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Clinical trials for Inheritance

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   44343   clinical trials with a EudraCT protocol, of which   7372   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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    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
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    17 result(s) found for: Inheritance. Displaying page 1 of 1.
    EudraCT Number: 2015-003184-11 Sponsor Protocol Number: R0002820 Start Date*: 2016-01-04
    Sponsor Name:Radboud University Medical Centre
    Full Title: Functional effects of botulinum toxin in the hip adductors and subsequent exercise in patients with hereditary spastic paraplegia: a pilot RCT
    Medical condition: Hereditary Spastic Paraplegia
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (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: 2010-023184-18 Sponsor Protocol Number: C10-44 Start Date*: 2012-01-25
    Sponsor Name:Inserm
    Full Title: PRE clinical mutation CARriers from families with DIlated cardiomyopathy and ACE inhibitors (PRECARDIA-INHERITANCE study) Preventive effect of ACE inhibitor (perindopril) on the onset or progres...
    Medical condition: dilated cardiomyopathy
    Disease: Version SOC Term Classification Code Term Level
    14.0 10007541 - Cardiac disorders 10056419 Dilated cardiomyopathy LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DK (Prematurely Ended) ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2006-002099-16 Sponsor Protocol Number: 06/Q0703/56 Start Date*: 2006-07-13
    Sponsor Name:King's College London
    Full Title: The effect of milk thistle extract (Legalon) versus tea on serum iron increase after a meal containing non-haem iron in hereditary haemochromatosis
    Medical condition: Type 1 hereditary haemochromatosis is associated with homozygous inheritance of the C282Y mutation in the HFE gene. The phenotype is characterised by increased serum iron levels, tissue iron accumu...
    Disease: Version SOC Term Classification Code Term Level
    8.1 10057873 Hereditary haemochromatosis LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2018-001762-42 Sponsor Protocol Number: 4658-402 Start Date*: 2020-04-03
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular ...
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: IE (Completed) GB (GB - no longer in EU/EEA) FR (Trial now transitioned) ES (Ongoing) SE (Prematurely Ended) DE (Trial now transitioned) DK (Trial now transitioned) NL (Trial now transitioned) IT (Trial now transitioned) PL (Trial now transitioned) GR (Trial now transitioned) NO (Trial now transitioned) HU (Trial now transitioned) CZ (Trial now transitioned) SI (Trial now transitioned) RO (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2005-001651-37 Sponsor Protocol Number: OGT 918-011 Start Date*: 2005-10-27
    Sponsor Name:Actelion Pharmaceuticals Ltd
    Full Title: A phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement ...
    Medical condition: Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 16 of the protocol: 1 BA...
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) ES (Completed) IT (Completed) DE (Completed) CZ (Completed) HU (Completed)
    Trial results: View results
    EudraCT Number: 2018-001983-49 Sponsor Protocol Number: UX023-CL205 Start Date*: 2019-10-10
    Sponsor Name:Ultragenyx Pharmaceutical Inc
    Full Title: An Open-Label, Phase 2 Study to Assess the Safety, Pharmacodynamics, and Efficacy of KRN23 in Children from 1 to 4 Years Old with X-linked Hypophosphatemia (XLH)
    Medical condition: XLH is a disorder of hypophosphatemia, renal phosphate wasting, and the most common inheritable form of rickets. In XLH patients, excess circulating fibroblast growth factor (FGF23) impair phosphat...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10016206 Familial hypophosphataemic rickets LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2005-005915-13 Sponsor Protocol Number: TRx-014-002 Start Date*: 2006-07-12
    Sponsor Name:TauRx Therapeutics PTE Ltd
    Full Title: An Open Pilot Study of Methlythioninium Chloride (MTC) in Frontotemporal Dementia and Related Dementia Syndromes
    Medical condition: Frontotemporal Dementia and related syndromes Prevention and reversal of tau protein aggregation is a novel approach to the treatment of patients with FTD and related syndromes and has the potent...
    Disease: Version SOC Term Classification Code Term Level
    8.1 10012267 PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: (No results available)
    EudraCT Number: 2014-005004-21 Sponsor Protocol Number: THOR-TUE-01 Start Date*: 2015-12-23
    Sponsor Name:Universitätsklinikum Tübingen, STZ eyetrial am Department für Augenheilkunde
    Full Title: THOR - Tübingen Choroideremia gene therapy trial open label Phase 2 clinical trial using an adeno-associated viral vector (AAV2) encoding Rab-escort protein 1 (REP1)
    Medical condition: Choroideremia (CHM)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000015185 10008791 Choroideremia LLT
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: DE (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2019-003190-26 Sponsor Protocol Number: BUR03 Start Date*: 2020-12-21
    Sponsor Name:Julius-Maximilian University of Würzburg
    Full Title: An investigator-sponsored Phase 3b Open-label Study of Anti-FGF23 Antibody Burosumab (KRN23) in Adult Patients with X-linked Hypophosphatemia (XLH) in GERmany - BurGER
    Medical condition: X-linked hypophosphatemia (XLH) is a disorder of renal phosphate wasting, and the most common heritable form of rickets. In XLH patients, high circulating levels of fibroblast growth factor 23 (FGF...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10077957 X-linked hypophosphatemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2019-000469-19 Sponsor Protocol Number: BUR-CL207 Start Date*: 2020-11-03
    Sponsor Name:Kyowa Kirin Pharmaceutical Development Ltd.
    Full Title: A Phase 1/2, Open-label, Multicenter, Non-randomized Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Pediatric Patients from Birth to Less than 1 Year of Ag...
    Medical condition: XLH is a rare, genetic disorder that is serious, chronically debilitating and represents an unmet medical need. This genetic deficiency is estimated to occur in about 1:20,000 live births (Burnett ...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10016206 Familial hypophosphataemic rickets LLT
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Completed) DE (Prematurely Ended) SE (Completed) AT (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2019-000601-77 Sponsor Protocol Number: 5051-201 Start Date*: 2019-12-11
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
    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
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: IE (Prematurely Ended) ES (Temporarily Halted) GB (GB - no longer in EU/EEA) BE (Trial now transitioned) NL (Restarted) DE (Trial now transitioned) IT (Trial now transitioned)
    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: 2014-000406-35 Sponsor Protocol Number: UX023-CL201 Start Date*: Information not available in EudraCT
    Sponsor Name:Ultragenyx Pharmaceuticals Inc.
    Full Title: A Randomized, open Label, Dose Finding, Phase 2 Study to Assess the Pharmacodynamics and Safety of the anti-FGF23 antibody, KRN23, in Pediatric Patients with X-linked Hypophosphatemia (XLH)
    Medical condition: X-linked hypophosphatemia (XLH) is a disorder of renal phosphate wasting, and the most common heritable form of rickets. In XLH patients, high circulating levels of fibroblast growth factor 23 (FG...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10016206 Familial hypophosphataemic rickets LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed) NL (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2012-005010-19 Sponsor Protocol Number: MPD-RC114 Start Date*: 2014-11-25
    Sponsor Name:Consorzio Mario Negri Sud
    Full Title: Exploring the Potential of Dual Kinase JAK 1/2 Inhibitor Ruxolitinib (INC424) with Reduced Intensity Allogeneic Hematopoietic Cell Transplantation in Patients with Myelofibrosis
    Medical condition: Primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF) and post-essential thrombocythemia myelofibrosis (PET-MF), collectively known as myelofibrosis (MF)
    Disease: Version SOC Term Classification Code Term Level
    18.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10028537 Myelofibrosis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: (No results available)
    EudraCT Number: 2015-001775-41 Sponsor Protocol Number: UX023-CL304 Start Date*: 2015-12-16
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: An Open-Label, Single-Arm, Phase 3 Study to Evaluate the Effects of KRN23 on Osteomalacia in Adults with X-linked Hypophosphatemia (XLH)
    Medical condition: XLH is a disorder of hypophosphatemia, renal phosphate wasting, and the most common inheritable form of rickets. In XLH patients, excess circulating fibroblast growth factor (FGF23) impair phospha...
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004850 10016206 Familial hypophosphataemic rickets LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DK (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2014-005529-11 Sponsor Protocol Number: UX023-CL303 Start Date*: 2016-02-01
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study with Open-Label Extension to Assess the Efficacy and Safety of KRN23 in Adults with X-linked Hypophosphatemia (XLH)
    Medical condition: XLH is a disorder of hypophosphatemia, renal phosphate wasting, and the most common inheritable form of rickets. In XLH patients, excess circulating fibroblast growth factor (FGF23) impair phospha...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10016206 Familial hypophosphataemic rickets LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (Completed) IE (Completed) FR (Completed) DK (Completed) IT (Completed)
    Trial results: View results
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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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