Anti-MAG neuropathy is a progressively disabling orphan rare disorder due to a monoclonal immunoglobulin M(IgM) gammopathy displaying reactivity toward MAG, a glycoprotein of the peripheral nervous system. Its prevalence is around 1/100000 and to date, no treatment has proven efficacy in this disease, including rituximab in 2 Randomized Controlled Trails(RCTs).
Disease duration of 5 years or less and documented clinical worsening (clinical or ENMG or disability) over the past 24 months
IgM gammopathy, either MGUS or Waldenstrom Macroglobulinemia (WM)
Demyelinating polyneuropathy according to European Federation of Neurological Societies/Peripheral Nerve Society guidelines for chronic inflammatory demyelinating polyneuropathy on nerve conduction studies.
Anti-MAG titre of 10 000 BTU or more
- Unable to give informed consent
History of severe allergic or anaphylactic reaction to chimeric monoclonal antibody
Hypersensitivity known to one of the compounds of polaramine or methylprednisolone
Previous treatment with rituximab
Parallel
Rituximab infusion
Drug2 infusions of 1 gram of rituximab at a 2 week interval (day 1 followed by day 15).
Placebo infusion
Drug2 infusions of placebo at a 2 week interval.
Premedications
DrugPremedications prior to rituximab or placebo infusions: * IV Dexchlorpheniramine Maleate IV: 10 mg * IV Methylprednisolone: 40 mg * PO Paracetamol : 1 gram
I-RODS score
Clinical response defined as a 4 points (or more) change of I-RODS between baseline and 12 months. I-RODS is a 24-item patient-reported outcome measure which maximum score is 48. It is a linearly weighted scale that specifically captures activity and social participation limitations in patients with inflammatory neuropathies, including Monoclonal Gammopathy of Unknown Significance (MGUS) related polyneuropathies.
Inflammatory Neuropathy Cause and Treatment (INCAT) disability score
The INCAT (Inflammatory Neuropathy Cause and Treatment) disability score is a measure of activity limitation with minimum score at 0 and maximum at 10.
Six minute walk test
Six minute walk test will be realized.
Timed 25- foot walk (FW) test
The T25-FW is a quantitative test of mobility and performance of leg function based on a timed outward journey of 25 steps, and a timed return journey of 25 steps. The score for the T25-FW is the average of the two completed trials
9 hole peg test
The nine hole peg test is a standardized, quantitative assessment used to measure finger dexterity. Scores are based on the time taken to complete the test activity, recorded in seconds
Click on the lead sponsor to view all of their trials.
Centre Hospitalier Universitaire de Saint Etienne
Lead sponsor
Ministry of Health, France
Collaborator
