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Clinical trials for Variant of uncertain significance

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
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    • 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   44338   clinical trials with a EudraCT protocol, of which   7368   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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    12 result(s) found for: Variant of uncertain significance. Displaying page 1 of 1.
    EudraCT Number: 2017-002767-17 Sponsor Protocol Number: D0816C00020 Start Date*: 2017-12-14
    Sponsor Name:AstraZeneca AB
    Full Title: A Phase IIIb, Single-arm, Open-label Multicentre Study of Olaparib Maintenance Monotherapy in Platinum Sensitive Relapsed non-Germline BRCA Mutated Ovarian Cancer Patients who are in Complete or Pa...
    Medical condition: Non Germline BRCA Mutated Ovarian Cancer
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10033128 Ovarian cancer PT
    Population Age: Adults, Elderly Gender: Female
    Trial protocol: CZ (Completed) SI (Completed) BG (Completed) GB (GB - no longer in EU/EEA) NL (Completed) ES (Completed) BE (Completed) AT (Completed) SE (Completed) PT (Completed) NO (Completed) DK (Completed) FI (Completed) PL (Completed) IT (Completed) RO (Ongoing)
    Trial results: View results
    EudraCT Number: 2021-002873-24 Sponsor Protocol Number: RM-493-035 Start Date*: 2022-05-16
    Sponsor Name:Rhythm Pharmaceuticals Inc.
    Full Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study: 5 Independent Sub-studies of Setmelanotide in Patients with POMC, PCSK1, LEPR, SRC1, SH2B1, and PCSK1 N221D Gene Defects in the Melano...
    Medical condition: Specific Gene Defects in the Melanocortin-4 Receptor Pathway, responsible for improper functions of certain messenger materials in the body. E.g Melanocyte-Stimulating Hormone (MSH)
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) ES (Ongoing) DE (Trial now transitioned) GR (Trial now transitioned) NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2021-000348-22 Sponsor Protocol Number: KER047-IR-201 Start Date*: 2021-08-10
    Sponsor Name:Keros Therapeutics, Inc.
    Full Title: A Phase 2, Open-label, Dose Escalation and Dose Expansion Study of KER-047 for the Treatment of IRIDA
    Medical condition: Iron-Refractory Iron-Deficiency Anemia (IRIDA)
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Prematurely Ended) ES (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2018-001213-32 Sponsor Protocol Number: MedOPP168 Start Date*: 2019-02-04
    Sponsor Name:Medica Scientia Innovation Research (MedSIR)
    Full Title: Effectiveness of olaparib plus trastuzumab in HER2-positive BRCA-mutated or Homologous Recombination Deficiency (HRD) advanced breast cancer patients – The OPHELIA Study –
    Medical condition: HER2-positive BRCAmutated or Homologous Recombination Deficiency (HRD) advanced breast cancer
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004864 10072737 Advanced breast cancer LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    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: 2022-003407-15 Sponsor Protocol Number: SRP-9001-104 Start Date*: 2023-08-08
    Sponsor Name:Sarepta Therapeutics, Inc
    Full Title: An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of SRP-9001 in association with imlifidase in Subjects with Duchenne Muscular Dystrophy with pre-exis...
    Medical condition: Duchenne muscular dystrophy
    Disease:
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: ES (Temporarily Halted)
    Trial results: (No results available)
    EudraCT Number: 2021-002855-12 Sponsor Protocol Number: RM-493-034 Start Date*: 2022-04-22
    Sponsor Name:Rhythm Pharmaceuticals Inc.
    Full Title: A 2-Stage (Open-Label Run-in followed by Randomized Withdrawal), Double-Blind, Placebo-Controlled, Phase 2 study of Setmelanotide in Patients with Specific Gene Defects in the Melanocortin-4 Rece...
    Medical condition: Specific Gene Defects in the Melanocortin-4 Receptor Pathway, responsible for improper functions of certain messenger materials in the body. E.g Melanocyte-Stimulating Hormone (MSH)
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Completed) DE (Completed) ES (Ongoing) NL (Completed) GR (Completed)
    Trial results: View results
    EudraCT Number: 2022-000691-19 Sponsor Protocol Number: BN43881 Start Date*: 2022-10-26
    Sponsor Name:F. Hoffmann-La Roche Ltd
    Full Title: A two-part, open-label systemic gene delivery study to evaluate the safety and expression of RO7494222 (SRP-9001) in subjects under the age of four with Duchenne muscular dystrophy
    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
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: ES (Ongoing) DE (Trial now transitioned) BE (Trial now transitioned) IT (Trial now transitioned) FR (Trial now transitioned)
    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: 2019-003374-91 Sponsor Protocol Number: SRP-9001-301 Start Date*: 2022-09-12
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBA...
    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
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: FR (Completed) BE (Completed) ES (Completed) DE (Completed) IT (Completed)
    Trial results: View results
    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: 2021-006597-23 Sponsor Protocol Number: UX143-CL301 Start Date*: Information not available in EudraCT
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: A Randomized, Double-Blind, Placebo-controlled, Phase 2/3 Study to Assess the Efficacy and Safety of Setrusumab in Subjects with Osteogenesis Imperfecta.
    Medical condition: Osteogenesis imperfecta (OI)
    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, Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) DK (Prematurely Ended) DE (Trial now transitioned) IT (Trial now transitioned) PL (Trial now transitioned) PT (Trial now transitioned)
    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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