Clinical trial NCT06742190: A Study to Assess the Efficacy and Safety of Empasiprubart Versus IVIg in Adults With Multifocal Motor Neuropathy
Official title: A Phase 3, Randomized, Double-Blinded, Double-Dummy Study Evaluating the Efficacy and Safety of Empasiprubart Versus Intravenous Immunoglobulin in Adults With Multifocal Motor Neuropathy
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Multifocal Motor Neuropathy (MMN)
Lead sponsor: argenx
Sponsor class: INDUSTRY
First posted: 2024-12-19
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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)

Primary outcomes:
measure: Change from baseline in MMN-RODS centile score at week 24
timeFrame: Up to 24 weeks
Secondary outcomes:
measure: Change from baseline in grip strength (3-day moving average) in the most affected hand at week 24
timeFrame: Up to 24 weeks
measure: Change from baseline in mMRC-14 sum score
timeFrame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)
measure: PGI-C actual value
timeFrame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)
measure: Change from baseline in CAP-PRI total score
timeFrame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)
measure: Percentage change from baseline in time to complete the 9-HPT with the dominant hand
timeFrame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)
measure: Incidence of AEs, AESIs and SAEs
timeFrame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)
measure: Serum concentrations over time of empasiprubart
timeFrame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)
measure: Percent changes from baseline in free C2 and total C2 over time
timeFrame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)
measure: Incidence of anti-drug antibodies (ADA) against empasiprubart in serum
timeFrame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)
measure: Incidence of NAb against empasiprubart in serum
timeFrame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)
measure: Change from baseline in grip strength (3-day moving average) of the least affected hand over time
timeFrame: Up to 24 weeks
measure: AUC of change from baseline in grip strength (3-day moving average) for the most and least affected hands
timeFrame: Up to 24 weeks
measure: Percentage change from baseline in time to complete the 9-HPT with the nondominant hand over time
timeFrame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)
measure: Change from baseline in sum scores for mMRC-10 and mMRC-14 restricted to the 2 most affected muscle groups over time
timeFrame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)
measure: Proportion of participants and shift from baseline over time by level of severity on PGI-S
timeFrame: Up to 24 weeks
measure: Change from baseline in Rasch-Transformed Fatigue Severity Scale (RT-FSS) score over time
timeFrame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)
measure: Change from baseline in physical component and mental component scores of 12-Item Short Form Survey (SF-12) over time
timeFrame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)
measure: Proportion of participants and shift from baseline by each dimension of the EQ-5D-5L scale
timeFrame: Up to 24 weeks (Part A), Up to 120 weeks (Part B)
measure: Changes from baseline in MMN-RODS centile score
timeFrame: Up to 120 weeks (part B)
measure: Changes from baseline in grip strength (3-day moving average; both hands)
timeFrame: Up to 120 weeks (part B)
measure: Actual values of PGI-S over time
timeFrame: Up to 120 weeks (part B)
Other outcomes:
Locations:
city: Pomona
country: United States
facility: The Neurology Group
state: California
city: Washington
country: United States
facility: Medstar Health Research Institute
state: District of Columbia
city: Coral Springs
country: United States
facility: Healthcare Innovations Institute, LLC
state: Florida
city: Amherst
country: United States
facility: Dent Neurologic Institute Amherst
state: New York
city: Austin
country: United States
facility: National Neuromuscular Research Institute
state: Texas
city: Houston
country: United States
facility: NeuroCarePlus
state: Texas
Study officials:
References: