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Clinical trials for Phakomatosis

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 ).  
     
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    18 result(s) found for: Phakomatosis. Displaying page 1 of 1.
    EudraCT Number: 2016-002408-13 Sponsor Protocol Number: NL58183.041.16 Start Date*: 2017-02-07
    Sponsor Name:UMC Utrecht
    Full Title: Bumetanide to Ameliorate Tuberous Sclerosis Complex Hyperexcitable Behaviors
    Medical condition: Tuberous Sclerosis Complex
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10045138 Tuberous sclerosis PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-002977-37 Sponsor Protocol Number: CRAD001M2X02B Start Date*: 2017-08-25
    Sponsor Name:Novartis Pharma AG
    Full Title: An open-label, multi-center long-term safety roll-over study in patients with tuberous sclerosis complex (TSC) and refractory seizures who are judged by the Investigator to benefit from continued t...
    Medical condition: Refractory seizures associated with tuberous sclerosis complex
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10045138 Tuberous sclerosis PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) HU (Completed) ES (Completed) BE (Completed) FR (Completed) PL (Trial now transitioned) IT (Completed)
    Trial results: (No results available)
    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: 2007-005978-30 Sponsor Protocol Number: FP2007/02 Start Date*: 2008-01-22
    Sponsor Name:FUNDACIÓ PUIGVERT
    Full Title: Ensayo clínico para evaluar la eficacia y seguridad de la rapamicina en los angiomiolipomas en pacientes con esclerosis tuberosa Clinical Trial to Determine the Efficacy and Safety of Rapamycin in ...
    Medical condition: Angiomiolipomas en pacientes con esclerosis tuberosa (angiomyolipoma of tuberous sclerosis patients)
    Disease: Version SOC Term Classification Code Term Level
    9.1 10045138 Tuberous sclerosis LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-002132-67 Sponsor Protocol Number: GWEP17005 Start Date*: 2021-10-22
    Sponsor Name:GW Research Ltd
    Full Title: An Open-Label, Randomized Trial to Assess the Safety, Pharmacokinetics, and Exploratory Efficacy of Adjunctive Cannabidiol Oral Solution (GWP42003-P) Compared with Standard of Care Antiseizure Medi...
    Medical condition: Tuberous Sclerosis Complex (TSC)
    Disease: Version SOC Term Classification Code Term Level
    21.0 10010331 - Congenital, familial and genetic disorders 10045138 Tuberous sclerosis LLT
    21.0 10010331 - Congenital, familial and genetic disorders 10080584 Tuberous sclerosis complex PT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: (No results available)
    EudraCT Number: 2013-003795-13 Sponsor Protocol Number: CRAD001M2305 Start Date*: 2014-12-10
    Sponsor Name:Novartis Pharma Services AG
    Full Title: Long-term follow-up study to monitor the growth and development of pediatric patients previously treated with everolimus in study CRAD001M2301
    Medical condition: Subependymal Giant Cell Astrocytoma (SEGA) associated with Tuberous Sclerosis Complex (TSC)
    Disease: Version SOC Term Classification Code Term Level
    21.0 100000004850 10045138 Tuberous sclerosis LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: BE (Completed) PL (Completed)
    Trial results: View results
    EudraCT Number: 2015-002154-12 Sponsor Protocol Number: GWEP1521 Start Date*: 2016-09-30
    Sponsor Name:GW Research Ltd.
    Full Title: A double-blind, randomized, placebo-controlled study to investigate the efficacy and safety of cannabidiol (GWP42003-P, CBD) as add-on therapy in patients with tuberous sclerosis complex who experi...
    Medical condition: Tuberous Sclerosis Complex (TSC)
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10045138 Tuberous sclerosis PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: ES (Completed) GB (GB - no longer in EU/EEA) PL (Completed) NL (Completed)
    Trial results: View results
    EudraCT Number: 2021-000838-34 Sponsor Protocol Number: NOE-TCS-201 Start Date*: 2023-04-04
    Sponsor Name:Noema Pharma AG
    Full Title: A Phase 2B, Multicenter, 30-week, Prospective, Cross-over, Doubleblind, Randomized, Placebo-controlled Study Followed by a 52-Week Open-label Extension Study to Evaluate the Efficacy and Safety of ...
    Medical condition: Tuberous Sclerosis Complex
    Disease: Version SOC Term Classification Code Term Level
    21.0 10010331 - Congenital, familial and genetic disorders 10080584 Tuberous sclerosis complex PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Trial now transitioned) ES (Ongoing) PL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2011-000860-90 Sponsor Protocol Number: CRAD001M2304 Start Date*: 2013-02-14
    Sponsor Name:Novartis Farmacéutica, S.A.
    Full Title: A three-arm, randomized, double-blind, placebo-controlled study of the efficacy and safety of two trough-ranges of everolimus as adjunctive therapy in patients with tuberous sclerosis complex (TSC)...
    Medical condition: Refractory partial-onset seizures associated with tuberous sclerosis complex (TSC)
    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, Elderly Gender: Male, Female
    Trial protocol: ES (Completed) IT (Completed) NL (Completed) DE (Completed) HU (Completed) GB (Completed) BE (Completed) GR (Completed) DK (Completed) IE (Completed)
    Trial results: View results
    EudraCT Number: 2010-024078-20 Sponsor Protocol Number: RSW2011 Start Date*: 2011-05-11
    Sponsor Name:Instituto Científico y Tecnológico de Navarra
    Full Title: Ensayo clínico fase II, aleatorizado, triple ciego, controlado intraindividualmente con placebo, para evaluar la eficacia y seguridad de la rapamicina tópica sola o asociada a láser de colorante pu...
    Medical condition: Malformación capilar cutánea en pacientes con síndrome de Sturge-Weber.
    Disease: Version SOC Term Classification Code Term Level
    13 10042265 Síndrome de Sturge-Weber PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: ES (Completed)
    Trial results: View results
    EudraCT Number: 2010-022583-13 Sponsor Protocol Number: CRAD001MIC02 Start Date*: Information not available in EudraCT
    Sponsor Name:Novartis Pharma Services AG
    Full Title: An open-label, multi-center, expanded access study of RAD001 in patients with subependymal giant cell astrocytomas (SEGA) associated with tuberous sclerosis complex (TSC). The EFFECTS STUDY: Everol...
    Medical condition: The study will evaluate the safety of RAD001 in patients with subependymal giant cell astrocytomas (SEGA) associated with Tuberous Sclerosis Complex (TSC).
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) ES (Completed) HU (Completed) CZ (Completed) FR (Ongoing) BE (Completed) GR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2007-006997-27 Sponsor Protocol Number: CRAD001M2301 Start Date*: 2011-06-01
    Sponsor Name:Novartis Pharma Services AG
    Full Title: A randomized, double-blind, placebo-controlled study of RAD001 in the treatment of patients with subependymal giant cell astrocytomas (SEGA) associated with Tuberous Sclerosis Complex (TSC)
    Medical condition: This study will evaluate the antitumor activity of RAD001 versus placebo in patients with subependymal giant cell astrocytomas (SEGA) associated with Tuberous Sclerosis Complex (TSC).
    Disease:
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) IT (Completed) GB (Completed) DE (Completed) NL (Completed)
    Trial results: View results
    EudraCT Number: 2020-005607-39 Sponsor Protocol Number: D134BC00001 Start Date*: 2021-10-05
    Sponsor Name:AstraZeneca AB
    Full Title: A Phase III, Multicentre, International Study with a Parallel, Randomised, Double blind, Placebo-controlled, 2 Arm Design to Assess the Efficacy and Safety of Selumetinib in Adult Participants with...
    Medical condition: Neurofibromatosis Type 1 (NF1) with Symptomatic, Inoperable Plexiform Neurofibromas (PN)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10029270 Neurofibromatosis, type 1 (von Recklinghausen's disease) LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Restarted) PL (Trial now transitioned) IT (Trial now transitioned) FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2013-003405-26 Sponsor Protocol Number: NF1-EXCEL Start Date*: 2014-09-03
    Sponsor Name:Erasmus MC
    Full Title: The effect of Lamotrigine on cognitive deficits associated with Neurofibromatosis type 1: a phase II randomized controlled multi-centre trial (NF1-EXCEL)
    Medical condition: Neurofibromatosis type 1
    Disease: Version SOC Term Classification Code Term Level
    17.0 100000004850 10029270 Neurofibromatosis, type 1 (von Recklinghausen's disease) LLT
    Population Age: Adolescents, Under 18 Gender: Male, Female
    Trial protocol: NL (Ongoing) BE (Completed) DK (Prematurely Ended) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2020-005648-52 Sponsor Protocol Number: D1346C00015 Start Date*: 2021-05-31
    Sponsor Name:AstraZeneca AB
    Full Title: A Phase I, Single-Arm, Sequential Study to Evaluate the Effect of Food on the Gastrointestinal Tolerability and Pharmacokinetics of Selumetinib after Multiple Doses in Adolescent Children with Neur...
    Medical condition: Neurofibromatosis Type 1 (NF1) Related Plexiform Neurofibromas (PN)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10029270 Neurofibromatosis, type 1 (von Recklinghausen's disease) LLT
    Population Age: Adolescents, Under 18 Gender: Male, Female
    Trial protocol: PL (Trial now transitioned) ES (Completed)
    Trial results: (No results available)
    EudraCT Number: 2012-005742-38 Sponsor Protocol Number: SANTA2012 Start Date*: 2013-05-02
    Sponsor Name:Central Manchester University Hospitals NHS Foundation Trust
    Full Title: Early phase triple blind placebo controlled RCT of simvastatin treatment for autism in young children with Neurofibromatosis Type 1
    Medical condition: Neurofibromatosis Type 1
    Disease: Version SOC Term Classification Code Term Level
    14.1 100000004850 10029270 Neurofibromatosis, type 1 (von Recklinghausen's disease) LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2020-005608-20 Sponsor Protocol Number: D1346C00004 Start Date*: 2021-11-24
    Sponsor Name:AstraZeneca AB
    Full Title: A Phase I/II, Single-Arm, Open label Study to Evaluate the Pharmacokinetics, Safety/Tolerability and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥ 1 to < 7 Years with Neurofibr...
    Medical condition: Neurofibromatosis Type 1 (NF1) Related Plexiform Neurofibromas (PN)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10029270 Neurofibromatosis, type 1 (von Recklinghausen's disease) LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) ES (Ongoing) NL (Prematurely Ended) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2021-001572-42 Sponsor Protocol Number: FCN-159-002 Start Date*: 2021-12-21
    Sponsor Name:Shanghai Fosun Pharmaceutical Industrial Development Co.,Ltd.
    Full Title: A multi-center, open-label, single-arm Phase I dose-escalation and Phase II dose-expansion study to evaluate the safety, tolerability, PK characteristics and anti-tumor activity of FCN-159 in adult...
    Medical condition: Participants diagnosed with Neurofibromatosis NF1-related plexiform neurofibromas (PN) and symptomatic with requirement of systematic therapy per investigator’s judgment. A PN is defined as a neuro...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10029270 Neurofibromatosis, type 1 (von Recklinghausen's disease) LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing) IT (Ongoing) PL (Completed)
    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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