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    Clinical Trial Results:
    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 transduction with a lentiviral vector carring FANCA gene and reinfusion in the patient

    Summary
    EudraCT number
    		 2011-006197-88
    Trial protocol
    		 ES  
    Global end of trial date
    30 Nov 2018

    Results information
    Results version number
    		 v1(current)
    This version publication date
    04 Oct 2021
    First version publication date
    04 Oct 2021
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    FANCOSTEM-1
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02931071
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    VHIR
    Sponsor organisation address
    Passeig Vall Hebron 119-129, Barcelona, Spain, 08035
    Public contact
    Joaquin Lopez Soriano, VHIR, 0034 934894865, joaquin.lopez.soriano@vhir.org
    Scientific contact
    Cristina Díaz de Heredia Rubio, VHIR, 0034 934893093, crdiaz@vhebron.net
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    30 Nov 2018
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    30 Nov 2018
    Global end of trial reached?
    Yes
    Global end of trial date
    30 Nov 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main objective is to assess the safety of cell mobilization after treatment with filgrastim and plerixafor in patients with Fanconi Anemia.
    Protection of trial subjects
    This study was conducted in compliance with the ethical principles originating in or derived from the Declaration of Helsinki and in compliance with all International Council for Harmonization (ICH) Good Clinical Practice (GCP) Guidelines. In addition, all local regulatory requirements were followed, in particular, those affording greater protection to the safety of trial subjects
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    01 Sep 2012
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Spain: 11
    Worldwide total number of subjects
    11
    EEA total number of subjects
    11
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    9
    Adolescents (12-17 years)
    2
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 -

    Pre-assignment period milestones
    Number of subjects started
    		 11
    Number of subjects completed
    		 11

    Period 1
    Period 1 title
    Overall trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 CD34+ cells mobilization, collection and immunoselection
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    Filgrastim
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection in vial
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    12 microgram/Kg/12 hours, subcutaneous days 0 to 5

    Investigational medicinal product name
    Plerixafor
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection in vial
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    240 microgram/kg/day subcutaneous at days 5 to 8

    Number of subjects in period 1
    		CD34+ cells mobilization, collection and immunoselection
    Started
    11
    Mobilization of CD34+ cells
    11
    Collection of CD34+ cells
    11
    Immunoselection of PB HSPCs
    11
    Completed
    11

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall trial
    Reporting group description
    		 -

    Reporting group values
    		 Overall trial Total
    Number of subjects
    		 11 11
    Age categorical
    Units: Subjects
        Children
    		 9 9
        Adolescent
    		 2 2
    Age continuous
    Units: years
        median (full range (min-max))
    		 5 (2 to 16) -
    Gender categorical
    Units: Subjects
        Female
    		 1 1
        Male
    		 10 10
    Chromosome fragility test
    Test with diepoxybutane or mitomycin C to confirm chromosome fragility for Fanconi anemia, descarding mosaicism
    Units: Subjects
        Yes
    		 11 11
    Neutrophils
    > or = 0.75 x 10^9 cells/L
    Units: cell/L
        median (full range (min-max))
    		 1.26 (0.76 to 2.4) -
    Platelet
    > or = to 30 x 10^9 cell/L
    Units: cells/L
        median (full range (min-max))
    		 76 (27 to 191) -
    Haemoglobin levels
    > or = to 8g/dL
    Units: g/dL
        median (full range (min-max))
    		 11.3 (10.4 to 12.8) -
    Subject analysis sets

    Subject analysis set title
    Efficacy and safety of cell yield
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    After screening, 11 patients were treated with filgrastim and plerixafor for mobilization of CD34+ cells. 9 patients satisfactorily mobilized cells and underwent cell collection. After collection, cells were sorted

    Subject analysis sets values
    		 Efficacy and safety of cell yield
    Number of subjects
    11
    Age categorical
    Units: Subjects
        Children
    9
        Adolescent
    2
    Age continuous
    Units: years
        median (full range (min-max))
    5 (2 to 16)
    Gender categorical
    Units: Subjects
        Female
    1
        Male
    10
    Chromosome fragility test
    Test with diepoxybutane or mitomycin C to confirm chromosome fragility for Fanconi anemia, descarding mosaicism
    Units: Subjects
        Yes
    11
    Neutrophils
    > or = 0.75 x 10^9 cells/L
    Units: cell/L
        median (full range (min-max))
    1.26 (0.76 to 2.4)
    Platelet
    > or = to 30 x 10^9 cell/L
    Units: cells/L
        median (full range (min-max))
    76 (27 to 191)
    Haemoglobin levels
    > or = to 8g/dL
    Units: g/dL
        median (full range (min-max))
    11.3 (10.4 to 12.8)

    End points

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    End points reporting groups
    Reporting group title
    CD34+ cells mobilization, collection and immunoselection
    Reporting group description
    		 -

    Subject analysis set title
    Efficacy and safety of cell yield
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    After screening, 11 patients were treated with filgrastim and plerixafor for mobilization of CD34+ cells. 9 patients satisfactorily mobilized cells and underwent cell collection. After collection, cells were sorted

    Primary: Safety

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    End point title
    		 Safety [1]
    End point description
    End point type
    Primary
    End point timeframe
    12 months follow-up after cell mobilization
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Goal of the study was to evaluate the safety of the procedure for mobilizing and collecting CD34 cells. There were no treatments in separate arms or periods to justify an statistical analysis. No procedure-associated serious adverse events were observed. Safety of the procedure is included in the adverse events section
    End point values
    		 CD34+ cells mobilization, collection and immunoselection
    Number of subjects analysed
    11
    Units: Lack of severe adverse effects
    11
    No statistical analyses for this end point

    Secondary: Patients with CD34 cell threshold mobilized

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    End point title
    		 Patients with CD34 cell threshold mobilized
    End point description
    > or = to 5 CD34 cell/microliter
    End point type
    Secondary
    End point timeframe
    After tretament with filgrastim and plerixafor
    End point values
    		 CD34+ cells mobilization, collection and immunoselection
    Number of subjects analysed
    11
    Units: cells/microL
        Yes
    9
    No statistical analyses for this end point

    Secondary: CD34 cell collected

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    End point title
    		 CD34 cell collected
    End point description
    Number of patients with efficient collection of cells after apheresis, > or = to 4x10^6 cells/kg body weight (projections of body weight after 5 years)
    End point type
    Secondary
    End point timeframe
    After apheresis collection
    End point values
    		 CD34+ cells mobilization, collection and immunoselection
    Number of subjects analysed
    9
    Units: Cells/Kg
        Yes
    4
    No statistical analyses for this end point

    Secondary: CD34 cell immunoselected

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    End point title
    		 CD34 cell immunoselected
    End point description
    Recovery of CD34 cells after immunoselection has to be > or = to 50%
    End point type
    Secondary
    End point timeframe
    After mobiliation, collection, and immunoselection
    End point values
    		 CD34+ cells mobilization, collection and immunoselection
    Number of subjects analysed
    9
    Units: percentage of cells
        Yes
    0
    No statistical analyses for this end point

    Secondary: Global efficacy of the procedure

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    End point title
    		 Global efficacy of the procedure
    End point description
    Patients achieving > or = 4x10^6 CD34 cells/kg, projecting body weight at 5 years
    End point type
    Secondary
    End point timeframe
    After completing the procedure
    End point values
    		 CD34+ cells mobilization, collection and immunoselection
    Number of subjects analysed
    11
    Units: Cells/Kg
        Yes
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    The whole study
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    2
    Reporting groups
    Reporting group title
    Drug-related
    Reporting group description
    		 -

    Reporting group title
    Total adverse events
    Reporting group description
    		 -

    Serious adverse events
    		 Drug-related Total adverse events
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 11 (0.00%)
    4 / 11 (36.36%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    General disorders and administration site conditions
    Fever of unknown origin
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Interstitial lung disease
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Varicella
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory infection
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Drug-related Total adverse events
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    7 / 11 (63.64%)
    9 / 11 (81.82%)
    Vascular disorders
    Hypotension
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    0 / 11 (0.00%)
    2 / 11 (18.18%)
         occurrences all number
    0
    2
    General disorders and administration site conditions
    Fever
         subjects affected / exposed
    2 / 11 (18.18%)
    4 / 11 (36.36%)
         occurrences all number
    2
    4
    Pain at administration site
         subjects affected / exposed
    1 / 11 (9.09%)
    1 / 11 (9.09%)
         occurrences all number
    1
    1
    Pain at central venous line access
         subjects affected / exposed
    0 / 11 (0.00%)
    3 / 11 (27.27%)
         occurrences all number
    0
    3
    Bleeding at central venous line access
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Throat pain
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Headache
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Blood and lymphatic system disorders
    Anemia
         subjects affected / exposed
    2 / 11 (18.18%)
    3 / 11 (27.27%)
         occurrences all number
    2
    3
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    2 / 11 (18.18%)
    5 / 11 (45.45%)
         occurrences all number
    2
    5
    Vomiting
         subjects affected / exposed
    2 / 11 (18.18%)
    4 / 11 (36.36%)
         occurrences all number
    2
    4
    Nausea
         subjects affected / exposed
    1 / 11 (9.09%)
    2 / 11 (18.18%)
         occurrences all number
    1
    2
    Respiratory, thoracic and mediastinal disorders
    Pleural effusion
         subjects affected / exposed
    0 / 11 (0.00%)
    1 / 11 (9.09%)
         occurrences all number
    0
    1
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    1 / 11 (9.09%)
    2 / 11 (18.18%)
         occurrences all number
    1
    2

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    01 Mar 2015
    Change of sponsor from Cristina Díaz de Heredia to VHIR Change of CRO from Quantum to Sermes Modification of hour of administration of plerixafor, closer to the apheresis procedure Modification of consent forms Modification in pharmaceutical form of filgrastrim to vials for injection Filgrastim datasheet was added to documentation
    06 Sep 2017
    Sample size and recruitment period were expanded

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    Although the threshold of CD34 immunoselected cells was not achieved, enough number of cells were obtained for gene correction and engraftment.

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/34485595
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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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