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    Clinical Trial Results:
    A Phase I/II Dose-escalation and Expansion Cohort Trial of Intracerebroventricular Radioimmunotherapy Using 177Lu-DTPA-omburtamab in Pediatric and Adolescent Patients with Recurrent or Refractory Medulloblastoma

    Summary
    EudraCT number
    		 2020-000670-22
    Trial protocol
    		 GB   DK  
    Global end of trial date
    11 Aug 2022

    Results information
    Results version number
    		 v1(current)
    This version publication date
    28 Oct 2023
    First version publication date
    28 Oct 2023
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    301
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04167618
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Y-mAbs Therapeutics Inc.
    Sponsor organisation address
    230 Park Avenue, Suite 3350, New York, United States, NY 10169
    Public contact
    GRS associate, 'Y-mAbs Therapeutics Inc, +45 70261414, clinicaltrials@ymabs.com
    Scientific contact
    GRS associate, 'Y-mAbs Therapeutics Inc, +45 70261414, clinicaltrials@ymabs.com
    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
    16 Dec 2022
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    11 Aug 2022
    Global end of trial reached?
    Yes
    Global end of trial date
    11 Aug 2022
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective of Part 1 (dose-escalation phase) of this trial is to explore the tolerability of up to 2 cycles of intracerebroventricular 177Lu-DTPA-omburtamab treatment in pediatric and adolescent patients with recurrent or refractory medulloblastoma. The MTD and/or the recommended Phase 2 dose for Part 2 will be determined. The primary objective of Part 2 (cohort-expansion phase) of this trial is to establish a safety profile of repeated dosing of 177Lu-DTPA-omburtamab in pediatric and adolescent patients with recurrent or refractory medulloblastoma.
    Protection of trial subjects
    This trial will be conducted in accordance with the protocol and with the following: • Consensus ethical principles derived from international guidelines including the Declaration of Helsinki and Council for International Organizations of Medical Sciences International Ethical Guidelines • Ethical considerations for clinical trials on medicinal products conducted with minors • Applicable International Council for Harmonisation (ICH) Good Clinical Practice (GCP) Guidelines • Applicable laws and regulations. The protocol, protocol amendments, patient information, ICF, investigator’s brochure, and other relevant documents (e.g., advertisements) must be submitted to an Institutional Review Board (IRB)/Ethics Committee (EC) by the investigator and reviewed and approved by the IRB/EC before the trial is initiated. • Any amendments to the protocol will require regulatory and IRB/EC approval before implementation of changes made to the trial design, except for changes necessary to eliminate an immediate hazard to trial patients.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    30 Sep 2021
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Denmark: 1
    Country: Number of subjects enrolled
    United States: 1
    Worldwide total number of subjects
    2
    EEA total number of subjects
    1
    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)
    1
    Adolescents (12-17 years)
    1
    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
    		 All screening evaluations must be completed and reviewed to confirm that potential patients meet all eligibility criteria. The investigator will maintain a screening log to record details of all patients screened and to confirm eligibility or record reasons for screening failure, as applicable.

    Period 1
    Period 1 title
    Part 1 - dose escalation phase (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 10 mCi 177Lu-DTPA-omburtamab
    Arm description
    		 Intracerebroventricular administration of 10 mCi 177Lu-DTPA-omburtamab for up to two cycles (Part 1).
    Arm type
    		 Experimental

    Investigational medicinal product name
    177Lu-DTPA-omburtamab (Biological, radiolabeled DPTA-omburtamab)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intracerebroventricular use
    Dosage and administration details
    Intracerebroventricular administration of 10 mCi 177Lu-DTPA-omburtamab for up to two cycles (Part 1).

    Arm title
    		 25 mCi 177Lu-DTPA-omburtamab
    Arm description
    		 Intracerebroventricular administration of 25 mCi 177Lu-DTPA-omburtamab for up to two cycles (Part 1).
    Arm type
    		 Experimental

    Investigational medicinal product name
    177Lu-DTPA-omburtamab (Biological, radiolabeled DPTA-omburtamab)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intracerebroventricular use
    Dosage and administration details
    Intracerebroventricular administration of 25 mCi 177Lu-DTPA-omburtamab for up to two cycles (Part 1).

    Number of subjects in period 1
    		10 mCi 177Lu-DTPA-omburtamab 25 mCi 177Lu-DTPA-omburtamab
    Started
    1
    1
    Completed
    0
    0
    Not completed
    1
    1
         early termination of trial
    1
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    10 mCi 177Lu-DTPA-omburtamab
    Reporting group description
    		 Intracerebroventricular administration of 10 mCi 177Lu-DTPA-omburtamab for up to two cycles (Part 1).

    Reporting group title
    25 mCi 177Lu-DTPA-omburtamab
    Reporting group description
    		 Intracerebroventricular administration of 25 mCi 177Lu-DTPA-omburtamab for up to two cycles (Part 1).

    Reporting group values
    		 10 mCi 177Lu-DTPA-omburtamab 25 mCi 177Lu-DTPA-omburtamab Total
    Number of subjects
    		 1 1 2
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0
        Children (2-11 years)
    		 1 0 1
        Adolescents (12-17 years)
    		 0 1 1
        Adults (18-64 years)
    		 0 0 0
        From 65-84 years
    		 0 0 0
        85 years and over
    		 0 0 0
    Age continuous
    Units: years
        arithmetic mean (full range (min-max))
    		 15 (15 to 15) 8 (8 to 8) -
    Gender categorical
    Units: Subjects
        Female
    		 0 0 0
        Male
    		 1 1 2
    Race
    Units: Subjects
        White
    		 1 1 2

    End points

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    End points reporting groups
    Reporting group title
    10 mCi 177Lu-DTPA-omburtamab
    Reporting group description
    		 Intracerebroventricular administration of 10 mCi 177Lu-DTPA-omburtamab for up to two cycles (Part 1).

    Reporting group title
    25 mCi 177Lu-DTPA-omburtamab
    Reporting group description
    		 Intracerebroventricular administration of 25 mCi 177Lu-DTPA-omburtamab for up to two cycles (Part 1).

    Primary: Dose Limiting Toxicities (DLTs) Part 1

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    End point title
    		 Dose Limiting Toxicities (DLTs) Part 1 [1]
    End point description
    Summary of DLTs in DLT evaluable subjects.
    End point type
    Primary
    End point timeframe
    Days 1 through 35 in cycle 1
    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: DLTs summarised
    End point values
    		 10 mCi 177Lu-DTPA-omburtamab 25 mCi 177Lu-DTPA-omburtamab
    Number of subjects analysed
    1
    1
    Units: participants
    0
    1
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From 1st dose to 5 weeks after last dose, up to 10 weeks (2 cycles).
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24.1
    Reporting groups
    Reporting group title
    10 mCi 177Lu-DTPA-omburtamab
    Reporting group description
    		 Intracerebroventricular administration of 10 mCi 177Lu-DTPA-omburtamab for up to two cycles (Part 1). 177Lu-DTPA-omburtamab: Biological, radiolabeled DPTA-omburtamab

    Reporting group title
    25 mCi 177Lu-DTPA-omburtamab
    Reporting group description
    		 Intracerebroventricular administration of 25 mCi 177Lu-DTPA-omburtamab for up to two cycles (Part 1). 177Lu-DTPA-omburtamab: Biological, radiolabeled DPTA-omburta

    Serious adverse events
    		 10 mCi 177Lu-DTPA-omburtamab 25 mCi 177Lu-DTPA-omburtamab
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 1 (100.00%)
    0 / 1 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Nervous system disorders
    Partial seizures
         subjects affected / exposed
    1 / 1 (100.00%)
    0 / 1 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 10 mCi 177Lu-DTPA-omburtamab 25 mCi 177Lu-DTPA-omburtamab
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 1 (100.00%)
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    1
    Blood albumin decreased
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    1
    Neutrophil count decreased
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    1
    Platelet count decreased
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    1
    White blood cell count decreased
         subjects affected / exposed
    0 / 1 (0.00%)
    1 / 1 (100.00%)
         occurrences all number
    0
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    19 Nov 2021
    .• Section 1.2.1 (Summary of clinical information) updated • Table 1 replaced • 2.2.2.1 & 2.2.2.2: Evaluation timepoints added to the endpoints • Added: Final assessment of the eligibility criteria is done prior to first dose • EOT visit changed to 5-6 weeks after the last dose • Follow-up visits re-scheduled as per first dose • Figure 1 updated • 3.1.1.1 simplified • Adapted description of clinical trials (Trial 03-133 and Trial 101) and the non-clinical summary • The primary endpoint of Part 1 changed to number of DLTs and Part 2 changed to number and severity of TEAEs • Inclusion and exclusion criteria adapted • Follow up changed to every 13 weeks
    20 Apr 2022
    • Allowing a longer screening period • Adapted exclusion criterion #6 • Allowing delay in dosing due to logistic reasons • Adapting the sentinel dosing, so that the time frame is until first treatment dose (and not the dosimetry dose). • Removing follow-up period from Part 1 • Removing efficacy related objectives and endpoints in Part 1 • Removing the analyses of ctDNA & B7-H3 (including the applicable objectives and endpoints) • Schedule of Assessments updated

    Interruptions (globally)
    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    11 Aug 2022
    The trial was terminated after two subjects - due to a business strategy decision
    -

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    The trial was terminated after 2 subjects due to a business strategy decision. At this point the maximum tolerated dose was not established.
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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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