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    Clinical Trial Results:
    A dose escalation and phase IIa study of TG6002 plus flucytosine in patients with unresectable colorectal cancer with liver metastases

    Summary
    EudraCT number
    		 2018-004103-39
    Trial protocol
    		 GB   FR  
    Global end of trial date
    23 Feb 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    23 Feb 2024
    First version publication date
    23 Feb 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    TG6002.03
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04194034
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    TRANSGENE S.A.
    Sponsor organisation address
    400 boulevard Gonthier d’Andernach - Parc d’innovation – CS80166, Illkirch-Graffenstaden, France, 67405
    Public contact
    Medical Affairs, TRANSGENE S.A., 33 388279155, clinical.trials@transgene.fr
    Scientific contact
    Medical Affairs, TRANSGENE S.A., 33 388279155, clinical.trials@transgene.fr
    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
    19 Apr 2023
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    31 Aug 2022
    Global end of trial reached?
    Yes
    Global end of trial date
    23 Feb 2023
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective of the phase I part is to determine the maximum tolerated dose (MTD) of TG6002 or maximal feasible dose (MFD) and the recommended dose for the Phase II part (RP2D) for TG6002 administered as an intrahepatic artery (IHA) infusions in combination with flucytosine (5-FC) in patients with unresectable advanced metastatic colorectal cancer (CRC) with liver metastases. Secondary objectives include the assessessment of the safety and tolerability of TG6002 administration by selective IHA infusion combined with oral 5-FC, the assessment of the study treatment efficacy and the assessment of TG6002 viral shedding.
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and in compliance with all International Conference on Harmonization Good Clinical Practice Guidelines. All the local regulatory requirements pertinent to safety of trial participants were followed.
    Background therapy
    		 Not applicable.
    Evidence for comparator
    		 Not applicable.
    Actual start date of recruitment
    17 Jan 2020
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Regulatory reason
    Long term follow-up duration
    		 5 Years
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United Kingdom: 7
    Country: Number of subjects enrolled
    France: 8
    Worldwide total number of subjects
    15
    EEA total number of subjects
    8
    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)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    10
    From 65 to 84 years
    5
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 First participant signed informed consent on 17 January 2020. Last participant last visit occurred on 1 August 2022, before long term follow-up.

    Pre-assignment
    Screening details
    		 Of 20 screened participants, 15 were included in the trial and 5 patients were excluded (4 because they were ineligible and 1 due to logistical constraints during the covid-19 pandemic).

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 1 x 10E6 pfu
    Arm description
    		 Cohort of patients administered with TG6002 intrahepatic artery (IHA) infusion at 1 x 10E6 pfu in combination with oral 5-FC at 200 mg/kg/day for 10 days.
    Arm type
    		 Experimental

    Investigational medicinal product name
    5-FC
    Investigational medicinal product code
    Other name
    Ancotil
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Patients were given oral 5-FC at 200 mg/kg/day from Day 5 to 14 (Cycle 1). If there was no disease progression or occurrence of toxicity that would jeopardize the patient study participation, the patient was proposed a second cycle of TG6002 plus 5-FC at the same dose given on Days 47 to 56.

    Investigational medicinal product name
    TG6002
    Investigational medicinal product code
    TG6002
    Other name
    Pharmaceutical forms
    Solution for injection/infusion
    Routes of administration
    Intraarterial use
    Dosage and administration details
    Patients were administered with TG6002 intrahepatic artery (IHA) infusion at 1 x 10E6 pfu (single dose on Day 1) in combination with oral 5-FC (Cycle 1). If there was no disease progression or occurrence of toxicity that would jeopardize the patient study participation, the patient was proposed a second cycle of TG6002 plus 5-FC at the same dose with an IHA infusion of TG6002 on Day 43 (+ 7 days) and oral 5-FC.

    Arm title
    		 1 x 10E7 pfu
    Arm description
    		 Cohort of patients administered with TG6002 intrahepatic artery (IHA) infusion at 1 x 10E7 pfu in combination with oral 5-FC at 200 mg/kg/day for 10 days.
    Arm type
    		 Experimental

    Investigational medicinal product name
    5-FC
    Investigational medicinal product code
    Other name
    Ancotil
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Patients were given oral 5-FC at 200 mg/kg/day from Day 5 to 14 (Cycle 1). If there was no disease progression or occurrence of toxicity that would jeopardize the patient study participation, the patient was proposed a second cycle of TG6002 plus 5-FC at the same dose given on Days 47 to 56.

    Investigational medicinal product name
    TG6002
    Investigational medicinal product code
    TG6002
    Other name
    Pharmaceutical forms
    Solution for injection/infusion
    Routes of administration
    Intraarterial use
    Dosage and administration details
    Patients were administered with TG6002 intrahepatic artery (IHA) infusion at 1 x 10E7 pfu (single dose on Day 1) in combination with oral 5-FC (Cycle 1). If there was no disease progression or occurrence of toxicity that would jeopardize the patient study participation, the patient was proposed a second cycle of TG6002 plus 5-FC at the same dose with an IHA infusion of TG6002 on Day 43 (+ 7 days) and oral 5-FC.

    Arm title
    		 1 x 10E8 pfu
    Arm description
    		 Cohort of patients administered with TG6002 intrahepatic artery (IHA) infusion at 1 x 10E8 pfu in combination with oral 5-FC at 200 mg/kg/day for 10 days.
    Arm type
    		 Experimental

    Investigational medicinal product name
    5-FC
    Investigational medicinal product code
    Other name
    Ancotil
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Patients were given oral 5-FC at 200 mg/kg/day from Day 5 to 14 (Cycle 1). If there was no disease progression or occurrence of toxicity that would jeopardize the patient study participation, the patient was proposed a second cycle of TG6002 plus 5-FC at the same dose given on Days 47 to 56.

    Investigational medicinal product name
    TG6002
    Investigational medicinal product code
    TG6002
    Other name
    Pharmaceutical forms
    Solution for injection/infusion
    Routes of administration
    Intraarterial use
    Dosage and administration details
    Patients were administered with TG6002 intrahepatic artery (IHA) infusion at 1 x 10E8 pfu (single dose on Day 1) in combination with oral 5-FC (Cycle 1). If there was no disease progression or occurrence of toxicity that would jeopardize the patient study participation, the patient was proposed a second cycle of TG6002 plus 5-FC at the same dose with an IHA infusion of TG6002 on Day 43 (+ 7 days) and oral 5-FC.

    Arm title
    		 1 x 10E9 pfu
    Arm description
    		 Cohort of patients administered with TG6002 intrahepatic artery (IHA) infusion at 1 x 10E9 pfu in combination with oral 5-FC at 200 mg/kg/day for 10 days.
    Arm type
    		 Experimental

    Investigational medicinal product name
    5-FC
    Investigational medicinal product code
    Other name
    Ancotil
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Patients were given oral 5-FC at 200 mg/kg/day from Day 5 to 14 (Cycle 1). If there was no disease progression or occurrence of toxicity that would jeopardize the patient study participation, the patient was proposed a second cycle of TG6002 plus 5-FC at the same dose given on Days 47 to 56.

    Investigational medicinal product name
    TG6002
    Investigational medicinal product code
    TG6002
    Other name
    Pharmaceutical forms
    Solution for injection/infusion
    Routes of administration
    Intraarterial use
    Dosage and administration details
    Patients were administered with TG6002 intrahepatic artery (IHA) infusion at 1 x 10E9 pfu (single dose on Day 1) in combination with oral 5-FC (Cycle 1). If there was no disease progression or occurrence of toxicity that would jeopardize the patient study participation, the patient was proposed a second cycle of TG6002 plus 5-FC at the same dose with an IHA infusion of TG6002 on Day 43 (+ 7 days) and oral 5-FC.

    Number of subjects in period 1
    		1 x 10E6 pfu 1 x 10E7 pfu 1 x 10E8 pfu 1 x 10E9 pfu
    Started
    3
    3
    3
    6
    Completed
    3
    3
    3
    4
    Not completed
    0
    0
    0
    2
         Adverse event, non-fatal
    -
    -
    -
    1
         For best supportive care
    -
    -
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    1 x 10E6 pfu
    Reporting group description
    		 Cohort of patients administered with TG6002 intrahepatic artery (IHA) infusion at 1 x 10E6 pfu in combination with oral 5-FC at 200 mg/kg/day for 10 days.

    Reporting group title
    1 x 10E7 pfu
    Reporting group description
    		 Cohort of patients administered with TG6002 intrahepatic artery (IHA) infusion at 1 x 10E7 pfu in combination with oral 5-FC at 200 mg/kg/day for 10 days.

    Reporting group title
    1 x 10E8 pfu
    Reporting group description
    		 Cohort of patients administered with TG6002 intrahepatic artery (IHA) infusion at 1 x 10E8 pfu in combination with oral 5-FC at 200 mg/kg/day for 10 days.

    Reporting group title
    1 x 10E9 pfu
    Reporting group description
    		 Cohort of patients administered with TG6002 intrahepatic artery (IHA) infusion at 1 x 10E9 pfu in combination with oral 5-FC at 200 mg/kg/day for 10 days.

    Reporting group values
    		 1 x 10E6 pfu 1 x 10E7 pfu 1 x 10E8 pfu 1 x 10E9 pfu Total
    Number of subjects
    		 3 3 3 6 15
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0 0 0
        Newborns (0-27 days)
    		 0 0 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0 0 0
        Children (2-11 years)
    		 0 0 0 0 0
        Adolescents (12-17 years)
    		 0 0 0 0 0
        Adults (18-64 years)
    		 2 1 2 5 10
        From 65-84 years
    		 1 2 1 1 5
        85 years and over
    		 0 0 0 0 0
    Age continuous
    Units: years
        median (full range (min-max))
    		 61 (59 to 70) 68 (59 to 77) 53 (37 to 69) 59.5 (50 to 78) -
    Gender categorical
    Units: Subjects
        Female
    		 0 1 0 3 4
        Male
    		 3 2 3 3 11
    ECOG performance status
    Units: Subjects
        Score 0
    		 3 2 3 1 9
        Score 1
    		 0 1 0 5 6
        Score 2
    		 0 0 0 0 0
        Score 3
    		 0 0 0 0 0
        Score 4
    		 0 0 0 0 0
    Tumor stage at initial diagnosis
    Units: Subjects
        Stage IA
    		 0 0 0 0 0
        Stage IB
    		 0 0 0 0 0
        Stage IIA
    		 0 0 0 0 0
        Stage IIB
    		 0 0 0 0 0
        Stage IIIA
    		 0 0 0 0 0
        Stage IIIB
    		 1 0 0 0 1
        Stage IIIC
    		 0 1 0 1 2
        Stage IV
    		 2 2 3 5 12
    Primary location of disease
    Units: Subjects
        Colon
    		 2 2 3 4 11
        Rectum
    		 1 1 0 2 4

    End points

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    End points reporting groups
    Reporting group title
    1 x 10E6 pfu
    Reporting group description
    		 Cohort of patients administered with TG6002 intrahepatic artery (IHA) infusion at 1 x 10E6 pfu in combination with oral 5-FC at 200 mg/kg/day for 10 days.

    Reporting group title
    1 x 10E7 pfu
    Reporting group description
    		 Cohort of patients administered with TG6002 intrahepatic artery (IHA) infusion at 1 x 10E7 pfu in combination with oral 5-FC at 200 mg/kg/day for 10 days.

    Reporting group title
    1 x 10E8 pfu
    Reporting group description
    		 Cohort of patients administered with TG6002 intrahepatic artery (IHA) infusion at 1 x 10E8 pfu in combination with oral 5-FC at 200 mg/kg/day for 10 days.

    Reporting group title
    1 x 10E9 pfu
    Reporting group description
    		 Cohort of patients administered with TG6002 intrahepatic artery (IHA) infusion at 1 x 10E9 pfu in combination with oral 5-FC at 200 mg/kg/day for 10 days.

    Primary: Safety and tolerability of TG6002 and 5-FC

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    End point title
    		 Safety and tolerability of TG6002 and 5-FC [1]
    End point description
    Patients were assessed for dose limiting toxicities (DLT) as well as for safety and tolerability of the treatment.
    End point type
    Primary
    End point timeframe
    Patients were assessed for dose limiting toxicity (DLTs) occurring within 28 days of administration of the first TG6002 infusion.
    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: Data for this end point were analyzed descriptively.
    End point values
    		 1 x 10E6 pfu 1 x 10E7 pfu 1 x 10E8 pfu 1 x 10E9 pfu
    Number of subjects analysed
    3
    3
    3
    6
    Units: Events
        Dose Limiting Toxicities (DLTs)
    0
    0
    0
    1
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    AEs and SAEs were recorded from the first IMP admin. up to 28 days after the last IMP admin. SAE related to study treatment were recorded with no time limitation. SAEs related to a protocol procedure were recorded from the time of ICF signature.
    Adverse event reporting additional description
    Adverse event information was collected by regular investigator assessment and regular laboratory testing.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    25.1
    Reporting groups
    Reporting group title
    1 x 10E6 pfu
    Reporting group description
    		 -

    Reporting group title
    1 x 10E7 pfu
    Reporting group description
    		 -

    Reporting group title
    1 x 10E8 pfu
    Reporting group description
    		 -

    Reporting group title
    1 x 10E9 pfu
    Reporting group description
    		 -

    Reporting group title
    Safety population
    Reporting group description
    		 Participants who received at least one dose of either IMP.

    Serious adverse events
    		 1 x 10E6 pfu 1 x 10E7 pfu 1 x 10E8 pfu 1 x 10E9 pfu Safety population
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    3 / 6 (50.00%)
    3 / 15 (20.00%)
         number of deaths (all causes)
    3
    3
    3
    4
    13
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    Injury, poisoning and procedural complications
    Vascular procedure complication
    Additional description: Pre-drug event related to other study procedure
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 6 (16.67%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Myocardial infarction
    Additional description: Myocardial infarction
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 6 (16.67%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    1 / 1
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Anaemia
    Additional description: Anaemia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 6 (16.67%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Diarrhoea
    Additional description: Diarrhoea
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 6 (16.67%)
    1 / 15 (6.67%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    1 / 1
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 1 x 10E6 pfu 1 x 10E7 pfu 1 x 10E8 pfu 1 x 10E9 pfu Safety population
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    3 / 3 (100.00%)
    3 / 3 (100.00%)
    2 / 3 (66.67%)
    6 / 6 (100.00%)
    14 / 15 (93.33%)
    Vascular disorders
    Hypertension
    Additional description: Hypertension
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    1 / 6 (16.67%)
    3 / 15 (20.00%)
         occurrences all number
    1
    1
    0
    1
    3
    General disorders and administration site conditions
    Pyrexia
    Additional description: Pyrexia
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    6 / 6 (100.00%)
    8 / 15 (53.33%)
         occurrences all number
    1
    0
    1
    9
    11
    Influenza like illness
    Additional description: Influenza like illness
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 6 (16.67%)
    2 / 15 (13.33%)
         occurrences all number
    1
    0
    0
    1
    2
    Fatigue
    Additional description: Fatigue
         subjects affected / exposed
    2 / 3 (66.67%)
    2 / 3 (66.67%)
    0 / 3 (0.00%)
    3 / 6 (50.00%)
    7 / 15 (46.67%)
         occurrences all number
    3
    2
    0
    3
    8
    Chills
    Additional description: Chills
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    3 / 6 (50.00%)
    3 / 15 (20.00%)
         occurrences all number
    0
    0
    0
    4
    4
    Chest discomfort
    Additional description: Chest discomfort
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 6 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    1
    Asthenia
    Additional description: Asthenia
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 6 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    1
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
    Additional description: Dyspnoea
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 6 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    2
    0
    0
    0
    2
    Psychiatric disorders
    Depression
    Additional description: Depression
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 6 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    1
    Anxiety
    Additional description: Anxiety
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    1 / 6 (16.67%)
    2 / 15 (13.33%)
         occurrences all number
    0
    1
    0
    1
    2
    Investigations
    Alanine aminotransferase increased
    Additional description: Alanine aminotransferase increased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 6 (16.67%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    1
    Amylase increased
    Additional description: Amylase increased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 6 (16.67%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    1
    Blood alkaline phosphatase increased
    Additional description: Blood alkaline phosphatase increased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 6 (16.67%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    1
    Aspartate aminotransferase increased
    Additional description: Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    2 / 6 (33.33%)
    3 / 15 (20.00%)
         occurrences all number
    0
    0
    1
    2
    3
    Blood bicarbonate increased
    Additional description: Blood bicarbonate increased
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 6 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    1
    Transaminases increased
    Additional description: Transaminases increased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 6 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    0
    1
    Mean cell volume abnormal
    Additional description: Mean cell volume abnormal
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 6 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    1
    Gamma-glutamyltransferase increased
    Additional description: Gamma-glutamyltransferase increased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 6 (16.67%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    1
    C-reactive protein increased
    Additional description: C-reactive protein increased
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 6 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    1
    Blood creatinine increased
    Additional description: Blood creatinine increased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 6 (16.67%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    1
    Blood creatine phosphokinase increased
    Additional description: Blood creatine phosphokinase increased
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 6 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    0
    1
    Nervous system disorders
    Headache
    Additional description: Headache
         subjects affected / exposed
    2 / 3 (66.67%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    2 / 6 (33.33%)
    4 / 15 (26.67%)
         occurrences all number
    6
    0
    0
    2
    8
    Blood and lymphatic system disorders
    Anaemia
    Additional description: Anaemia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 6 (16.67%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    1
    Gastrointestinal disorders
    Abdominal pain
    Additional description: Abdominal pain
         subjects affected / exposed
    3 / 3 (100.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 6 (0.00%)
    4 / 15 (26.67%)
         occurrences all number
    6
    1
    0
    0
    7
    Nausea
    Additional description: Nausea
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 6 (16.67%)
    2 / 15 (13.33%)
         occurrences all number
    3
    0
    0
    2
    5
    Frequent bowel movements
    Additional description: Frequent bowel movements
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 6 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    1
    Dysphagia
    Additional description: Dysphagia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 6 (16.67%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    1
    Diarrhoea
    Additional description: Diarrhoea
         subjects affected / exposed
    1 / 3 (33.33%)
    2 / 3 (66.67%)
    0 / 3 (0.00%)
    1 / 6 (16.67%)
    4 / 15 (26.67%)
         occurrences all number
    1
    2
    0
    1
    4
    Constipation
    Additional description: Constipation
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    1 / 6 (16.67%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    1
    1
    2
    Abdominal pain upper
    Additional description: Abdominal pain upper
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    1 / 6 (16.67%)
    3 / 15 (20.00%)
         occurrences all number
    1
    1
    0
    1
    3
    Vomiting
    Additional description: Vomiting
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    2 / 6 (33.33%)
    3 / 15 (20.00%)
         occurrences all number
    0
    1
    0
    2
    3
    Hepatobiliary disorders
    Hepatic pain
    Additional description: Hepatic pain
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    1 / 6 (16.67%)
    2 / 15 (13.33%)
         occurrences all number
    0
    1
    0
    1
    2
    Skin and subcutaneous tissue disorders
    Hyperhidrosis
    Additional description: Hyperhidrosis
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    1 / 6 (16.67%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    1
    1
    2
    Night sweats
    Additional description: Night sweats
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 6 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
    Additional description: Arthralgia
         subjects affected / exposed
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 6 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    0
    0
    1
    Back pain
    Additional description: Back pain
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 6 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    1
    Pain in extremity
    Additional description: Pain in extremity
         subjects affected / exposed
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 6 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    0
    1
    Myalgia
    Additional description: Myalgia
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 6 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    1
    1
    0
    0
    2
    Infections and infestations
    Cystitis
    Additional description: Cystitis
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 6 (16.67%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    1
    Metabolism and nutrition disorders
    Hypokalaemia
    Additional description: Hypokalaemia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 3 (33.33%)
    0 / 6 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    0
    1
    Hyperkalaemia
    Additional description: Hyperkalaemia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 6 (16.67%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    1
    Decreased appetite
    Additional description: Decreased appetite
         subjects affected / exposed
    1 / 3 (33.33%)
    1 / 3 (33.33%)
    0 / 3 (0.00%)
    0 / 6 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    1
    1
    0
    0
    2
    Hyponatraemia
    Additional description: Hyponatraemia
         subjects affected / exposed
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    0 / 3 (0.00%)
    1 / 6 (16.67%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    14 May 2019
    Protocol version 2.0 dated 14-May-2019: addition of dermatological examination.
    24 Sep 2020
    Protocol version 3.0 dated 24-Sep-2020 : addition of 2 dose limiting toxicities, addition of TPOXX as rescue medication, addition of the collection of the history of smallpox vaccination and race/ethnicity data, modification of some inclusion criteria (excluding the patients at risk of hypertensive peaks, and correcting the hemoglobin limit), and addition of other EU countries.
    16 Dec 2020
    Protocol version 4.0 dated 16-Dec-2020: modification of some inclusion criteria (clarifying the absence of therapeutic alternatives and the standard anti-cancer treatment, modifying total bilirubin limit, adding alkaline phosphatase limit), addition of exclusion criteria (related to other malignancies, prior gene therapy, history of several reaction to smallpox vaccination, and specifying a visit with the cardiologist), and clarification some concomitant medication restrictions.
    18 Jan 2021
    Protocol version 5.0 dated 18-Jan-2021: update the study calendar and specify an exclusion criterion excluding protected adult.

    Interruptions (globally)
    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    23 Feb 2023
    The trial was prematurely terminated per Sponsor decision on 23 February 2023 after the completion of the phase I portion and before the commencement of the phase II, for reasons other than safety.
    -

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported.
    For support, Contact us.
    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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