Facioscapulohumeral muscular dystrophy (FSHD)
Conditions
Interventions
Product Code: RO720-4239/F01-01
Pharmaceutical Form: Solution for injection
Current Sponsor code: RO7204239
Other descriptive name: GYM329
Concentration unit: mg/ml milligram(s)/millilitre
Concentrati
Sponsors
F.Hoffmann-La Roche Ltd
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Age >=18 and =65 years) yes F.1.3.1 Number of subjects for this age range 1
Exclusion criteria
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 ß2-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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: • To evaluate the pharmacodynamic effects of RO7204239 compared with placebo, using magnetic resonance imaging (MRI) • To evaluate the safety of RO7204239 compared with placebo ;Secondary Objective: • 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 ;Primary 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;Timepoint(s) of evaluation of this end point: 1. At Week 52 2-8. Up to approximately 2.5 years | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary 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;Timepoint(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 | — |
Countries
Denmark, Italy, United Kingdom, United States
Contacts
Public ContactTrial Information Support Line-TISL
F. Hoffmann-La Roche Ltd
Outcome results
None listed