Multifocal Motor Neuropathy MedDRA version: 21.1 Level: PT Classification code 10065579 Term: Multifocal motor neuropathy System Organ Class: 10029205 - Nervous system disorders
Conditions
Sponsors
argenx BV
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Participants are eligible to be included in the trial only if all of the following criteria apply: 1. Capable of providing signed informed consent, and complying with protocol requirements. Participants must be able to read and write. 2. Must have completed the double-blinded treatment period of the ARGX-117-2002 trial and considered to be eligible for treatment with ARGX-117 3. Agrees to use contraceptive measures consistent with local regulations and the following: a. Male participants: must use an acceptable contraceptive method that should be maintained at minimum until 12 months after last dose of Investigational Medicinal Product (IMP). b. Female participants (women) of childbearing potential must have a negative urine pregnancy test at baseline before IMP can be administered. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 36 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 12
Exclusion criteria
Exclusion criteria: Participants will be excluded from the trial if any of the following criteria apply: 1. Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection 2. Clinical evidence of other significant serious diseases, have had a recent major surgery, or who have any other condition, in the opinion of the investigator, that could confound the results of the trial or put the participant at undue risk. a. Germany only: A SARS-CoV-2 rapid antigen test must be completed or a negative rapid antigen test not older than 48 hours (depending on local requirements) must be presented upon site entry during DV1 while COVID-19 is considered an epidemic. Rapid antigen testing will only be mandatory while required by local regulations. 3. Currently participating in another interventional clinical study 4. Pregnant or lactating or intend to become pregnant during the trial or within 12 months after last dose of the IMP.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the long-term safety and tolerability of ARGX-117 in adult participants with MMN;Secondary Objective: • To evaluate the long-term efficacy of ARGX-117 on muscle strength and/or motor function in adult participants with MMN • To evaluate the long-term efficacy of ARGX-117 on functional ability, arm and hand function, quality of life (QOL), and fatigue in adult participants with MMN • To evaluate the long-term effect of ARGX-117 on health-related productivity and work productivity • To assess the PK, PD, and immunogenicity of ARGX-117;Primary end point(s): • Safety outcomes based on AE monitoring and other safety assessments;Timepoint(s) of evaluation of this end point: continuously | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Modified Medical Research Council (mMRC) 1.Value and change from baseline in the mMRC-10 sum score 2.Proportion of participants showing a deterioration of at least 2 points in mMRC-10 sum score 3.Value and change from baseline in the mMRC-14 sum score 4.Proportion of participants showing a deterioration of at least 2 points in the mMRC-14 sum score 5.Proportion of participants showing a deterioration of 1 or more points in at least 2 muscle groups as assessed by the mMRC-14 sum score 6.Proportion of participants with no deterioration in 2 or more muscle groups as assessed by mMRC-14 sum score 7.Value and change from baseline in the 2 most important muscle groups as assessed by the mMRC-14 sum score • Grip strength (GS) 8.Values, change, and percent change from baseline in GS 9.Proportion of participants with a decline of >30% in GS 10. Proportion of participants with a GS decrease of 8 kilopascals (kPa) or more 11.Values and change from baseline in the Rasch-built overall disability scale for MMN (MMN-RODS) 12.Values and change from baseline in the average time for the upper extremity (arm and hand) function (9-Hole Peg Test [9-HPT], or timed pegboard test) 13.Proportion of participants by level of severity on each dimension of EQ-5D-5L 14.Value and change from baseline in EQ-5D-5L visual analog scale (VAS) 15.Values and change from baseline in the Chronic Acquired Polyneuropathy Patient-Reported Index (CAP-PRI) 16.Values and change from baseline in the 9-item Fatigue Severity Scale (FSS) 17.Proportion of participants by level of change as assessed by the Patient Global Impression of Change (PGIC) 18.Proportion of participants by level of severity of MMN as assessed by the Patient Global Impression of Severity (PGIS) 19.Values for work-related and household chore activities of the Health-Related Productivity Questionnaire (HRPQ) 20.Serum concentrations and PK parameters for ARGX-117 21.Values and change from baseline in free C2, total C2 | — |
Countries
Austria, Belgium, Canada, France, Germany, Italy, Netherlands, Poland, Spain, United Kingdom, United States
Contacts
Public ContactRegulatory
argenx BV
Outcome results
None listed