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   43850   clinical trials with a EudraCT protocol, of which   7282   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:2019-000177-23
    Sponsor's Protocol Code Number:P170930J
    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:2019-04-12
    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 number2019-000177-23
    A.3Full title of the trial
    IMMUNOTHERAPY WITH INTRAVENOUS IMMUNOGLOBULINS, CYCLOPHOSPHAMIDE AND METHYLPREDNISOLONE IN PATIENTS WITH PARANOPLASTIC SENSITIVE NEURONOPATHIES WITH ANTI-HU ANTIBODIES
    IMMUNOTHERAPIE PRECOCE PAR IMMUNOGLOBULINES INTRAVEINEUSES, CYCLOPHOSPHAMIDE ET METHYLPREDNISOLONE CHEZ DES PATIENTS AVEC NEURONOPATHIES SENSITIVES PARANEOPLASIQUES A ANTICORPS ANTI-HU
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Treatment with 3 drugs against sensitive neuropathies
    Traitement par 3 médicaments contre les neuropathies sensitives
    A.3.2Name or abbreviated title of the trial where available
    NESPA
    NESPA
    A.4.1Sponsor's protocol code numberP170930J
    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 SponsorASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
    B.1.3.4CountryFrance
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportMinistère de la santé
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
    B.5.2Functional name of contact pointSophie COURTIAL-DESTEMBERT
    B.5.3 Address:
    B.5.3.1Street AddressDRCI Hôpital St Louis, 1 av. Claude Vellefaux
    B.5.3.2Town/ cityParis
    B.5.3.3Post code75010
    B.5.3.4CountryFrance
    B.5.4Telephone number+33140275591
    B.5.5Fax number+33144841701
    B.5.6E-mailsophie.courtial-destembert@aphp.fr
    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 Yes
    D.2.1.1.1Trade name Privigen 100 mg/ml
    D.2.1.1.2Name of the Marketing Authorisation holderCSL Behring GmbH
    D.2.1.2Country which granted the Marketing AuthorisationFrance
    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.3.4Pharmaceutical form Solution 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) 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 Yes
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    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 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.2.1.2Country which granted the Marketing AuthorisationFrance
    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.3.1Product namecyclophosphamide
    D.3.4Pharmaceutical form Powder and solvent for solution 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 Yes
    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) 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 No
    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: 3
    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.2.1.2Country which granted the Marketing AuthorisationFrance
    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.3.1Product nameméthylprednisolone
    D.3.4Pharmaceutical form Powder and solvent for solution 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 Yes
    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) 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 No
    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
    Paraneoplastic Neurological Syndromes
    Syndromes Neurologiques Paranéoplasiques
    E.1.1.1Medical condition in easily understood language
    neuron disease
    Maladie des neurones
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Evaluation of the 3-Month Efficacy of Early Immunotherapy with IgIV, Cyclophosphamide and Methylprednisolone in Patients with Paraneoplastic Sensory Neuronopathy with anti-Hu antibody
    Evaluation de l’efficacité à 3 mois d’une immunothérapie précoce à base d’IgIV, Cyclophosphamide et Méthylprednisolone chez des patients atteints d’une neuronopathie sensitive paranéoplasique à anticorps anti-Hu
    E.2.2Secondary objectives of the trial
    Evaluation :
    -of effectiveness at 6 months of study immunotherapy
    -of the percentage of patients alive and without tumor progression at 6 months
    -treatment tolerance
    Evaluation :
    -de l’efficacité à 6 mois de l’immunothérapie à l’étude
    -du pourcentage des patients vivants et sans progression tumorale à 6 mois
    -de la tolérance du traitement
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -Age ≥ 18 years
    -Neuropathic sensory "possible" according to the criteria of Camdessanché et al.
    -Dominant ataxia table (central nervous system involvement and / or neuromuscular junction is allowed, provided it has a minor impact on the patient's disability)
    -Anti-Hu antibody positive in blood and / or cerebrospinal fluid
    -Outpatient (modified Rankin score 2 or 3)
    -First neurological symptoms for less than 3 months
    -Free, informed, written and signed consent
    -Affiliation to a social security scheme or beneficiary (except AME)
    -Age ≥ 18 ans
    -Neuronopathie sensitive « possible » selon les critères de Camdessanché et al.
    -Tableau dominant d’ataxie sensitive (une atteinte du système nerveux central et/ou de la jonction neuromusculaire est admise, à condition qu’il ait un impact mineur sur le handicap du patient)
    -Anticorps anti-Hu positifs dans le sang et/ou le liquide céphalorachidien
    -Patient ambulatoire (Echelle modified Rankin Score (mRS) 2 ou 3)
    -Début des symptômes neurologiques depuis moins de 3 mois
    -Consentement libre, éclairé et écrit signé
    -Affiliation à un régime de sécurité sociale ou bénéficiaire (sauf AME)
    E.4Principal exclusion criteria
    -Possible hypersensitivity to any of the study treatments, their metabolites, or any of the excipients
    -Absolute contraindications to intravenous immunoglobulin : selective IgA deficiency, known thrombophilia
    -Absolute contraindications to cyclophosphamide: vaccination against yellow fever in the 3 months preceding inclusion, acute urinary tract infection, acute spinal cord failure
    -Over two courses of IVIG administered within 3 months before recruitment
    -Other concomitant immunotherapy
    -Other cause of immunodepression (acquired or congenital)
    -Treatment with checkpoint inhibitors in progress or completed less than 3 months ago
    - Women of childbearing age without effective contraception, pregnant or lactating
    -Psychiatric history or general illness that may contraindicate the treatment
    -Patients unable to perform the follow-up required by the study
    -Patients under guardianship or curatorship
    -Patient deprived of liberty by a judicial or administrative decision
    -Hypersensibilité connue à un des traitements à l’étude, à leurs métabolites, ou à l’un des excipients
    -Contre-indications absolues aux IgIV : déficit sélective en IgA, thrombophilie connue
    -Contre-indications absolues au cyclophosphamide : vaccination contre fièvre jaune dans les trois mois précédents l’inclusion, infection urinaire aiguë, insuffisance médullaire aigue
    -Plus de deux cures d’IgIV administrés dans les 3 mois avant le recrutement
    -Autre immunothérapie concomitante
    -Autre cause d’immunodépression (acquise ou congénitale)
    -Traitement avec inhibiteurs de checkpoint en cours ou complété moins de 3 mois auparavant
    -Femme en âge de procréer sans contraception efficace, enceinte ou allaitante
    -Antécédents psychiatriques ou de maladies générales pouvant contre-indiquer le traitement
    -Patients ne pouvant effectuer le suivi requis par l’étude
    -Patients sous tutelle ou curatelle
    -Patient privé de liberté par une décision judiciaire ou administrative
    E.5 End points
    E.5.1Primary end point(s)
    Percentage of patients with clinical improvement on the ONLS (Overall Neuropathy Limitations Scale) at 3 months
    Pourcentage des patients avec amélioration clinique sur l’échelle ONLS (Overall Neuropathy Limitations Scale) à 3 mois
    E.5.1.1Timepoint(s) of evaluation of this end point
    3 months
    3 mois
    E.5.2Secondary end point(s)
    -The percentage of patients with clinical improvement on the ONLS scale at 6 months
    -The percentage of patients with improvement of the ataxic component on the Score of Ataxia scale at 3 and 6 months
    -The percentage of patients with improvement in neuropathic pain on the Numeric Rating Scale (NRS) at 3 and 6 months
    -The percentage of patients with functional improvement on the modified Rankin Score (mRS) at 3 and 6 months
    -The percentage of patients with functional improvement on the Barthel Index (BI) at 3 and 6 months
    -The percentage of patients alive and without tumor progression at 6 months
    -The tolerance to treatment will be evaluated by the frequency and severity of expected and unexpected adverse effects recorded during treatment
    -Le pourcentage de patients avec amélioration clinique sur l’échelle ONLS à 6 mois
    -Le pourcentage de patients avec amélioration de la composante ataxique sur l’échelle Score of Ataxia à 3 et 6 mois
    -Le pourcentage de patients avec amélioration de la douleur neuropathique sur l’échelle Numeric Rating Scale (NRS) à 3 et 6 mois
    -Le pourcentage de patients avec amélioration fonctionnelle sur le modified Rankin Score (mRS) à 3 et 6 mois
    -Le pourcentage de patients avec amélioration fonctionnelle sur le Barthel Index (BI) à 3 et 6 mois
    -Le pourcentage de patients vivants et sans progression tumorale à 6 mois
    -La tolérance au traitement sera évaluée par la fréquence et la sévérité des effets indésirables attendus et inattendus enregistrés pendant le traitement
    E.5.2.1Timepoint(s) of evaluation of this end point
    3 and 6 months
    3 et 6 mois
    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 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) 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) 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 No
    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
    Etude prospective de phase II, ouverte, non comparative, multicentrique
    Prospective phase II study, open, non-comparative, multicenter
    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 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 concerned7
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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
    LVLS
    Dernière visite du dernier patient
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    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: 11
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 10
    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 No
    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 state
    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 Decision2021-04-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-07-04
    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-Fri Apr 19 19:06:27 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA