UMIN-CTR Clinical Trial

Basic information
Public title	An open-label continuation trial of tocilizumab for familial Mediterranean fever with colchicine ineffective or intolerance
Acronym	An open-label continuation trial of tocilizumab for familial Mediterranean fever
Scientific Title	An open-label continuation trial of tocilizumab for familial Mediterranean fever with colchicine ineffective or intolerance
Scientific Title:Acronym	An open-label continuation trial of tocilizumab for familial Mediterranean fever
Region	Japan

Condition
Condition	familial Mediterranean fever
Classification by specialty	Clinical immunology Pediatrics
Classification by malignancy	Others
Genomic information	YES

Objectives
Narrative objectives1	To assess the long-term safety of Tocilizumab in FMF patients
Basic objectives2	Safety
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	Safety Adverse event
Key secondary outcomes	Efficacy Number of fever attacks

Base
Study type	Interventional

Study design
Basic design	expanded access
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	Tocilizumab
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	12 years-old <=
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	(1) Patients who completed the 24-week study drug administration period of the preceding trial. (2) Patients who got adequate explanation about the contents of explanatory documents and other matters concerning clinical trials, understand the contents thereof, and obtain written consent based on free will about participation in this trial.
Key exclusion criteria	Patients applicable to even one of the following are excluded from the subject (1) Women who are breast-feeding, pregnant, or may become pregnant (2) Patients with a history of hypersensitivity to the components of tocilizumab (3) Patients whose corticosteroids are routinely used (excluding topical treatments such as external preparations) due to diseases other than familial Mediterranean fever and which are judged inappropriate by investigators or clinical trial doctors (4) Patients who have interstitial pneumonia and who are judged inappropriate by investigators or clinical trial doctors (5) Patients who have complicated serious diseases and who are judged inappropriate by investigators or clinical trial doctors as clinical trials (6) Patients whose investigator or clinical trial doctor judged that the condition in the preceding study is inappropriate for continued administration (7) Patients judged inappropriate by investigators or clinical trial doctors
Target sample size	24

Research contact person
Name of lead principal investigator	1st name Atsushi Middle name Last name Kawakami
Organization	Nagasaki University Graduate School of Biomedical Sciences
Division name	Department of Immunology and Rheumatology, Division of Advanced Preventive Medical Sciences
Zip code	8528501
Address	Sakamoto 1-7-1, Nagasaki
TEL	095-819-7260
Email	atsushik@nagasaki-u.ac.jp

Public contact
Name of contact person	1st name Tomohiro Middle name Last name Koga
Organization	Nagasaki University Graduate School of Biomedical Sciences
Division name	Department of Immunology and Rheumatology, Division of Advanced Preventive Medical Sciences
Zip code	8528501
Address	Sakamoto 1-7-1, Nagasaki
TEL	095-819-7260
Homepage URL
Email	tkoga@nagasaki-u.ac.jp

Sponsor
Institute	Department of Immunology and Rheumatology, Division of Advanced Preventive Medical Sciences, Nagasaki University Graduate School of Biomedical Sciences
Institute
Department

Funding Source
Organization	Japan Agency for Medical Research and Development
Organization
Division
Category of Funding Organization	Japanese Governmental office
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funder(s)	CHUGAI PHARMACEUTICAL CO., LTD.

IRB Contact (For public release)
Organization	Institutional Review Board, Nagasaki University Hospital
Address	1-7-1 Sakamoto, Nagasaki 852-8501
Tel	095-819-7256
Email	2745@ml.nagasaki-u.ac.jp

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	長崎大学病院（長崎県）、千葉大学医学部附属病院（千葉県）、福島県立医科大学附属病院（福島県）、九州大学病院（福岡県）、京都大学医学部附属病院（京都府）、金沢大学附属病院（金沢県）、信州大学医学部附属病院（長野県）、横浜市立大学附属病院（神奈川県）、北海道大学病院（北海道）

Other administrative information
Date of disclosure of the study information	2018 Year 05 Month 30 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	2018 Year 05 Month 30 Day
Date of IRB	2018 Year 05 Month 31 Day
Anticipated trial start date	2018 Year 09 Month 01 Day
Last follow-up date	2021 Year 02 Month 19 Day
Date of closure to data entry	2021 Year 04 Month 30 Day
Date trial data considered complete	2021 Year 05 Month 06 Day
Date analysis concluded	2021 Year 07 Month 28 Day

Other

Other related information

The manuscript title An open-label continuation trial of tocilizumab for familial Mediterranean fever with colchicine ineffective or intolerance : Study protocol for investigator-initiated, multicenter, open-label trial The authorship list in the study protocol Tomohiro Koga, MD, PhD, Naoko Hagimori, PhD, Shuntaro Sato, PhD, Shinpei Morimoto, PhD, Naoki Hosogaya, MD, PhD, Chizu Fukushima, MD, PhD, Hiroshi Yamamoto, Atsushi Kawakami, MD, PhD

Management information
Registered date	2018 Year 05 Month 11 Day
Last modified on	2021 Year 05 Month 12 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000037116