A Study to Assess the Effect of Empasiprubart on Nerve Morphology by Ultrasound in Adults With Multifocal Motor Neuropathy

Phase 1Not Yet RecruitingNCT07610564

argenx60 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Multifocal Motor Neuropathy (MMN)

Interventions

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Outcomes

Primary Outcomes

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)

CSA : cross-sectional area

Time frame: Up to 48 weeks

Secondary Outcomes

Change from baseline in GS in both hands over time

The grip strength (GS) will be measured using a vigorimeter

Time frame: Up to 48 weeks

Change from baseline in MMN-RODS over time

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)

Time frame: Up to 48 weeks

Change from baseline in mMRC-14 sum score over time

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

Time frame: Up to 48 weeks

Percentage change from baseline in time to complete the 9-HPT with both hands over time

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).

Time frame: Up to 48 weeks

Change from baseline in nerve CSA over time as measured by ultrasound imaging of the median nerve and brachial plexus

CSA : cross-sectional area

Time frame: Up to 48 weeks

Serum concentrations of empasiprubart over time

Time frame: Up to 60 weeks

Percentage change from baseline in free C2 and total C2 over time

C2 : complement component 2

Time frame: Up to 60 weeks

Incidence of AEs and AESIs

AE : adverse event ; AESI : adverse event of special interest

Time frame: Up to 60 weeks

Incidence of SAEs

SAE : Serious adverse event

Time frame: Up to 60 weeks

A Study to Assess the Effect of Empasiprubart on Nerve Morphology by Ultrasound in Adults With Multifocal Motor Neuropathy

Overview

Conditions

Interventions

Eligibility

Outcomes

Primary Outcomes

Secondary Outcomes

Central Contacts