臨床研究等提出・公開システム

Date of registration	June. 08, 2022
Last modified on	Dec. 06, 2024
Trial ID	jRCT2031220113

Scientific Title	Observational study of children with achondroplasia
Public Title	Observational study of 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		June. 08, 2022
Actual date of first enrollment		Oct. 03, 2022
Target sample size		14
Study Type		Observational
Key inclusion & exclusion criteria	Inclusion Criteria	(1) 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 trial in the assent document (if possible) (2) Has achondroplasia, diagnosed by genetic diagnosis (3) Aged 5 to 14 years, inclusive, at study entry (4) Able to lead a normal life without assistance (5) Able to perform all the tests described in the protocol ,judged by Investigator
	Exclusion Criteria	(1) Has short stature condition other than achondroplasia (2) Has congenital diseases other than achondroplasia (3) Has history of treatment with growth hormone or VOXZOGO within 12 weeks before study entry (4) Has history of limb-lengthening surgery (5) With a closed growth plate (6) Has any of the following disorders that require surgical treatment on MRI or CT scans, or has clinically significant neurological symptoms or obstructive sleep apnea syndrome caused by these disorders: foramen magnum stenosis, spinal stenosis, hydrocephalus, spinal cord and cauda equina compression, etc (However, patients who have undergone surgical treatment for these disorders at least 26 weeks (6 months) before study entry and have no clinical problems can be enrolled) (7) Has a history of allergies against the following products containing RBM-007 components (e.g., nucleic acid, polyethylene glycol, mannitol): prescribed medicines, vaccines, over-the-counter medicines, cosmetics, foods, etc (8) Has history of cardiac or vascular disease (9) Shows clinically significant abnormalities in the clinical laboratory test result, which is related to hepatic function or renal function (10) Had a fracture of the long bones or spine (except for fracture of digits or toes) (11) Has history of hip surgery, severe hip dysplasia or serious hip injury (12) Highly likely to be unable to complete the trial, judged by the Investigator (13) Used any other IP or investigational medical device within 26 weeks (6 months) before study entry (14) Inadequate as a patient for this trial 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
Primary Outcome(s)		(1) Height growth velocity for 26 weeks (6 months) (2) Change in body weight, head circumference and chest circumference (3) Change in BMI (4) Change in serum ALP and NT-proCNP (5) Changes 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) Bone age (7) Morphological shape of the femoral growth plate (8) Condition of elbow and hip joints based on clinical findings (9) Tanner Staging Classification (10) Adverse event

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	July. 26, 2022

Name of Certified Review Board	Institutional Review Board of Osaka University Hospital
Address	2-15, Yamdaoka, Suita, Osaka, Tokyo
Telephone	+81-6-6879-5111
E-Mail	jim-chiken@hp-crc.med.osaka-u.ac.jp
Approval Status	Approval
Date of approval	July. 26, 2022

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
E-Mail	tikenkanri@jikei.ac.jp
Approval Status	Approval
Date of approval	July. 26, 2022

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	July. 26, 2022

Name of Certified Review Board	Hyogo Prefectural Kobe Children's Hospital Institutional Review Board
Address	1-6-7, Minatojima-minamimachi, Chuo-ku, Kobe, Tokyo
Telephone	+81-78-945-7300
Approval Status	Approval
Date of approval	July. 26, 2022

Name of Certified Review Board	Pediatric Clinical Trials Network Central Institutional Review Board
Address	2-10-1, Okura, Setagaya-ku, Tokyo, Tokyo
Telephone	+81-3-5494-7297
E-Mail	jctn_cirb@ncchd.go.jp
Approval Status	Approval
Date of approval	July. 26, 2022

Name of Certified Review Board	Kurashiki Central Hospital Institutional Review Board
Address	1-1-1 Miwa,Kurashiki,Okayama, Tokyo
Telephone	+81-86-422-0210
E-Mail	igakuken@kchnet.or.jp
Approval Status	Approval
Date of approval	July. 26, 2022

Plan to share IPD	No

Countries of Recruitment（Except Japan）	none

History of Changes

No	Publication date
12	Dec. 06, 2024	(this page)	Changes
11	Aug. 01, 2024	Detail	Changes
10	April. 17, 2024	Detail	Changes
9	Jan. 22, 2024	Detail	Changes
8	Oct. 31, 2023	Detail	Changes
7	June. 24, 2023	Detail	Changes
6	May. 17, 2023	Detail	Changes
5	Feb. 23, 2023	Detail	Changes
4	Dec. 23, 2022	Detail	Changes
3	Dec. 14, 2022	Detail	Changes
2	Aug. 26, 2022	Detail	Changes
1	June. 08, 2022	Detail

List of change history