A Phase 3, double-blind, randomized, placebo-controlled study evaluating efficacy of SAR445088
Study Description
Eligibility
Inclusion Criteria:
Participants are eligible to be included in the study only if all of the following criteria apply:
- Participant must have CIDP or possible CIDP criteria, based on European Academy of Neurology (EAN)/ Peripheral Nerve Society (PNS) Task Force CIDP guidelines, second revision (2021).
- Participant must have either typical CIDP, or one of the following two CIDP variants: motor CIDP, multifocal CIDP (also known as Lewis Sumner Syndrome). Diagnosis must be confirmed by the adjudication committee.
- Participant must be refractory to either immunoglobulin therapy or corticosteroid therapy, as defined below.
a. Immunoglobulinrefractory subgroup: Historic evidence of failure or inadequate response to immunoglobulin therapy prior to screening, defined as no clinically meaningful improvement or persistent INCAT score greater than or equal to 2 after a minimum of:
I. One dose of IVIg of 2 g/kg, followed by a second dose of 2 g/kg or two doses of 1 g/kg, with a separation of approximately 3 weeks between doses (each dose can be divided over 2 to 5 days), as indicated in the EAN/PNS 2021 guidelines OR
II. SCIg maintenance therapy with at least 0.2 g/kg weekly for 5 weeks
b. Corticosteroidrefractory subgroup:
- Historic evidence of failure or inadequate response to corticosteroid therapy prior to screening, defined as no clinically meaningful improvement or persistent INCAT score greater than or equal to 2 after a minimum of 12 weeks of corticosteroid therapy. Corticosteroid regimen can be daily oral prednisone/prednisolone, at least 60 mg, equivalent to methylprednisolone 48 mg, tapered over 6 to 8 months, or alternative regimens, e.g. pulsed high-dose corticosteroid treatment (40 mg/day oral dexamethasone or 500 mg/day IV methylprednisolone, each daily for 4 days per month for 6 months), as indicated in the EAN/PNS 2021 guidelines A clinically meaningful improvement is defined as one or more of the following:
a. A greater than or equal to 1 point decrease in adjusted INCAT disability score
b. An increase in IRODS total score greater than or equal to 4 points
c. An increase in MRC Sum score greater than or equal to 3 points
d. An improvement in hand grip strength of greater than or equal to 8 kilopascals or
e. Equivalent improvement based on information from medical records and per the Investigator's judgment
-Participant has an adjusted INCAT score of 2 to 9
(a score of 2 should be exclusively from the leg disability component of INCAT).
I. Any allowed immunosuppressant drugs (azathioprine, cyclosporine, or mycophenolate mofetil) have been taken for greater than or equal to 6 months at a stable dose for greater than or equal to 3 months prior to Screening
II. Participant may be receiving low-dose oral corticosteroids less than or equal to 20 mg/day of prednisone [or equivalent dose for other oral corticosteroids]), but only if taken at a stable dose for greater than or equal to 3 months prior to Screening
III. Participant must have active disease, defined by a CIDP disease activity score (CDAS) of greater than or equal to 2 points at Screening
Participant must have documented vaccinations against encapsulated bacterial pathogens given within 5 years of screening or initiated a minimum of 14 days prior to first dose of study intervention
IV. All participants must agree to use contraception methods during and after the study as required.
V. Contraceptive use by men and women participating in the study should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- Male participants are eligible to participate if they agree to the following during the study intervention period and for at least 55 weeks after the last dose of study medication:
- Refrain from donating or cryopreserving sperm PLUS
Note: Other protocol defined Inclusion/Exclusion criteria may apply
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