UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Treatment of Lambert-Eaton myasthenic syndrome with 3,4-diaminopyridin
Title of the study (Brief title)	Treatment of Lambert-Eaton myasthenic syndrome with 3,4-diaminopyridin
Region	Japan

Condition
Condition	Lambert-Eaton myasthenic syndrome
Classification by specialty	Neurology
Classification by malignancy	Malignancy
Genomic information	NO

Objectives
Narrative objectives1	Lambert-Eaton myasthenic syndrome(LEMS) is an uncommon autoimmune disease that is characterized by muscle weakness and dysautonomia. LEMS is a disorder of reduced acetylcholine release from the presynaptic nerve terminals. Antibodies directed against the voltage-gated calcium channel interfere with the normal calcium flux required for the release of acetylcholine. 3,4-diaminopyridine(3,4-DAP) is used to treat LEMS in European countries. The mechanism of action is a significant prolongation of the nerve terminal membrane depolarization, which enhances calcium entry and thereby improves the release of acetylcholine. The purpose of this study is to assess the effectiveness of 3,4-DAP in LEMS.
Basic objectives2	Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	The compound muscle action potential(CMAP) and the degree of increase in CMAP amplitude after high frequency receptive nerve stimulation conducted 1, 2, 4 months after treatment.
Key secondary outcomes

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	3,4-diaminopyridine
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit	20 years-old <=
Age-upper limit	85 years-old >=
Gender	Male and Female
Key inclusion criteria	Patients who are electrically diagnosed to have Lambert-Eaton myasthenia syndrome or anti- voltage-gated calcium channel antibody positive patients.
Key exclusion criteria	Minors, pregnant women, or patients with a past history of epilepsy
Target sample size	10

Research contact person
Name of lead principal investigator	Wataru Sako
Organization	Tokushima University
Division name	Department of Neurology
Address	2-50-1, Kuramoto-Cho, Tokushima city, Tokushima prefecture
TEL	088-633-7207
Email	dwsako@tokushima-u.ac.jp

Public contact
Name of contact person	Takahiro Furukawa
Organization	Tokushima University
Division name	Department of Neurology
Address	2-50-1, Kuramoto-Cho, Tokushima city, Tokushima prefecture
TEL	088-633-7207
Homepage URL
Email	tfurukawa@tokushima-u.ac.jp

Sponsor
Institute	Tokushima University
Institute
Department

Funding Source
Organization	Tokushima University
Organization
Division
Category of Funding Organization	Other
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funｄer(s)

Secondary IDs
Secondary IDs	NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions

Other administrative information
Date of disclosure of the study information	2016 Year 09 Month 14 Day

Progress
Recruitment status	Enrolling by invitation
Date of protocol fixation	2016 Year 09 Month 14 Day
Anticipated trial start date	2016 Year 11 Month 01 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Related information
URL releasing protocol
Publication of results	Unpublished
URL releasing results
Results
Other related information

Management information
Registered date	2016 Year 09 Month 14 Day
Last modified on	2018 Year 03 Month 19 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000027642