The purpose of this ongoing study is to gather information about how safe and effective an experimental MLD gene therapy is in children with pre-symptomatic early onset (Late Infantile - Early Juvenile) MLD.
Only children with pre-symptomatic early onset MLD will be eligible to participate in this study.
In addition to a confirmed MLD diagnosis, early onset status must be confirmed by EITHER:
a) Previous MLD diagnosis in an older sibling, whose age of symptom onset was 6 years or younger
b) In the absence of an older affected sibling, the child must be 6 years of age or younger and confirmed to have an early onset form of MLD. More detailed information about this study, including the selection criteria, can found on clinicaltrials.gov.
The study is being conducted at Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET) in Milan, Italy.
Participation in this study will last up to 8 years. During this time, study participants will undergo various tests, visit the study site in Milan and study staff will monitor any changes to health through the different phases of the study:
As part of the study, study participants will receive the gene therapy and all the study tests and procedures at no cost. Reasonable costs of travelling/accommodation related to study visits will be reimbursed for both study participants and their caregiver.
If you have questions or would like more information about this MLD gene therapy study, please ask your physician to contact the study investigator Prof. Aiuti at Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET), Milan, Italy or Orchard’s clinical trial team at firstname.lastname@example.org.