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    Summary
    EudraCT Number:2018-003898-94
    Sponsor's Protocol Code Number:BN40955
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2019-06-11
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2018-003898-94
    A.3Full title of the trial
    AN OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG TERM SAFETY AND TOLERABILITY OF INTRATHECALLY ADMINISTERED RO7234292 (RG6042) IN PATIENTS WITH HUNTINGTON’S DISEASE
    ESTUDIO DE EXTENSIÓN ABIERTO PARA EVALUAR LA SEGURIDAD Y LA TOLERABILIDAD A LARGO PLAZO DE RO7234292 (RG6042) ADMINISTRADO POR VÍA INTRATECAL EN PACIENTES CON ENFERMEDAD DE HUNTINGTON
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Open-Label Extension Study to Evaluate the Long-Term Safety and Tolerability of Intrathecally Administered RO7234292 (RG6042) in Patients with Huntington’s Disease
    Estudio de extension abierto para evaluar la seguridad y la tolerabilidad a largo plazo de RO7234292 (RG6042) administrado por via intratecal en pacientes con Enfermedad de Huntington
    A.4.1Sponsor's protocol code numberBN40955
    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 SponsorRoche Farma S.A(Soc. Unipersonal) que realiza el ensayo en España y actúa como representante de F. Hoffmann-La Roche LTD
    B.1.3.4CountrySwitzerland
    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 supportF.Hoffmann La-Roche Ltd - Basel
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationF. Hoffmann-La Roche LTD
    B.5.2Functional name of contact pointTrial Information Support Line-TISL
    B.5.3 Address:
    B.5.3.1Street AddressGrenzacherstrasse 124
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4070
    B.5.3.4CountrySwitzerland
    B.5.6E-mailglobal.rochegenentechtrials@roche.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/3/15/1453
    D.3 Description of the IMP
    D.3.2Product code Ro 723-4292/F02
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntrathecal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNnot available
    D.3.9.1CAS number 1709886-74-7
    D.3.9.2Current sponsor codeRO7234292/F02
    D.3.9.3Other descriptive nameRG6042, formerly ISIS 443139, IONIS-HTTRx
    D.3.9.4EV Substance CodeSUB173711
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number6
    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 Yes
    D.3.11.13.1Other medicinal product typemodified antisense oligonucleotide
    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
    Huntington's disease (HD)
    Enfermedad de Huntington (EH)
    E.1.1.1Medical condition in easily understood language
    Huntington's disease is an inherited disease that causes the progressive breakdown (degeneration) of nerve cells in the brain
    La enfermedad de Huntington es una enfermedad hereditaria que causa el colapso progresivo (degeneración) de las células nerviosas en el cerebro.
    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
    To evaluate the safety and tolerability of RO7234292
    Evaluar la seguridad y tolerabilidad de RO7234292
    E.2.2Secondary objectives of the trial
    Not applicable
    No aplica
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Prior enrollment in a Roche sponsored or Genentech-sponsored study in HD for the RO7234292 development program that made provision for entry into an OLE study
    - Ability and willingness to comply with the study protocol including the visit schedule and all assessments, in the investigator's judgment (apart from patients joining from Study BN40697 who are unable to undergo MRI)
    - For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive measures, during the treatment period and for 5 months after the final dose of study drug
    - For men: agreement to remain abstinent (refrain from heterosexual intercourse) or use a condom, and agreement to refrain from donating sperm during the treatment period and for 5 months after the final dose of study drug to avoid exposing the embryo
    -Participación previa en un estudio sobre la EH patrocinado por Roche o Genentech para el
    programa de desarrollo de RO7234292 en el que estuviera prevista la participación en una ERA.
    -Capacidad y voluntad de cumplir el protocolo del estudio, incluido el calendario de visitas y
    todas las evaluaciones, según el criterio del investigador (aparte de los pacientes procedentes
    del estudio BN40697 que no puedan someterse a una RM).
    -Mujeres en edad fértil: deberán comprometerse a practicar la abstinencia (no mantener
    relaciones sexuales) o a usar métodos anticonceptivos, durante el periodo de tratamiento y por 5 meses después de la última dosis del fármaco del estudio.
    -Varones: compromiso de practicar la abstinencia sexual (abstenerse de mantener
    relaciones heterosexuales) o de utilizar preservativo y compromiso de no donar semen, durante el periodo de tratamiento y durante al menos 5 meses después de la última dosis del
    fármaco del estudio para no exponer al embrión.
    E.4Principal exclusion criteria
    - Withdrawal of consent from the preceding study
    - Permanent discontinuation of RO7234292 for any drug-related safety concern during the preceding study or meeting of any study treatment discontinuation criteria specified in the preceding study at the time of enrollment into this study
    - An ongoing, unresolved, clinically significant medical problem that in the judgment of the investigator would make it unsafe for the patient to participate in this study
    - Antiplatelet or anticoagulant therapy within 14 days prior to inclusion or anticipated use during the study, including, but not limited to, aspirin (unless <=81 mg/day), clopidogrel, dipyridamole, warfarin, dabigatran, rivaroxaban, and apixaban
    - History of bleeding diathesis or coagulopathy
    - Platelet count less than the lower limit of normal
    - Concurrent participation in any therapeutic clinical trial (other than the preceding study)
    - Study treatment (RO7234292) is commercially marketed in the patient’s country for the patient-specific disease and is accessible to the patient
    - Pregnant or breastfeeding, or intending to become pregnant during the study or within 5 months after the final dose of study drug
    -Retirada del consentimiento para el estudio anterior.
    -Suspensión permanente del tratamiento con RO7234292 por cualquier problema de seguridad relacionado con el fármaco durante el estudio precedente, o cumplimiento de alguno de los criterios de suspensión del tratamiento del estudio especificados en el
    estudio precedente en el momento de la inclusión en el presente estudio.
    -Un problema médico en curso, no resuelto y clínicamente importante que, en opinión del investigador, haría que no fuera segura la participación del paciente en el estudio.
    -Tratamiento con antiagregantes plaquetarios o anticoagulantes en los 14 días previos a la inclusión o uso previsto durante el estudio, entre ellos, ácido acetilsalicílico (a menos que
    sea <= 81 mg/día), clopidogrel, dipiridamol, warfarina, dabigatrán, rivaroxabán y apixabán.
    -Antecedentes de diátesis hemorrágica o coagulopatía.
    -Recuento de plaquetas por debajo del límite inferior de la normalidad
    -Participación simultánea en algún ensayo clínico terapéutico (diferente del estudio precedente).
    -El tratamiento del estudio (RO7234292) está comercializado en el país del paciente para su enfermedad específica y es accesible para el paciente.
    -Embarazo o lactancia, o intención de quedarse embarazada durante el estudio o en los 5 meses siguientes a la última dosis del fármaco del estudio.
    E.5 End points
    E.5.1Primary end point(s)
    1. Incidence and severity of adverse events, with severity determined according to the Adverse Event Severity Grading Scale
    2. Change from baseline in targeted vital signs
    3. Change from baseline in physical findings
    4. Change from baseline in neurological findings
    5. Change from baseline in behavioral findings, as assessed by the Columbia-Suicide Severity Rating Scale
    6. Change from baseline in cognition
    7. Change from baseline in targeted ECG results
    8. Change from baseline in targeted clinical laboratory test results
    1. Incidencia e intensidad de los acontecimientos adversos, con determinación de la intensidad según la Escala de Gradación de la Intensidad de los Acontecimientos Adversos
    2. Variación con respecto al momento basal de constantes vitales seleccionadas.
    3. Variación con respecto al momento basal de los signos físicos.
    4. Variación con respecto al momento basal de los signos neurológicos.
    5. Variación con respecto al momento basal de los signos conductuales, evaluados mediante
    la Escala de Valoración del Riesgo de Suicidio de Columbia (C-SSRS).
    6. Variación con respecto al momento basal del estado cognitivo.
    7. Variación con respecto al momento basal de los resultados de ECG seleccionados.
    8. Variación con respecto al momento basal de los resultados de pruebas analíticas seleccionadas.
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Up to 5 years
    2-8. Baseline to up to 5 years
    1. Hasta un máximo de 5 años
    2-8. Desde basal hasta un máximo de 5 años
    E.5.2Secondary end point(s)
    Not applicable
    No aplica
    E.5.2.1Timepoint(s) of evaluation of this end point
    Not applicable
    No aplica
    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 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
    PD Biomarker effects
    Immunogenicity
    Efectos FD de los biomarcadores
    Inmunogenicidad
    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 No
    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.8.2.4Number 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 concerned9
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA53
    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
    Australia
    Austria
    Canada
    Chile
    Denmark
    France
    Germany
    Italy
    Netherlands
    New Zealand
    Poland
    Russian Federation
    Spain
    Switzerland
    United Kingdom
    United States
    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
    UVUP
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years5
    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 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: 855
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 45
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state138
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 545
    F.4.2.2In the whole clinical trial 900
    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)
    The Sponsor will offer continued access to Roche IMP (RO7234292) free of charge to eligible patients in accordance with the Roche Global Policy on Continued Access to Investigational Medicinal Product as outlined in the protocol section 4.3.4.
    El promotor ofrecerá acceso continuado al IMP de Roche (RO7234292) gratuitamente a los pacientes elegibles de acuerdo con la política global de Roche de acceso continuado al fármaco en investigación tal y como se refleja en la sección 4.3.4 del protocolo
    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 Decision2019-06-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-06-18
    P. End of Trial
    P.End of Trial StatusOngoing
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