臨床研究等提出・公開システム

Date of registration	Sept. 16, 2022
Last modified on	July. 01, 2026
Trial ID	jRCT2031220338

Scientific Title	A long-term study of RBM-007 in children with achondroplasia
Public Title	A long-term study of RBM-007 in children with achondroplasia

Contact for Scientific Queries	Name	Nonaka Yosuke
	Affiliation	RIBOMIC Inc.
	Address	3-16-13 Shirokanedai, Minato-ku, Tokyo 108-0071, Japan
	Telephone	+81-3-3440-3303
	E-mail	y.nonaka@ribomic.com
Contact for Public Queries	Name	Nonaka Yosuke
	Affiliation	RIBOMIC Inc.
	Address	3-16-13 Shirokanedai, Minato-ku, Tokyo 108-0071, Japan
	Telephone	+81-3-3440-3303
	E-mail	y.nonaka@ribomic.com

Recruitment status	Not Recruiting

Anticipated date of first enrollment		Sept. 16, 2022
Actual date of first enrollment		Dec. 21, 2023
Target sample size		12
Study Type		Interventional
Study Design	allocation	non-randomized controlled trial
	masking	open(masking not used)
	control	uncontrolled control
	assignment	single assignment
	purpose	treatment purpose
Key inclusion & exclusion criteria	Inclusion Criteria	(1) Has completed the early phase II study, has completed the safety and efficacy evaluation (height growth velocity, etc.) by Investigator, and has been judged to have no safety issues (2) Able to obtain written informed consent from surrogate decision maker (parents, relatives or legal representative) before study entry Able to obtain written informed assent from the patient after explaining the contents of this study in the assent document (if possible) (3) Able to perform all the tests and administration of investigational product described in the protocol, judged by Investigator
	Exclusion Criteria	(1) Inadequate as a patient for this study based on examinations, observations, etc. at the end of the early phase II study (2) Highly likely to be unable to complete this study due to poor compliance treated with RBM-007 or poor implementation of regular examinations and observation items, judged by the Investigator (3) Diagnosed with a closed growth plate in the examination at the end of the early phase II study (4) Has a positive pregnancy test at the end of the early phase II study (only female patients) (5) Inadequate as a patient for this study for any other reason than above, considered by the Investigator
	Age Minimum	5age old over
	Age Maximum	14age old under
	Gender	Both
Health Condition(s) or Problem(s) Studied		Achondroplasia
Intervention(s)		Administration of RBM-007 at a dose of 0.3 or 0.6 mg/kg, every 1 to 4 weeks. The administration period is 1 to 5 years (including the administration period of the early phase II study) or the period until the height reaches the average height of healthy children, whichever is shorter.
Primary Outcome(s)		(1) Comparison of height growth velocity in the observational study and height growth velocity after the early phase II study entry (2) Change in serum ALP and NT-proCNP (3) Change in body weight, head circumference and chest circumference (4) Change in BMI (5) Change in body proportion ratios (ratio of upper arm length to forearm length, ratio of thigh length to lower leg length, ratio of arm span to height) (6) Condition of the elbow and hip joints based on clinical findings (7) Bone age (8) Morphological shape of the femoral growth plate (9) Tanner Staging Classification (10) Adverse event Note: The following assessments were discontinued and were no longer performed from Protocol Version 6.0 onward: i) Serum ALP and NT-proCNP levels ii) Changes in head circumference and chest circumference iii) Ratio of upper arm length to forearm length, and ratio of thigh length to lower leg length

Primary Sponsor	RIBOMIC Inc.

Name of Certified Review Board	Institute of Science Tokyo Hospital Institutional Review Board
Address	1-5-45 Yushima, Bunkyo-ku, Tokyo, Tokyo
Telephone	+81-3-5803-5612
E-Mail	tiken.crc@tmd.ac.jp
Approval Status	Approval
Date of approval	Oct. 24, 2023

Name of Certified Review Board	Osaka City General Hospital Institutional Review Board
Address	2-13-22, Miyakojimahondori, Osaka Shi Miyakojima Ku, Osaka, Tokyo
Telephone	+81-6-6929-3269
E-Mail	chiken@osakacity-hp.or.jp
Approval Status	Approval
Date of approval	Oct. 24, 2023

Name of Certified Review Board	Jikei University Hospital Drug Clinical Trial Review Committee
Address	3-19-18, Nishi-Shinbashi, Minato-ku, Tokyo, Tokyo
Telephone	+81-3-3433-1111
Approval Status	Approval
Date of approval	Oct. 24, 2023

Name of Certified Review Board	Pediatric Clinical Trials Network Central Institutional Review Board
Address	2-10-1, Okura, Setagaya-ku, Tokyo, Tokyo, Tokyo
Telephone	+81-3-5494-7297
E-Mail	jctn_cirb@ncchd.go.jp
Approval Status	Approval
Date of approval	Oct. 24, 2023

Name of Certified Review Board	Kurashiki Central Hospital Institutional Review Board
Address	1-1-1 Miwa,Kurashiki,Okayama, Okayama, Tokyo
Telephone	+81-86-422-0210
E-Mail	igakuken@kchnet.or.jp
Approval Status	Approval
Date of approval	Oct. 24, 2023

Name of Certified Review Board	Hyogo Prefectural Kobe Children's Hospital Institutional Review Board
Address	1-6-7, Minatojima-minamimachi, Chuo-ku, Kobe, Hyogo, Tokyo
Telephone	+81-78-945-7300
Approval Status	Approval
Date of approval	Oct. 24, 2023

Plan to share IPD	No

Countries of Recruitment（Except Japan）	none

History of Changes

No	Publication date
9	July. 01, 2026	(this page)	Changes
8	Feb. 26, 2025	Detail	Changes
7	Dec. 06, 2024	Detail	Changes
6	Aug. 01, 2024	Detail	Changes
5	April. 18, 2024	Detail	Changes
4	Jan. 09, 2024	Detail	Changes
3	Oct. 31, 2023	Detail	Changes
2	May. 20, 2023	Detail	Changes
1	Sept. 16, 2022	Detail

List of change history