A multicenter, open-label study to investigate the efficacy and safety of GB-0998 for the treatment of neurological neuropathy in patients with Eosinophilic Granulomatosis with Polyangiitis(EGPA).

JRCT ID: jRCT2041220136

Registered date:06/02/2023

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: EGPA patients with residual neurological neuropathy after corticosteroid therapy
• Date of first enrollment: 15/01/2023
• Target sample size: 25
• Study type: Interventional

Intervention(s)

The use of the following drugs and therapies is prohibited throughout the period from informed consent until 4 weeks after the start of study treatment (or until the discontinuation).
-Human immunoglobulin products other than the study drug
-Cyclophosphamide
-Rituximab (recombinant) products
-Whole blood products
-Fresh frozen human plasma
-Plasmapheresis (including immunoadsorption therapy)
-Other study drugs

Outcome(s)

• Primary Outcome: Change from baseline (before the start of study treatment) in a sum score of the MMT to 4 weeks after the start of study treatment

Secondary Outcome

-Change from baseline (before the start of study treatment) in a sum score of the MMT sum score to 2
weeks after the start of study treatment
-Changes from baseline (before the start of study treatment) in the number of muscles with an MMT score of be equal to or lower than 3 to 2 and 4 weeks after the start of study treatment
-Changes from baseline (before the start of study treatment) in the Pain VAS to 2 and 4 weeks after the start of study treatment
-Changes from baseline (before the start of study treatment) in the numbness grade to 2 and 4 weeks after
the start of study treatment
-Changes from baseline (before the start of study treatment) in the BVAS to 2 and 4 weeks after the start of study treatment
-Changes from baseline (before the start of study treatment) in the Modified Barthel Index to 2 and 4 weeks after the start of study treatment

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

-Men and women aged 18 years and older at the time of informed consent.
-Patients who have received an explanation of the study through the informed consent form approved by the Institutional Review Board and have signed the form prior to any study-related procedure specified in the
protocol.
-Patients with a diagnosis of "definite EGPA" based on the diagnostic criteria of the Ministry of Health and Welfare of Japan (1998).
-Patients with residual neuropathy after the following corticosteroid therapies 1) and 2) for EGPA prior to
informed consent.
1) Induction therapy with 40 mg/day or more of prednisolone (or an equivalent dosage of another
corticosteroid) for at least 4 weeks, followed by a reduction of dosage and then
2) Maintenance therapy with 5 to 20 mg/day of prednisolone (or an equivalent dosage of another
corticosteroid) for at least 4 weeks.
-Patients with the manual muscle testing (MMT) of a score of be equal to 3 or less on at least one item AND a sum MMT score of less than 130 at provisional enrollment and at definitive enrollment.

Exclude criteria

-Patients whose a sum score of the MMT at the definitive enrollment improved by at least 10% of room for
improvement in a sum score of the MMT at the provisional enrollment. (the score at the definitive enrollment - the score at the provisional enrollment)/(144 -the score at the provisional enrollment) not lower than 0.1).
-Patients who have used any prohibited drug (defined separately) during the period between informed
consent and the start of study drug administration.
-Patients with muscle atrophy considered ineligible for this drug.
-Patients who have received a total of 2.0 g/kg of human immunoglobulin G to treat an autoimmune or
inflammatory disease within 12 months before informed consent.
-Patients who have received cyclophosphamide within 4 weeks before informed consent.
-Patients who have newly started mepolizumab within 24 weeks before informed consent.

Related Information

• Primary Sponsor: Amano Koichi
• Secondary Sponsor: Kanme Shinya