Continuous dosing study of DYD-301 tablet in Japanese patients with Lambert Eaton Myasthenic Syndrome (Expanded Access Program)

JRCT ID: jRCT2031220702

Registered date:14/03/2023

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Lambert Eaton Myasthenic Syndrome(LEMS)
• Date of first enrollment: 14/04/2023
• Target sample size: 10
• Study type: Interventional

Intervention(s)

DYD-301 tablet (amifampridine phosphate) will be administered in the range of 15 mg to 80 mg daily in 3 to 4 divided doses.
If the investigator determines that the subject has no safety concerns, the dose can be increased up to 100 mg daily in 5 divided doses. The maximum single dosage is 30 mg..Duration of administration is approximately 2 years.

Outcome(s)

• Primary Outcome: Adverse events/ Adverse drug reaction, Vital sign, Laboratory test, ECGs
• Secondary Outcome: Change from baseline in SGI score ,CGI-I score

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) Patients who participated in the DYD-301-11 (LMS-005) study and completed the study without discontinuation
2) Patient is >=17year old male or female with LEMS
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

Exclude criteria

The subjects who meet any of the following criteria will be excluded in the study:
1) Patients is, in the opinion of the Investigator, continued administration of DYD-301 tablets is not appropriate from the viewpoint of safety and efficacy and suitable in any other way to participate in the study.

Related Information

• Primary Sponsor: Toide Kenji