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    Summary
    EudraCT Number:2021-005086-41
    Sponsor's Protocol Code Number:B1030-101
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-05-13
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedGermany - PEI
    A.2EudraCT number2021-005086-41
    A.3Full title of the trial
    A First-in-Human, Phase 1/2, Dose Escalation Study of BOXR1030 T cells in Subjects With Advanced GPC3-Positive Solid Tumors.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Cell therapy clinical trial of BOXR1030 in GPC3 positive liver, squamous lung, Merkel cell cancer, and myxoid/round cell liposarcoma
    A.3.2Name or abbreviated title of the trial where available
    BOXR1030 Clinical Trial in GPC3 Positive Tumors
    A.4.1Sponsor's protocol code numberB1030-101
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05120271
    A.5.4Other Identifiers
    Name:IND NumberNumber:27903
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorSOTIO Biotech Inc
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportSOTIO Biotech a.s.
    B.4.2CountryCzechia
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSOTIO Biotech Inc
    B.5.2Functional name of contact pointClinical Trial information
    B.5.3 Address:
    B.5.3.1Street Address180 Canal Street, Suite 300
    B.5.3.2Town/ cityBoston
    B.5.3.3Post codeMA 02114
    B.5.3.4CountryUnited States
    B.5.6E-mailB1030_101@sotio.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.2Product code BOXR1030
    D.3.4Pharmaceutical form Dispersion for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms Yes
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Histologically confirmed advanced unresectable or metastatic hepatocellular carcinoma (HCC), squamous cell carcinoma (SCC) of the lung, myxoid/round cell liposarcoma, or Merkel cell carcinoma (MCC) with GPC3 overexpression by IHC, with a cytoplasmic/membranous H-score >30 for study eligibility. In the event of a tumor agnostic expansion cohort, other indications may be investigated.
    E.1.1.1Medical condition in easily understood language
    Patients with advanced, unresectable liver, squamous lung, and Merkel cell cancer, and myxoid/round cell liposarcoma that have tumor that is positive for GPC3 protein
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10077738
    E.1.2Term Hepatocellular carcinoma metastatic
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level PT
    E.1.2Classification code 10025120
    E.1.2Term Lung squamous cell carcinoma recurrent
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10073139
    E.1.2Term Round cell liposarcoma
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10064025
    E.1.2Term Merkel cell carcinoma
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    - To characterize the safety of BOXR1030 in the first 6 months after dosing in subjects with GPC3+ advanced malignancies

    - To determine the RP2D of BOXR1030 in GPC3+ solid tumors
    E.2.2Secondary objectives of the trial
    - To assess short-term antitumor activity of BOXR1030 in GPC3+ tumors, using RECIST 1.1 criteria (Schwartz et al., 2016)

    - To characterize BOXR1030 T-cell population, as well as expansion and persistence, in blood

    - To evaluate increases from baseline in inflammatory markers and cytokines as potential correlates of safety and/or antitumor activity

    - To characterize the long-term safety of BOXR1030 up to 15 years after administration
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Aged 18 to 80 years at time of enrollment

    2. Able to provide a recent tumor specimen taken since the time of the subject’s most recent systemic anticancer therapy, for GPC3 expression assessment by IHC.

    3. Histologically confirmed advanced unresectable or metastatic hepatocellular carcinoma (HCC), squamous cell carcinoma (SCC) of the lung, myxoid/round cell liposarcoma, or MCC with GPC3 overexpression by IHC, with a cytoplasmic/membranous H-score >30 for study eligibility.

    4. Documentation of disease progression or refractory disease or intolerance to prior lines of standard-of-care (SOC) therapies. Patients with tumors with genetic alterations and mutations who have approved targeted therapies available for their cancer will need to have been treated with such approved therapies or refused such approved targeted therapy for their cancer prior to enrolling in this study.

    5. Life expectancy >16 weeks

    6. Have adequate organ/renal function as defined by the protocol

    7. Left ventricular ejection fraction (LVEF) ≥50% by multiple-gated acquisition (MUGA) scan or echocardiogram (ECHO)

    8. Eastern Cooperative Oncology Group performance status of 0 to 1

    9. For subjects with HCC:
    9.1 Child-Pugh Score of A
    9.2 No fibrolamellar carcinoma or mixed hepatocellular cholangiocarcinoma histology
    9.3 No moderate or severe ascites

    10. A minimum of 2 sites of disease, including at least 1 site that is measurable by RECIST Version 1.1 criteria (Schwartz et al., 2016) to ensure sufficient disease for response assessment. At least 1 of the other lesions must be considered adequate for protocol-required
    tumor biopsy. Consider prioritization of percutaneous lesions that are palpable, or guided by imaging if necessary, and exclude biopsies of any lesions that are in proximity to vital visceral, cardio-pulmonary, or any neurovascular structures. A single site of disease is considered adequate to allow for response assessment and protocol-required tumor biopsies if it measures at least 2 cm in the shortest axis.

    11.1 Adequate wash-out of prior systemic therapy for underlying malignancy, relative to leukapheresis:
    11.1.a Last dose of any antineoplastic treatment must be at least 2 weeks before leukapheresis.
    11.1.b Last dose of any investigational agent must be at least 3 half-lives of the treatment, or 28 days, before leukapheresis (whichever is shorter).
    11.2 Adequate wash-out of prior systemic therapy for underlying malignancy, relative to LD chemotherapy:
    11.2.a The last dose of systemic nitrosourea or systemic mitomycin-C must be at least 6 weeks before the first dose of LD chemotherapy in this study.
    11.2.b For all other for systemic cytotoxic chemotherapy, the wash-out must be the duration of a full cycle of that therapy.
    11.2.c For biologic therapy (eg, antibodies) the wash-out must be either the duration of the biologic agent dosing interval, or 3 weeks, whichever is shorter.
    11.2.d For small molecule therapies, the wash-out must be 5 half-lives of the drug.
    11.2.e For any investigational agent, the wash-out must be 3 half-lives of the investigational agent, or 4 weeks, whichever is shorter.
    Note: Local radiation of lesions is allowed if indicated for palliation; locally treated lesions will be considered non-target lesions. Hormone ablation is also allowed as clinically indicated.

    12. Subjects or their legally acceptable representative must be able and willing to:
    12.1 Provide Institutional Review Board/Independent Ethics Committee (IRB/IEC)-approved written informed consent in accordance with regulatory and institutional guidelines. This
    must be obtained before the performance of any protocol-related procedures that are not part of normal patient care.
    12.2 Comply with the study protocol and with the planned biopsy procedures.

    13. Willing and able to commit to study assessments and visit schedule, including availability of a caregiver to conduct daily neurological assessments for 28 days after BOXR1030 administration.

    14.1 For women of childbearing potential (defined as physiologically capable of becoming pregnant), confirmation of a negative serum pregnancy test and agreement to use of highly effective contraception per Clinical Trials Facilitation and Coordination Group (CTFG) criteria from screening through the first 12 months after BOXR1030 administration.
    14.2 For men with partners of childbearing potential, agreement to use effective barrier contraception from screening through the first 12 months after BOXR1030 administration.
    E.4Principal exclusion criteria
    1. Prior treatment with adoptive cell therapy

    2. History of allogenic hematopoietic stem cell transplant (HSCT).

    3. Untreated central nervous system (CNS) tumors or brain metastasis unless they are asymptomatic, were treated and patients have neurologically returned to baseline. Imaging obtained for the purpose of CNS metastases management performed within 28 days prior to Day 1 must document radiographic stability of CNS lesions and be performed after completion of any CNS directed therapy. CNS evaluation for asymptomatic patients is not required for the study. Patients with leptomeningeal metastases are excluded.

    4. Patients who have not recovered to ≤ Grade 1 or baseline from all AEs due to previous therapies (patients with ≤ Grade 2 neuropathy that has been stable for at least 4 weeks or ≤Grade 2 endocrine-related AEs that has been stable for at least 4 weeks on replacement
    therapy).

    5. Planned use of any antineoplastic treatment or investigational agent from the time of the first dose of LD chemotherapy through the end of study participation, except for allowed local radiation of lesions for palliation (to be considered non-target lesions after treatment) and hormone ablation.

    6. Uncontrolled or life-threatening symptomatic concomitant disease including clinically significant gastrointestinal bleeding or pulmonary hemorrhage within 4 weeks before screening, known symptomatic human immunodeficiency virus (HIV) positive with an
    acquired immunodeficiency syndrome (AIDS)-defining opportunistic infection within the last year, or a current CD4 count <350 cells/uL, symptomatic active hepatitis B or C checked at screening, or active tuberculosis. Patients with HIV are eligible if:
    6.1 They have received antiretroviral therapy (ART) as clinically indicated for at least 4 weeks prior to starting study treatment
    6.2 They continue on ART as clinically indicated while enrolled on study
    6.3 CD4 counts and viral load are monitored per standard of care by a local health care provider

    7. Has undergone a major surgery within 3 weeks of starting study treatment or has inadequate healing or recovery from complications of surgery prior to starting study treatment.

    8. Has received prior radiotherapy within 2 weeks of start of study treatment. Subjects must have recovered from all radiation-related toxicities, not require corticosteroids, and not have had severe radiation pneumonitis. A 1-week wash-out is permitted for palliative radiation (≤2 weeks of radiotherapy) to non-CNS disease.

    9. Potentially life-threatening second malignancy requiring systemic treatment within the last 3 years (ie, patients with a history of prior malignancy are eligible if treatment was completed
    at least 3 years before entering the Treatment Period and the patient has no evidence of disease) or which would impede evaluation of treatment response. Hormone ablation therapy
    is allowed within the last 3 years. Patients with history of prior early stage basal/squamous cell skin cancer or non-invasive or in situ cancers that have undergone definitive treatment at any time are eligible.

    10. Clinically significant (i.e. active) cardiovascular disease: cerebral vascular accident/stroke (<6 months prior to enrollment), myocardial infarction (<6 months prior to enrollment), unstable angina, congestive heart failure (≥ New York Heart Association Classification Class II), or the presence of any condition that can increase proarrhythmic risk (eg, hypokalemia, bradycardia, heart block) including any new, unstable, or serious cardiac arrhythmia
    requiring medication, or other baseline arrhythmia that might interfere with interpretation of electrocardiograms (ECGs) on study (eg, bundle branch block). Patients with QTcF >450 msec for males and >470 msec for females on screening ECG are excluded. Any
    patients with a bundle branch block will be excluded with QTcF >450 msec. Males who are on stable doses of concomitant medication with known prolongation of QTcF (eg, selective
    serotonin re-uptake inhibitor antidepressants) will only be excluded for QTcF >470 msec.

    11. Has an active infection requiring systemic therapy.

    12. A woman of child-bearing potential (WOCBP) who has a positive pregnancy test prior to treatment.

    13. Is breastfeeding or expecting to conceive or father children within the projected duration of the study, starting with the Screening Visit through 6 months after the last dose of study
    treatment.

    14. Unable to receive any of the agents used in this study due to history of severe immediate hypersensitivity reaction (eg, hypersensitivity to dimethyl sulfoxide [DMSO]).

    15. Known allergy or contraindication to any of the LD chemotherapy or prophylaxis medications required during the study.

    16. Any other significant co-morbid disease or condition which, in the judgment of the Investigator, would put the subject at undue risk (eg, cirrhotic liver disease).
    E.5 End points
    E.5.1Primary end point(s)
    - DLT

    - RP2D

    - Incidence and severity of treatment-emergent AEs and clinically significant abnormalities of laboratory values
    E.5.1.1Timepoint(s) of evaluation of this end point
    DLT = 28days
    RP2D = 28 days
    Rest = 6 months
    E.5.2Secondary end point(s)
    - Investigator-assessed responses defined according to
    RECIST 1.1 criteria: ORR; BOR; DOR; PFS

    - OS

    - BOXR1030 T-cell levels in blood

    - BOXR1030 T-cell characterization in blood

    - Levels of inflammatory markers including, but not limited to, CRP, serum ferritin, ESR, and triglycerides

    - Levels of cytokines and other analytes in blood including, but not limited to, IFN-g, IL-1a, IL-1b, IL-6, IL-8, IL-10, GM-CSF, and TNF-a

    - Incidence and severity of select AEs

    - RCR qPCR results
    E.5.2.1Timepoint(s) of evaluation of this end point
    6 and 12 weeks
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans Yes
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA10
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    United States
    France
    Germany
    United Kingdom
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LPLV as defined by protocol
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 50
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 48
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state25
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 70
    F.4.2.2In the whole clinical trial 98
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Subjects will continue in the Long-term Follow-up Period (with decreased study visit frequency and limited
    study assessments) for up to 15 years after BOXR1030 administration.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-12-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-09-01
    P. End of Trial
    P.End of Trial StatusOngoing
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