Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

  • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).


    The EU Clinical Trials Register currently displays   43801   clinical trials with a EudraCT protocol, of which   7263   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2020-003200-14
    Sponsor's Protocol Code Number:JR-141-GS31
    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:2021-06-15
    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 number2020-003200-14
    A.3Full title of the trial
    A Phase III study of JR-141 in Mucopolysaccharidosis type II (Hunter Syndrome) patients.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase III study of JR-141 in Hunter Syndrome patients
    A.3.2Name or abbreviated title of the trial where available
    STARLIGHT
    A.4.1Sponsor's protocol code numberJR-141-GS31
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04573023
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorJCR Pharmaceuticals Co., Ltd.
    B.1.3.4CountryJapan
    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 supportJCR Pharmaceuticals Co., Ltd.
    B.4.2CountryJapan
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationJCR Pharmaceuticals Co., Ltd.
    B.5.2Functional name of contact pointInternational Project Unit
    B.5.3 Address:
    B.5.3.1Street Address3-19 Kasuga-cho, Ashiya
    B.5.3.2Town/ cityHyogo
    B.5.3.3Post code659-0021
    B.5.3.4CountryJapan
    B.5.4Telephone number+81 797328582
    B.5.5Fax number+81797343897
    B.5.6E-mailclinical_development@jp.jcrpharm.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/3/19/2140
    D.3 Description of the IMP
    D.3.1Product namepabinafusp alfa
    D.3.2Product code JR-141
    D.3.4Pharmaceutical form Lyophilisate and solvent for suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSolution for injection (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPabinafusp alfa
    D.3.9.1CAS number 2140211-48-7
    D.3.9.3Other descriptive nameJR-141
    D.3.9.4EV Substance CodeSUB193542
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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 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 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.IMP: 2
    D.1.2 and D.1.3IMP RoleComparator
    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 Yes
    D.2.1.1.1Trade name Elaprase
    D.2.1.1.2Name of the Marketing Authorisation holderTakeda Pharmaceuticals International AG Ireland Branch
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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/3/01/078
    D.3 Description of the IMP
    D.3.1Product nameElaprase
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNIdursulfase
    D.3.9.1CAS number 50936-59-9
    D.3.9.4EV Substance CodeSUB22927
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    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 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 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
    Mucopolysaccharidosis type II
    E.1.1.1Medical condition in easily understood language
    Hunter syndrome
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10056889
    E.1.2Term Mucopolysaccharidosis II
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    * To demonstrate the efficacy of JR-141 on CNS symptoms in MPS II patients
    * To demonstrate the efficacy of JR-141 on somatic symptoms in MPS II patients
    * To evaluate the safety of JR-141 in MPS II patients
    * To evaluate the PKs of JR-141 in MPS II patients

    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. A patient who voluntarily signs an IRB or Independent Ethics Committee (IEC)-approved written ICF. If the patient is aged under 18
    years (aged under 16 years in the UK) at the time of enrollment or willingness to participate in the study cannot be confirmed due to MPS
    II-related intellectual disability, the patient's legally acceptable representative (e.g., his parents or guardians) may sign the informed
    consent on behalf of the patient. Written informed assent should be obtained from the patient, wherever possible.
    2. Patients with confirmed diagnosis of MPS II, based on all of the following criteria:
    1) Deficient activity of IDS in leucocytes, plasma or fibroblasts defined
    by 10% or less of the lower limit of the measuring laboratory normal
    range unless the hospital or laboratory has established different criteria
    2) Documented mutation identified in the IDS gene
    3) Increased levels of urinary glycosaminoglycans (GAGs) (or uronic acid) or clinical symptoms and signs consistent with MPS II (such as
    dysostosis multiplex, coarse facies, cardiac valve disease, neuronopathic, chronic pulmonary disease, hernias, kyphosis, joint contractures, carpal tunnel syndrome, etc)
    3. Naïve patients or patients who are receiving stable enzyme replacement therapy (ERT) with idursulfase for more than 12 weeks
    before starting administration of JR-141 or idursulfase for this study.
    4. Cohort A
    - Patients aged 36-42 months old at the time of ICF signing: patients
    must have a standard score on the cognitive domain measured by the
    BSID-III of 85 or less at screening
    - Patients aged 43-71 months old at the time of ICF signing: patients
    must EITHER have
    1. A development quotient (DQ) on the cognitive domain measured by the BSID-III between 20 and 85 at screening
    2. A composite standard score on Nonverbal Index (NVI) measured by the KABC-II of 85 or less at screening for only who are able to perform
    the KABC-II
    - Patients aged 30-35 months old at the time of randomization and who are judged as having the severe phenotype by the Expert Board based on
    presence of one of the following mutations in the IDS gene and other information such as high CSF HS concentrations:
    1) Large deletion or rearrangement
    2) Small insertions or deletions that are out of frame
    3) Missense mutations, nonsense mutations, in frame inserts or deletions that involve neuronopathic disease in either another family or
    the patient's family members.
    5. Cohort B
    - Patients 6 years of age or older at the time of ICF signing.
    - Intelligence quotient (IQ) measured by the Wechsler test (WISC-V, or
    WAIS-IV) is 70 or higher at screening.
    - Enrollment of subjects in Cohort B is contingent on the availability in
    that country of a validated country-specific version of the test (either
    WISC-V, WIAS-IV, or T.O.V.A.).
    - Patients with 1SD deficiency in the omission errors or variability domains of the T.O.V.A. test or Processing Speed or Working memory on
    the Wechsler tests at screening
    6. Patients or patients whose partners are of child-bearing potential agree to use a medically accepted, highly effective method of contraception being
    use of condoms from the time of informed consent to 90 days after the final administration or vasectomy at least 13 weeks prior to signing
    informed consent. Alternatively, true abstinence is acceptable when it is in line with the subject's preferred and usual lifestyle. If a subject is
    usually not sexually active but becomes active, they, must comply with the contraceptive requirements detailed above. Study participants are
    strongly encouraged to discuss with their Partners the use of an additional approved method of effective contraception such as:
    • Partner's use of combined (estrogen and progestogen-containing)
    hormonal contraception associated with inhibition of ovulation:
    - oral
    - intravaginal
    - transdermal
    • Partner's use of progestogen-only hormonal contraception:
    - oral
    - injectable/implantable
    - intrauterine hormone-releasing system (IUS)
    • Partner's use of implantable intrauterine device (IUD)
    • Surgical sterilization (for example, vasectomy or bilateral tubal
    occlusion)
    • Partner's use of female cap or diaphragm (double barrier)

    7. For subjects with hearing impairment requiring hearing aid(s), every effort has been made to encourage compliance with the use of
    functioning hearing aid(s) before baseline neurocognitive assessments, and parent/legally acceptable representative or subject agrees to
    encourage wearing them during the study and on neurocognitive testing days.


    E.4Principal exclusion criteria
    1. A patient with a history of engrafted hematopoietic stem cell transplantation (HSCT), with successful engraftment.
    2. A patient who has received gene therapy treatment at any point.
    3. A patient who is judged by the principal investigator or sub-investigator as being unable to undergo lumbar puncture, including
    those who has difficulties in taking position for lumbar puncture due to joint contracture or those who is likely to experience breathing
    difficulties during the lumbar puncture process.
    4. A patient who is enrolled in another clinical study that involves clinical investigations or use of any investigational product (drug or device) within 4 months before obtaining informed consent.
    5. A patient who is unable to comply with the protocol (e.g. is unable to return for safety evaluations or is otherwise unlikely to complete the study) as determined by the principal investigator or sub-investigator.
    6. A patient who is judged by the principal investigator or sub-investigator to be ineligible to participate in the study due to a history of serious drug allergy or sensitivity including anesthesia or hypersensitivity to any component of JR-141 or idursulfase.
    7. A patient who has a known or suspected local or general infection or
    is at risk of abnormal bleeding due to medical conditions* or therapies
    the investigator classifies as causing the patient to be ineligible to
    participate in the study.
    8. A patient who has documented mutation of other genes, including loci adjacent to the IDS gene (e.g., fragile X mental retardation [FMR1] or
    AF4/FMR2 family member 2[i.e., AFF2 or FMR2]), that are known to be associated with developmental delay, seizures, or other significant CNS
    disorders.
    9. A patient who has documented loss of activity of sulfatases other than IDS, indicating multiple sulfatase deficiency.
    10. A patient who has had a ventriculoperitoneal (VP) shunt placed or any other brain surgery, or has a clinically significant VP shunt
    malfunction within 30 days of screening (Patients may be rescreened after the 30-day waiting period has elapsed).
    11. A patient who is full time employee of the Sponsor or research site personnel directly affiliated with this study or their immediate family
    members, defined as a spouse, parent, child, or sibling, whether biological or legally adopted.
    12. A patient who otherwise is judged by the principal investigator or sub-investigator to be ineligible to participate in the study.
    * Medical Conditions:
    1. Clinically significant multiple or severe drug allergies, intolerance to topical corticosteroids, or severe post-treatment hypersensitivity reactions (including, but not limited to, erythema multiforme major, linear immunoglobulin A [IgA] dermatosis, toxic epidermal necrolysis, and exfoliative dermatitis)
    2. Evidence or history of significant active bleeding or coagulation disorder or use of non-steroidal anti-inflammatory drugs or other drugs that affect coagulation or platelet function within 14 days prior to lumbar catheter insertion
    3. Allergy to lidocaine (Xylocaine®) or its derivatives
    E.5 End points
    E.5.1Primary end point(s)
    - Change in level of CSF HS from baseline to Week 53 in Cohort A
    - Change in the raw scores from baseline to Week 105 measured by the
    BSID-III (cognitive domain) in Cohort A

    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 53 and Week 105
    E.5.2Secondary end point(s)
    - Change in the growth scores of cognitive domain measured by the
    BSID-III from baseline to Week 105 in Cohort A
    - Change in the age equivalent scores of adaptive behavior measured by
    the VABS-II -from baseline to Week 105 in Cohort A
    - Relative change in liver volume relative to body weight from baseline
    to Week 53 in Cohort A and Cohort B
    - Relative change in spleen volume relative to body weight from baseline
    to Week 53 in Cohort A and Cohort B
    - Relative change in distance walked using the 6-minute walk test from
    baseline to Week 53 in Cohort B
    E.5.2.1Timepoint(s) of evaluation of this end point
    Central Nervous System symptom: Week 105
    Somatic symptom: Week 53
    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 Yes
    E.6.13.1Other scope of the trial description
    Immunogenicity, genetics
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Assessor blinded study
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 EEA11
    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
    Argentina
    Colombia
    Brazil
    United Kingdom
    United States
    France
    Germany
    Italy
    Poland
    Spain
    Türkiye
    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
    The end of the study is defined as the date of the last visit of the last subject in the study or last scheduled procedure shown in the Schedule of Activities for the last subject in the trial globally. Eligible subjects who complete the trial will be invited to participate in the follow on JR-141 long term trial.
    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 months7
    E.8.9.1In the Member State concerned days16
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months2
    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 Yes
    F.1.1Number of subjects for this age range: 75
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 65
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 5
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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
    If the patient is aged under 18 years or willingness to participate in the study cannot be confirmed due to MPS II-related intellectual disability, informed permission from the patient’s legally acceptable representative needs to be obtained instead.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 39
    F.4.2.2In the whole clinical trial 80
    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)
    On completion of this JR-141-GS31 study, a follow on study will be set as a Global Phase III Extension Study in patients with MPS II, who have participated in the current study and provide consent to participate.
    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 Decision2021-11-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-05-16
    P. End of Trial
    P.End of Trial StatusOngoing
    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.

    European Medicines Agency © 1995-2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA