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    The EU Clinical Trials Register currently displays   42886   clinical trials with a EudraCT protocol, of which   7063   are clinical trials conducted with subjects less than 18 years old.
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    Summary
    EudraCT Number:2016-004184-39
    Sponsor's Protocol Code Number:BP39054
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2018-10-01
    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 number2016-004184-39
    A.3Full title of the trial
    AN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS OF RO7034067 IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of RO7034067 in adult and pediatric patients with spinal muscular atrophy
    A.4.1Sponsor's protocol code numberBP39054
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/284/2017
    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
    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
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameRO7034067
    D.3.2Product code RO7034067/F13
    D.3.4Pharmaceutical form Powder for oral solution
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNRisdiplam
    D.3.9.1CAS number 1825352-65-5
    D.3.9.2Current sponsor codeRO7034067/F13
    D.3.9.3Other descriptive nameRO7034067
    D.3.9.4EV Substance CodeSUB179686
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number60
    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
    Spinal Muscular Atrophy (SMA)
    E.1.1.1Medical condition in easily understood language
    Spinal muscular atrophy, a genetic progressive neuromuscular disease characterized by profound weakness and muscle atrophy that is the leading genetic cause of death in babies and toddlers
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10041582
    E.1.2Term Spinal muscular atrophy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    •To evaluate the safety and tolerability of RO7034067
    •To investigate the pharmacokinetics (PK) of RO7034067 and metabolites as appropriate
    E.2.2Secondary objectives of the trial
    •To investigate the PK/ pharmacodynamics (PD) relationship of RO7034067. The PD investigations will include analyses of Survival of motor neuron (SMN) mRNA splice forms and SMN protein
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -Males and females 6 months to 60 years of age inclusive (at screening)
    - Confirmed diagnosis of 5q-autosomal recessive SMA, including:
    •Genetic confirmation of homozygous deletion or heterozygosity predictive of loss of function of the SMN1 gene.
    •Clinical history, signs, or symptoms attributable to SMA
    - Previous enrollment in Study BP29420 (Moonfish) with the splicing modifier RO6885247 or previous treatment with nusinersen (defined as having received >= 4 doses of nusinersen, provided that the last dose was received >=90 days prior to screening) or olesoxime (provided that the last dose was received <=12 months and >= 90 days prior to screening)
    - Adequately recovered from any acute illness at the time of screening and considered well enough to participate in the opinion of the Investigator
    - Women of childbearing potential: Negative blood pregnancy test at screening, agreement to remain abstinent or use contraceptive measures, and agreement to refrain from donating eggs for at least 28 days after the final dose of study drug For men: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive measures and agreement to refrain from donating sperm
    -For patients aged 2 years or younger at screening:
    •Receiving adequate nutrition and hydration (with or without gastrostomy) at the time of screening, in the opinion of the Investigator
    •Medical care meets local accepted standard of care, in the opinion of the Investigator
    •Would be able to complete all study procedures, measurements and visits, and the parent or caregiver of the patient has adequately supportive psychosocial circumstances, in the opinion of the Investigator
    •Parent or caregiver of patient is willing to consider nasogastric, naso-jejunal or gastrostomy tube placement, as recommended by the Investigator, during the study to maintain safe hydration, nutrition and treatment delivery
    •Parent or caregiver of patient is willing to consider the use of non-invasive ventilation, as recommended by the Investigator during the study
    E.4Principal exclusion criteria
    -Inability to meet study requirements
    -Concomitant participation in any investigational drug or device study
    -Previous participation in any investigational drug or device study, with the exception of studies with olesoxime or nusinersen,within 90 days prior to screening, or 5 half-lives of the drug, whichever is longer
    -Any history of gene or cell therapy
    -Unstable gastrointestinal, renal, hepatic, endocrine, or cardiovascular system diseases as considered to be clinically significant by the Investigator
    -Inadequate venous or capillary blood access for the study procedures, in the opinion of the Investigator
    -For patients aged < 2 years, hospitalization for a pulmonary event within 2 months prior to screening and pulmonary function not fully recovered at the time of screening
    -Lactating women
    -Suspicion of regular consumption of drugs of abuse
    -For adults and adolescents only, i.e., aged > 12 years, positive urine test for drugs of abuse or alcohol at the screening or Day − 1 visit
    -Presence of clinically significant ECG abnormalities before study drug administration
    -Presence of clinically significant cardiovascular, blood pressure, and heart rate measures
    -History of malignancy if not considered cured
    -For patients aged > 6 years, significant risk for suicidal behavior, in the opinion of the Investigator as assessed by the Columbia-Suicide Severity Rating Scale (C-SSRS)
    -Any major illness within one month before the screening examination or any febrile illness within one week prior to screening and up to first dose administration
    -Use of any OCT-2 and MATE substrates within 2-weeks before dosing including the mother, if breastfeeding the patient
    -Use of the following medications within 90 days prior to enrollment: riluzole, valproic acid, hydroxyurea, sodium phenylbutyrate, butyrate derivatives, creatine, carnitine, growth hormone, anabolic steroids, probenecid, agents anticipated to increase or decrease muscle strength, agents with known or presumed histone deacetylase (HDAC) inhibitory effect, and medications with known phototoxicity liabilities (e.g., oral retinoids including over-the-counter formulations, amiodarone. Use of inhaled corticosteroids is allowed
    -Recently initiated treatment for SMA (within < 6 weeks prior to enrollment) with oral salbutamol or another β2-adrenergic agonist taken orally is not allowed. Patients who have been on oral salbutamol (or another β2-adrenergic agonist) for >= 6 weeks before enrollment and have shown good tolerance are allowed. The dose of β2-adrenergic agonist should remain stable as much as possible for the duration of the study. Use of inhaled β2-adrenergic agonists (e.g., for the treatment of asthma) is allowed
    -Any prior use of chloroquine, hydroxychloroquine, retigabin, vigabatrin or thioridazine is not allowed. Use of other medications known to or suspected of causing retinal toxicity within one year prior to enrollment is not allowed
    -Clinically significant abnormalities in laboratory test results
    -Donation or loss of blood >= 10% of blood volume within three months prior to screening
    -Ascertained or presumptive hypersensitivity (e.g., anaphylactic reaction) to RO7034067 or to the constituents of its formulation
    -Concomitant disease or condition that could interfere with, or treatment of which might interfere with, the conduct of the study, or that would, in the opinion of the Investigator, pose an unacceptable risk to the patient in this study
    -Recent history (less than one year) of ophthalmological diseases
    E.5 End points
    E.5.1Primary end point(s)
    1.Incidence and severity of adverse events and serious adverse events with severity determined according to National Cancer Institute Common Terminology Criteria for Adverse Events scale, Version 4.0
    2.Incidence of treatment discontinuations due to adverse events
    3.Incidence of abnormal vital sign, laboratory, and ECG abnormalities
    4.Incidence of clinically significant findings on ophthalmological examination
    5.Incidence of clinically significant findings on neurological examination
    6.Physical examination including examination of the skin, mouth, pharynx and larynx
    7.Incidence of emergence or worsening of symptoms as measured by the Columbia-Suicide Severity Rating Scale (C-SSRS) (adult version for adults and adolescents, pediatric version for patients aged 6-11 years)
    8.Mean plasma concentrations of RO7034067 and metabolites
    9.Peak plasma concentration (Cmax)
    10.Area under the curve (AUC)
    11.Concentration at the end of a dosing interval (Ctrough) to assess steady-state
    E.5.1.1Timepoint(s) of evaluation of this end point
    1-6. Up to 5 years
    7. Aged 6 to 60 Years: Screening, Day-1, Day 182, 364, 546, 728, at extension phase and follow-up visit 1 (Week 112)
    8-11. Aged 2 to 60 Years: Days 1, 14, 28, 91, 183, 273, 365, 456, 637, 729, at early withdrawal and at follow-up visit 1;
    Aged 6 month to 2 Years: Days 1, 14, 28, 91, 182, 273,364, 456, 546, 637, 728, every 26 weeks during the extension phase and at early withdrawal
    E.5.2Secondary end point(s)
    1.SMN mRNA in blood
    2.SMN protein levels in blood
    E.5.2.1Timepoint(s) of evaluation of this end point
    1.Aged 2 to 60 Years: Days -1, 1, 28, 91, 183, 365, 729, at early withdrawal, and at follow-up visit 1; Patients Aged 6 Months to <2 Years: Day 1, 28, 182, 364 and 728 and at early withdrawal

    2.Aged 2 to 60 Years: Day -1, Day 28, 91, 183, 365, 729, at early withdrawal, and at follow-up visit 1; Patients Aged 6 Months to <2 Years: Day 1, 28, 182, 364 and 728 and at early withdrawal

    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 Yes
    E.6.5Efficacy No
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    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
    Tolerability
    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 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.8.3 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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA20
    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
    Belgium
    France
    Germany
    Italy
    Netherlands
    Poland
    Switzerland
    United Kingdom
    United States
    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
    Last Patient Last Visit
    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 months5
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    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: 62
    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: 6
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 56
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 63
    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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Pediatric patients may provide according to local regulation, informed assent informed consent for study participation will be obtained from parents
    or legal guardian
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state24
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 100
    F.4.2.2In the whole clinical trial 125
    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 post-trial access to the study drug free of charge to eligible patients in accordance with the Roche Global Policy on Continued Access to Investigational Medicinal Product.

    The Roche Global Policy on Continued Access to Investigational Medicinal Product is available at the following Web site:
    http://www.roche.com/policy_continued_access_to_investigational_medicines.pdf
    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 Decision2018-12-11
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial Status
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