This is a multi-center, Phase 2 trial of ublituximab as first-line combination therapy in early, active autoantibody positive immune-mediated necrotizing myopathy.
The primary objective is to estimate the effect of ublituximab as first add-on combination therapy at 24 weeks compared to placebo in treating early, active autoantibody positive immune-mediated necrotizing myopathy using the validated 2016 American College of Rheumatology/ European League Against Rheumatism (ACR/EULAR) Myositis Response Criteria, as measured by the Total Improvement Score (TIS)
Participant must be able to understand and provide informed consent.
Age 18 years or older at disease onset.
Definite or probable IIM per the 2017 EULAR/ACR classification criteria.
Anti-SRP
End-stage myositis with end-organ involvement that poses additional risk to the participant or confounds the assessment of the participant in the study. Conditions include but are not limited to advanced symptomatic interstitial lung disease (e.g., Forced Vital Capacity (FVC) <60% and/or requiring oxygen therapy) or severe dysphagia.
Irreversible muscle involvement and/or severe atrophy that will pose additional risk to the participant or confound the assessment of the participant in the study. This includes Muscle Damage VAS ≥ 3 cm at screening, documented history of severe atrophy of multiple muscle groups (based on MRI), and/or wheelchair bound.
Uncontrolled interstitial lung disease or any other uncontrolled IIM manifestation that in the opinion of the investigator would be likely to require treatment with prohibited medication during the study.
Diagnosis of other inflammatory or noninflammatory myopathies, including antibody-negative IMNM, inclusion body myositis, overlap myositis, metabolic myopathies, muscular dystrophies or family history of muscular dystrophy, drug induced, cancer-associated myositis, or endocrine-based myositis (except statin induced anti-HMGCR associated IMNM).
Parallel
Ublituximab
DrugInitial dose includes 150 mg dose followed by 450 mg two weeks later; maintenance dose is 450 mg
Placebo for Ublituximab
Drug0.9% sodium chloride injection
The primary endpoint is the Total Improvement Score (TIS) at Week 24 reflecting the change from baseline
TIS is a composite endpoint based on improvement in the 6 Disease Activity Core Set Measure (CSM) scores and ranges from 0 to 100 (2016 American College of Rheumatology \[ACR\] Myositis Response Criteria/European League Against Rheumatism \[EULAR\]). A higher score indicates more improvement.
Change in mean Total Improvement Score (TIS) value
Defined as achieving the Total Improvement Score (TIS)
Proportion of participants achieving minimal improvement response (TIS >= 20 points)
Defined as achieving The Total Improvement Score (TIS) \> =20 points (minimum improvement)
Proportion of participants achieving moderate improvement response (TIS >= 40 points)
Defined as achieving The Total Improvement Score (TIS) \> =40 points (moderate improvement)
Median Time to achieving minimal improvement response (TIS >= 20 points)
Defined as the time to achieving The Total Improvement Score (TIS) \> =20 points (minimum improvement)
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National Institute of Allergy and Infectious Diseases (NIAID)
Lead sponsor
Autoimmunity Centers of Excellence
Collaborator
TG Therapeutics
Collaborator
Rho Federal Systems Division, Inc.
Collaborator
