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    Summary
    EudraCT Number:2020-003200-14
    Sponsor's Protocol Code Number:JR-141-GS31
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-06-17
    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 ConcernedFrance - ANSM
    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.
    Étude de Phase III du JR-141 chez des patients atteints d’une mucopolysaccharidose de type 2 (Maladie de Hunter).
    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
    Étude de Phase III du JR-141 chez des patients atteints d'une maladie de Hunter
    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 pointClinical Development Department
    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-mailkaihatsu@jcrpharm.co.jp
    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.3.9.3Other descriptive nameJR-141
    D.3.9.4EV Substance CodeSUB193542
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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
    Mucopolysaccharidose de type 2
    E.1.1.1Medical condition in easily understood language
    Hunter syndrome
    Maladie de Hunter
    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 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
    * Démontrer l’efficacité du JR-141 sur les symptômes du SNC chez les patients MPS II
    * Démontrer l’efficacité du JR-141 sur les symptômes somatiques chez les patients MPS II
    * Évaluer la tolérance du JR-141 chez les patients MPS II
    * Évaluer la pharmacocinétique (PK) du JR-141 chez les patients MPS II
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. A patient from whom a voluntarily signed written IRB or Independent Ethics Committee (IEC)-approved ICF can be obtained. Informed permission from the patient’s legally accepted representative (e.g. his parent[s] or guardian[s]) needs to be obtained for a patient who is under 18 years of age (aged under 16 years in the UK) at the time of assent and/or for a patient where willingness to participate in the study cannot be confirmed due to MPS II-related intellectual disability. In these cases, written informed consent or assent should still be obtained from the patient, whenever possible
    2. Males with confirmed diagnosis of MPS II, based on the documented mutation identified in the IDS gene and meeting one of the following criteria:
    1) Deficient activity of IDS in leucocyte or fibroblasts
    2) Increased levels of urinary glycosaminoglycans (GAGs) (or uronic acid) measured before the start of specific therapy
    3. Naïve patients or patients who are receiving stable enzyme replacement therapy (ERT) with idursulfase for more than 12 weeks before starting administration of the JR-141 or idursulfase for this study.
    4. Cohort A
    - Males aged 36-71 months old at the time of ICF signed and whose development quotient (DQ) on the cognitive domain by the BSID-III or the nonverbal index by the KABC-II is between 55-75 at screening; OR
    - Males 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 either active site residues or have been identified to be associated with severe, neuronopathic disease in either another family or other family members.
    5. Cohort B
    - Males aged 6 years or older at the time of ICF signed and whose intelligence quotient (IQ) on a Wechsler test (either WPPSI-IV, WISC-V, or WAIS-IV) is 70 and higher at screening
    - 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. Male patients whose partners are of child-bearing potential agree to use a medically accepted, highly effective method of contraception being a condom plus an approved method of effective contraception from the time of informed consent.
    The following methods are acceptable:
    • 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)
    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, with their partner, must comply with the contraceptive requirements detailed above.
    1. Pour un patient en ayant la capacité, signature volontaire du FCE écrit approuvé par un Comité de protection des personnes (CPP). Pour un patient de moins de 18 ans (âgé de moins de 16 ans au Royaume-Uni) au moment de l’assentiment et/ou pour un patient présentant un déficit intellectuel lié à la MPS-II ne permettant pas de confirmer sa volonté de participer à l’étude, obtention de l’autorisation éclairée du représentant légalement accepté du patient (par exemple, son ou ses parent[s] ou tuteur[s]) Dans ces cas, un consentement ou un assentiment éclairé écrit devra toujours être obtenu auprès du patient, si possible.
    2. Patients de sexe masculin ayant un diagnostic confirmé de MPSII, fondée sur la documentation d’une mutation identifiée dans le gène de l’IDS et réunissant un des critères suivants :
    1) Activité déficiente de l’IDS dans les leucocytes ou les fibroblastes.
    2) Taux urinaires élevés de glycosaminoglycanes (GAG) (ou d’acide uronique) mesuré avant le début d’un traitement spécifique.
    3. Patients naïfs de tout traitement ou patients ayant reçu un traitement enzymatique substitutif (TES) par idursulfase pendant plus de 12 semaines avant le début de l’administration du JR-141 ou de l’idursulfase dans cette étude.
    4. Cohorte A
    − Patients de sexe masculin âgés de 36 à 71 mois au moment de la signature du FCE et dont le quotient de développement (QD) sur le domaine cognitif de l’échelle BSID-III ou l’indice non verbal de l’échelle KABC-II est compris entre 55 et 75 à la sélection OU
    − Patients de sexe masculin âgés de 30 à 35 mois au moment de la randomisation et jugés par le comité d’experts comme présentant le phénotype sévère, fondé sur la présence d’une des mutations suivantes dans le gène de l’IDS et d’autres informations telles que la présence de concentrations élevées d’HS dans le LCR :
    1) Délétion ou réarrangement important(e).
    2) Petites insertions ou délétions hors cadre.
    3) Mutations faux-sens, mutation non-sens, insertions ou délétions décalantes impliquant des résidus de site actif ou identifiées comme associées à une atteinte neuronopathique sévère dans une autre famille ou d’autres membres de la famille.
    5. Cohorte B
    − Patients de sexe masculin âgés de 6 ans et plus au moment de la signature du FCE, dont le quotient intellectuel (QI) évalué par un test de Wechsler (WPPSI-IV, WISC-V ou WAIS-IV) est de 70 ou plus au moment de la sélection.
    - Déficit d’1 ET dans les domaines d’erreurs d’omission ou de variabilité du test T.O.V.A. ou les indices de vitesse de traitement ou de mémoire de travail de l’échelle Wechsler au moment de la sélection.
    6. Les patients de sexe masculin dont les partenaires sont en âge de procréer doivent accepter d’utiliser un moyen de contraception hautement efficace, médicalement accepté, en associant un préservatif à un moyen de contraception efficace approuvé, à partir de la signature du consentement éclairé. Les moyens de contraception suivants sont acceptables :
    • Utilisation par la partenaire d’une contraception hormonale combinée (contenant des oestrogènes et des progestatifs) associée à une inhibition de l’ovulation :
    − Orale.
    − Intravaginale.
    − Transdermique.
    • Utilisation par la partenaire d’une contraception hormonale uniquement progestative :
    − Orale.
    − Injectable/implantable.
    − Système intra-utérin à libération hormonale (SIU).
    • Utilisation par la partenaire d’un dispositif intra-utérin (DIU).
    • Stérilisation chirurgicale (par exemple, vasectomie ou occlusion bilatérale des trompes).
    • Utilisation par la partenaire d’un diaphragme ou d’une cape cervicale (double barrière).
    Sinon, l’abstinence réelle de rapports sexuels hétérosexuels est une forme acceptable de contraception lorsqu’elle correspond au mode de vie préférentiel et habituel du patient. Si un patient n’a généralement pas d’activité sexuelle mais devient sexuellement actif, il doit, avec sa partenaire, respecter les exigences détaillées ci-dessus concernant la contraception
    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 have difficulties in taking position for lumbar puncture due to joint contracture or those who are likely 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 a medical condition* or therapy.
    8. 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
    1. Antécédents de greffe de cellules souches hématopoïétiques (GCSH), avec réussite de la greffe.
    2. Antécédents de traitement par thérapie génique, à n’importe quel moment.
    3. Patient jugé par l’investigateur principal ou le co-investigateur comme ne pouvant pas subir de ponction lombaire, y compris les patients ayant des difficultés à prendre la position pour une ponction lombaire en raison de rétractions articulaires ou ceux qui sont susceptibles de présenter des difficultés respiratoires pendant la ponction lombaire.
    4. Recrutement dans une autre étude clinique impliquant des évaluations cliniques ou l’utilisation d’un produit expérimental (médicament ou dispositif) dans les 4 mois avant l’obtention du formulaire de consentement éclairé.
    5. Incapacité du patient à respecter le protocole (par exemple, incapacité à se rendre dans le centre d’étude pour des évaluations de tolérance ou non susceptible de terminer l’étude, pour une autre raison), d’après le jugement de l’investigateur principal ou du co-investigateur.
    6. Patient jugé par l’investigateur principal ou le co-investigateur comme inéligible pour la participation à l’étude en raison d’antécédents d’allergie médicamenteuse grave ou de problèmes de sensibilité y compris anesthésie ou d’une hypersensibilité à un des composants du JR-141 ou de l’idursulfase.
    7. Infection générale ou locale connue ou suspectée ou risque de saignement anormal lié à une pathologie médicale* ou un traitement.
    8. Patient jugé par l’investigateur principal ou le co-investigateur comme inéligible pour la participation à l’étude pour toute autre raison.
    *Pathologies médicales :
    1. Allergies médicamenteuses multiples ou sévères cliniquement significatives, intolérance aux corticoïdes topiques ou réactions d’hypersensibilité sévères post-thérapeutiques (y compris notamment : érythème polymorphe majeur, dermatose à immunoglobuline A [IgA] linéaire, nécrolyse épidermique toxique, et dermatite exfoliative)
    2. Présence ou antécédents de saignement ou trouble de la coagulation actif significatif ou utilisation d’un anti-inflammatoire non stéroïdien ou d’autres médicaments interférant sur la coagulation ou la fonction plaquettaire dans les 14 jours avant la pose du cathéter lombaire
    3. Allergie à la lidocaïne (Xylocaïne®) ou ses dérivés
    E.5 End points
    E.5.1Primary end point(s)
    •Level of CSF HS at Week 53 and Week 105 in Cohort A
    •Change in age equivalent scores of neurocognitive testing from baseline to Week 53 and Week 105 by the BSID-III (cognitive domain) or the KABC-II (nonverbal index (NVI)) in Cohort A
    • Relative change in the liver volume relative to body weight from baseline to Week 53 in Cohort A and Cohort B
    • Relative change in the spleen volume relative to body weight from baseline to Week 53 in Cohort A and Cohort B
    • Relative change in the distance walked in 6-minute walk test from baseline to Week 53 in Cohort B
    - Taux d’HS dans le LCR à la Semaine 53 et la Semaine 105 dans la Cohorte A.
    - Modification des scores obtenus à âge équivalent aux tests neurocognitifs, entre l’inclusion et la Semaine 53 et la Semaine 105, évalués par l’échelle d’évaluation BSID-III (domaine cognitif) ou l’échelle KABC-II (indice non verbal (INV)) dans la Cohorte A.
    -Modification relative du volume du foie par rapport au poids corporel, entre l’inclusion et la Semaine 53, dans la Cohorte A et la Cohorte B.
    - Modification relative du volume de la rate par rapport au poids corporel, entre l’inclusion et la Semaine 53, dans la Cohorte A et la Cohorte B.
    - Modification relative de la distance parcourue au cours du test de marche de 6 minutes, entre l’inclusion et la Semaine 53, dans la Cohorte B.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Cohort A: Week 53 & Week 105
    Cohort B: Week 53
    Cohort A: semaine 53 & semaine 105
    Cohort B: semaine 53
    E.5.2Secondary end point(s)
    Cohort A:
    • Change in age equivalent scores of adaptive behavior from baseline to Week 53 and Week 105 by the VABS-II in Cohort A
    • Relative change in liver volume relative to body weight from baseline to Week 105 in Cohort A

    Cohort B:
    • Change in level of CSF HS from baseline to Week 53 in Cohort B
    • Change in the standard scores on omission error and variability domain by the T.O.V.A or in the composite scores on Processing Speed or Working Memory by the Wechsler tests from baseline to Week 53 in Cohort B
    • Absolute change in the absolute FVC from baseline to Week 53 in Cohort B
    Cohort A:
    • Modification des scores à âge équivalent de comportement socio adaptatif, entre l’inclusion et la Semaine 53 et la Semaine 105, évalués par l’échelle VABS-II dans la Cohorte A.
    • Modification relative du volume du foie par rapport au poids corporel, entre l’inclusion et la Semaine 105, dans la Cohorte A.

    Cohort B:
    • Variation du taux d’HS dans le LCR entre l’inclusion et la Semaine 53, dans la Cohorte B.
    • Modifications des scores standard obtenus dans le domaine des erreurs d’omission et de variabilité de l’évaluation T.O.V.A. ou modifications des scores composites obtenus aux indices de vitesse de traitement ou de mémoire de travail de l’évaluation Wechsler, entre l’inclusion et la Semaine 53, dans la Cohorte B.
    • Variation absolue de la CVF absolue, entre l’inclusion et la Semaine 53, dans la Cohorte B.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Cohort A: Week 53 & Week 105
    Cohort B: Week 53
    Cohort A: semaine 53 & semaine 105
    Cohort B: semaine 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 No
    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 Yes
    E.6.13.1Other scope of the trial description
    Immunogenicity, genetics
    Immunogénicité, génétique
    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 Yes
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    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) 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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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
    Brazil
    United States
    France
    Germany
    United Kingdom
    E.8.7Trial has a data monitoring committee No
    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.
    La fin de l'étude est définie comme la date de la dernière visite du dernier patient de l'étude ou de la dernière procédure prévue indiquée dans le programme d'activités pour le dernier patient de l'essai. Les patients éligibles qui termineront l'essai seront invités à participer à l'essai de suivi à long terme du JR-141.
    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 months10
    E.8.9.1In the Member State concerned days15
    E.8.9.2In all countries concerned by the trial years3
    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 Yes
    F.1.1Number of subjects for this age range: 26
    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: 13
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 13
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 12
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 12
    F.2 Gender
    F.2.1Female No
    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 No
    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 state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 26
    F.4.2.2In the whole clinical trial 50
    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.
    À la fin de cette étude JR-141-GS31, une étude de suivi sera mise en place, étude d'extension de phase III chez les patients atteints de MPS II, qui ont participé à l'étude en cours et qui ont donné leur consentement pour y participer.
    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 Decision2021-10-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-05-04
    P. End of Trial
    P.End of Trial StatusOngoing
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