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    Summary
    EudraCT Number:2018-003898-94
    Sponsor's Protocol Code Number:BN40955
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2020-10-08
    Trial results View 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 ConcernedNetherlands - Competent Authority
    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
    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
    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 SponsorF.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 INNtominersen
    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)
    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
    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
    E.2.2Secondary objectives of the trial
    Not applicable
    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

    The following eligibility criteria only apply to patients who were eligible for Study BN40423 (GENERATION HD1) but were not randomized due to logistical challenges resulting from the COVID-19 pandemic
    - Age 25-65 years
    - Manifest HD diagnosis, defined as a diagnostic confidence level (DCL) score of 4
    - Independence Scale (IS) score >= 70
    - Genetically confirmed disease by direct DNA testing with a CAP score > 400
    - Ability to read the words "red", "blue", and "green" in native language
    - Ability to walk unassisted without a cane or walker and move about without a wheelchair on a daily basis as determined at screening and baseline visit
    - Body mass index 16-32 kg/m2; total body weight > 40 kg
    - Ability to undergo and tolerate MRI scans
    - Ability to tolerate blood draws and lumbar punctures
    - Creatinine clearance (CrCl) >= 60 mL/min (Cockcroft-Gault formula)
    - Ability and willingness, in the investigator's judgment, to comply with all aspects of the protocol including completion of interviews and questionnaires for the duration of the study
    - Stable medical, psychiatric, and neurological status for at least 12 weeks prior to screening and at the time of enrollment
    - 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 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. Men must refrain from donating sperm during this same period
    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

    The following eligibility criteria only apply to patients who were eligible for Study BN40423 (GENERATION HD1) but were not randomized due to logistical challenges resulting from the COVID-19 pandemic
    - History of attempted suicide or suicidal ideation with plan that required hospital visit and/or change in level of care within 12 months prior to screening
    - Current active psychosis, confusional state, or violent behavior
    - Any serious medical condition or clinically significant laboratory, or vital sign abnormality or claustrophobia at screening that, in the investigator's judgment, precludes the patient's safe participation in and completion of the study - History known to the investigator or presence of an abnormal ECG that is clinically significant in the investigator's opinion, including complete left bundle branch block, second- or third degree atrioventricular heart block, or evidence of prior myocardial infarction - Lifetime clinical diagnosis of chronic migraines
    - Pregnant or breastfeeding, or intending to become pregnant during the study or within 5 months after the final dose of study drug
    - Presence of an implanted shunt for the drainage of CSF or an implanted CNS catheter
    - Positive for hepatitis C virus (HCV) or hepatitis B surface antigen (HBsAg) at screening
    - Known HIV infection
    - Current or previous use of an antisense oligonucleotide (including small interfering RNA)
    - Current or previous use of anti-psychotics prescribed for a primary independent psychotic disorder, cholinesterase inhibitors, memantine, amantadine, or riluzole within 12 weeks from initiation of study treatment
    - Treatment with investigational therapy within 4 weeks or 5 drug elimination half lives prior to screening, whichever is longer
    - Antiplatelet or anticoagulant therapy within 14 days prior to screening or anticipated use during the study
    - History of bleeding diathesis or coagulopathy - Platelet count less than the lower limit of normal
    - History of gene therapy, cell transplantation, or any experimental brain surgery
    - Concurrent participation in any therapeutic clinical trial.
    - Drug and/or alcohol abuse or psychological or physiological dependency within 12 months prior to screening, as per the investigator's judgment
    - Poor peripheral venous access
    - Scoliosis or spinal deformity or surgery making IT injection not feasible in an outpatient setting and potentially interfering with distribution of RO7234292 up the neuraxis
    - An infection requiring oral or IV antibiotics within 14 days prior to screening and prior to randomization
    - Antiretroviral medications, including antiretroviral medication taken as prophylaxis within 12 months of study enrollment
    - Malignancy within 5 years prior to screening, except basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated
    - Preexisting intra-axial or extra-axial lesions as assessed by a centrally read MRI scan during the screening period
    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
    9. Relationship between lab parameter change and safety targeted endpoints
    10. Relationship between MRI parameter change and safety targeted endpoints
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Up to 6 years
    2-10. Baseline to up to 6 years
    E.5.2Secondary end point(s)
    Not applicable
    E.5.2.1Timepoint(s) of evaluation of this end point
    Not applicable
    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
    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 concerned2
    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
    Australia
    Canada
    Chile
    New Zealand
    Russian Federation
    United States
    Austria
    Denmark
    France
    Germany
    Italy
    Netherlands
    Poland
    Spain
    Switzerland
    United Kingdom
    Argentina
    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
    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: 1045
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 55
    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 state25
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 638
    F.4.2.2In the whole clinical trial 1050
    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.
    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 Decision2020-10-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-06-04
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-03-30
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    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.

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