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Clinical trials for Fanconi anemia

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   44334   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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    10 result(s) found for: Fanconi anemia. Displaying page 1 of 1.
    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: 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: 2004-004914-18 Sponsor Protocol Number: CD-001 Start Date*: 2005-01-21
    Sponsor Name:ISTITUTO GIANNINA GASLINI
    Full Title: A pilot multicentric study for the use of the anti-TNF alfa in patients with: 1) Fanconi Anemia with marrow failure with no bone marrow compatible donor 2) In patients with acquired aplastic anem...
    Medical condition: Marrow failure in patients refractary/non eligible to conventional treatments
    Disease:
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-003226-16 Sponsor Protocol Number: BP-404 Start Date*: 2017-02-03
    Sponsor Name:Bellicum Pharmaceuticals, Inc.
    Full Title: Follow-up of phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR αβ+ T cells in pediatric patients affected by hematological disorders
    Medical condition: Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplastic syndromes;Congenital immune deficiencies;Severe aplastic anemia;Fanconi anemia; Osteopetrosis;Selected cases of hemoglobinopath...
    Disease: Version SOC Term Classification Code Term Level
    19.0 10005329 - Blood and lymphatic system disorders 10018849 Haematological disorders NEC HLGT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2014-000584-41 Sponsor Protocol Number: BP-004 Start Date*: 2014-10-23
    Sponsor Name:Bellicum Pharmaceuticals, Inc.
    Full Title: Phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR αβ+ T cells in pediatric patients affected by hematological disorders
    Medical condition: Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplastic syndromes;Congenital immune deficiencies;Severe aplastic anemia;Fanconi anemia; Osteopetrosis;Selected cases of hemoglobinopath...
    Disease: Version SOC Term Classification Code Term Level
    16.1 10005329 - Blood and lymphatic system disorders 10018849 Haematological disorders NEC HLGT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Completed) GB (GB - no longer in EU/EEA) ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2015-003531-35 Sponsor Protocol Number: CICL670ARU01 Start Date*: 2016-03-07
    Sponsor Name:Novartis Pharmaceuticals
    Full Title: 1 Year, Open-label Multicenter Evaluation of Efficacy, Safety of Deferasirox in Patients MDS, Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload.
    Medical condition:
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004850 10054658 Thalassemia LLT
    19.0 100000004864 10028534 Myelodysplastic syndrome NOS LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2018-001489-41 Sponsor Protocol Number: P00003466 Start Date*: 2018-07-02
    Sponsor Name:Karolinska Universitetssjukhuset
    Full Title: Radiation- and alkylator-free hematopoietic cell transplantation for bone marrow failure due to dyskeratosis congenita / telomere disease
    Medical condition: Dyskeratosis congenita / telomere disease
    Disease: Version SOC Term Classification Code Term Level
    20.0 10042613 - Surgical and medical procedures 10001756 Allogenic bone marrow transplantation therapy PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: SE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2011-004868-30 Sponsor Protocol Number: PNET5MB Start Date*: 2013-10-31
    Sponsor Name:University Medical Center Hamburg-Eppendorf
    Full Title: AN INTERNATIONAL PROSPECTIVE TRIAL ON MEDULLOBLASTOMA (MB) IN CHILDREN OLDER THAN 3 TO 5 YEARS WITH WNT BIOLOGICAL PROFILE (PNET 5 MB – LR and PNET 5 MB – WNT-HR), AVERAGE-RISK BIOLOGICAL PROFILE (...
    Medical condition: Children with medulloblastoma. Medulloblastoma is a highly cellular malignant embryonal neoplasm.
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10027107 Medulloblastoma PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) BE (Trial now transitioned) IT (Trial now transitioned) FR (Trial now transitioned) FI (Trial now transitioned) SE (Trial now transitioned) ES (Ongoing) GB (GB - no longer in EU/EEA) AT (Ongoing) NO (Completed) CZ (Trial now transitioned) NL (Trial now transitioned) DK (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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