IRB Study Number 25-274
Status Recruiting
Phase Phase 3
Location Cleveland Clinic Main Campus
Institute Neurological Institute
Description
This study aims to evaluate the effect (efficacy) and safety of the study drug empasiprubart compared with intravenous immunoglobulin (IVIg) treatment in adults with multifocal motor neuropathy (MMN).
Participation includes a screening period, 2 treatment periods (Part A and Part B) and a safety follow-up period and will last about 49 months. You may visit the study site up to 45 times.
Inclusion Criteria
Is at least 18 years of age
Is capable of providing signed informed consent and complying with protocol requirements
Agrees to use contraceptive measures consistent with local regulations and the study protocol
Women of Child Bearing Potential (WOCBP) must have a negative serum pregnancy test at screening and at baseline before receiving study drug
Has a confirmed diagnosis of definite or probable MMN at screening according to the EFNS/PNS 2010 guidelines and confirmed by the MCC
Has responded to IVIg in the past 5 years
Is receiving IVIg at a treatment interval of once every 2, 3, 4, or 5 weeks, and a dose of 0.4 to 2.0 g/kg body weight per cycle
Minimum converted weekly IVIg dose of ≥0.125 g/kg
Is receiving an optimal IVIg maintenance regimen (as assessed by the investigator, with no changes in frequency, and no change in dose >10%) for at least 8 weeks before screening (or at least 10 weeks for participants receiving IVIg once every 5 weeks) and will receive the same regimen until baseline (day 1) with at least 2 recorded IVIg cycles during screening
Has a maximum MMN-RODS centile score of 90 at screening and confirmed at baseline (day 1)
Has documented immunization against encapsulated bacterial pathogens (N meningitidis and S pneumoniae) within 5 years of screening or is willing to receive immunization at least 14 days before first IMP administration.
Exclusion Criteria
- Besides the indication under study, known autoimmune disease (eg, SLE) or any other medical condition (eg, diabetic neuropathy, CIDP, inflammatory arthritis, or osteoarthritis affecting the hand) that would confound the study results or put the participant at undue risk
- Clinical signs or symptoms suggestive of neuropathies other than MMN, such as motor neuron disease (eg, bulbar signs, brisk reflexes) or other inflammatory neuropathies (eg, sensory neuropathy)
- History of malignancy unless resolved by adequate treatment with no evidence of recurrence for at least 3 years before the first IMP administration. Participants with concurrent instances or a history of the following carcinomas will be eligible if adequately treated: Basal cell or squamous cell skin cancer; Carcinoma in situ of the cervix; Carcinoma in situ of the breast; Incidental histologic finding of prostate cancer (TNM stage T1a or T1b)
- Serious or severe active infection that is not sufficiently resolved before baseline in the investigator’s opinion
- Positive serum test at screening for active infection with any of the following: a.) HBV indicative of an acute or chronic infection unless associated with a negative HBV DNA test result; b.) HCV based on HCV antibody assay unless a negative RNA test result is available; c.) HIV based on test results of a CD4 count <200 cells/mm3; d.) Having an AIDS-defining condition; e.) HIV based on test results of a CD4 count >/= 200 cells/mm3 but not adequately treated with antiviral therapy
- Recent major surgery (including a splenectomy at any time) within 3 months of screening or intention to have major surgery during the study
- Different study drug received in another clinical study <12 weeks or <5 half-lives (whichever is longer) before screening
- Current participation in another interventional clinical study
- Known hypersensitivity to the study drug or 1 of its ingredients
- History (within 12 months before screening) of or current alcohol, drug, or medication abuse as assessed by the investigator
- Pregnant or lactating state or intention to become pregnant during the study
- Previous participation in an empasiprubart clinical study and at least 1 dose of study drug received
- Received cyclophosphamide or mycophenolate mofetil <6 months before screening
- Received any complement inhibitors <6 months before screening
- Received rituximab and/or any other B-cell line-depleting agent <6 months before screening
- Any known complement deficiency
- Severe psychiatric disorder (eg, severe depression, psychosis, bipolar disorder); history of suicide attempt in the previous 12 months; or current suicidal ideation
