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    Summary
    EudraCT Number:2021-006255-34
    Sponsor's Protocol Code Number:BN43703
    National Competent Authority:Denmark - DHMA
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-07-25
    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 ConcernedDenmark - DHMA
    A.2EudraCT number2021-006255-34
    A.3Full title of the trial
    A PHASE II, MULTICENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO EVALUATE THE PHARMACODYNAMICS, SAFETY, TOLERABILITY, PHARMACOKINETICS, AND EFFICACY OF RO7204239 IN PARTICIPANTS WITH FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate the Pharmacodynamics, Safety, Tolerability, Pharmacokinetics, and Efficacy of RO7204239 Compared with Placebo in Participants with Facioscapulohumeral Muscular Dystrophy
    A.4.1Sponsor's protocol code numberBN43703
    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
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.2Product code RO720-4239/F01-01
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeRO7204239
    D.3.9.3Other descriptive nameGYM329
    D.3.9.4EV Substance CodeSUB217042
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number80
    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 No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboConcentrate for solution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Facioscapulohumeral muscular dystrophy (FSHD)
    E.1.1.1Medical condition in easily understood language
    FSHD is a group of heritable diseases that cause degeneration of muscle and progressive weakness, in which the muscles of the face, shoulder blades, and upper arms are among the most affected.
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    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
    • To evaluate the pharmacodynamic effects of RO7204239 compared with placebo, using magnetic resonance imaging (MRI)
    • To evaluate the safety of RO7204239 compared with placebo
    E.2.2Secondary objectives of the trial
    • To evaluate additional pharmacodynamic effects of RO7204239 compared to placebo, using serum samples and MRI
    • To evaluate pharmacokinetics (PK) parameters for RO7204239
    • To evaluate immune response to RO7204239
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Age >=18 and <=65 years at the time of signing the Informed Consent Form
    • Genetic confirmation of FSHD1 or FSHD2
    • Clinical findings consistent with FSHD according to the investigator’s clinical judgment
    • Ability to walk unassisted 10 meters (Timed 10-Meter Walk Test [10MWT])
    • Ricci Clinical Severity Scale score ≥2.5 and ≤ 4
    • Agreement to maintain the same frequency and intensity of physiotherapy, occupational therapy, and other forms of exercise during the clinical study
    • Ability and willingness to comply with the study protocol and to complete all study procedures, measurements, and visits
    • For female participants of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraception, during treatment with study drug and for 17 months after the final dose of RO7204239
    • For male participants: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive methods, and agree to refrain from donating sperm, during treatment with study drug and for 4 months after the final dose of RO7204239
    E.4Principal exclusion criteria
    • Pregnancy or breastfeeding, or intention of becoming pregnant during the study or within 17 months after the final dose of RO7204239
    • Current or previous treatment (or receipt) of anti-myostatin therapies
    • Treatment with any investigational therapy within 90 days prior to screening, or 5 drug-elimination half-lives of the drug, whichever is longer
    • Contraindications to MRI scans, difficulties maintaining a prolonged supine position, or any other clinical history or examination finding that would pose a potential hazard in combination with MRI
    • Presence of clinically significant ECG abnormalities from average of triplicate measurement at screening indicating a safety risk for participants
    • Presence of clinically significant cardiovascular disease indicating a safety risk for participants
    • Presence of clinically significant abnormal findings in echocardiography at screening, with the exception of mitral valve prolapse, which does not exclude participants from the study
    • Any major illness within 1 month before screening
    • Ascertained or presumptive hypersensitivity to RO7204239, or to the constituents of its formulation
    • Concurrent disease or a medical condition or abnormality in clinical laboratory tests that could interfere with, or treatment of which might interfere with, the conduct of the study, or that would pose an unacceptable risk to the participant in this study
    • History of malignancy
    • Any clinically relevant history of anaphylactic reaction requiring inotropic support
    • Any abnormal skin conditions, pigmentation, or lesions in the area intended for SC injection (abdomen) and that would prevent visualization of potential injection-site reactions to RO7204239
    • Immobilization, surgical procedures, fracture, or trauma to the upper or lower limbs within 90 days prior to screening or longer, if judged by the investigator that it may affect motor function assessment
    • Any planned surgery that may affect a participant’s motor function assessment, including participants who have had surgery of scapular fixation within the 12 months preceding screening or that are planned during the study
    • Substance abuse within 12 months prior to screening or are at risk of substance abuse per investigator’s judgment
    • Use of the following medications within 90 days prior to enrollment:
    – Salbutamol or another β2-adrenergic agonist taken orally
    – Creatine
    – Recombinant human growth hormone
    – Recombinant human insulin growth factor-1
    – Testosterone, oxandrolone or other anabolic steroid
    – Chronic oral or parenteral use of corticosteroids (inhaled corticosteroid use is allowed) unless required to manage injection reactions
    – Agents anticipated to increase or decrease muscle volume or strength
    E.5 End points
    E.5.1Primary end point(s)
    1. Percent change from baseline in contractile muscle volume (CMV) of quadriceps femoris muscles, as assessed by MRI bilaterally
    2. Incidence, severity, and causal relationship of adverse events, with severity determined according to National Cancer Institute Common Terminology Criteria for Adverse Events v5.0
    3. Change from baseline in vital signs, physical findings, ECG, echocardiogram, and clinical laboratory results
    4. Incidence of local and systemic injection reactions
    5. Incidence of abnormal laboratory findings
    6. Incidence of abnormal ECG parameters
    7. Incidence of abnormal echocardiographic parameters
    8. Incidence of abnormal vital signs
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. At Week 52
    2-8. Up to approximately 2.5 years
    E.5.2Secondary end point(s)
    1. Change from baseline in serum concentrations of total and free latent myostatin, and mature myostatin
    2. Percent change from baseline in CMV of 36 muscles based on whole body MRI (excluding muscles with severe fat replacement)
    3. Change from baseline in fat fraction of 36 muscles based on whole body MRI (excluding muscles with severe fat replacement)
    4. Percent change from baseline in CMV of quadriceps femoris muscles, as assessed by MRI bilaterally, at Week 28 of treatment
    5. Change from baseline in fat fraction of quadriceps femoris muscles, as assessed by MRI bilaterally
    6. Percent change from baseline in CMV of tibialis anterior muscles, as assessed by MRI bilaterally
    7. Change from baseline in fat fraction of tibialis anterior muscles, as assessed by MRI bilaterally
    8. Percent change from baseline in CMV of biceps brachii muscles, as assessed by MRI bilaterally
    9. Change from baseline in fat fraction of biceps brachii muscles, as assessed by MRI bilaterally
    10. Percent change from baseline in the contractile cross-sectional area (CSA) of skeletal muscle in the proximal lower limb muscles, as assessed by MRI bilaterally
    11. Change from baseline in the fat fraction of proximal lower limb muscles, as assessed at a single mid femur slice by MRI bilaterally
    12. Serum concentrations of RO7204239 at specified timepoints
    13. Cmax of RO7204239 at specified timepoints
    14. Area under the concentration-time curve (AUC) of RO7204239 at specified timepoints
    15. Ctrough of RO7204239 at specified timepoints
    16. Prevalence of anti-drug antibodies (ADAs) at baseline and incidence of ADAs during the study
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Through 2 years
    2-3. At Weeks 28 and 52
    4. At Week 28
    5-11. At Weeks 28 and 52
    12-15. Through 2 years
    16. Baseline up to approximately 2 years
    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 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 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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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 Yes
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned Information not present in EudraCT
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States No
    E.8.5.1Number of sites anticipated in the EEA3
    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
    United States
    Denmark
    Italy
    United Kingdom
    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
    The end of this study is defined as the date of the last visit of the last
    participant in the study or the date at which the last data point
    required for statistical analysis (i.e., for the final analysis) or safety
    follow-up is received from the last participant in the study, whichever
    occurs later.
    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 months11
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months11
    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: 47
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 0
    F.4.2.2In the whole clinical trial 48
    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 RO7204239 free of charge to eligible participants in accordance with the Roche Global Policy on Continued Access to Investigational Medicinal Product. For detailed information refer to protocol BN43703, section 6.6 Continued access to study treatment after the end of the study.
    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 Decision2022-08-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-08-26
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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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
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