MMN, Multifocal Motor Neuropathy (MMN)
Conditions
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. More information can be found here: https://clinicaltrials.argenx.com/empassion
Interventions
BIOLOGICALEmpasiprubart
Intravenous infusion of empasiprubart
BIOLOGICALIVIG (Intravenous Immunoglobulin)
Intravenous infusion of IVIg
A placebo resembling the empasiprubart treatment
OTHERIVIg-placebo
A placebo resembling the IVIg treatment
Sponsors
argenx
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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
Exclusion criteria
* 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)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from baseline in grip strength (4-week average) in the most affected hand at week 24 | Up to 24 weeks |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 |
| 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) | — |
| 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) | — |
| 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. |
| Change from baseline in grip strength (3-day moving average) in the most affected hand at week 24 | Up to 24 weeks | — |
Countries
Australia, Austria, Belgium, Brazil, Canada, China, Czechia, Denmark, France, Germany, Greece, Italy, Japan, Latvia, Lithuania, Netherlands, Norway, Poland, Portugal, Serbia, Slovakia, Slovenia, Spain, Switzerland, United Kingdom, United States
Outcome results
None listed