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A safety and efficacy extension of study HGT-MLD-070 in Children with Metachromatic Leukodystrophy recieving enzyme (HGT-1110) replacement by intrathecal injection
EU CTIS ID: 2024-514403-34-00
What this study is testing
The primary objective of this study is to collect long-term safety data in patients with MLD who are receiving HGT-1110 and have participated in Study HGT-MLD-070 through Week 40.
- Phase I and Phase II (Integrated)- Other
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Patient has participated in Study HGT-MLD-070 through Week 40.
- Patient must have no safety or medical issues that contraindicate participation.
- The patient, patient's parent or legally authorized representative(s) must provide written informed consent and/or assent (if applicable) prior to performing any study-related activities.
You likely can't join if
- The patient is unable to comply with the protocol (eg, is unable to return for safety evaluations, or is otherwise unlikely to complete the study) as determined by the Investigator.
- 6d. The patient is at risk of abnormal bleeding due to a medical condition or therapy.
- 6e. The patient has one or more spinal abnormalities that could complicate safe implantation or fixation.
- 6f. The patient has a functioning CSF shunt device.
- 6g. The patient has shown intolerance to an implanted device.
- Undergoes bone marrow transplantation, hematopoietic stem cell transplantation, or gene therapy at any point during the study.
See the full eligibility criteria
- Patient has participated in Study HGT-MLD-070 through Week 40.
- Patient must have no safety or medical issues that contraindicate participation.
- The patient, patient's parent or legally authorized representative(s) must provide written informed consent and/or assent (if applicable) prior to performing any study-related activities.
- The patient is unable to comply with the protocol (eg, is unable to return for safety evaluations, or is otherwise unlikely to complete the study) as determined by the Investigator.
- 6d. The patient is at risk of abnormal bleeding due to a medical condition or therapy.
- 6e. The patient has one or more spinal abnormalities that could complicate safe implantation or fixation.
- 6f. The patient has a functioning CSF shunt device.
- 6g. The patient has shown intolerance to an implanted device.
- Undergoes bone marrow transplantation, hematopoietic stem cell transplantation, or gene therapy at any point during the study.
- The patient has any known or suspected hypersensitivity to agents used for anesthesiaor is thought to be at an unacceptably high risk for associated potential complications of airway compromise or other conditions.
- The patient is pregnant or breastfeeding.
- The patient 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 within 6 months prior to study enrollment or at any time during the study.
- The patient has a condition that is contraindicated as described in the SOPH-A-PORT Mini S IDDD Instructions for Use, including:
- 6a. The patient has had, or may have, an allergic reaction to the materials of construction of the SOPH-A-PORT Mini S device.
- 6b. The patient’s body size is too small to support the size of the SOPH-A-PORT Mini S Access Port, as judged by the investigator.
- 6c. The patient has a known or suspected local or general infection.
The study team makes the final eligibility decision.
Where it's taking place
- Japan
- United Kingdom
- Brazil
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Japan; United Kingdom; Brazil. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.