A Study to Assess the Efficacy and Safety of Empasiprubart Versus IVIg in Adults With Multifocal Motor Neuropathy
- Conditions
- Multifocal Motor Neuropathy (MMN)
- Interventions
- Biological: IVIG (Intravenous Immunoglobulin)Other: IVIg-placeboOther: Empasiprubart-placebo
- Registration Number
- NCT06742190
- Lead Sponsor
- argenx
- Brief Summary
The main purpose of this study is to compare empasiprubart and IVIg in adult patients with MMN. The study consists of a double-blinded part A (empasiprubart, IVIg) and an open-label part B (empasiprubart). The maximum study duration for participants is up to 49 months.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 100
- Is at least 18 years of age and the local legal age of consent for clinical studies
- Has a confirmed diagnosis of definite or probable MMN at screening according to the EFNS/PNS 2010 guidelines
- 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
- Is receiving a maintenance regimen (no change in frequency, and no change in dose >10%) of IVIg for at least 8 weeks before screening (or at least 10 weeks for participants receiving IVIg once every 5 weeks)
- Minimum converted weekly IVIg dose of ≥0.125 g/kg
- 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 study drug administration
- Besides the indication under study, known autoimmune disease (eg, SLE) or any other medical condition 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)
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Part A - empasiprubart + IVIg-placebo Empasiprubart During the double blinded - double dummy part A, participants receive empasiprubart and a placebo resembling the IVIg treatment in this arm Part B - empasiprubart Empasiprubart After completion of part A, participants can proceed to part B where they receive empasiprubart (no IVIg) Part A - empasiprubart + IVIg-placebo IVIg-placebo During the double blinded - double dummy part A, participants receive empasiprubart and a placebo resembling the IVIg treatment in this arm Part A - IVIg + empasiprubart-placebo IVIG (Intravenous Immunoglobulin) During the double blinded - double dummy part A, participants receive IVIg and a placebo resembling the empasiprubart treatment in this arm Part A - IVIg + empasiprubart-placebo Empasiprubart-placebo During the double blinded - double dummy part A, participants receive IVIg and a placebo resembling the empasiprubart treatment in this arm
- Primary Outcome Measures
Name Time Method Change from baseline in MMN-RODS centile score at week 24 Up to 24 weeks The 25-Item Rasch-Built Overall Disability Scale for MMN (MMN-RODS) is a questionnaire about the relationship between daily activities and the participants' health
- Secondary Outcome Measures
Name Time Method Change from baseline in physical component and mental component scores of 12-Item Short Form Survey (SF-12) over time Up to 24 weeks (Part A), Up to 120 weeks (Part B) Change from baseline in grip strength (3-day moving average) in the most affected hand at week 24 Up to 24 weeks Change from baseline in mMRC-14 sum score Up to 24 weeks (Part A), Up to 120 weeks (Part B) The modified Medical Research Council (mMRC)-14 is a questionnaire where each muscle group is scored from 0 (paralysis) to 5 (normal strength). A higher value indicates better muscle strength. The total score is based on the sum of both the left and right side of the body.
PGI-C actual value Up to 24 weeks (Part A), Up to 120 weeks (Part B) The Patient Global Impression of Change (PGI-C) is a 7-point scale depicting a participant's rating of overall improvement. The lower the score, the better the improvement.
Change from baseline in CAP-PRI total score Up to 24 weeks (Part A), Up to 120 weeks (Part B) The Chronic Acquired Polyneuropathy Patient-Reported Index (CAP-PRI) includes the assessment of 15 items. Items will be scored 0 (not at all), 1 (a little bit), or 2 (a lot), yielding a total score that ranges from 0 to 30.
Percentage change from baseline in time to complete the 9-HPT with the dominant hand Up to 24 weeks (Part A), Up to 120 weeks (Part B) The 9-Hole Peg Test (9-HPT) is a quantitative measure of upper extremity (arm and hand) function. Both the dominant and nondominant hands will be tested twice (2 consecutive trials of the dominant hand, followed immediately by 2 consecutive trials of the nondominant hand).
Incidence of AEs, AESIs and SAEs Up to 24 weeks (Part A), Up to 120 weeks (Part B) Serum concentrations over time of empasiprubart Up to 24 weeks (Part A), Up to 120 weeks (Part B) Percent changes from baseline in free C2 and total C2 over time Up to 24 weeks (Part A), Up to 120 weeks (Part B) Incidence of anti-drug antibodies (ADA) against empasiprubart in serum Up to 24 weeks (Part A), Up to 120 weeks (Part B) Incidence of NAb against empasiprubart in serum Up to 24 weeks (Part A), Up to 120 weeks (Part B) Change from baseline in grip strength (3-day moving average) of the least affected hand over time Up to 24 weeks AUC of change from baseline in grip strength (3-day moving average) for the most and least affected hands Up to 24 weeks Percentage change from baseline in time to complete the 9-HPT with the nondominant hand over time Up to 24 weeks (Part A), Up to 120 weeks (Part B) The 9-Hole Peg Test (9-HPT) is a quantitative measure of upper extremity (arm and hand) function. Both the dominant and nondominant hands will be tested twice (2 consecutive trials of the dominant hand, followed immediately by 2 consecutive trials of the nondominant hand).
Change from baseline in sum scores for mMRC-10 and mMRC-14 restricted to the 2 most affected muscle groups over time Up to 24 weeks (Part A), Up to 120 weeks (Part B) The modified Medical Research Council (mMRC) is a questionnaire where each muscle group is scored from 0 (paralysis) to 5 (normal strength). A higher value indicates better muscle strength. The total score is based on the sum of both the left and right side of the body.
Proportion of participants and shift from baseline over time by level of severity on PGI-S Up to 24 weeks Patient Global Impression of Severity (PGIS) is a 7-point scale depicting a participant's rating of overall illness severity. Higher scores mean a higher severity.
Change from baseline in Rasch-Transformed Fatigue Severity Scale (RT-FSS) score over time Up to 24 weeks (Part A), Up to 120 weeks (Part B) Proportion of participants and shift from baseline by each dimension of the EQ-5D-5L scale Up to 24 weeks (Part A), Up to 120 weeks (Part B) Changes from baseline in MMN-RODS centile score Up to 120 weeks (part B) The 25-Item Rasch-Built Overall Disability Scale for MMN (MMN-RODS) is a questionnaire about the relationship between daily activities and the participants' health
Changes from baseline in grip strength (3-day moving average; both hands) Up to 120 weeks (part B) Actual values of PGI-S over time Up to 120 weeks (part B) Patient Global Impression of Severity (PGIS) is a 7-point scale depicting a participant's rating of overall illness severity. Higher scores mean a higher severity.
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Trial Locations
- Locations (6)
The Neurology Group
🇺🇸Pomona, California, United States
Medstar Health Research Institute
🇺🇸Washington, District of Columbia, United States
Healthcare Innovations Institute, LLC
🇺🇸Coral Springs, Florida, United States
Dent Neurologic Institute Amherst
🇺🇸Amherst, New York, United States
National Neuromuscular Research Institute
🇺🇸Austin, Texas, United States
NeuroCarePlus
🇺🇸Houston, Texas, United States