UMIN-CTR Clinical Trial

Basic information
Public title	Treatment of MuSK antibody-positive myasthenia gravis with rituximab: an exploratory clinical study
Acronym	Treatment of MuSK antibody-positive myasthenia gravis with rituximab: an exploratory clinical study
Scientific Title	Treatment of MuSK antibody-positive myasthenia gravis with rituximab: an exploratory clinical study
Scientific Title:Acronym	Treatment of MuSK antibody-positive myasthenia gravis with rituximab: an exploratory clinical study
Region	Japan

Condition
Condition	MuSK antibody-positive myasthenia gravis
Classification by specialty	Neurology
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	To evaluate the effects of rituximab as treatment in MuSK antibody-positive myasthenia gravis.
Basic objectives2	Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	We assess the clinical state of patients using a Myasthenia gravis foundation of America post-intervention status (MGFA-PIS), Quantitative MG score for Disease Severity (QMG score), and the amounts of waning of repetitive stimulation tests. A patient is considered to have responded if they have attained an MGFA-PIS of improved, Minimal Manifestations, Pharmacologic Remission or Complete Stable Remission. The effectiveness evaluation is established by the response rate.
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	Administration of Rituximab at the standard dose of 375 mg/m2 every week for 4 consecutive weeks and then monthly for the next 3 months.
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	80 years-old >
Gender	Male and Female
Key inclusion criteria	(1) MuSK antigody-positive MG patients who did not attain remission with oral steroid and immunosuppressors, and need hospitalization for plasma pheresis or high-dose intravenous immunoglobulin. (2) The patients who are given information about this study and decide to undergo the study and express the consent in consent forms.
Key exclusion criteria	(1) Patients who have severe hypersensitivity or anaphylactic reaction to rituximab or mouse-derived products. (2) Pregnant women or women of possibility of pregnancy. (3) Lactating women. (4) Patients of HBs antigen positive or patients who had HBV infection in their past. (5) Patients of HBs antigen negative and HBc antibody or HBs antibody positive, and detectable HBV-DNA. (6) Patients who have received any other experimental drug or investigational product within three months before the start of study treatment in the present study. (7) Patients who are considered inadequate for this study by Principal investigator.
Target sample size	5

Research contact person
Last name of lead principal investigator	1st name Middle name Last name Yasuo Terao
Organization	University of Tokyo
Division name	Department of Neurology
Zip code
Address	7-3-1 Hongo Bunkyo-ku, Tokyo
TEL	03-3815-5411
Email	yasuo.terao@gmail.com

Public contact
1st name of contact person	1st name Middle name Last name Yasuo Terao
Organization	University of Tokyo
Division name	Department of Neurology
Zip code
Address	7-3-1 Hongo Bunkyo-ku, Tokyo
TEL	03-3815-5411
Homepage URL
Email	yasuo.terao@gmail.com

Sponsor
Institute	Department of Neurology University of Tokyo, Graduate School of Medicine
Institute
Department

Funding Source
Organization	Department of Neurology University of Tokyo, Graduate School of Medicine
Organization
Division
Category of Funding Organization	Self funding
Nationality of Funding Organization	Japan

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

IRB Contact (For public release)
Organization
Address
Tel
Email

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	2013 Year 10 Month 23 Day

Related information
URL releasing protocol
Publication of results	Unpublished

Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status	Completed
Date of protocol fixation	2013 Year 10 Month 15 Day
Date of IRB	2013 Year 10 Month 08 Day
Anticipated trial start date	2013 Year 10 Month 15 Day
Last follow-up date	2018 Year 04 Month 25 Day
Date of closure to data entry	2018 Year 04 Month 25 Day
Date trial data considered complete	2018 Year 04 Month 25 Day
Date analysis concluded	2018 Year 04 Month 25 Day

Other
Other related information

Management information
Registered date	2013 Year 10 Month 21 Day
Last modified on	2019 Year 04 Month 28 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000014094