臨床研究等提出・公開システム

Japanese

Date of registration	Aug. 26, 2024
Last modified on	Nov. 15, 2024
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	Ibaraki 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	Ibaraki 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	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 or high doses once a week.
Health Condition(s) Keyword
Intervention(s) Keyword
Health Condition(s) Code
Intervention(s) Code
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.

Source of Monetary Support / Secondary Sponsor
Secondary Sponsor

Source of Monetary Support
Secondary Sponsor

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
E-Mail
Approval Status	Approval
Date of approval	Sept. 02, 2024

Plan to share IPD	No
Plan description

Secondary ID(s)
Issuing Authority

Countries of Recruitment（Except Japan）	none

History of Changes

No	Publication date
3	Nov. 15, 2024	(this page)	Changes
2	Sept. 18, 2024	Detail	Changes
1	Aug. 26, 2024	Detail

List of change history