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    Summary
    EudraCT Number:2021-004141-20
    Sponsor's Protocol Code Number:APHP210360
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-05-24
    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 number2021-004141-20
    A.3Full title of the trial
    Innovative Imaging and cognitive BIOmarkers to predict Huntington’s Disease progression
    Imagerie Innovante et BIOmarqueurs cognitifs pour prédire la progression de la maladie de Huntington
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Innovative Imaging and cognitive BIOmarkers to predict Huntington’s Disease progression
    Imagerie Innovante et BIOmarqueurs cognitifs pour prédire la progression de la maladie de Huntington
    A.3.2Name or abbreviated title of the trial where available
    I2BIO-HD
    I2BIO-HD
    A.4.1Sponsor's protocol code numberAPHP210360
    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 supportDirection générale de l’offre de soin (PRTS 2020)
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAssistance Publique - Hôpitaux Paris - Direction de la Recherche Clinique et de l'Innovation
    B.5.2Functional name of contact pointproject Manager
    B.5.3 Address:
    B.5.3.1Street Address1 avenue Claude Vellefaux
    B.5.3.2Town/ cityPARIS
    B.5.3.3Post code75010
    B.5.3.4CountryFrance
    B.5.4Telephone number13344841747
    B.5.6E-mailcandy.estevez@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 No
    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 name[18F]-MNI-659
    D.3.2Product code [18F]-MNI-659
    D.3.4Pharmaceutical form Solution for injection
    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) 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 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 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
    Patients with Huntington's disease : symptomatic (HD) and pre-symptomatic (pre-HD).
    Patients atteints de maladie de Huntington : symptomatiques (MH) et pré-symptomatiques (preMH).
    E.1.1.1Medical condition in easily understood language
    Patients with symptomatic and pre-symptomatic Huntington's disease
    Patients atteints de la maladie de de Huntington symptomatique et pré-symptomatiques
    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 20.0
    E.1.2Level PT
    E.1.2Classification code 10070668
    E.1.2Term Huntington's disease
    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
    Define a multidomain composite score to measure the clinical evolution of patients, from the presymptomatic stages, in the context of clinical trials
    DeĢfinir un score composite multidomaine pour mesurer les évolutions cliniques des patients, dès les stades présymptomatiques, dans le cadre d’essais cliniques.
    E.2.2Secondary objectives of the trial
    1) Identify patient profiles and HD progression trajectories (unsupervised analyses);
    2) Identify the best predictive biomarkers, individually or in combination, and establish their prognostic value on disease progression (supervised analyses).
    1) Identifier des profils de patients et des trajectoires de progression de la MH (analyses non supervisées) ;
    2) Identifier les meilleurs biomarqueurs prédicteurs, isolément ou combinés, et établir leur valeur pronostique sur l'évolution de la maladie (analyses supervisées).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Age 18-65
    - Information and collection of written consent
    - Affiliation to a social security scheme, beneficiary or beneficiary

    Controls:
    - TFC (Total Functional Capacity) = 13
    - TMS (Total Motor Score ) < 6
    - No known genetic disease or family ascendant carrying the gene for the Huntington's disease mutation.


    Symptomatic HD patients:
    - Number of CAG ≥ 40
    - CAP (CAG Age Product score) score ≥ 250
    - 10 ≤ TFC ≤ 13
    - TMS >5 if TFC=13
    - Diagnostic confidence level =4
    - Age onset of disease > 20 years
    - Patients physically able to sign

    Presymptomatic preMH patients:
    - Number of CAG ≥ 40
    - CAP score ≥250
    - TFC = 13
    - TMS < 6
    - Patients physically able to sign
    - Age 18-65 ans
    - Information et recueil du consentement écrit
    - Affiliation à un régime de sécurité sociale, bénéficiaire ou ayant droit

    Contrôles :
    - TFC = 13
    - TMS < 6
    - Sans maladie génétique connue ni ascendant familiaux porteurs du gène de la mutation maladie de Huntington
    Les contrôles seront inscrits dans le fichier national des personnes qui se prêtent à des recherches interventionnelles impliquant la personne humaine .


    Patients symptomatiques MH :
    - Nombre de CAG ≥ 40
    - CAP score ≥ 250
    - 10 ≤ TFC ≤ 13
    - TMS >5 si TFC=13
    - Niveau de confiance du diagnostic =4
    - Age début de la maladie > 20
    - Patients en capacité physique de signer

    Patients présymptomatiques preMH :
    - Nombre de CAG ≥ 40
    - CAP score ≥250
    - TFC = 13
    - TMS < 6
    - Patients en capacité physique de signer
    E.4Principal exclusion criteria
    - Participant under guardianship or curatorship
    - Neurological or psychiatric disorder unrelated to HD
    - Intercurrent illness that may impact participants' performance
    - Progressive chronic neurological disease
    - Claustrophobia
    - Brain damage not related to HD
    - Pacemaker, intracorporeal metal, intracerebral clip
    - Catheters with metal components (Swan-Ganz catheter), metal fragments such as bullets, shotgun pellets and metal shrapnel, cerebral artery aneurysm clips, magnetic dental implants, tissue expander, artificial limb, hearing aid, piercing such as pacemaker
    - Known hypersensitivity to the radiopharmaceutical preparation (excipients in the radiopharmaceutical preparation)
    - Pregnant or breastfeeding woman
    - Person under AME
    - Person deprived of liberty
    - Person participating or having participated in an interventional study for less than 3 months or without time limit in a trial of neural transplants or gene therapy.
    - Person participating or having participated in a research protocol with injection of a radiopharmaceutical for less than 12 months.
    - Participant sous tutelle ou curatelle
    - Trouble neurologique ou psychiatrique non relié à la MH
    - Maladie intercurrente pouvant impacter les performances des participants
    - Maladie neurologique chronique évolutive
    - Claustrophobie
    - Lésion cérébrale non reliée à la MH
    - Pacemaker, métal intracorporel, clip intracérébral, tout corps étranger métallique
    - Cathéters avec des composants métalliques (cathéter de Swan-Ganz), fragments métalliques tels que balles, plombs de fusil de chasse et éclats d'obus métalliques, clips d'anévrisme de l'artère cérébrale, implants dentaires magnétiques, expanseur tissulaire, membre artificiel, prothèse auditive, piercing tel que stimulateur cardiaque,
    - Hypersensibilité connue à la préparation radiopharmaceutique (excipients dans la préparation radiopharmaceutique)
    - Femme enceinte, allaitante
    - Personne sous AME
    - Personne privée de liberté
    - Personne participant ou ayant participé à une étude interventionnelle depuis moins de 3 mois ou sans limite de délai à un essai de greffes neuronales ou de thérapie génique.
    - Personne participant ou ayant participé à un protocole de recherche avec injection d’un radiopharmaceutique depuis moins de 12 mois.

    E.5 End points
    E.5.1Primary end point(s)
    The construction of the composite score will be based on a sequential approach, aiming to i) calculate for each candidate measure the longitudinal evolution in absolute and relative value and in terms of effect size; ii) select the measures with the largest effect sizes (i.e. Cohen’s d) in HD and preMH patients, as well as evolutions significantly different from those observed in controls; iii) carry out a principal component analysis (PCA) on the basis of the best individual scores previously identified in order to define a composite score, a linear combination of the individual variables.
    The candidate measures for constructing the composite score will include measures related to cognition, motor skills, functional status, mood, behaviors and paraclinical imaging parameters.
    La construction du score composite s’appuiera sur une approche séquentielle, visant à i) calculer pour chaque mesure candidate l’évolution longitudinale en valeur absolue, relative et en termes de taille d'effets ; ii) sélectionner les mesures ayant les tailles d'effet (i.e. Cohen’s d) les plus élevées chez les patients MH et preMH, ainsi que des évolutions significativement différentes de celles observées chez des contrôles (volontaires sains) ; iii) réaliser une analyse en composantes principales (ACP) sur la base des meilleurs scores individuels identifiés précédemment afin de définir un score composite, combinaison linéaire des variables individuelles.
    Les mesures candidates à la construction du score composite incluront les mesures liées à la cognition, la motricité, l’état fonctionnel, l’humeur, les comportements et paramètres paracliniques d’imagerie.
    E.5.1.1Timepoint(s) of evaluation of this end point
    period of 23 months compared to Month 1
    période de 23 mois en comparaison à la visite 1 mois de référence
    E.5.2Secondary end point(s)
    1) The analysis of patient progression profiles and trajectories will be based on socio-demographic characteristics, as well as on initial clinical and paraclinical scores and their evolution over time. The clustering analyzes will be based on statistical validation indices in order to determine the optimal number of clusters and inform on the quality of the groupings.
    2) The identification of the best predictive biomarkers of an unfavorable progression of the disease will be carried out using conventional regression and machine learning methods. The discrimination and calibration performances will be systematically evaluated and compared for each of the models built.
    1) L’analyse des profils et trajectoires de progression des patients s’appuiera sur les caractéristiques sociodémographiques, ainsi que sur les scores cliniques et paracliniques initiaux et leur évolution au cours du temps. Les analyses de clustering s’appuieront sur des indices statistiques de validation afin de déterminer le nombre optimal de clusters et informer sur la qualité des regroupements.
    2) L’identification des meilleurs biomarqueurs prédicteurs d’une évolution défavorable de la maladie sera réalisée en s’appuyant sur des méthodes de régression conventionnelle et d’apprentissage automatique. Les performances de discrimination et de calibration seront systématiquement évaluées et comparées pour chacun des modèles construits.

    E.5.2.1Timepoint(s) of evaluation of this end point
    Month 1,Month 12 ans Month 24
    M1 et M12 et M24
    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 No
    E.6.5Efficacy No
    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 Yes
    E.6.13.1Other scope of the trial description
    Definition of a composite score as a tool for predicting disease progression
    Définition d'un score composite comme outil de prédiction de l'évolution de la maladie
    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 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
    Étude de cohorte prospective, patient et contrôles
    prospective cohort study, patients and controls
    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 concerned2
    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 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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months46
    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: 100
    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 Yes
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2022-05-24. Yes
    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 state100
    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
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-08-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-06-30
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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