Long-term study of DYD-301 tablet in Japanese patients with Lambert Eaton Myasthenic Syndrome

JRCT ID: jRCT2031210552

Registered date:17/01/2022

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: Lambert Eaton Myasthenic Syndrome(LEMS)
• Date of first enrollment: 02/03/2022
• Target sample size: 10
• Study type: Interventional
• Intervention(s): Subjects receive DYD-301tablet orally

Outcome(s)

• Primary Outcome: Efficacy endpoint : Change from baseline (Visit 3) in QMG score at Visit 6

Secondary Outcome

Efficacy endpoint:
Change from baseline (Visit 3) in each QMG sub-score at Visit 6
Change from baseline (Visit 3) in SGI score at Visit 6
CGI-I score at Visit 6

Key inclusion & exclusion criteria

• Age minimum: >= 17age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

The subjects are eligible to be included in the study only if all of the following criteria apply:
1) Patient is 17year old or over, male or female with LEMS
2) Patients diagnosed with LEMS by VGCC antibody (P/Q antibody) test or EMG
3) Patients who have agreed to use properly contraception themselves or their female partners to avoid becoming pregnant, if they or their female partners have child bearing potential.
4) Patients (or legal representatives of minor patients) who have received sufficient explanation about the objective and content of the clinical study, expected efficacy, pharmacological action, risks, etc., and who have obtained understanding and obtained written consent from the patient
5) Patients with a QMG score of 8 of higher at the timing of screening and is ambulatory at least any supports or assistances from a device (only for treatment naive patients)
6) If the patient is receiving peripherally acting cholinesterase inhibitors (e.g., pyridostigmine), patients who can keep stable dose of cholinesterase inhibitors for at least 7 days prior to the start of the titration period and throughout the treatment period.
7) If the patient is receiving permitted oral steroid with immunosuppressive activity (prednisolone or other corticosteroid), patients who can keep stable dose of permitted oral steroid for at least 30 days prior to the start of the titration period and throughout the treatment period.

Exclude criteria

The subjects who meet any of the following criteria will be excluded in the study:
1) Patients with a history of epilepsy or seizures (excluding febrile seizures) or who are currently treating with anticonvulsants
2) Patients who have active brain metastases at the time of screening
3) Patients with a history of brain tumors, uterine tumors, schwannomas and/or endometrial tumors
4) Patients who have not completed anticancer treatment at least three months (90days) prior to the screening
5) Patients who are in pregnant or lactating
6) Patients who cannot discontinue immunosuppressive drugs and immunomodulatory therapy (e.g., mycophenolate, azathioprine, cyclosporine, intravenous immunoglobulin) within 4 weeks before the start of the screening period
7) Patients who are treated with rituximab within 12 months prior to the start of the screening
8) Patients with a history of severe renal dysfunction, or patients with suspected severe renal dysfunction based on screening test results (especially creatinine clearance of less than 30 mL / min)
9) Patients with a history of severe hepatic impairment or patients with suspected severe hepatic impairment on screening lab test, specifically ALT, ALT and/or total bilirubin exceeds three times the upper limit of normal
10) Patients received other investigational drugs within 1 month before consent was obtained, and patients who had not completed other clinical trials before consent was obtained
11) Patients who have hypersensitivity or suspected hypersensitivity to the study drug ( including 3,4-DAP which is not study drug)
12) Patients who have the drugs known to lower the epileptic threshold (eg, antidepressants such as tricyclics and bupropion; neuroleptics such as phenothiazine, butyrophenones, clozapine, olanzapine, quetiapine; centrally active cholinesterase inhibitors such as donepezil, rivastigmine, galantamine; mefloquine) within 7 days prior to the start of Screening.
13) Patients who have changed the doses using selective serotonin reuptake inhibitor (SSRI) class antidepressants (ie.escitalopram, sertraline, paroxetine, fluvoxamine) and tramadol within 90 days prior to the start of the screening.
14) Patients is, in the opinion of the Investigator, unsuitable in any other way to participate in the study.

Related Information

• Primary Sponsor: Toide Kenji