IGNITE
Research type
Research Study
Full title
A Phase 2 Safety, Tolerability, and Proof-of-Concept Study of VGL101 in Patients With Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia (ALSP)
IRAS ID
1007099
Contact name
Supicha Kridaratikorn
Contact email
Sponsor organisation
Vigil Neuroscience, Inc.
Eudract number
2022-001979-13
Research summary
Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) is a rare, inherited, neurological disease that affects an estimated 10,000 people in the United States, with about 1000 to 2000 new cases annually. Similar epidemiology exists for Europe and Japan.
Adults are typically diagnosed in their forties. Diagnosis is done through genetic testing with associated clinical and imaging criteria.
Symptoms include cognitive dysfunction, neuropsychiatric symptoms, and motor impairment. These symptoms typically exhibit rapid progression, which causes significant patient and caregiver burden. The average life expectancy is approximately 6 to 7 years after diagnosis.
There are currently no approved products to treat ALSP.
ALSP is caused by genetic mutations in a type of protein found on the immune cells located in the brain and central nervous system. As a result of these mutations, these cells do not work properly, leading to loss of white matter in the brain and other associated CNS deficiencies. VGL101 is a special type of protein called an antibody that is expected to override the mutations found in people with ALSP and stop or slow the loss of white matter in the brain.REC name
East of England - Essex Research Ethics Committee
REC reference
23/EE/0026
Date of REC Opinion
21 Apr 2023
REC opinion
Further Information Favourable Opinion