Chronic inflammatory demyelinating polyradiculoneuritis and multifocal motor neuropathy
Conditions
Interventions
Patients with CIDP/MMN
1. Acute treatment
GGL is administered as human immunoglobulin at a dose of 400 mg (4 mL)/kg body weight intravenously for 5 consecutive days. If the patient meets the criteria*
Sponsors
Yamamoto Akihiko
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: (1) CIDP patients only: Patients aged 18 years or older at the time of informed consent (2) MMN patients only: Patients aged 16 years or older at the time of informed consent (3) CIDP patients only: Patients diagnosed with CIDP as defined in the 2021 EAN/PNS Guideline (excluding patients with possible CIDP) (4) MMN patients only: Patients diagnosed with MMN as defined in the 2010 EFNS/PNS Guideline (excluding patients with possible MMN) (5) Patients with progressive or recurrent CIDP (acute treatment cohort) or MMN (acute treatment cohort) (regardless of history of treatment with IVIg/SCIg) (6) Patients with CIDP (maintenance therapy cohort): Patients on IVIg/SCIg maintenance therapy who have responded to IgG administration (Patients receiving IVIg 0.4 to 1.2 g/kg every 2 to 6 weeks for the past 12 weeks from the time of eligibility confirmation or SCIg 80 to 480 mg/kg every 1 to 2 weeks for the past 12 weeks from the time of eligibility confirmation) (7) Patients with MMN (maintenance therapy cohort): Patients on IVIg maintenance therapy who have responded to IgG administration (i.e., have previously shown partial or complete improvement in neurological symptoms and disabilities) (Patients receiving IVIg 0.4 to 1.2 g/kg every 2 to 6 weeks for the past 12 weeks from the time of eligibility confirmation) (8) Patients with CIDP (acute treatment cohort): Patients with an INCAT (Inflammatory Neuropathy Cause and Treatment) score between 2 and 9 of disability at the time of eligibility confirmation (but with a lower limb disability score of 2 or more, if the upper limb disability score is 1) (9) Patients with CIDP (maintenance therapy cohort): Patients with an INCAT score between 0 and 7 at the time of eligibility confirmation and a previous disability with an INCAT score between 2 and 9 (but with a lower limb disability score of 2 or more, if the upper limb disability score is 1) (10) In the case of using therapeutic agents * for CIDP and MMN within 30 days prior to eligibility confirmation, the patients who continue without adding and increasing dosage and require treatment with the test product *Immunosuppressants including steroids, azathioprine, tacrolimus, cyclosporine, cyclophosphamide, methotrexate, mycophenolate mofetil, fingolimod, eculizumab
Exclusion criteria
Exclusion criteria: Patients with CIDP (1) Patients with sensory CIDP (2) Patients with autoimmune nodopathy (3) Patients with autoimmune/inflammatory neuropathy such as multifocal motor neuropathy (MMN), Guillain-Barre syndrome, POEMS syndrome, IgM paraproteinemia including MAG antibody-associated neuropathy (4) Patients with hereditary neuropathy such as Charcot-Marie-Tooth disease, hereditary sensory and autonomic neuropathy (HSAN) (5) Patients with neuropathy secondary to internal diseases such as diabetes mellitus, cancer, collagen disease, amyloidosis, or infection such as Borrelia burgdorferi infection (Lyme disease), diphtheria (Eligible if there is no diabetic neuropathy, HbA1c level is stable and blood glucose control can be maintained appropriately) (6) Patients with drugs, biological products, chemotherapy or toxicant induced neuropathy Patients with MMN (7) Patients with CIDP (8) Patients with other neuromuscular diseases (e.g., hereditary motor neuropathy, motor neuron disease, neuromuscular junction disease, muscle disease, diabetic neuropathy, cervical spondylosis, lumbar spondylosis, hereditary neuropathy with liability to pressure palsies, peripheral nerve tumor, vasculitic neuropathy, sarcoid neuropathy, etc.). However, patients with those diseases that are considered NOT to affect outcomes or evaluations during the study in the investigator's opinion can be enrolled. Patients with CIDP/MMN (9) Patients who have received maintenance therapy with IVIg/SCIg for more than 52 weeks (maintenance therapy cohort only) (however, patients who received maintenance therapy with IVIg/SCIg for more than 52 weeks may also be enrolled if immunoglobulin administrations are considered necessary based on medical judgment). (10) Patients with central demyelinating diseases such as multiple sclerosis (11) Patients with chronic illnesses or central nervous system disorders (e.g. stroke, Parkinson's disease, amyotrophic lateral sclerosis, etc.) that may cause neurological symptoms or signs. However, patients with those diseases that are considered NOT to affect outcomes or evaluations during the study in the investigator's opinion can be enrolled. (12) Patients who have an active malignancy requiring chemotherapy and/or radiation therapy, or have a medical history of malignancy and have been in complete remission for less than 2 years prior to eligibility confirmation. Exceptions to this exclusion criterion are appropriately treated basal cell or squamous cell carcinoma of the skin, intraepithelial carcinoma of the cervix, and stable prostate cancer not requiring treatment. (13) For the most recent IVIg treatment, those who received >1.2 g/kg and =0.75 g/kg and =0.375 g/kg at the time of eligibility confirmation. *Blood IVIg level is calculated based on the dosage of IVIg treatment within 15 weeks before eligibility confirmation and its half-life (21 days). (14) Patients who received SCIg in excess of 480 mg/kg as a single dose 4-5 weeks (22-35 days) prior to eligibility confirmation, patients who received SCIg in excess of 240 mg/kg as a single dose (480 mg/kg total) 3-5 weeks (15-35 days) prior to eligibility confirmation, patients who received SCIg in excess of 120 mg/kg as a single dose (480 mg/kg
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Patients with CIDP - Proportion of patients with improvement in the adjusted INCAT score after the first course of acute treatment - Proportion of patients with worsening on the adjusted INCAT score during the first phase of maintenance therapy Patients with MMN Medical Research Council (MRC) sum scores and changes from baseline in the MRC scores in the first course of acute treatment and in the first phase of maintenance therapy | — |
Secondary
| Measure | Time frame |
|---|---|
| [Efficacy] Patients with CIDP - MRC sum scores and changes from baseline in the first course of acute treatment and the first phase of maintenance therapy - Proportion of patients with improvement in the adjusted INCAT score after the second course of acute treatment - Rasch-built Overall Disability Scale (R-ODS) scores and changes from baseline in the first course of acute treatment and in the first phase of maintenance therapy - INCAT Sensory Sum scores (ISS) and changes from baseline in the first course of acute treatment and in the first phase of maintenance therapy - Time to worsening of the adjusted INCAT score during the first phase of maintenance therapy Patients with MMN - Guy's Neurological Disability Scale (GNDS) sum scores and changes from baseline in the first course of acute treatment and in the first phase of maintenance therapy -Time to worsening of MRC sum scores during the first phase of maintenance therapy Patients with CIDP/MMN - Maximum grip strength (Martin hand dynamometer) and changes in grip strength from baseline in the first course of acute treatment and in the first phase of maintenance therapy - Compound muscle action potential (CMAP) amplitude following stimulation at the most severely affected proximal motor nerve site and changes in CMAP amplitude from baseline during the first course of acute treatment and during the first phase of maintenance therapy - Serum IgG levels and changes from baseline during the first course of acute treatment and during the first phase of maintenance therapy | — |
Contacts
Public ContactMasatoshi Yamashita
KM Biologics Co., Ltd.
Outcome results
None listed