臨床研究等提出・公開システム

Japanese

Date of registration	May. 23, 2025
Last modified on	May. 23, 2025
Trial ID	jRCT2021250004

Scientific Title	A randomized, double-blind, placebo-controlled phase III study to evaluate the efficacy, safety, and tolerability of remibrutinib in patients with generalized Myasthenia Gravis, followed by an open-label extension phase (CLOU064O12301)
Public Title	A study to evaluate the efficacy, safety, and tolerability of remibrutinib tablets compared with placebo tablets in patients aged 18 to 75 with generalized Myasthenia Gravis who are receiving standard treatment. (CLOU064O12301)

Contact for Scientific Queries	Name	Maruyama Hideki
	Affiliation	Novartis Pharma. K.K.
	Address	Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan
	Telephone	+81-120-003-293
	E-mail	rinshoshiken.toroku@novartis.com
Contact for Public Queries	Name	Maruyama Hideki
	Affiliation	Novartis Pharma. K.K.
	Address	Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan
	Telephone	+81-120-003-293
	E-mail	rinshoshiken.toroku@novartis.com

Recruitment status	Recruiting

Anticipated date of first enrollment		May. 30, 2025
Actual date of first enrollment
Target sample size		14
Study Type		Interventional
Study Design	allocation	randomized controlled trial
	masking	double blind
	control	placebo control
	assignment	parallel assignment
	purpose	treatment purpose
Key inclusion & exclusion criteria	Inclusion Criteria	-Adult patients with gMG (age 18-75 years) -Confirmed diagnosis of Myasthenia Gravis Foundation of America (MGFA) Class II-IV gMG at screening and likely not in need of a respirator for the duration of the study, as judged by the Investigator -Documented evidence of positive serologic testing for AChR+ antibody or MuSK+ antibody at screening, OR seronegative for both AChR and MuSK antibodies at screening -Baseline MG-ADL score >= 6 with >= 50% of the total score due to non-ocular symptoms -Participants who have been on a stable dose of standard-of-care treatment as specified in the protocol -Able to safely swallow the study medication according to investigator clinical judgement based on a bedside swallowing test or another formal swallowing test in line with local practice, both at Screening and Baseline
	Exclusion Criteria	-Prior to baseline have been treated with intravenous immunoglobulins or plasma exchange (IVIg/PLEX) in the past month, with rituximab in the past 6 months, eculizumab in the past 2 months, ravulizumab or other complement inhibitors in the past 3 months, efgartigimod or other anti-FcRn therapies in the past 3 months, or had a thymectomy in the past 6 months or a planned thymectomy during the trial period -Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for 1 week after stopping of study treatment
	Age Minimum	18age old over
	Age Maximum	75age old under
	Gender	Both
Health Condition(s) or Problem(s) Studied		generalized Myasthenia Gravis
Intervention(s)		Remibrutinib arm Core Part: Remibrutinib tablet taken orally [Extension Part: Open-label remibrutinib tablet taken orally] Placebo arm Core Part: Placebo tablet taken orally [Extension Part: Open-label remibrutinib tablet taken orally]
Health Condition(s) Keyword
Intervention(s) Keyword
Health Condition(s) Code
Intervention(s) Code
Primary Outcome(s)		Change from baseline to Month 6 in Myasthenia Gravis Activity of Daily Living (MG-ADL) total score
Secondary Outcome(s)

Primary Sponsor	Novartis Pharma. K.K.

Source of Monetary Support / Secondary Sponsor
Secondary Sponsor

Source of Monetary Support
Secondary Sponsor

Name of Certified Review Board	General Hanamaki Hospital Institutional Review Board
Address	4-56 Otayacho, Hamanaki, Iwate
Telephone	+81-198-23-3311
E-Mail
Approval Status	Approval
Date of approval	Mar. 18, 2025

Plan to share IPD	Yes
Plan description	Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.

Plan to share IPD

Yes

Plan description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.

Secondary ID(s)	NCT06744920
Issuing Authority	Clinical Trials.gov

Countries of Recruitment（Except Japan）	US/France/Germany/Poland/China/Spain/India/Canada/Taiwan/Australia/Korea/Romania/Belgium/Netherland