The purpose of this study is to collect long-term safety data in participants with metachromatic leukodystrophy (MLD) who are receiving HGT-1110 and have participated in Study HGT-MLD-070 (NCT01510028) through Week 40.
Who can participate
Age range0 Years ā 13 Years
SexALL
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Inclusion criteria
ā. Participant has participated in Study HGT-MLD-070 (NCT01510028) through Week 40.
ā. Participant must have no safety or medical issues that contraindicate participation.
ā. The participant, participant's parent(s), or legally authorized representative(s) must provide written informed consent and/or assent (if applicable) prior to performing any study-related activities.
Exclusion criteria
ā. The participant is unable to comply with the protocol (example, is unable to return for safety evaluations, or is otherwise unlikely to complete the study) as determined by the investigator.
ā. Undergoes bone marrow transplant (BMT), hematopoietic stem cell transplantation (HSCT), or gene therapy at any point during the study.
ā. The participant has any known or suspected hypersensitivity to agents used for anesthesia or is thought to be at an unacceptably high risk for associated potential complications of airway compromise or other conditions.
ā. The participant is pregnant or breastfeeding.
ā. The participant is enrolled in another clinical study that involves clinical investigations or use of any investigational product (drug or drug delivery device) other than those used in HGT-MLD-070 (NCT01510028) within 6 months prior to study enrollment or at any time during the study.
ā. The participant has a condition that is contraindicated as described in the SOPH-A-PORT Mini SIDDD Instructions for Use (IFU), including:
ā
What they're measuring
1
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Timeframe: Baseline to Follow-up (Week 628)
2
Presence of Anti-HGT-1110 Antibodies in Cerebrospinal Fluid (CSF) and Serum
Timeframe: Baseline until end of the study (Week 624)