Metachromatic Leukodystrophy
Conditions
Interventions
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-
Sponsors
Nonomura Hidenori
Eligibility
Sex/Gender
All
Inclusion 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
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Not applicable due to Post-Trial Access (PTA) Program | — |
Secondary
| Measure | Time frame |
|---|---|
| Not applicable due to Post-Trial Access (PTA) Program | — |
Countries
Argentina, Brazil, Canada, Czech Republic, Denmark, France, Germany, Greece, Italy, Japan, Poland, Spain, United Kingdom, United States
Contacts
Public ContactContact for Clinical Trial Information
Takeda Pharmaceutical Company Limited
Outcome results
None listed