NCT05677659

A Study of VGL101 in Patients With Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia

Terminated Phase 2 Last updated 11 June 2025

What this trial tests

Phase 2 trial testing VGL101 in ALSP in 20 participants. Terminated before completion.

Timeline

14 December 2022

Primary endpoint
14 February 2025

4 June 2025

Quick facts

Lead sponsor	Vigil Neuroscience, Inc.
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	20
Start date	14 December 2022
Primary completion	14 February 2025
Estimated completion	4 June 2025
Sites	10 locations across France, Netherlands, United Kingdom, Germany, United States

Drugs / interventions tested

• VGL101 — full drug profile →

Conditions studied

• ALSP — all drugs for ALSP →

Sponsor

Vigil Neuroscience, Inc. — full company profile →

Who can join

18 and older, any sex, with ALSP. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This is a multicenter, open-label study to assess the safety and tolerability of iluzanebart (also referred to as VGL101) in subjects with documentation of a gene mutation in the CSF1R gene for the treatment of adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) and to evaluate the effects of iluzanebart on imaging and biomarkers of disease progression in subjects with ALSP. Participants will receive infusions of iluzanebart approximately every 4 weeks for 1 year. The study includes a 52-week, open-label Core Study, followed by a Long-Term Extension (LTE), which provides subjects who complete the original 52-week study (Core Study) with the option to continue treatment for up to an additional 2 years.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Preclinical and first-in-human evaluation of AL002, a novel TREM2 agonistic antibody for Alzheimer's disease.
   Long H, Simmons A, Mayorga A, Burgess B, et al · · 2024 · cited 59× · PMID 39444037 · DOI 10.1186/s13195-024-01599-1
2. New insight on microglia activation in neurodegenerative diseases and therapeutics.
   Xu Y, Gao W, Sun Y, Wu M. · · 2023 · cited 22× · PMID 38188026 · DOI 10.3389/fnins.2023.1308345
3. <i>CSF1R</i>-Related Disorder: Prevalence of <i>CSF1R</i> Variants and Their Clinical Significance in the UK Population.
   Wade C, Runeckles K, Chataway J, Houlden H, et al · · 2024 · cited 15× · PMID 39040919 · DOI 10.1212/nxg.0000000000200179
4. Brain abnormalities, neurodegeneration, and dysosteosclerosis (BANDDOS): new cases, systematic literature review, and associations with CSF1R-ALSP.
   Dulski J, Souza J, Santos ML, Wszolek ZK. · · 2023 · cited 12× · PMID 37349768 · DOI 10.1186/s13023-023-02772-9
5. Genetic testing in adults with neurologic disorders: indications, approach, and clinical impacts.
   Dratch L, Azage M, Baldwin A, Johnson K, et al · · 2024 · cited 11× · PMID 37891417 · DOI 10.1007/s00415-023-12058-6
6. Rescue of in vitro models of CSF1R-related adult-onset leukodystrophy by iluzanebart: mechanisms and therapeutic implications of TREM2 agonism.
   Larson KC, Gergits FW, Renoux AJ, Weisman EJ, et al · · 2025 · cited 8× · PMID 39891235 · DOI 10.1186/s12974-025-03346-1
7. Hematopoietic Stem Cell Transplantation in an International Cohort of Colony Stimulating Factor-1 Receptor (CSF1R)-Related Disorder.
   Yska HAF, Golse M, Beerepoot S, Hayer S, et al · · 2025 · cited 6× · PMID 40646711 · DOI 10.1002/mds.30282
8. TREM2&lt;sup&gt;+&lt;/sup&gt; macrophages: a key role in disease development.
   Wang X, Wang Y, Yang L, Zhang Y, et al · · 2025 · cited 4× · PMID 40242752 · DOI 10.3389/fimmu.2025.1550893