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Date of registration	Aug. 26, 2024
Last modified on	Aug. 26, 2026
Trial ID	jRCT2071240053

Scientific Title	Phase I study of weekly infusion of JR-441 in patients with mucopolysaccharidosis type IIIA
Public Title	Phase I study of weekly infusion of JR-441 in patients with mucopolysaccharidosis type IIIA

Contact for Scientific Queries	Name	Takada Naoki
	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	Development Division Clinical Operation Unit
	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	Not Recruiting

Anticipated date of first enrollment		Oct. 01, 2024
Actual date of first enrollment		Oct. 08, 2024
Target sample size		3
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 1 year or older and under 18 years at the time of signing ICF - A subject with a diagnosis of MPS IIIA at the time of ICF - A subject with a minimal body weight of 10 kg at the time of screening
	Exclusion Criteria	- Prior experience to gene therapy or Hematopoietic stem cell transplantation 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 The above information is not intended to contain all considerations relevant to a patient's potential participation in this clinical trial.
	Age Minimum	1age old over
	Age Maximum	18age old not
	Gender	Both
Health Condition(s) or Problem(s) Studied		Mucopolysaccharidosis IIIA
Intervention(s)		JR-441 is administered intravenously at low, medium, or high doses once a week.
Primary Outcome(s)		- Incidence and severity of treatment-emergent adverse events - Change in safety laboratory tests - Change in vital signs - Abnormality in 12-lead electrocardiogram [Timeframe: Over the study period]
Secondary Outcome(s)		- Immunogenicity of JR-441 in plasma, as measured by the expression of anti-drug antibodies against JR-441 - Changes from baseline in oncentration of heparan sulfate in serum and urine [Timeframe: Over the study period] - Plasma drug concentrations and pharmacokinetics parameters [Timeframe: Multiple time points, including the point of administration of the maximum dose] - Changes from baseline in concentrations of heparan sulfate, ganglioside-monosialic acid 2, and ganglioside-monosialic acid 3 in cerebrospinal fluid [Timeframe: Baseline to Week 53]

Primary Sponsor	JCR Pharmaceuticals Co., Ltd.

Name of Certified Review Board	Local Incorporated Administrative Agency, Fukuoka City Hospital Organization, Fukuoka Children's Hospital Institutional Review Board
Address	5-1-1 Kashiiteriha Higashi-ku, Fukuoka-shi, Fukuoka
Telephone	+81-92-682-7000
Approval Status	Approval
Date of approval	Sept. 02, 2024

Name of Certified Review Board	Tokyo Medical University Hospital Institutional Review Board
Address	6-7-1 Nishishinjuku, Shinjuku-ku, Tokyo, Fukuoka
Telephone	+81-3-3342-6111
E-Mail	adm_crsc@tokyo-med.ac.jp
Approval Status	Approval
Date of approval	Sept. 02, 2024

Name of Certified Review Board	Institutional Review Board of National Center for Child Health and Development
Address	2-10-1 Okura, Setagaya-ku, Tokyo, Fukuoka
Telephone	+81-3-3416-0181
E-Mail	seiiku-chiken@ncchd.go.jp
Approval Status	Approval
Date of approval	Sept. 02, 2024

Name of Certified Review Board	Fukuoka University Chikushi Hospital Institutional Review Board
Address	1-1-1 Zokumyoin, Chikushino City, Fukuoka, Fukuoka
Telephone	+81-570-02-7777
E-Mail	chikushirinsho@adm.fukuoka-u.ac.jp
Approval Status	Approval
Date of approval	Sept. 02, 2024

Plan to share IPD	No

Countries of Recruitment（Except Japan）	none

History of Changes

No	Publication date
7	Aug. 26, 2026	(this page)	Changes
6	April. 24, 2026	Detail	Changes
5	April. 22, 2025	Detail	Changes
4	April. 10, 2025	Detail	Changes
3	Nov. 15, 2024	Detail	Changes
2	Sept. 18, 2024	Detail	Changes
1	Aug. 26, 2024	Detail

List of change history