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    Clinical Trial Results:
    Phase I/II Study of CaspaCIDe T Cells (BPX-501; Rivogenlecleucel) From an HLA Partially Matched Family Donor After Negative Selection of TCRαβ+ T Cells in Paediatric Patients Affected by Haematological Disorders.

    Summary
    EudraCT number
    2014-000584-41
    Trial protocol
    IT   GB   ES  
    Global end of trial date

    Results information
    Results version number
    v1(current)
    This version publication date
    11 Feb 2023
    First version publication date
    11 Feb 2023
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    BP-004
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT02065869
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Bellicum Pharmaceuticals, Inc.
    Sponsor organisation address
    3730 Kirby Drive, Suite 1200 , Houston, United States, 77098
    Public contact
    Rivogenlecleucel Study Team, Bellicum Pharmaceuticals, Inc., +1 (832) 384 1100, clinicaltrials@bellicum.com
    Scientific contact
    Rivogenlecleucel Study Team, Bellicum Pharmaceuticals, Inc., +1 (832) 384 1100, clinicaltrials@bellicum.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    EMEA-001870-PIP01-15 EMEA-001869-PIP01-15
    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
    Interim
    Date of interim/final analysis
    07 Sep 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    08 Feb 2019
    Global end of trial reached?
    No
    General information about the trial
    Main objective of the trial
    This is a Phase I/II study evaluating the safety and feasibility of BPX-501 T cells infused after partially mismatched, related, TCR alpha beta T cell depleted hematopoietic stem cell transplant (HSCT) in pediatric patients. The purpose of this clinical trial is to determine whether BPX-501 infusion can enhance immune reconstitution in those patients with hematologic disorders, with the potential for reducing the severity and duration severe acute graft versus host disease (GvHD). The trial will also evaluate the treatment of GvHD by the infusion of dimerizer drug (AP1903/rimiducid) in those subjects who present with GVHD who progress or do not respond to standard of care treatment.
    Protection of trial subjects
    The study was conducted according to the study protocol, the ethical principles of the declaration of Helsinki, the International Council for Harmonisation (ICH) Good Clinical Practice (GCP) Guidelines, the EU Clinical Trial Directive (2001/120/EG), the Italian Ministry of Health decree of 15 Jul 1997, the Food and Drug Administration (FDA), and other international regulatory agencies. All investigators agreed to conduct all aspects of this study in accordance with national and local laws and regulations. Before study onset, the original protocol, the informed consent form (ICF) and any other written information regarding this study were reviewed by the relevant Independent Ethics Committee (IEC). Written informed consent was obtained from the patient and/or their parent/legal guardian and donor before the performance of any study-specific procedure. The patient/donor or legal guardian was given clear explanations about the nature, scope and possible consequences and risks of the clinical study by the investigator. Information was provided both in writing (patient information sheet [PIS]) and verbally with ample opportunity provided to ask questions and decide whether to participate in this study. It was also made clear to patients that they could withdraw from the trial at any time without giving a reason. Patients who turned 18 years of age during the study completed an adult ICF at that time. The ICFs along with a declaration on data privacy were signed and dated by both the informing physician and the donor/patient or their legal guardian before the beginning of the study. A copy of the signed ICF was given to the patient. To ensure medical confidentiality and data protection, the signed ICFs were stored in the investigator's site file and retained within it.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    09 Apr 2014
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    Safety
    Long term follow-up duration
    15 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: 22
    Country: Number of subjects enrolled
    Italy: 162
    Worldwide total number of subjects
    184
    EEA total number of subjects
    162
    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)
    43
    Children (2-11 years)
    105
    Adolescents (12-17 years)
    35
    Adults (18-64 years)
    1
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    -

    Pre-assignment
    Screening details
    Screening activities occurred after consenting. The screening procedures included medical and cancer history, physical and neurological exams, vital signs, scans, bone marrow aspirate, and laboratory tests (chimerism, hematology, chemistry, immune function flow panel, immunoglobulins, infectious disease titers, HLA typing, HAMA, RCR, pregnancy test

    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
    BPX-501 T Cells and Rimiducid
    Arm description
    TCR alpha beta depleted graft infusion with addback of BPX-501 T cells (rivogenlecleucel). Rimiducid/AP1903: Dimerizer drug administered to subjects who develop Grade III-IV acute GVHD, Grade II gut/liver acute GVDH or Grade I/II skin-only acute GvHD which is non-responsive after 7 days of standard of care treatment.BPX-501 T cells: 1x10E6 cells/kg infused on Day 0 Rimiducid: 0.4mg/kg administered IV to treat GVHD
    Arm type
    Experimental

    Investigational medicinal product name
    Rivogenlecleucel
    Investigational medicinal product code
    Other name
    BPX-501
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    In Phase 1, rivogenlecleucel was administered via intravenous infusion at 3 different escalating doses (0.25×10^6, 0.5×10^6 and 1×10^6 cells/kg recipient total body weight) in all patient populations. Two further escalation doses of 2×10^6 and 4×10^6 cells/kg recipient total body weight were evaluated in patients with malignant disease only. In Phase 2, patients (with either malignant or non-malignant haematological diseases) were infused with rivogenlecleucel at the 1×10^6 cells/kg dose.

    Investigational medicinal product name
    Rimiducid
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    0.4 mg/kg rimiducid was administered via intravenous infusion to patients who received rivogenlecleucel and developed GvHD which progressed or did not respond within 7 days to standard of care treatment

    Number of subjects in period 1
    BPX-501 T Cells and Rimiducid
    Started
    184
    Patients Receiving Rivogenlecleucel
    171
    ITT Population
    142 [1]
    Patients Receiving ≥ 1 Dose of Rimiducid
    16 [2]
    Completed
    149
    Not completed
    35
         Death
    7
         No rivogenlecleucel cells infused (non-evaluable)
    7
         Disease relapse
    16
         Unknown
    1
         Lost to follow-up
    4
    Notes
    [1] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: 184 subjects were enrolled in the study. 171 subjects received BPX-501 T cells and rimiducid. Of these subjects, 142 were placed in the intent-to-treat population i.e. patients treated with haematopoietic stem cell transplantation (HSCT) and who received a dose of BPX-501 at 1x10^6 cells/kg. 16 subjects received at least 1 dose of rimiducid for the treatment of acute or chronic GvHD refractory to standard of care treatment
    [2] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: 184 subjects were enrolled in the study. 171 subjects received BPX-501 T cells and rimiducid. Of these subjects, 142 were placed in the intent-to-treat population i.e. patients treated with haematopoietic stem cell transplantation (HSCT) and who received a dose of BPX-501 at 1x10^6 cells/kg. 16 subjects received at least 1 dose of rimiducid for the treatment of acute or chronic GvHD refractory to standard of care treatment

    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
    184 184
    Age categorical
    Units: Subjects
        In utero
    0
        Preterm newborn infants (gestational age < 37 wks)
    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)
    0
        From 65-84 years
    0
        85 years and over
    0
    Age continuous
    Units: years
        median (full range (min-max))
    5.8 (0.1 to 18.1) -
    Gender categorical
    Units: Subjects
        Female
    83 83
        Male
    101 101

    End points

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    End points reporting groups
    Reporting group title
    BPX-501 T Cells and Rimiducid
    Reporting group description
    TCR alpha beta depleted graft infusion with addback of BPX-501 T cells (rivogenlecleucel). Rimiducid/AP1903: Dimerizer drug administered to subjects who develop Grade III-IV acute GVHD, Grade II gut/liver acute GVDH or Grade I/II skin-only acute GvHD which is non-responsive after 7 days of standard of care treatment.BPX-501 T cells: 1x10E6 cells/kg infused on Day 0 Rimiducid: 0.4mg/kg administered IV to treat GVHD

    Primary: Event-free Survival (EFS) at 180 Days After Transplant

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    End point title
    Event-free Survival (EFS) at 180 Days After Transplant [1]
    End point description
    Events included transplant-related mortality (TRM) / non-relapse mortality (NRM), severe GvHD (acute Grades 2-4 organ or extensive chronic GvHD) and life-threatening infections (Grade 4). Time to the first event only is represented in the primary endpoint, if a subsequent event occurred in the same patient this was not captured in this outcome.
    End point type
    Primary
    End point timeframe
    180 days after transplant
    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: No comparator in the study so statistical analysis not feasible. Kaplan-Meier technique was used to estimate event free survival at Day 180.
    End point values
    BPX-501 T Cells and Rimiducid
    Number of subjects analysed
    142
    Units: Kaplan Meier EFS Estimate (%)
        number (confidence interval 95%)
    90.8 (84.6 to 94.5)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Within 180 days post BPX-501 or 30 days post Rimiducid
    Adverse event reporting additional description
    Analysis of safety, regardless of study treatment and in relation to BPX-501, was conducted with the BPX-501 Safety Population (patients who received HSCT and subsequently received a dose of BPX-501) Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population (patients who received at least 1 dose of rimiducid)
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    18.0
    Reporting groups
    Reporting group title
    Rivogenlecleucel
    Reporting group description
    Analysis of safety, regardless of study treatment and in relation to rivogenlecleucel (BPX-501), was conducted with the rivogenlecleucel Safety Population (patients who received HSCT and subsequently received any dose of rivogenlecleucel)

    Reporting group title
    Rimiducid
    Reporting group description
    Rimiducid/AP1903 (0.4 mg/kg) is a dimerizer drug administered via intravenous infusion to subjects who develop Grade III-IV acute GVHD, Grade II gut/liver acute GVDH or Grade I/II skin-only acute GvHD which is non-responsive after 7 days of standard of care treatment. Analysis of safety in relation to rimiducid treatment was performed with the Rimiducid Population, (patients who received at least 1 dose of rimiducid)

    Serious adverse events
    Rivogenlecleucel Rimiducid
    Total subjects affected by serious adverse events
         subjects affected / exposed
    52 / 171 (30.41%)
    7 / 16 (43.75%)
         number of deaths (all causes)
    7
    0
         number of deaths resulting from adverse events
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Juvenile chronic myelomonocytic leukaemia
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    Vascular disorders
    Hypotension
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pelvic venous thrombosis
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Device damage
         subjects affected / exposed
    2 / 171 (1.17%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Malaise
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Medical device complication
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pyrexia
         subjects affected / exposed
    7 / 171 (4.09%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 7
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Immune system disorders
    Acute graft versus host disease
         subjects affected / exposed
    4 / 171 (2.34%)
    2 / 16 (12.50%)
         occurrences causally related to treatment / all
    2 / 4
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Acute respiratory distress syndrome
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    Cardiopulmonary failure
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    Pneumonitis
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pulmonary haemorrhage
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    1 / 1
    0 / 0
    Pulmonary hypertension
         subjects affected / exposed
    1 / 171 (0.58%)
    1 / 16 (6.25%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory distress
         subjects affected / exposed
    1 / 171 (0.58%)
    1 / 16 (6.25%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac disorders
    Pericardial effusion
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumopericardium
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Central nervous system lesion
         subjects affected / exposed
    1 / 171 (0.58%)
    1 / 16 (6.25%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Encephalopathy
         subjects affected / exposed
    1 / 171 (0.58%)
    1 / 16 (6.25%)
         occurrences causally related to treatment / all
    0 / 1
    1 / 1
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    Miller Fisher syndrome
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Posterior reversible encephalopathy syndrome
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Autoimmune haemolytic anaemia
         subjects affected / exposed
    2 / 171 (1.17%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cytopenia
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Haemolytic anaemia
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Histiocytosis haematophagic
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Neutropenia
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Thrombocytopenia
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Thrombotic microangiopathy
         subjects affected / exposed
    2 / 171 (1.17%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Eye disorders
    Diplopia
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    2 / 171 (1.17%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal haemorrhage
         subjects affected / exposed
    1 / 171 (0.58%)
    1 / 16 (6.25%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 1
         deaths causally related to treatment / all
    1 / 1
    0 / 0
    Haematemesis
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pancreatitis
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Parotid gland enlargement
         subjects affected / exposed
    1 / 171 (0.58%)
    1 / 16 (6.25%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Vomiting
         subjects affected / exposed
    2 / 171 (1.17%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Cystitis haemorrhagic
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Adenovirus infection
         subjects affected / exposed
    2 / 171 (1.17%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Bacterial sepsis
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cytomegalovirus infection
         subjects affected / exposed
    3 / 171 (1.75%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Device related infection
         subjects affected / exposed
    3 / 171 (1.75%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Encephalitis
         subjects affected / exposed
    2 / 171 (1.17%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    1 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Escherichia infection
         subjects affected / exposed
    2 / 171 (1.17%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastroenteritis rotavirus
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infection
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Klebsiella infection
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pharyngitis
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pseudomonas infection
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory tract infection
         subjects affected / exposed
    1 / 171 (0.58%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Sepsis
         subjects affected / exposed
    1 / 171 (0.58%)
    1 / 16 (6.25%)
         occurrences causally related to treatment / all
    1 / 1
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Septic shock
         subjects affected / exposed
    1 / 171 (0.58%)
    1 / 16 (6.25%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    1 / 1
    0 / 0
    Staphylococcal bacteraemia
         subjects affected / exposed
    1 / 171 (0.58%)
    1 / 16 (6.25%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Staphylococcal infection
         subjects affected / exposed
    1 / 171 (0.58%)
    1 / 16 (6.25%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Staphylococcal sepsis
         subjects affected / exposed
    2 / 171 (1.17%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Varicella zoster virus infection
         subjects affected / exposed
    2 / 171 (1.17%)
    0 / 16 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Rivogenlecleucel Rimiducid
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    140 / 171 (81.87%)
    9 / 16 (56.25%)
    Cardiac disorders
    Right ventricular failure
         subjects affected / exposed
    1 / 171 (0.58%)
    1 / 16 (6.25%)
         occurrences all number
    1
    1
    Nervous system disorders
    Encephalopathy
         subjects affected / exposed
    1 / 171 (0.58%)
    1 / 16 (6.25%)
         occurrences all number
    1
    1
    Central nervous system lesion
         subjects affected / exposed
    1 / 171 (0.58%)
    1 / 16 (6.25%)
         occurrences all number
    1
    1
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    33 / 171 (19.30%)
    0 / 16 (0.00%)
         occurrences all number
    33
    0
    Immune system disorders
    Chronic graft versus host disease
         subjects affected / exposed
    5 / 171 (2.92%)
    1 / 16 (6.25%)
         occurrences all number
    5
    1
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    20 / 171 (11.70%)
    0 / 16 (0.00%)
         occurrences all number
    20
    0
    Gastrointestinal haemorrhage
         subjects affected / exposed
    1 / 171 (0.58%)
    1 / 16 (6.25%)
         occurrences all number
    1
    1
    Nausea
         subjects affected / exposed
    10 / 171 (5.85%)
    0 / 16 (0.00%)
         occurrences all number
    10
    0
    Parotid gland enlargement
         subjects affected / exposed
    1 / 171 (0.58%)
    1 / 16 (6.25%)
         occurrences all number
    1
    1
    Vomiting
         subjects affected / exposed
    20 / 171 (11.70%)
    0 / 16 (0.00%)
         occurrences all number
    20
    0
    Respiratory, thoracic and mediastinal disorders
    Pulmonary hypertension
         subjects affected / exposed
    1 / 171 (0.58%)
    1 / 16 (6.25%)
         occurrences all number
    1
    1
    Respiratory distress
         subjects affected / exposed
    2 / 171 (1.17%)
    1 / 16 (6.25%)
         occurrences all number
    2
    1
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    12 / 171 (7.02%)
    0 / 16 (0.00%)
         occurrences all number
    12
    0
    Rash
         subjects affected / exposed
    21 / 171 (12.28%)
    0 / 16 (0.00%)
         occurrences all number
    21
    0
    Skin discolouration
         subjects affected / exposed
    1 / 171 (0.58%)
    1 / 16 (6.25%)
         occurrences all number
    1
    1
    Psychiatric disorders
    Confusional state
         subjects affected / exposed
    1 / 171 (0.58%)
    1 / 16 (6.25%)
         occurrences all number
    1
    1
    Infections and infestations
    Acute graft versus host disease
         subjects affected / exposed
    48 / 171 (28.07%)
    3 / 16 (18.75%)
         occurrences all number
    48
    3
    Adenovirus infection
         subjects affected / exposed
    11 / 171 (6.43%)
    0 / 16 (0.00%)
         occurrences all number
    11
    0
    Cytomegalovirus infection A
         subjects affected / exposed
    34 / 171 (19.88%)
    0 / 16 (0.00%)
         occurrences all number
    34
    0
    Human herpesvirus 6 infection
         subjects affected / exposed
    9 / 171 (5.26%)
    0 / 16 (0.00%)
         occurrences all number
    9
    0
    Sepsis
         subjects affected / exposed
    1 / 171 (0.58%)
    1 / 16 (6.25%)
         occurrences all number
    1
    1
    Septic shock
         subjects affected / exposed
    1 / 171 (0.58%)
    1 / 16 (6.25%)
         occurrences all number
    1
    1
    Staphylococcal infection
         subjects affected / exposed
    1 / 171 (0.58%)
    1 / 16 (6.25%)
         occurrences all number
    1
    1
    Metabolism and nutrition disorders
    Hypokalaemia
         subjects affected / exposed
    2 / 171 (1.17%)
    1 / 16 (6.25%)
         occurrences all number
    2
    1

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    23 Oct 2014
    Version 2.0
    17 Dec 2014
    Version 3.0
    05 Mar 2015
    Version 4.0
    30 Oct 2015
    Version 5.1 (Italy)
    15 Nov 2015
    Version 6.0 (Italy)
    22 Apr 2016
    Version 7.0 (Italy)
    08 Nov 2016
    Version 8.0 (Italy)
    10 Feb 2017
    Version 9.0 (Italy)
    10 Feb 2017
    Version 5.0 (UK)
    02 Apr 2018
    Version 10.0 (Italy)
    02 Apr 2018
    Version 6.0 (UK)
    29 Oct 2018
    Version 11.0 (Italy)
    01 Nov 2018
    Version 7.0 (UK)
    30 Jun 2020
    Version 8.0 (UK)

    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.
    None reported
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