multifocal motor neuropathy,MMN
Conditions
Interventions
Test group:Intervention: Intravenous immunoglobulin (IVIg)
Sponsors
Peking Union Medical College Hospital
Eligibility
Sex/Gender
All
Age
18 Years to 75 Years
Inclusion criteria
Inclusion criteria: 1. Must have given written informed consent before any study-related activities are carried out and must be able to understand the full nature and purpose of the study, including possible risks and adverse effects. 2. Adult males and females, 18 to 75 years of age (inclusive) at Screening. 3. Weight range between 40 to 120 kg at Screening. 4. A diagnosis of definite or probable MMN per the European Federation of Neurological Societies (EFNS) / Peripheral Nerve Society (PNS)(EFNS/PNS) 2010 guidelines at Screening. 5. No history of clinically significant findings in serum chemistry, hematology, coagulation, urinalysis tests, or ECG. 6. Be willing and able to comply with all study assessments and adhere to the protocol schedule and restrictions.
Exclusion criteria
Exclusion criteria: 1. History or presence of significant medical/surgical condition including any acute illness or major surgery considered to be clinically significant or that could have potential impact on safety/efficacy or study procedures in the opinion of the Investigator. 2. Any coexisting conditions, which may interfere with outcome assessments, such as severe diabetic neuropathy, chronic inflammatory demyelinating polyneuropathy, inflammatory arthritis or osteoarthritis affecting the hand (among others). 3. Concurrent or previous use of rituximab. If a participant has previously used this medication, the last dose must be at least 6 months prior to Day 1. 4. Concurrent use of cyclophosphamide, mycophenolate mofetil, azathioprine, cyclosporine, corticosteroids or other. If a participant has previously used these medications, the last dose must be prior to Day 1. 5. Currently or previously on complement inhibitors, including in a clinical trial setting. 6. History of positive test results for active human immunodeficiency virus (HIV-1 or HIV-2), hepatitis B surface antigen (HBsAg), or hepatitis C virus (HCV) antibodies. Participants who have no evidence of cirrhosis, have completed a curative intent regimen for HCV, and are deemed by a gastroenterologist to have no active HCV will not be excluded. 7. Known diagnosis of systemic lupus erythematosus (SLE) or family history (defined as a parent, sibling, or child) of SLE; 8. History of active malignancy within 5 years prior to Screening, except basal cell carcinoma of the skin, curatively resected squamous cell carcinoma of the skin, cervical carcinoma in situ curatively treated or low-grade prostate adenocarcinoma for which appropriate management is observation alone. 9. Clinically significant drug or alcohol abuse in the opinion of the Investigator. 10. Participation in another clinical study of an investigational drug within 90 days or 5 half-lives of the investigational agent (whichever is longer) prior to Day 1. 11. Any other overlapping condition for which the condition or treatment of the condition may affect the study assessments or outcomes. 12. Any other condition, including mental illness or prior therapy, that in the opinion of the Investigator would make the participant unsuitable for this study, including inability to cooperate fully with the requirements of the study protocol or likelihood of noncompliance with any study requirements.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The average score of the Rasch-Built Multifarious Motor Neuron Disease Overall Disability Scale (MMN-RODS) after treatment for 12 to 24 weeks and the change compared to baseline;Change in the average score of the Medical Research Council total score (MRC-14) from baseline after 12 to 24 weeks of treatment;Average grip strength after treatment for 12 to 24 weeks, changes compared to baseline and percentage change; | — |
Secondary
| Measure | Time frame |
|---|---|
| Change in ANA titer after treatment for 12 to 24 weeks compared to baseline.;Change in VAS score from baseline after treatment for 12 to 24 weeks.;The average change in the corrected inflammatory neuropathy cause and treatment scale (INCAT) disability score from baseline after 12 to 24 weeks of treatment.; | — |
Countries
China
Contacts
Public ContactMingsheng Liu
Peking Union Medical College Hospital
Outcome results
None listed