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: 1. Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol (including consent for the use and disclosure of research related health information). Participants must be able to read and write and be willing and able to comply with the trial protocol procedures (including attending the required trial visits) 2. Male/female at least 18 years of age at the time the ICF is signed 3. Probable or definite MMN according to the EFNS/PNS 2010 guidelines at screening confirmed by the MCC 4. Receiving a stable IVIg regimen before screening and both of the following: a) IVIg treatment interval of 2 to 5 weeks b) IVIg dose of 0.4 to 2.0 g per kg body weight and infusion 5. IVIg treatment dependency confirmation by the MCC at screening or at IMV1, based on 1 of the following: a) Recently initiated IVIg treatment (less than 3 months): - Clinical improvement following IVIg initiation documented in the participant’s medical record b) Maintenance therapy with IVIg (longer than 3 months), based on 1 of the following: - Clinical deterioration following IVIg withdrawal, IVIg dose reduction, or IVIg delayed administration within 12 months prior to screening (documented in the participant’s medical record) - Clinical deterioration following IVIg delayed administration during the IVDP 6. Immunization with the first meningococcal vaccine and pneumococcal vaccine, and the single Haemophilus influenza type B vaccine must be performed at least 14 days before IMP administration at V1 according to local country-specific immunization schedules. A documented history of vaccination against Neisseria meningitides, Haemophilus influenza type B, and streptococcus pneumonia will be permitted 7. Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies a) Male participants must agree to not donate sperm from the time the ICF is signed until 12 months after the last IMP administration b) Women of childbearing potential (WOCBP) must have a negative serum pregnancy test at screening and 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: 1. Any coexisting condition which may interfere with the outcome assessments (eg, diabetic neuropathy, CIDP, inflammatory arthritis, or osteoarthritis affecting the hand) 2. Clinical signs or symptoms suggestive for neuropathies other than MMN such as motor neuron disease (eg, bulbar signs or brisk reflexes) or other inflammatory neuropathies (eg, sensory neuropathy) 3. Severe psychiatric disorder (such as severe depression, psychosis, bipolar disorder), history of suicide attempt, or current suicidal ideation that in the opinion of the investigator could create undue risk to the participant or could affect adherence with the trial protocol. 4. Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection during the screening and/or IVMP. 5. Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of MMN or put the participant at undue risk (eg, SLE). 6. History of malignancy unless resolved by adequate treatment with no evidence of recurrence for =3 years before the first administration of the IMP. Participants with the following carcinomas will be eligible: a. Adequately treated basal cell or squamous cell skin cancer b. Carcinoma in situ of the cervix c. Carcinoma in situ of the breast or d. Incidental histological finding of prostate cancer (TNM stage T1a or T1b) 7. 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 8. Prior/concomitant therapy a. Cyclophosphamide and/or rituximab and/or eculizumab and/or mycophenolate mofetil within 3 months prior to screening b. Use of an investigational product within 3 months or 5 half-lives (whichever is longer) before the first dose of the IMP. 9. Positive serum test at screening for an active viral infection with any of the following conditions: a. Hepatitis B virus (HBV) that is indicative of an acute or chronic infection (https://www.cdc.gov/hepatitis/HBV/PDFs/SerologicChartv8.pdf) b. Hepatitis C virus (HCV) based on HCV antibody assay c. HIV based on test results that are associated with an AIDS-defining condition or a CD4 count <200 cells/mm3 10. Current or history of (ie, within 12 months of screening) alcohol, drug, or medication abuse 11. Known hypersensitivity reaction to 1 of the components of the IMP or any of its excipients 12. Female participants with a positive serum or urine pregnancy test, lactating females, and those who intend to become pregnant during the trial or within 9 months after last dose of the IMP
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and tolerability of ARGX-117 compared to placebo in adult participants previously stabilized with IVIg;Secondary Objective: - To evaluate the efficacy of ARGX-117 compared to placebo on muscle strength and/or motor function in adult participants previously stabilized with IVIg - To evaluate the efficacy of ARGX-117 on functional ability, arm and hand function, quality of life, and fatigue in adult participants with MMN - To evaluate the effect of ARGX-117 on health-related productivity and work productivity - To evaluate medication treatment satisfaction - To assess the PK, PD, and immunogenicity of ARGX-117;Primary end point(s): Safety outcomes based on adverse event (AE) monitoring and other safety assessments;Timepoint(s) of evaluation of this end point: Days 1, 4, 8, 15, 22, 29, 43, 57, 71, 78, 85, 99, 102 and 113 + continuous monitoring (CM) | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: A & S: Days 1, 4, 8, 15, 22, 29, 43, 57, 71, 78, 85, 99, 102 and 113 B: Days 4, 8, 15, 22, 29, 43, 57, 71, 78, 85, 99, 102 and 113 C, D, E, F, G: Days 1, 8, 15, 22, 29, 43, 57, 71, 85, 99 and 113 H, I & J: Days 1, 4, 8, 15, 22, 29, 43, 57, 71, 78, 85, 99, 102 and 113 + cm K & L: Days 1, 8, 15, 22, 29, 43, 57, 71, 85, 99, and 113 M, N & Q: Days 1, 15, 29, 57, 85 and 113 O: Days 1, 15, 29, 43, 57, 71, 85, 99 and 113 P: Days 1, 15, 29, 43, 57, 71, 85, 99 and 113 R: Days 1, 15, 29, 43, 57, 71, 85, 99, 113 T: Days 1, 8, 15, 22, 29, 43, 57, 85 and 113 U: Days 1 and 113;Secondary end point(s): A. Time to the first retreatment with IVIg since the final IVIg treatment of the IVIg monitoring period B. Difference between the time to the first retreatment with IVIg (cycle) and the second time to retreatment with IVIg C. AUC of the change from baseline in mMRC-10 sum score D. Change from baseline in the 2 most important muscle groups as assessed by the mMRC-14 sum score E. Value and change from baseline in the mMRC-14 sum score F. 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 G. Percentage of time with no deterioration in 2 or more muscle groups as assessed by mMRC-14 sum score H. AUC of the change from baseline in GS I. Proportion of participants with a GS decrease of 8 kilopascal (kPa) or more over 3 consecutive days J. Values, change, and percent change from baseline in GS K. Values and change from baseline in the Rasch-built overall disability scale for MMN (MMN-RODS©) L. Values and change from baseline in upper extremity (arm and hand) function (9-Hole Peg Test [9-HPT], or timed Peg Board Test) M. Values and change from baseline in quality of life using the Euro-Quality of Life 5 Dimensions 5 Levels (EQ-5D-5L) scale N. Values and change from baseline in the chronic acquired polyneuropathy patient-reported index (CAP-PRI) O. Values and chang | — |
Countries
Austria, Belgium, Canada, Denmark, France, Germany, Italy, Netherlands, Poland, Spain, United Kingdom, United States
Contacts
Public ContactRegulatory
argenx BV
Outcome results
None listed