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Clinical trials for Fanconi Syndrome

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   43881   clinical trials with a EudraCT protocol, of which   7295   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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    40 result(s) found for: Fanconi Syndrome. Displaying page 1 of 2.
    1  2  Next»
    EudraCT Number: 2009-012564-13 Sponsor Protocol Number: Cystadrops®/09/choc-study Start Date*: 2013-01-03
    Sponsor Name:Orphan Europe SARL
    Full Title: Cysteamine Hydrochloride for nephropathic Cystinosis, open-label Phase III pivotal study
    Medical condition: Nephropatic cystinosis patients with cystine corneal deposits
    Disease: Version SOC Term Classification Code Term Level
    16.0 100000004850 10071112 Nephropathic cystinosis LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2014-005264-14 Sponsor Protocol Number: P130103 Start Date*: Information not available in EudraCT
    Sponsor Name:ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
    Full Title: NA
    Medical condition: Fanconi Anemia
    Disease: Version SOC Term Classification Code Term Level
    18.1 100000004850 10055206 Fanconi's anemia LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2018-002502-31 Sponsor Protocol Number: RP-L102-0118 Start Date*: 2018-10-31
    Sponsor Name:Rocket Pharmaceuticals, Inc.
    Full Title: A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemi...
    Medical condition: Fanconi anemia (subtype A)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10055206 Fanconi's anemia LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: ES (Ongoing) GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2018-002984-24 Sponsor Protocol Number: CYT-C2-001 Start Date*: 2019-10-25
    Sponsor Name:RECORDATI Rare Diseases SARL
    Full Title: Open-label, Single-arm, Multicenter Study to Assess the Safety of Cystadrops® in Pediatric Cystinosis Patients from 6 Months to Less Than 2 Years Old [SCOB2 Study]
    Medical condition: Corneal cystine crystal deposits in pediatric patients with nephropathic cystinosis from 6 months to less than 2 years old
    Disease:
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Ongoing) DE (Ongoing) BE (Completed) IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2011-006197-88 Sponsor Protocol Number: FANCOSTEM-1 Start Date*: 2012-03-22
    Sponsor Name:Cristina Díaz de Heredia Rubio
    Full Title: Clinical Phase II Trial to evaluate efficacy and safety of CD34+ cells mobilization and collection after treatment with plerixafor and filgrastim in patients with Fanconi anemia for subsequent tra...
    Medical condition: Fanconi anemia
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10055206 Fanconi's anemia LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: ES (Completed)
    Trial results: View results
    EudraCT Number: 2011-006100-12 Sponsor Protocol Number: FANCOLEN-1 Start Date*: 2013-04-12
    Sponsor Name:FUNDACION PARA LA INVESTIGACION BIOMEDICA DEL HOSPITAL UNIVERSITARIO NIÑO JESUS
    Full Title: Clinical Trial Phase I / II to evaluate the safety and efficacy of the infusion of autologous CD34+ cells transduced with a lentiviral vector carrying the FANCA gene (orphan drug) for patients with...
    Medical condition: Fanconi anemia (Subtype A)
    Disease: Version SOC Term Classification Code Term Level
    15.1 100000004850 10055206 Fanconi's anemia LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2010-022370-14 Sponsor Protocol Number: 1015202 Start Date*: Information not available in EudraCT
    Sponsor Name:Centre Hospitalier de Toulouse
    Full Title: Evaluation de la tolérance d'une administration intra-nasale d'ocytocine chez des nourrissons présentant un syndrome de Prader-Willi et de son effet sur la succion et la prise alimentaire.
    Medical condition: Syndrome de Prader Willi
    Disease: Version SOC Term Classification Code Term Level
    12.1 10036476 Prader-Willi syndrome LLT
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2014-004272-29 Sponsor Protocol Number: 14-MI-10 Start Date*: 2015-03-30
    Sponsor Name:Great Ormond Street Hospital NHS foundation Trust
    Full Title: Phase II clinical trial to evaluate safety and efficacy of mobilisation and collection of CD34+ cells after treatment with plerixafor and filgrastim in patients with Fanconi anaemia for subsequent ...
    Medical condition: Fanconi Anaemia
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2011-003432-32 Sponsor Protocol Number: P100116 Start Date*: Information not available in EudraCT
    Sponsor Name:ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
    Full Title:
    Medical condition:
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10036476 Prader-Willi syndrome PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2010-023179-25 Sponsor Protocol Number: Start Date*: 2011-09-13
    Sponsor Name:Aintree University Hospital NHS Foundation Trust [...]
    1. Aintree University Hospital NHS Foundation Trust
    2. UNIVERSITY OF LIVERPOOL
    Full Title: Effects of exenatide on appetite and ghrelin levels in patients with Prader-Willi Syndrome
    Medical condition: Ghrelin levels in patients with Prader Willi Syndrome and healthy controls and response of ghrelin levels to a single exenatide injection compared with placebo (0.9% sodium chloride) injection.
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10036476 Prader-Willi syndrome PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2017-003423-30 Sponsor Protocol Number: 2017-RCT-OX Start Date*: 2017-12-12
    Sponsor Name:Dutch Growth Research Foundation
    Full Title: Randomized, double-blind, placebo-controlled oxytocin and dose-response trial in children with Prader-Willi syndrome. Effects on social behaviour.
    Medical condition: Prader-Willi syndrome
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10036476 Prader-Willi syndrome PT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-003820-22 Sponsor Protocol Number: 2016-Longterm-OX Start Date*: 2016-12-22
    Sponsor Name:Dutch Growth Research Foundation
    Full Title: Intranasal administration of oxytocin in children with Prader-Willi Syndrome. A randomized, open-label, cross-over trial of different treatment regimens of oxytocin administration. Effects on eatin...
    Medical condition: Prader-Willi syndrome
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10036476 Prader-Willi syndrome PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: NL (Not Authorised)
    Trial results: (No results available)
    EudraCT Number: 2013-004134-15 Sponsor Protocol Number: 2013-OX Start Date*: 2014-07-15
    Sponsor Name:Dutch Growth Research Foundation
    Full Title: Intranasal administration of oxytocin in children and young adults with Prader-Willi Syndrome. A randomized, double-blind, placebo-controlled trial. Effects on satiety and food intake, and social b...
    Medical condition: Prader-Willi syndrome
    Disease: Version SOC Term Classification Code Term Level
    17.0 10010331 - Congenital, familial and genetic disorders 10036476 Prader-Willi syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: NL (Completed)
    Trial results: (No results available)
    EudraCT Number: 2007-006305-25 Sponsor Protocol Number: 2007-006305-25 Start Date*: 2008-02-15
    Sponsor Name:Karolinska University Hospital
    Full Title: Effects of rimonabant (Acomplia) on obesity in adults with Prader-Willi Syndrome
    Medical condition: Obese adults with Prader Willi Syndrome
    Disease: Version SOC Term Classification Code Term Level
    9.1 10036476 Prader-Willi syndrome LLT
    9.1 10029883 Obesity LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: SE (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2017-002164-41 Sponsor Protocol Number: END-GH-2017 Start Date*: 2017-07-03
    Sponsor Name:Fundació Parc Taulí
    Full Title: Growth hormone therapy in adults with Prader-Willi syndrome: Effect on muscle tone assessed by functional magnetic resonance imaging (fMRI) and its relation to muscle strenght and body composition.
    Medical condition: Patients with Prader-Willi Syndrome (SPW) with Growth hormone deficit.
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10036476 Prader-Willi syndrome PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2007-004716-31 Sponsor Protocol Number: GR8009 Start Date*: 2008-03-05
    Sponsor Name:Dutch Growth Foundation
    Full Title: Effects of growth hormone treatment after final height in Prader-Willi Syndrome: A double-blind multicenter, cross-over study on the effects of growth hormone versus placebo on body composition and...
    Medical condition: Prader-Willi Syndrome
    Disease: Version SOC Term Classification Code Term Level
    9.1 10036476 Prader-Willi syndrome LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (Completed)
    Trial results: (No results available)
    EudraCT Number: 2007-000469-39 Sponsor Protocol Number: B9R-HL-GDGN Start Date*: 2007-05-02
    Sponsor Name:Oy Eli Lilly Finland Ab
    Full Title: An Open Label Follow-up Study of Patients Who Participated in Clinical Study B9R-HL-GDDV. A Phase IV, one-arm follow-up study to assess final adult results of GH treatment in patients with the Pra...
    Medical condition: Prader-Willi syndrome
    Disease: Version SOC Term Classification Code Term Level
    9.1 10036476 Prader-Willi syndrome LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FI (Completed)
    Trial results: View results
    EudraCT Number: 2013-004437-33 Sponsor Protocol Number: 13687203 Start Date*: 2014-03-18
    Sponsor Name:Centre Hospitalier de Toulouse
    Full Title: Effets de l'administration intranasale répétée d'ocytocine chez des patients adultes présentant un syndrome de Prader-Willi.
    Medical condition: Prader-Willi syndrom
    Disease: Version SOC Term Classification Code Term Level
    18.0 10010331 - Congenital, familial and genetic disorders 10036476 Prader-Willi syndrome PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2019-000735-61 Sponsor Protocol Number: SP-NAC19 Start Date*: 2021-04-06
    Sponsor Name:Dutch Growth Research Foundation
    Full Title: N- acetylcysteine treatment for skin picking in children and young adults with PWS: a randomized, controlled, cross-over trial.
    Medical condition: Prader-Willi syndrome
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10036476 Prader-Willi syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2012-005325-67 Sponsor Protocol Number: 1239102 Start Date*: 2013-04-30
    Sponsor Name:Centre Hospitalier de Toulouse
    Full Title: Evaluation of tolerance, suckling and food intake after repeated nasals administrations of Oxytocin in PWS infants
    Medical condition: Prader Willi Syndrom
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10036476 Prader-Willi syndrome PT
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: FR (Ongoing)
    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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