• Adrenoleukodystrophy (ALD) Gene Therapy Clinical Trial

    Gene Therapy

    Phone: 617-632-5064  

    Adrenoleukodystrophy (ALD) is a rare and progressive genetic disorder, usually diagnosed in boys, that affects the nervous system and the adrenal glands (small glands found on top of the kidneys). The cells of a child with ALD are missing the gene for an enzyme called ALDP, which helps break down fatty acids.

    Children who meet the following criteria may be eligible to take part in our ALD gene therapy trial:

    • Be male, aged 17 years or younger at time of consent
    • Have active cerebral ALD as defined by elevated VLCFA levels
    • Have active central nervous system (CNS) disease established by central radiographic review of brain MRI demonstrating:
    • Loes score between 0.5 and 9 (inclusive) on the 34-point scale
    • Gadolinium enhancement of demyelinating lesions on MRI
    • Lack availability of a 10/10 human leukocyte antigen (HLA)-matched sibling donor
    Learn more