臨床研究等提出・公開システム

Date of registration	Dec. 18, 2025
Last modified on	Jan. 23, 2026
Trial ID	jRCT2041250146

Scientific Title	Post-Trial Access (PTA) Program for Patients in Japan with Metachromatic Leukodystrophy who Received SHP611 While Participating either in the SHP611-201 or the HGT-MLD-071 Study
Public Title	Post-Trial Access (PTA) Program of SHP611

Contact for Scientific Queries	Name	Nonomura Hidenori
	Affiliation	Takeda Pharmaceutical Company Limited
	Address	1-1, Doshomachi 4-chome, Chuo-ku, Osaka
	Telephone	+81-6-6204-2111
	E-mail	smb.Japanclinicalstudydisclosure@takeda.com
Contact for Public Queries	Name	Contact for Clinical Trial Information
	Affiliation	Takeda Pharmaceutical Company Limited
	Address	1-1, Doshomachi 4-chome, Chuo-ku, Osaka
	Telephone	+81-6-6204-2111
	E-mail	smb.Japanclinicalstudydisclosure@takeda.com

Recruitment status	Not Recruiting

Anticipated date of first enrollment		Jan. 22, 2026
Actual date of first enrollment		Jan. 22, 2026
Target sample size		4
Study Type		Other
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. The patient must be a resident of Japan and must be receiving SHP611 through either the SHP611-201 or the HGT-MLD-071 study. 2. The patient must derive continued benefit from SHP611 treatment in the opinion of their parent(s) or legally authorized representative(s) in consultation with their treating physician. 3. The patient must have no safety or medical issues that, in the opinion of the treating physician, would contraindicate participation. 4. The patient's parent(s) or legally authorized representative(s) must provide consent through the Information Sheet for participation in the program.
	Exclusion Criteria	Any patients with metachromatic leukodystrophy (MLD) not enrolled in either parent studies (SHP611-201 or the HGT-MLD-071) will not be eligible for participation in this program.
	Age Minimum	No limit
	Age Maximum	No limit
	Gender	Both
Health Condition(s) or Problem(s) Studied		Metachromatic Leukodystrophy
Intervention(s)		The treated patients will continue their weekly or every-other-week doses of the drug as prescribed to them by the treating physician in either the SHP611-201 (weekly) or the HGT-MLD-071 (every-other-week) study. Patients will be able to receive SHP611 through this program until there are either any safety concerns or they no longer benefit from the treatment in the opinion of their parents or legally authorized representative(s) in consultation with their treating physician.
Primary Outcome(s)		Not applicable due to Post-Trial Access (PTA) Program
Secondary Outcome(s)		Not applicable due to Post-Trial Access (PTA) Program

Primary Sponsor	Takeda Pharmaceutical Company Limited

Secondary Sponsor

Name of Certified Review Board	Kanazawa University Hospital Institutional Review Board
Address	13-1 Takara-machi, Kanazawa, Ishikawa, Japan, Ishikawa
Telephone	+81-76-265-2090
Approval Status	Approval
Date of approval	Dec. 11, 2025

Plan to share IPD	No
Plan description	De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be reidentified (due to the limited number of study participants/study sites).

Countries of Recruitment（Except Japan）	Argentina/Brazil/Canada/Czech Republic/Denmark/France/Germany/Greece/Italy/Poland/Spain/United Kingdom/United States

History of Changes

No	Publication date
2	Jan. 23, 2026	(this page)	Changes
1	Dec. 18, 2025	Detail

List of change history