NIPH Clinical Trials Search

Post-Trial Access (PTA) Program of SHP611

Basic Information

Recruitment status	Not Recruiting
Health condition(s) or Problem(s) studied	Metachromatic Leukodystrophy
Date of first enrollment	22/01/2026
Target sample size	4
Countries of recruitment	Argentina,Japan,Brazil,Japan,Canada,Japan,Czech Republic,Japan,Denmark,Japan,France,Japan,Germany,Japan,Greece,Japan,Italy,Japan,Poland,Japan,Spain,Japan,United Kingdom,Japan,United States,Japan
Study type	Other
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.

Outcome(s)

Primary Outcome	Not applicable due to Post-Trial Access (PTA) Program
Secondary Outcome	Not applicable due to Post-Trial Access (PTA) Program

Key inclusion & exclusion criteria

Age minimum	Not applicable
Age maximum	Not applicable
Gender	Both
Include 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.
Exclude 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.