A Study to Assess the Efficacy and Safety of Empasiprubart Compared to Intravenously Administered Immunoglobulin in Adults with Multifocal Motor Neuropathy

Australia, Austria, Belgium, Canada, China, Czechia, Denmark, Estonia, France, Germany, Greece, Italy, Japan, Korea, Democratic People's Republic of, Latvia, Lithuania, Netherlands, Norway, Poland, Portugal, Serbia, Slovakia, Slovenia, Spain, Sweden, Switzerland, United Kingdom, United States (ICTRP)

ICTRP Studien-ID
CTIS2024-516473-72-00 (ICTRP)

Offizieller Titel (Genehmigt von der Ethikkommission)
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 (BASEC)

Wissenschaftlicher Titel
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. - ARGX-117-2302 (ICTRP)

Öffentlicher Titel
A study to assess the efficacy and safety of Empasiprubart versus IVIg in adults with Multifocal Motor Neuropathy (ICTRP)

Untersuchte Krankheit(en)
Multifocal Motor Neuropathy
MedDRA version: 21.1Level: PTClassification code: 10065579Term: Multifocal motor neuropathy Class: 100000004852Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]Therapeutic area: Diseases [C] - Nervous System Diseases [C10] (ICTRP)

Untersuchte Intervention
Product Name: HUMAN NORMAL IMMUNOGLOBULIN (IV), Product Code:SUB12041MIG, Pharmaceutical Form: SOLUTION FOR INFUSION, Other descriptive name: Human normal immunoglobulin for intravenous use (IVIG) , Strength: , Product Name: ARGX-117, Product Code:PRD10384929, Pharmaceutical Form: CONCENTRATE FOR SOLUTION FOR INFUSION, Other descriptive name: , Strength: , Product Name: Placebo solution for IV infusion, Product Code:N/A, Pharmaceutical Form: N/A, Other descriptive name: N/A , Strength: , Pharmaceutical form of the placebo: N/A , Product Name: Placebo solution for IV infusion, Product Code:N/A, Pharmaceutical Form: N/A, Other descriptive name: N/A , Strength: , Pharmaceutical form of the placebo: N/A , Product Name: HUMAN NORMAL IMMUNOGLOBULIN (IV), Product Code:SUB12041MIG, Pharmaceutical Form: SOLUTION FOR INFUSION, Other descriptive name: Human normal immunoglobulin for intravenous use (IVIG) , Strength: (ICTRP)

Studientyp
Interventional clinical trial of medicinal product (ICTRP)

Studiendesign
Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Number of treatment arms in the trial: 2 (ICTRP)

Ein-/Ausschlusskriterien
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 andS pneumoniae) within 5 years of screening or is willing to receive immunization at least 14 days before first study drug administration (ICTRP)

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)

Primäre und sekundäre Endpunkte
Main Objective: Part A: To demonstrate the efficacy of empasiprubart compared to IVIg in improving muscle strength;Secondary Objective: Part A - To demonstrate the efficacy of empasiprubart compared to IVIg on functional ability, Part A - To demonstrate the efficacy of empasiprubart compared to IVIg on motor function, Part A - To demonstrate the efficacy of empasiprubart compared to IVIg on manual dexterity, Part A - To evaluate the safety and tolerability of empasiprubart compared to IVIg, Part A - To evaluate the PK, PD, and immunogenicity of empasiprubart, Part A - To evaluate the efficacy of empasiprubart compared to IVIg on muscle strength, motor function, manual dexterity, and patient-reported outcomes, Part A - To evaluate the effect on health-related quality of life for empasiprubart compared to IVIg, Part B - To evaluate the long-term efficacy of empasiprubart in improving functional ability, muscle strength, motor function, manual dexterity, quality of life, and patient-reported outcomes, Part B - To evaluate the long-term safety and tolerability of empasiprubart, Part B - To evaluate the PK, PD, and immunogenicity of empasiprubart, Part B - To evaluate the effect of empasiprubart on long-term health-related quality of life;Primary end point(s): Change from baseline in GS (3 day moving average) in the most affected hand at week 24 (ICTRP)

Secondary end point(s):Part B - Actual values of and percentage change from baseline in time to complete the 9-HPT with the dominant and nondominant hands over time;Secondary end point(s):Part A - Change from baseline in MMN-RODS centile score at week 24;Secondary end point(s):Part A - Change from baseline in mMRC-14 sum score at week 24;Secondary end point(s):Part A - PGI-C actual value over time;Secondary end point(s):Part A - Change from baseline in CAP-PRI total score over time;Secondary end point(s):Part A - Percentage change from baseline in time to complete the 9-HPT with the dominant hand at week 24;Secondary end point(s):Part A - Incidence and severity of AEs and AESIs, Incidence of SAEs;Secondary end point(s):Part A - Clinically meaningful changes in laboratory parameters, vital signs, and ECG results;Secondary end point(s):Part A - Serum concentrations over time and PK parameters of empasiprubart;Secondary end point(s):Part A - Values and percentage change from baseline in free C2 and total C2 over time;Secondary end point(s):Part A - Incidence and prevalence of anti-drug antibodies (ADA) against empasiprubart in serum;Secondary end point(s):Part A - Incidence and prevalence of NAb against empasiprubart in serum;Secondary end point(s):Part A - Change from baseline in GS (3-day moving average) of the least affected hand over time and AUC of change from baseline in GS (daily average) for both hands; percentage change from baseline in GS (3-day moving average) for both hands;Secondary end point(s):Part A - Percentage change from baseline in time to complete the 9-HPT with the nondominant hand over time;Secondary end point(s):Part A - Change from baseline in sum scores for mMRC-10 and mMRC-14 restricted to the 2 most affected muscle groups over time;Secondary end point(s):Part A - Proportion of participants and shift from baseline over time by level of severity on PGI-S;Secondary end point(s):Part A - Change from baseline in Rasch-Transformed Fatigue Severity Scale (RT-FSS) score over time;Secondary end point(s):Part A - Change from baseline in physical component and mental component scores of 12-Item Short Form Survey (SF-12) over time;Secondary end point(s):Part A - Proportion of participants and shift from baseline by each dimension of the EQ5D-5L scale, change from baseline in the EQ-5D-5L visual analog scale over time, and change from baseline in EQ-5D-5L valuation index;Secondary end point(s):Part B - Actual values of and changes from baseline in MMN-RODS centile score, CAPPRI total score, grip strength (daily average; both hands), mMRC-10 sum score, mMRC-14 sum score, and mMRC-14 sum score restricted to the 2 most affected muscle groups over time;Secondary end point(s):Part B - Actual values of PGI-C and PGI-S over time;Secondary end point(s):Part B - Incidence and severity of AEs and AESIs, Incidence of SAEs;Secondary end point(s):Part B - Clinically meaningful changes in laboratory parameters, vital signs, and ECG results;Secondary end point(s):Part B - Serum concentrations over time and PK parameters of empasiprubart;Secondary end point(s):Part B - Values and percentage change from baseline in free C2 and total C2 over time;Secondary end point(s):Part B - Incidence and prevalence of ADA against empasiprubart in serum;Secondary end point(s):Part B - Incidence and prevalence of NAb against empasiprubart in serum;Secondary end point(s):Part B - Change from baseline in RT-FSS score over time;Secondary end point(s):Part B - Change from baseline in physical component and mental component scores of SF-12 over time;Secondary end point(s):Part B - Proportion of participants and shift from baseline by each dimension of the EQ5D-5L scale and change from baseline in the EQ-5D-5L visual analog scale over time (ICTRP)

Registrierungsdatum
26.03.2025 (ICTRP)

Einschluss des ersten Teilnehmers
04.09.2025 (ICTRP)

Sekundäre Sponsoren
nicht verfügbar

Weitere Kontakte
Chief Scientific Officer, ClinicalTrials@argenx.com, 3293103400, Argenx (ICTRP)

Sekundäre IDs
NCT06742190 (ICTRP)

Angaben zur Verfügbarkeit von individuellen Teilnehmerdaten
No (ICTRP)

Weitere Informationen zur Studie
https://euclinicaltrials.eu/search-for-clinical-trials/?lang=en&EUCT=2024-516473-72-00 (ICTRP)