臨床研究等提出・公開システム

Japanese

Date of registration	July. 30, 2024
Last modified on	July. 30, 2024
Trial ID	jRCT2071240043

Scientific Title	An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
Public Title	An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB

Contact for Scientific Queries	Name	Wakita Ryo
	Affiliation	JCR Pharmaceuticals Co., Ltd.
	Address	11-18, Kusunoki-cho, Ashiya-shi, 659-0015, Hyogo
	Telephone	+81-797-32-8582
	E-mail	clinical_development@jp.jcrpharm.com
Contact for Public Queries	Name	Wakita Ryo
	Affiliation	JCR Pharmaceuticals Co., Ltd.
	Address	11-18, Kusunoki-cho, Ashiya-shi, 659-0015, Hyogo
	Telephone	+81-797-32-8582
	E-mail	clinical_development@jp.jcrpharm.com

Recruitment status	Pending

Anticipated date of first enrollment		Aug. 01, 2024
Actual date of first enrollment
Target sample size		10
Study Type		Interventional
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	-Chronological age of <18 years -Confirmed diagnosis of MPS III
	Exclusion Criteria	-Prior experience to gene therapy or HSCT with successful engraftment -Past use of another investigational drug or product in last 4 months or 5 half-lives (whichever is longer) before signing ICF -Current participation in a clinical trial or past participation (within 30 days of enrolment into this study) in a study involving invasive procedures -Past use of Genistein or Kineret (anakinra) within 4 months before signing ICF - Serious drug allergy or hypersensitivity -Contraindication for lumbar puncture or MRI - History of bleeding disorder or current use of medications that, in the opinion of the investigator, place them at risk of bleeding following lumbar puncture The above information is not intended to contain all considerations relevant to a patient's potential participation in this clinical trial.
	Age Minimum	No limit
	Age Maximum	18age old not
	Gender	Both
Health Condition(s) or Problem(s) Studied		Mucopolysaccharidosis IIIB
Intervention(s)		JR-446 will be administered intravenously at multiple doses. Once the optimal dose is determined, all subjects will receive the same dose intravenously.
Health Condition(s) Keyword
Intervention(s) Keyword
Health Condition(s) Code
Intervention(s) Code
Primary Outcome(s)		1.To evaluate the tolerability of JR-446 in MPSIIIB patients Incidence and severity of infusion-associated reactions, including anaphylaxis [Time Frame: up to 4 years (multiple visits)] 2. To explore the potential efficacy of JR-446 Change from baseline in heparan sulfate levels in cerebrospinal fluid (CSF) [Time Frame: up to 4 years (multiple visits)]
Secondary Outcome(s)		1. Change from baseline in cognitive function [Time Frame: up tp 4 years (multiple visits)] 2. Change from baseline in HS concentration in serum and urine [Time Frame: up tp 4 years (multiple visits)] 3. Plasma drug concentration [Time Frame: up tp 4 years (multiple visits)]

Primary Sponsor	JCR Pharmaceuticals Co., Ltd.

Source of Monetary Support / Secondary Sponsor
Secondary Sponsor

Source of Monetary Support	MEDIPAL HOLDINGS CORPORATION
Secondary Sponsor	Applicable

Name of Certified Review Board	University of the Ryukyus Hospital Institutional Review Board
Address	207 Banchi, Uehara, Nishihara-cho,Nakagami-gun, Okinawa
Telephone	+81-98-895-3331
E-Mail
Approval Status
Date of approval

Name of Certified Review Board	Hiroshima University hospital IRB
Address	1-2-3, Kasumi, Minami-ku, Okinawa
Telephone	+81-82-257-5596
E-Mail	hugcp@hiroshima-u.ac.jp
Approval Status
Date of approval

Name of Certified Review Board	Center for Clinical Research and Development National Center for Child Health and Development
Address	2-10-1Okura,Setagaya-ku, Okinawa
Telephone	+81-3-3416-0181
E-Mail	seiiku-chiken_irb@ncchd.go.jp
Approval Status
Date of approval

Plan to share IPD	No
Plan description

Secondary ID(s)	NCT06488924
Issuing Authority	ClinicalTrials.gov

Countries of Recruitment（Except Japan）	none