical condition: HUNTINGTON’S DISEASE
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10070668 Huntington's disease PT
    19.0 10010331 - Congenital, familial and genetic disorders 10020469 Huntington's chorea LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) GB (Completed)
    Trial results: View results
    EudraCT Number: 2022-000596-40 Sponsor Protocol Number: 112930 Start Date*: 2023-01-10
    Sponsor Name:Radboudumc
    Full Title: Effect of dapaglifozin on serum magnesium in HNF1β patients with renal hypomagnesemia
    Medical condition: Renal hypomagnesemia in ADTKD-HNF1β patients
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2016-004291-21 Sponsor Protocol Number: 33IC30_166855 Start Date*: 2018-01-15
    Sponsor Name:Insel Gruppe AG - Inselspital Universitätsklinik für Kardiologie
    Full Title: Effect of phosphodiesterase-5 inhibition with Tadalafil on SystEmic Right VEntricular size and function – a multi-center, double-blind, randomized, placebo-controlled clinical trial – SERVE Trial
    Medical condition: Adults with congenital heart disease (CHD) and a right ventricle (RV) in subaortic (systemic) position.
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: AT (Completed)
    Trial results: View results
    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: 2013-003532-68 Sponsor Protocol Number: FIBCON Start Date*: 2014-03-11
    Sponsor Name:Guy's and St Thomas NHS Foundation Trust
    Full Title: Fibrinogen concentrate supplementation in the management of bleeding during paediatric cardiopulmonary bypass: a phase 1B/2A, open label dose escalation study (Version 1.0, Jan 28, 2014)
    Medical condition: Neonates and infants who are at risk of mediastinal bleeding following cardiopulmonary bypass surgery for congenital heart disease
    Disease: Version SOC Term Classification Code Term Level
    16.1 100000004849 10055817 Haemorrhage intrapericardial LLT
    16.1 100000004850 10010495 Congenital heart disease NOS LLT
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2011-001319-30 Sponsor Protocol Number: CH/2011/3670 Start Date*: 2012-04-04
    Sponsor Name:University Hospitals Bristol NHS Foundation Trust
    Full Title: A randomized, double-blind, parallel group, placebo-controlled trial of metformin in tuberous sclerosis complex.
    Medical condition: Tuberous Sclerosis Complex
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10045138 Tuberous sclerosis PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2017-002124-24 Sponsor Protocol Number: ROPROP Start Date*: 2017-12-22
    Sponsor Name:Universität Zürich
    Full Title: Oral Propranolol for prevention of threshold retinopathy of prematurity
    Medical condition: Retinopathy of prematurity
    Disease:
    Population Age: Preterm newborn infants, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: DE (Trial now transitioned)
    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: 2013-001479-18 Sponsor Protocol Number: GENiSIS2013 Start Date*: 2014-06-09
    Sponsor Name:Manchester University NHS Foundation Trust
    Full Title: A Phase III, Double Blinded, Randomised, Placebo Controlled Clinical Trial of High Dose Oral Genistein Aglycone in Patients with Sanfilippo Syndrome (Mucopolysaccharidosis III)
    Medical condition: Sanfilippo syndrome (Mucopolysaccharidosis III)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10056918 Sanfilippo's syndrome LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2013-001998-24 Sponsor Protocol Number: ME3827/1-1 Start Date*: 2014-10-09
    Sponsor Name:Saarland University
    Full Title: A prospective, multicenter, double blind, placebo-controlled, two-arm parallel group phase 3 trial to evaluate the effect of early postnatal additional high dose oral vitamin A supplementation of 5...
    Medical condition: Preventing bronchopulmonary dysplasia (BPD) or death in extremely-low-birth-weight (ELBW) infants.
    Disease:
    Population Age: Preterm newborn infants, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) AT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2012-000387-19 Sponsor Protocol Number: VX11-770-110 Start Date*: 2012-06-11
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis who Have the R117H-CFTR Mutation
    Medical condition: cystic fibrosis
    Disease: Version SOC Term Classification Code Term Level
    16.0 10010331 - Congenital, familial and genetic disorders 10011762 Cystic fibrosis PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2009-010261-23 Sponsor Protocol Number: VX08-770-104 Start Date*: 2011-06-06
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Safety and Efficacy of VX-770 in Subjects Aged 12 Years and Older With Cystic Fibrosis who are Homozygo...
    Medical condition: Cystic Fibrosis
    Disease: Version SOC Term Classification Code Term Level
    13.1 10010331 - Congenital, familial and genetic disorders 10011762 Cystic fibrosis PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2012-000388-26 Sponsor Protocol Number: VX12-770-111 Start Date*: 2012-07-23
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Phase 3, Two-Part, Randomized, Double-Blind, Placebo-Controlled, Crossover Study With an Open-Label Period to Evaluate the Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Who Ha...
    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, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: BE (Completed)
    Trial results: View results
    EudraCT Number: 2016-002578-11 Sponsor Protocol Number: 917741 Start Date*: 2016-09-16
    Sponsor Name:Alder Hey Children's NHS Foundation Trust
    Full Title: The cystic fibrosis (CF) anti-staphylococcal antibiotic prophylaxis trial (CF START); a randomised registry trial to assess the safety and efficacy of flucloxacillin as a longterm prophylaxis agent...
    Medical condition: Cystic fibrosis
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10011762 Cystic fibrosis PT
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
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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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