Lenalidomide in Anti-MAG Neuropathy: Phase 1b Study
Status:
Active, not recruiting
Trial end date:
2023-12-30
Target enrollment:
Participant gender:
Summary
Anti-myelin-associated glycoprotein (MAG) is a rare autoimmune disorder of the peripheral
nerves that presents with weakness, gait imbalance, and loss of sensation. It almost always
occurs in the setting of excess protein buildup in the body in the form of immunoglobulin
monoclonal (IgM) gammopathy. Anti-MAG neuropathy currently has no established therapies. It
is diagnosed through blood tests (anti-MAG and IgM), nerve conduction studies (which showed
marked velocity slowing), and clinical exam findings.The efficacy of lenalidomide has been
demonstrated in anti-MAG peripheral neuropathy with two separate dosing regimens: 25mg on
days 1-21 of each 28 day cycle in conjunction with oral dexamethasone 20mg/day on days 1-4 of
each cycle as well as at 5mg on days 1-21 of each cycle without oral dexamethasone. This
phase 1 study aims to determine the maximum tolerated dose (MTD) of Lenalidomide in patients
with anti-MAG neuropathy. We will explore preliminary efficacy and postulate that this drug
is effective in this subset of patients, using preselected, specifically tailored outcome
measures that encompass quality of life, neurologic function, serum protein levels, and
focused measures of proprioception.