Multifocal Motor Neuropathy (MMN)
Conditions
Brief summary
The main purpose of the study is to evaluate empasiprubart use in adults living with Multifocal Motor Neuropathy (MMN). The researchers will evaluate the effect of empasiprubart on the thickness of the nerves, muscle strength, movement, and on the potential improvement of grip strength of the participants in handling objects. The study consists of an open-label treatment period where all participants will receive empasiprubart intravenously (IV). After the last dose of empasiprubart participants will enter a safety follow-up period for up to 15 months. The total study duration for each participant will be approximately 29 months. More information can be found here: clinicaltrials.argenx.com/empasound
Interventions
BIOLOGICALEmpasiprubart IV
Intravenous infusions of empasiprubart
Sponsors
argenx
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Is at least 18 years old * Diagnosed with MMN * Has an MMN-RODS centile score of ≤90 * Has confirmed nerve thickening in at least 1 nerve per ultrasound measurement
Exclusion criteria
* Besides the indication under study, known autoimmune disease or any medical condition that, in the investigator's opinion, would interfere with an accurate assessment of clinical symptoms of MMN or puts the participant at undue risk.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in nerve CSA at week 24 as measured by ultrasound imaging of the median nerve and brachial plexus (individual nerves and average sum score) | Up to 48 weeks | CSA : cross-sectional area |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in GS in both hands over time | Up to 48 weeks | The grip strength (GS) will be measured using a vigorimeter |
| Change from baseline in MMN-RODS over time | Up to 48 weeks | The MMN Rasch-Built Overall Disability Scale (MMN-RODS) is a disease-specific patient-reported outcome instrument that captures activity limitations in patients with MMN. The total raw score will be transformed to a centile score, ranging from 0 (worst outcome) to 100 (best outcome) |
| Change from baseline in mMRC-14 sum score over time | Up to 48 weeks | 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 |
| Percentage change from baseline in time to complete the 9-HPT with both hands over time | Up to 48 weeks | 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 nerve CSA over time as measured by ultrasound imaging of the median nerve and brachial plexus | Up to 48 weeks | CSA : cross-sectional area |
| Serum concentrations of empasiprubart over time | Up to 60 weeks | — |
| Percentage change from baseline in free C2 and total C2 over time | Up to 60 weeks | C2 : complement component 2 |
| Incidence of AEs and AESIs | Up to 60 weeks | AE : adverse event ; AESI : adverse event of special interest |
| Incidence of SAEs | Up to 60 weeks | SAE : Serious adverse event |
Countries
Georgia
Contacts
CONTACTSabine Coppieters, MD
Outcome results
None listed