A Study to Assess the Efficacy and Safety of Empasiprubart Versus IVIg in Adults With Multifocal Motor Neuropathy

Phase 3RecruitingNCT06742190

argenx115 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

115

Conditions

Multifocal Motor Neuropathy (MMN)MMN

Interventions

EmpasiprubartBIOLOGICAL

Intravenous infusion of empasiprubart

IVIG (Intravenous Immunoglobulin)BIOLOGICAL

Intravenous infusion of IVIg

Empasiprubart-placeboOTHER

A placebo resembling the empasiprubart treatment

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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)

Locations (108)

Outcomes

Primary Outcomes

Change from baseline in grip strength (3-day moving average) in the most affected hand at week 24

Time frame: Up to 24 weeks

Secondary Outcomes

Change from baseline in MMN-RODS centile score at week 24

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

Time frame: Up to 24 weeks

Change from baseline in mMRC-14 sum score

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 24 weeks (Part A), Up to 120 weeks (Part B)

PGI-C actual value

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.

Time frame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)

Change from baseline in CAP-PRI total score

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.

Time frame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)

Percentage change from baseline in time to complete the 9-HPT with the dominant hand

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).

Central Contacts

Sabine Coppieters, MD

CONTACT

857-350-4834 clinicaltrials@argenx.com

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.

A placebo resembling the IVIg treatment

HonorHealth Neurology - Bob Bove Neuroscience Institute - Neurology

Scottsdale, Arizona, United States

RECRUITING

The Neurology Group

Pomona, California, United States

RECRUITING

Samir Macwan, M.D., Inc.

Rancho Mirage, California, United States

RECRUITING

University of California San Francisco

San Francisco, California, United States

RECRUITING

University of Colorado - Anschutz Cancer Pavilion (Neuro Onc Clinic)

Aurora, Colorado, United States

RECRUITING

Yale University School of Medicine

New Haven, Connecticut, United States

RECRUITING

Medstar Health Research Institute

Washington D.C., District of Columbia, United States

RECRUITING

EZR Research LLC

Boca Raton, Florida, United States

RECRUITING

Healthcare Innovations Institute, LLC

Coral Springs, Florida, United States

RECRUITING

University of Miami Miller School of Medicine

Miami, Florida, United States

RECRUITING

...and 98 more locations

Time frame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)

Incidence of AEs, AESIs and SAEs

Time frame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)

Serum concentrations over time of empasiprubart

Time frame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)

Percent changes from baseline in free C2 and total C2 over time

Time frame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)

Incidence of anti-drug antibodies (ADA) against empasiprubart in serum

Time frame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)

Incidence of NAb against empasiprubart in serum

Time frame: 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

Time frame: Up to 24 weeks

AUC of change from baseline in grip strength (3-day moving average) for the most and least affected hands

Time frame: Up to 24 weeks

Percentage change from baseline in time to complete the 9-HPT with the nondominant hand over time

Time frame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)

Change from baseline in sum scores for mMRC-10 and mMRC-14 restricted to the 2 most affected muscle groups over time

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.

Time frame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)

Proportion of participants and shift from baseline over time by level of severity on PGI-S

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.

Time frame: Up to 24 weeks

Change from baseline in Rasch-Transformed Fatigue Severity Scale (RT-FSS) score over time

Time frame: 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

Time frame: 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

Time frame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)

Changes from baseline in MMN-RODS centile score

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

Time frame: Up to 120 weeks (part B)

Changes from baseline in grip strength (3-day moving average; both hands)

Time frame: Up to 120 weeks (part B)

Actual values of PGI-S over time

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.

Time frame: Up to 120 weeks (part B)