HUMANISED IGG1 MONOCLONAL ANTIBODY AGAINST HUMAN LATENT MYOSTATIN for Facioscapulohumeral muscular dystrophy

Inclusion criteria

• {"criterion_text":"- 1. Age ≥ 18 and ≤ 65 years at the time of signing the Informed Consent Form\n- 2. Genetic confirmation of FSHD1 or FSHD2\n- 3. Clinical findings consistent with FSHD according to the investigator’s clinical judgment\n- 4. Ability to walk unassisted 10 meters (Timed 10-Meter Walk Test [10MWT])\n- 5. Ricci Clinical Severity Scale score ≥ 2.5 and ≤ 4\n- 6. Agreement to maintain the same frequency and intensity of physiotherapy, occupational therapy, and other forms of exercise during the clinical study"}

Exclusion criteria

• {"criterion_text":"- 1. Pregnancy or breastfeeding, or intention of becoming pregnant during the study or within 17 months after the final dose of RO7204239\n- 2. Current or previous treatment (or receipt) of anti-myostatin therapies\n- 3. Treatment with any investigational therapy within 90 days prior to screening, or 5 drug-elimination half-lives of the drug, whichever is longer\n- 4. 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\n- 5. Presence of clinically significant ECG abnormalities from average of triplicate measurement at screening indicating a safety risk for participants\n- 6. 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"}

Primary endpoints

• {"endpoint_text":"- 1. Percent change from baseline in contractile muscle volume (CMV) of quadriceps femoris muscles, as assessed by MRI bilaterally","definition_or_measurement_approach":"Assessment by MRI bilaterally measuring percent change from baseline in contractile muscle volume (CMV) of quadriceps femoris muscles."}
• {"endpoint_text":"- 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","definition_or_measurement_approach":"Safety reporting: incidence, severity and causality of adverse events; severity graded per NCI CTCAE v5.0."}
• {"endpoint_text":"- 3. Change from baseline in vital signs, physical findings, ECG, echocardiogram, and clinical laboratory results","definition_or_measurement_approach":"Clinical assessments comparing change from baseline in vital signs, physical exam findings, ECG, echocardiography and laboratory tests."}
• {"endpoint_text":"- 4. Incidence of local and systemic injection reactions","definition_or_measurement_approach":"Recording and reporting incidence of local and systemic injection reactions."}
• {"endpoint_text":"- 5. Incidence of abnormal laboratory findings","definition_or_measurement_approach":"Recording incidence of clinically significant abnormal laboratory results compared to baseline."}
• {"endpoint_text":"- 6. Incidence of abnormal ECG parameters","definition_or_measurement_approach":"Monitoring and reporting incidence of abnormal ECG parameters compared to baseline."}
• {"endpoint_text":"- 7. Incidence of abnormal echocardiographic parameters","definition_or_measurement_approach":"Monitoring and reporting incidence of abnormal echocardiographic findings compared to baseline."}
• {"endpoint_text":"- 8. Incidence of abnormal vital signs","definition_or_measurement_approach":"Monitoring and reporting incidence of clinically significant changes in vital signs from baseline."}

Secondary endpoints

• {"endpoint_text":"- 1. Change from baseline in serum concentrations of total and free latent myostatin, and mature myostatin","definition_or_measurement_approach":"Laboratory measurement of serum concentrations at specified timepoints; change from baseline."}
• {"endpoint_text":"- 2. Percent change from baseline in CMV of 36 muscles based on whole body MRI (excluding muscles with severe fat replacement)","definition_or_measurement_approach":"Whole-body MRI quantification of contractile muscle volume (36 muscles), percent change from baseline; excludes muscles with severe fat replacement."}
• {"endpoint_text":"- 3. Change from baseline in fat fraction of 36 muscles based on whole body MRI (excluding muscles with severe fat replacement)","definition_or_measurement_approach":"Whole-body MRI measurement of muscle fat fraction for 36 muscles; change from baseline; excludes muscles with severe fat replacement."}
• {"endpoint_text":"- 4. Percent change from baseline in CMV of quadriceps femoris muscles, as assessed by MRI bilaterally","definition_or_measurement_approach":"Bilateral MRI assessment of quadriceps CMV; percent change from baseline."}
• {"endpoint_text":"- 5. Change from baseline in fat fraction of quadriceps femoris muscles, as assessed by MRI bilaterally","definition_or_measurement_approach":"Bilateral MRI assessment of quadriceps fat fraction; change from baseline."}
• {"endpoint_text":"- 6. Percent change from baseline in CMV of tibialis anterior muscles, as assessed by MRI bilaterally","definition_or_measurement_approach":"Bilateral MRI assessment of tibialis anterior CMV; percent change from baseline."}
• {"endpoint_text":"- 7. Change from baseline in fat fraction of tibialis anterior muscles, as assessed by MRI bilaterally","definition_or_measurement_approach":"Bilateral MRI assessment of tibialis anterior fat fraction; change from baseline."}
• {"endpoint_text":"- 8. Percent change from baseline in CMV of biceps brachii muscles, as assessed by MRI bilaterally","definition_or_measurement_approach":"Bilateral MRI assessment of biceps brachii CMV; percent change from baseline."}
• {"endpoint_text":"- 9. Change from baseline in fat fraction of biceps brachii muscles, as assessed by MRI bilaterally","definition_or_measurement_approach":"Bilateral MRI assessment of biceps brachii fat fraction; change from baseline."}
• {"endpoint_text":"- 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","definition_or_measurement_approach":"MRI assessment of contractile CSA in proximal lower limb muscles bilaterally; percent change from baseline."}
• {"endpoint_text":"- 11. Change from baseline in the fat fraction of proximal lower limb muscles, as assessed at a single mid femur slice by MRI bilaterally","definition_or_measurement_approach":"MRI fat fraction measurement at a single mid-femur slice bilaterally; change from baseline."}
• {"endpoint_text":"- 12. Serum concentrations of RO7204239 at specified timepoints","definition_or_measurement_approach":"PK sampling to measure serum concentrations of RO7204239 at specified timepoints."}
• {"endpoint_text":"- 13. Cmax of RO7204239 at specified timepoints","definition_or_measurement_approach":"Pharmacokinetic measurement of maximum observed concentration (Cmax)."}
• {"endpoint_text":"- 14. Area under the concentration-time curve (AUC) of RO7204239 at specified timepoints","definition_or_measurement_approach":"Pharmacokinetic calculation of AUC over specified time intervals."}
• {"endpoint_text":"- 15. Ctrough of RO7204239 at specified timepoints","definition_or_measurement_approach":"Measurement of trough concentration (Ctrough) at specified timepoints."}
• {"endpoint_text":"- 16. Prevalence of anti-drug antibodies (ADAs) at baseline and incidence of ADAs during the study","definition_or_measurement_approach":"Immunogenicity assessments to detect ADAs at baseline and during study; report prevalence and incidence."}

Denmark

Earliest CTIS Part Ii Submission Date

18-01-2024

Latest Decision Or Authorization Date

19-04-2024

Processing Time Days

92

Number Of Sites

1

Number Of Participants

17

Italy

Earliest CTIS Part Ii Submission Date

18-01-2024

Latest Decision Or Authorization Date

23-02-2024

Processing Time Days

36

Number Of Sites

2

Number Of Participants

10

Primary sponsor

Full Name

F. Hoffmann-La Roche AG

Organisation Type

Pharmaceutical company

Country Of Registered Address

Switzerland

Contract research organisations

Name

Icon Development Solutions LLC

Responsibilities

code 4 (as listed in sponsor third-party duties); contact BA_submissions@iconplc.com

Name

Biotel Research LLC

Responsibilities

code 4 (as listed in sponsor third-party duties); contact johnda.snyder@gobio.com

Name

Fortrea Inc.

Responsibilities

Other Third Party Duty (as listed); contact roche-pm@fortrea.com

Third parties

• {"country":"United Kingdom","full_name":"Q2q Communications Limited","duties_or_roles":"Meeting Organizer","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Bioniks","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Q Squared Solutions Limited","duties_or_roles":"code 4","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Signant Health Global LLC","duties_or_roles":"Other Third Party Duty; code 3","organisation_type":"Pharmaceutical company"}
• {"country":"France","full_name":"Sysnav","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Publicis Healthcare Communications Group Limited","duties_or_roles":"Medical Communications","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Fortrea Inc.","duties_or_roles":"Other Third Party Duty","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Icon Development Solutions LLC","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Other Third Party Duty","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Biotel Research LLC","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}