Ublituximab in Autoantibody Positive Immune Mediated Necrotizing Myopathy
United States30 participantsStarted 2026-02-26
Plain-language summary
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)
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. 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.
✓. Diagnosis of IMNM, meeting the 2016 ENMC classification criteria and having either of the following antibodies:
✓. Anti-SRP
✓. Anti-HMGCR
✓. Early disease as defined as onset of objective muscle weakness assessed by a physician and/or CK elevation attributed to IMNM within 1 year of the time of informed consent.
✓. Muscle weakness as assessed by an MMT-8 score \< 142 of 150 and CK \> 1.5x ULN along with abnormality in at least 1 of the following 4 CSMs at screening:
Exclusion criteria
✕. 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.
What they're measuring
1
The primary endpoint is the Total Improvement Score (TIS) at Week 24 reflecting the change from baseline
Timeframe: Baseline to Week 24
Trial details
NCT IDNCT07103746
SponsorNational Institute of Allergy and Infectious Diseases (NIAID)
✕. 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).
✕. Severe liver disease, such as signs of ascites or hepatic encephalopathy.
✕. History of malignancy (excluding non-melanoma skin cancer) unless cured by adequate treatment, with no evidence of recurrence for ≥ 5 years from the time of informed consent.
✕. Recent or ongoing bacterial, viral, or fungal infection requiring systemic treatment within 14 days of the time of informed consent.
✕. Current suppressive therapy for any chronic infections including herpes simplex virus (HSV).