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    Summary
    EudraCT Number:2020-001590-68
    Sponsor's Protocol Code Number:03-133
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2020-11-26
    Trial results View 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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2020-001590-68
    A.3Full title of the trial
    Phase I Study Of Intrathecal Radioimmunotherapy Using 131I-Omburtamab for Central Nervous System/Leptomeningeal Neoplasms
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Phase I Study Of Intrathecal Radioimmunotherapy Using 131I-Omburtamab for Central Nervous System/Leptomeningeal Neoplasms
    A.4.1Sponsor's protocol code number03-133
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/322/2020
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorY-mAbs Therapeutics 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 supportY-mAbs Therapeutics Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationY-mAbs Therapeutics Inc
    B.5.2Functional name of contact pointclinical department
    B.5.3 Address:
    B.5.3.1Street Address230 Park Avenue, Suite 3350
    B.5.3.2Town/ cityNew York, NY
    B.5.3.3Post code10169
    B.5.3.4CountryUnited States
    B.5.4Telephone number+16468858477
    B.5.6E-mailRAB@ymabs.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/17/1839
    D.3 Description of the IMP
    D.3.1Product name131I-omburtamab
    D.3.2Product code 131I-murine 8H9
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPIntracerebroventricular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNomburtamab
    D.3.9.1CAS number 1895083-75-6
    D.3.9.2Current sponsor code131I-omburtamab
    D.3.9.3Other descriptive name131I-BURTOMAB
    D.3.9.4EV Substance CodeSUB188981
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number0.8 to 1.2
    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) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    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 Yes
    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 Yes
    D.3.11.10Medicinal product containing genetically modified organisms No
    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
    Treatment of pediatric neuroblastoma patients with CNS relapse as
    evidenced by CNS/LM metastases
    E.1.1.1Medical condition in easily understood language
    Treatment of pediatric neuroblastoma patients with CNS relapse as
    evidenced by CNS/LM metastases
    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 PT
    E.1.2Classification code 10073130
    E.1.2Term Central nervous system neuroblastoma
    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 assess the overall survival at 3 years after the first treatment dose of 131I-omburtamab.
    E.2.2Secondary objectives of the trial
    To evaluate efficacy in terms of CNS/LM progression-free survival (CNS/LM PFS) at 12 month
    To evaluate long term safety.
    To evaluate pharmacokinetics and dosimetry after first dosimetry dose
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patients must have a histologically confirmed diagnosis of a malignancy known to be Omburtamab reactive. Omburtamab expression must be confirmed by immunohistochemical staining of tumor and assessed by the Department of Pathology or by immunofluorescence of bone marrow except for patients confirmed to have neuroblastoma5 or an embryonal tumor (such as medulloblastoma, retinoblastoma, rhabdomyosarcoma and DSRCT).
    2. Patients must have CNS/ leptomeningeal disease which is refractory to conventional therapies or for which no conventional therapy exists OR a recurrent brain tumor with a predilection for leptomeningeal dissemination (PNET, rhabdoid tumor).
    3. Patients must have no rapidly progressing or deteriorating neurologic examination.
    4. Patients must have an absolute neutrophil count (ANC) > 1000/ul and a platelet count > 50,000/ul.
    5. Patients may have active malignancy outside the central nervous system.
    6. Both pediatric and adult patients of any age are eligible.
    7. Signed informed consent indicating awareness of the investigational nature of this program.
    8. Patients with stored stem cells will be treated at the escalating dose while patients with no stem cells will be treated at the 50 mCi dose. Neuroblastoma patients can be treated at the 50 mCi dose with or without stored stem cells.
    E.4Principal exclusion criteria
    1. Patients with obstructive or symptomatic communicating hydrocephalus.
    2. Patients with an uncontrolled life-threatening infection.
    3. Patients who are pregnant: Pregnant women are excluded for fear of danger to the fetus. Therefore, negative pregnancy test is required for all women of child-bearing age, and appropriate contraception is required during the study period.
    4. Patients who have received cranial or spinal irradiation less than 3 weeks prior to the start of this protocol.
    5. Patients who have received systemic chemotherapy (corticosteroids and immunotherapies not included) less than 3 weeks prior to the start of this protocol.
    6. Severe major organ toxicity. Specifically, renal, cardiac, hepatic, pulmonary, and gastrointestinal system toxicity should all be less than grade 2. Patients with stable neurological deficits (because of their brain tumor) are not excluded. Patients with <= 3 hearing loss are not excluded.
    E.5 End points
    E.5.1Primary end point(s)
    Overall survival rate at 3 years
    E.5.1.1Timepoint(s) of evaluation of this end point
    3 years
    E.5.2Secondary end point(s)
    CNS/LM progression-free survival (CNS/LM PFS) at 12 month
    Long term safety (measured throughout the entire study)
    Pharmacokinetics and dosimetry after first dosimetry dose
    E.5.2.1Timepoint(s) of evaluation of this end point
    See section E.5.2 for a specification of the timepoints of evaluations for each of the secondary endpoints
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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 No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Dose-escalation
    E.7.2Therapeutic exploratory (Phase II) No
    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 Yes
    E.8.1.7.1Other trial design description
    Dose-escalation
    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.8.3 Will this trial be conducted at a single site globally? Yes
    E.8.4 Will this trial be conducted at multiple sites globally? No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    United States
    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
    End of trial is 15 July 2019 (not LPLV).

    Protocol 03-133 stopped for enrolment of patients whose primary cancer is neuroblastoma in December 2018. The non-neuroblastoma patients part of the trial continued thereafter, and stopped for enrolment in July 2019. Due to long-term follow-up, LPLV has not been defined for the overall protocol.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years15
    E.8.9.2In all countries concerned by the trial months5
    E.8.9.2In all countries concerned by the trial days10
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 93
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 5
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 84
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 4
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Patients could be between the ages of birth and 18 years and therefore informed consent from legal guardian(s) and/or child must be obtained in accordance with local regulation. Pediatric patients must provide assent as required by local regulations.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 93
    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)
    None
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.3.4Network Country United States
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
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