Illuminate

• Research type

  Research Study

• Full title

  A Natural History Study of Patients With Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia (ALSP)

• IRAS ID

  303729

• Contact name

  Julie Carafa

• Contact email

  Julie.Carafa@parexel.com

• Sponsor organisation

  Vigil Neuroscience, Inc.

• Duration of Study in the UK

  3 years, 4 months, 22 days

• Research summary

  Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) is a rare, rapidly progressing, genetic, neurodegenerative (nerve function loss/death) disease caused by mutations in the CSF1R gene. There are no definitive treatment options and limited information on the natural history of the disease. The structural, genetic, and neuropathophysiological (nervous system physiology) abnormalities of ALSP lead to the onset of neurologic symptoms, such as moderate to severe motor (movement) and neuropsychiatric (mental) impairments.
  Vigil Neuroscience, Inc. is developing VGL101, a monoclonal antibody (man-made immune system protein) for the treatment of ALSP. This natural history (observational) study will collect data to contribute to the development of future novel therapies, including VGL101, that focus on the neuropathophysiological features that underlie ALSP and that are essential to reverse, delay, or stop progression of this debilitating disorder.
  About 50 male and female adult participants at about 15 centres worldwide will participate in this study (approximately 38 with ALSP and 12 carriers of the CSF1R gene mutation ) for 24 months. Clinic visits to assess disease status will be conducted at Screening/Baseline and at Months 6, 12, 18, and 24. Each clinic visit will include clinical assessments and MRI imaging. Blood and cerebral spinal fluid (CSF) for biomarker analysis will be collected at Screening/Baseline and at Months 12 and 24. Adverse events (AEs) and concomitant medications and procedures will be recorded throughout the 24-month study.

• REC name

  West Midlands - Coventry & Warwickshire Research Ethics Committee

• REC reference

  21/WM/0224

• Date of REC Opinion

  23 Nov 2021

• REC opinion

  Further Information Favourable Opinion