EMPASIPRUBART for Multifocal motor neuropathy

Inclusion criteria

• {"criterion_text":"- Is at least 18 years of age and the local legal age of consent for clinical studies\n- Has a confirmed diagnosis of definite or probable MMN at screening according to the EFNS/PNS 2010 guidelines\n- Has responded to IVIg in the past 5 years.\n- 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\n- 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)\n- Minimum converted weekly IVIg dose of ≥0.125 g/kg\n- 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

• {"criterion_text":"- 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\n- 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 endpoints

• {"endpoint_text":"- Change from baseline in GS (3 day moving average) in the most affected hand at week 24","definition_or_measurement_approach":"Change from baseline in grip strength (GS), measured as a 3-day moving average in the most affected hand, assessed at Week 24."}

Secondary endpoints

• {"endpoint_text":"- Part A - Change from baseline in MMN-RODS centile score at week 24","definition_or_measurement_approach":"Change from baseline in MMN-RODS centile score measured at Week 24."}
• {"endpoint_text":"- Part A - Change from baseline in mMRC-14 sum score at week 24","definition_or_measurement_approach":"Change from baseline in modified MRC-14 sum score measured at Week 24."}
• {"endpoint_text":"- Part A - PGI-C actual value over time","definition_or_measurement_approach":"Patient Global Impression of Change (PGI-C) actual values collected over time."}
• {"endpoint_text":"- Part A - Change from baseline in CAP-PRI total score over time","definition_or_measurement_approach":"Change from baseline in CAP-PRI total score measured over time."}
• {"endpoint_text":"- Part A - Percentage change from baseline in time to complete the 9-HPT with the dominant hand at week 24","definition_or_measurement_approach":"Percentage change from baseline in time to complete 9-Hole Peg Test (9-HPT) with dominant hand at Week 24."}
• {"endpoint_text":"- Part A - Incidence and severity of AEs and AESIs, Incidence of SAEs","definition_or_measurement_approach":"Incidence and severity of adverse events (AEs), adverse events of special interest (AESIs), and incidence of serious adverse events (SAEs) as recorded during Part A."}
• {"endpoint_text":"- Part A - Clinically meaningful changes in laboratory parameters, vital signs, and ECG results","definition_or_measurement_approach":"Clinically meaningful changes in laboratory parameters, vital signs, and ECG results assessed during Part A."}
• {"endpoint_text":"- Part A - Serum concentrations over time and PK parameters of empasiprubart","definition_or_measurement_approach":"Serum concentration-time profiles and pharmacokinetic (PK) parameters of empasiprubart measured over time."}
• {"endpoint_text":"- Part A - Values and percentage change from baseline in free C2 and total C2 over time","definition_or_measurement_approach":"Values and percent change from baseline for free C2 and total C2 measured over time."}
• {"endpoint_text":"- Part A - Incidence and prevalence of anti-drug antibodies (ADA) against empasiprubart in serum","definition_or_measurement_approach":"Incidence and prevalence of anti-drug antibodies (ADA) against empasiprubart measured in serum."}
• {"endpoint_text":"- Part A - Incidence and prevalence of NAb against empasiprubart in serum","definition_or_measurement_approach":"Incidence and prevalence of neutralizing antibodies (NAb) against empasiprubart measured in serum."}
• {"endpoint_text":"- 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","definition_or_measurement_approach":"Change from baseline in grip strength (3-day moving average) of least affected hand over time; 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."}
• {"endpoint_text":"- Part A - Percentage change from baseline in time to complete the 9-HPT with the nondominant hand over time","definition_or_measurement_approach":"Percentage change from baseline in time to complete 9-HPT with the non-dominant hand measured over time."}
• {"endpoint_text":"- Part A - Change from baseline in sum scores for mMRC-10 and mMRC-14 restricted to the 2 most affected muscle groups over time","definition_or_measurement_approach":"Change from baseline in summed mMRC-10 and mMRC-14 scores restricted to the two most affected muscle groups, measured over time."}
• {"endpoint_text":"- Part A - Proportion of participants and shift from baseline over time by level of severity on PGI-S","definition_or_measurement_approach":"Proportion of participants and shift from baseline over time by PGI-S severity levels."}
• {"endpoint_text":"- Part A - Change from baseline in Rasch-Transformed Fatigue Severity Scale (RT-FSS) score over time","definition_or_measurement_approach":"Change from baseline in RT-FSS score measured over time."}
• {"endpoint_text":"- Part A - Change from baseline in physical component and mental component scores of 12-Item Short Form Survey (SF-12) over time","definition_or_measurement_approach":"Change from baseline in SF-12 physical and mental component scores measured over time."}
• {"endpoint_text":"- 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","definition_or_measurement_approach":"Proportion and shift from baseline by each EQ-5D-5L dimension; change in EQ-5D-5L visual analogue scale and valuation index over time."}
• {"endpoint_text":"- 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","definition_or_measurement_approach":"Actual values and changes from baseline for listed measures (MMN-RODS, CAPPRI, grip strength, mMRC scores) collected during Part B over time."}
• {"endpoint_text":"- 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","definition_or_measurement_approach":"Actual values and percentage change from baseline in time to complete 9-HPT with dominant and non-dominant hands during Part B over time."}
• {"endpoint_text":"- Part B - Actual values of PGI-C and PGI-S over time","definition_or_measurement_approach":"Actual PGI-C and PGI-S values collected over time during Part B."}
• {"endpoint_text":"- Part B - Incidence and severity of AEs and AESIs, Incidence of SAEs","definition_or_measurement_approach":"Incidence and severity of AEs and AESIs and incidence of SAEs as recorded during Part B."}
• {"endpoint_text":"- Part B - Clinically meaningful changes in laboratory parameters, vital signs, and ECG results","definition_or_measurement_approach":"Clinically meaningful changes in laboratory parameters, vital signs and ECG results assessed during Part B."}
• {"endpoint_text":"- Part B - Serum concentrations over time and PK parameters of empasiprubart","definition_or_measurement_approach":"Serum concentrations and PK parameters of empasiprubart measured over time during Part B."}
• {"endpoint_text":"- Part B - Values and percentage change from baseline in free C2 and total C2 over time","definition_or_measurement_approach":"Values and percent change from baseline in free and total C2 measured over time during Part B."}
• {"endpoint_text":"- Part B - Incidence and prevalence of ADA against empasiprubart in serum","definition_or_measurement_approach":"Incidence and prevalence of anti-drug antibodies (ADA) against empasiprubart measured in serum during Part B."}
• {"endpoint_text":"- Part B - Incidence and prevalence of NAb against empasiprubart in serum","definition_or_measurement_approach":"Incidence and prevalence of neutralizing antibodies (NAb) against empasiprubart measured in serum during Part B."}
• {"endpoint_text":"- Part B - Change from baseline in RT-FSS score over time","definition_or_measurement_approach":"Change from baseline in RT-FSS score measured over time during Part B."}
• {"endpoint_text":"- Part B - Change from baseline in physical component and mental component scores of SF-12 over time","definition_or_measurement_approach":"Change from baseline in SF-12 physical and mental component scores measured over time during Part B."}
• {"endpoint_text":"- 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","definition_or_measurement_approach":"Proportion and shift by EQ-5D-5L dimensions and change in EQ-5D-5L VAS over time during Part B."}

Methods

• Country-specific recruitment arrangements documented (K1 recruitment arrangements per country).
• Patient flyers (country-specific patient flyers listed for many countries).
• Patient letters (country-specific patient letters listed).
• Recruitment brochures (country-specific recruitment brochures).
• Appointment reminder cards.
• Site flyers / Site-and-Patient-Advocacy contact lists.
• Email communications for study contact/scouting (SC-Email-Communication, Scout email communications).
• Digital/web content (TrialMax web content, ScoutPass materials / TrialMax-related web material).
• Patient emergency cards and study brochures (for participant information and recruitment support).

Primary sponsor

Full Name

Argenx

Organisation Type

Pharmaceutical company

Country Of Registered Address

Belgium

Contract research organisations

Name

IQVIA Limited

Responsibilities

Pharmacovigilance

Name

PPD Development LP

Responsibilities

Multiple operational and study management responsibilities (multiple sponsorDuties codes listed)

Name

PPD Denmark Filial Af PPD Scandinavia AB Sverige

Responsibilities

Operational support (local PPD duties)

Name

PPD Global Ltd.

Responsibilities

Operational support (local PPD duties)

Third parties

• {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"Pharmacovigilance","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Mayo Collaborative Services LLC","duties_or_roles":"Biomarker tests C1 and C5","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United Kingdom","full_name":"Clinical Outcomes Solutions Limited","duties_or_roles":"Patient Exit Interview","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"Multiple operational duties (codes provided in sponsorDuties: 1, 11, 12, 13, 2, 4, 5, 8, 9) as listed in source","organisation_type":"Pharmaceutical company"}
• {"country":"Denmark","full_name":"PPD Denmark Filial Af PPD Scandinavia AB Sverige","duties_or_roles":"Duties (codes provided in sponsorDuties: 1, 12, 2)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"ECG","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Cytel Inc.","duties_or_roles":"IDMC; statistical support (codes: 10, 15 IDMC, 6)","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Belgium","full_name":"PPD Global Central Labs","duties_or_roles":"Central lab activities (code 4)","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"Azenta Germany GmbH","duties_or_roles":"Long term storage of samples","organisation_type":"Pharmaceutical company"}
• {"country":"Switzerland","full_name":"Celerion Switzerland AG","duties_or_roles":"PK, Immunogenicity (ADA, Nab) and PD total C2 analysis; central lab support (codes: 15 and 4)","organisation_type":"Pharmaceutical company"}
• {"country":"Finland","full_name":"SYRINX Bioanalytics Oy","duties_or_roles":"PD analysis (free C2)","organisation_type":"Pharmaceutical company"}
• {"country":"Greece","full_name":"PPD Global Ltd.","duties_or_roles":"Operational duties (codes: 1, 12, 2)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"E-data capture support (code 3)","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Ireland","full_name":"Signant Health Global Solutions Limited","duties_or_roles":"E-data capture (eCOA)","organisation_type":"Pharmaceutical company"}
• {"country":"Ireland","full_name":"Accellacare Limited","duties_or_roles":"External nurse activities","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"Azenta Germany GmbH (duplicate entry present for long term storage)","duties_or_roles":"Long term storage of samples (listed elsewhere)","organisation_type":"Pharmaceutical company"}