臨床研究等提出・公開システム

Date of registration	Aug. 22, 2024
Last modified on	May. 08, 2025
Trial ID	jRCT2031240290

Scientific Title	An observational study of real-world outcomes in Japanese children with achondroplasia initiating Voxzogo before the age of 2 years
Public Title	An observational study of real-world outcomes in Japanese children with achondroplasia initiating Voxzogo before the age of 2 years

Contact for Scientific Queries	Name	Tokuoka Hirofumi
	Affiliation	BioMarin Pharmaceutical Japan K.K.
	Address	16th Floor JR Miraina Tower, 4-1-6, Shinjuku, Shinjuku-ku, Tokyo
	Telephone	+81-70-1465-7861
	E-mail	hirofumi.tokuoka@bmrn.com
Contact for Public Queries	Name	Trial Specialist
	Affiliation	BioMarin Pharmaceutical Inc.
	Address	770 Lindaro Street, San Rafael, CA, USA
	Telephone	1-800-983-4587
	E-mail	medinfo@bmrn.com

Recruitment status	Recruiting

Anticipated date of first enrollment		Dec. 06, 2024
Target sample size		50
Study Type		Observational
Study Design	allocation	single arm study
	masking	open(masking not used)
	control	uncontrolled control
	assignment	single assignment
	purpose	treatment purpose
Key inclusion & exclusion criteria	Inclusion Criteria	1. Documented diagnosis of ACH as per standard of care. 2. Have initiated Voxzogo treatment, or plan to initiate Voxzogo treatment within 3 months, before the age of 2 years, in accordance with local standard clinical practice and local label with full medical record available. 3. Parent(s) or guardian(s) must be willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any research-related procedure. 4. Have available medical history from the start of Voxzogo treatment, or 6 months of age, whichever is earlier, to enable collection of data elements of interest for the study (including anthropomorphic measurements, medical and surgical outcomes of interest). 5. Participants who are on Voxzogo treatment must be enrolled in 111-604 in parallel and provide consent to share safety information via 111-604.
	Exclusion Criteria	Currently enrolled in an interventional study (e.g., studies which require medical device use or drug therapy or with protocol required procedures) or prior enrollment in another interventional study(s) or receiving investigational agent(s)
	Age Minimum	No limit
	Age Maximum	2age old not
	Gender	Both
Health Condition(s) or Problem(s) Studied		achondroplasia
Primary Outcome(s)		Event rate of ACH-related surgeries of interest
Secondary Outcome(s)		Event rate of comorbidities including obstructive or central sleep apnea, foramen magnum stenosis, otitis media Event rate of continuous positive airway pressure (CPAP) and bilevel positive airway pressure (BiPAP) therapy Changes over time in anthropometric measurements including: Age at achievement of gross motor developmental milestones

Primary Sponsor	BioMarin Pharmaceutical Japan K.K.

Name of Certified Review Board	Review Board of Human Rights and Ethics for Clinical Studies Institutional Review Board
Address	2-2-1 Kyobashi, Chuo-ku, Tokyo, Japan, Tokyo
Approval Status	Approval
Date of approval	Sept. 19, 2024

Name of Certified Review Board	The Institutional Review Board, Juntendo University Hospital
Address	2-2-1 Kyobashi, Chuo-ku, Tokyo, Japan, Tokyo
Approval Status	Approval
Date of approval	Sept. 19, 2024

Name of Certified Review Board	Sendai City Hospital Institutional Review Board
Address	1-1-1 Asutonagamachi, Taihaku-ku, Sendai-shi, Miyagi, Japan, Tokyo
Approval Status	Approval
Date of approval	Sept. 19, 2024

Name of Certified Review Board	Hiroshima City Hiroshima Citizens Hospital Institutional Review Board
Address	7-33 Motomachi, Naka-ku, Hiroshima-shi, Hiroshima, Japan, Tokyo
Approval Status	Approval
Date of approval	Sept. 19, 2024

Name of Certified Review Board	Tohoku University Hospital Institutional Review Board
Address	1-1 Seiryo-machi, Aoba-ku, Sendai-shi, Miyagi, Japan, Tokyo
Approval Status	Approval
Date of approval	Sept. 19, 2024

Name of Certified Review Board	Nihon University Hospitals' Joint Institutional Review Board
Address	30-1, Oyaguchi Kami-cho, Itabashi-ku, Tokyo, Japan, Tokyo
Approval Status	Approval
Date of approval	Sept. 19, 2024

Name of Certified Review Board	Pediatric Central Institutional Review Board
Address	2-10-1 Okura, Setagaya-ku, Tokyo, Japan, Tokyo
Approval Status	Approval
Date of approval	Sept. 19, 2024

Name of Certified Review Board	Japan Community Health care Organization Osaka Hospital Institutional Review Board
Address	4-2-78 Fukushima, Fukushima-ku, Osaka-shi, Osaka, Japan, Tokyo
Approval Status	Approval
Date of approval	Sept. 19, 2024

Name of Certified Review Board	University of the Ryukyus Hospital Institutional Review Board
Address	1076, Kiyuna, Ginowan-shi, Okinawa, Japan, Tokyo
Approval Status	Approval
Date of approval	Sept. 19, 2024

Name of Certified Review Board	Medical Corporation Cattleyakai Mano Medical Clinic IRB
Address	1-8-1 Ebisu, Shibuya-ku, Tokyo, Japan, Tokyo
Approval Status	Approval
Date of approval	Sept. 19, 2024

Name of Certified Review Board	Kumamoto University Hospital Institutional Review Board
Address	1-1-1, Honjo, Chuo-ku, Kumamoto-shi, Kumamoto, Japan, Tokyo
Approval Status	Approval
Date of approval	Sept. 19, 2024

Name of Certified Review Board	The Institutional Review Board, The University of Osaka Hospital
Address	2-15 Yamadaoka, Suita-shi, Osaka, Japan, Tokyo
Approval Status	Approval
Date of approval	Sept. 19, 2024

Name of Certified Review Board	The Institutional Review Board, The University of Tokyo Hospital
Address	7-3-1 Hongo, Bunkyo-ku, Tokyo, Japan, Tokyo
Approval Status	Approval
Date of approval	Sept. 19, 2024

Plan to share IPD	No

Secondary ID(s)	NA

Countries of Recruitment（Except Japan）	none

History of Changes

No	Publication date
2	May. 08, 2025	(this page)	Changes
1	Aug. 22, 2024	Detail

List of change history