An Open-Label Extension of Study HGT-MLD-070 Evaluating Long Term Safety and Efficacy of Intrathecal Administration of HGT-1110 in Patients With Metachromatic Leukodystrophy

HGT-MLD-071

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.

2013-05-23

2024-12-31

2024-12-31

Active (not recruiting)

Early phase I

24

Inclusion Criteria: 1. Participant has participated in Study HGT-MLD-070 (NCT01510028) through Week 40. 2. Participant must have no safety or medical issues that contraindicate participation. 3. 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: 1. 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. 2. Undergoes bone marrow transplant (BMT), hematopoietic stem cell transplantation (HSCT), or gene therapy at any point during the study. 3. 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. 4. The participant is pregnant or breastfeeding. 5. 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. 6. The participant has a condition that is contraindicated as described in the SOPH-A-PORT Mini SIDDD Instructions for Use (IFU), including: 1. The participant has had, or may have, an allergic reaction to the materials of construction of the SOPH-A-PORT Mini S device. 2. The participant's body size is too small to support the size of the SOPH-A-PORT Mini S Access Port, as judged by the investigator. 3. The participant has a known or suspected local or general infection. 4. The participant is at risk of abnormal bleeding due to a medical condition or therapy. 5. The participant has one or more spinal abnormalities that could complicate safe implantation or fixation. 6. The participant has a functioning CSF shunt device. 7. The participant has shown an intolerance to an implanted device.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 24, 'type': 'ACTUAL'}}

2023-07-21