Ongoing Clinical Trials for Leukoencephalopathy

Currently, there is 1 ongoing clinical trial investigating treatment for leukoencephalopathy, specifically focusing on a rare form called Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia (ALSP). This trial is being conducted across three European countries and is testing an investigational drug called VGL101 (also known as Iluzanebart).

Clinical trial locations

• France
  • Study on the Safety and Tolerability of VGL101 for Adults with Adult-Onset Leukoencephalopathy
• Germany
  • Study on the Safety and Tolerability of VGL101 for Adults with Adult-Onset Leukoencephalopathy
• Netherlands
  • Study on the Safety and Tolerability of VGL101 for Adults with Adult-Onset Leukoencephalopathy

Study on the Safety and Tolerability of VGL101 for Adults with Adult-Onset Leukoencephalopathy

This clinical trial is investigating VGL101 (also known as Iluzanebart), a medication delivered directly into the bloodstream through intravenous infusion. The study focuses on Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia (ALSP), a rare brain disease affecting the white matter, which is essential for transmitting signals in the brain. The condition causes degeneration of myelin (the protective covering of nerve fibers), leading to cognitive decline, movement problems, and behavioral changes.

Main inclusion criteria:

• Must be at least 18 years old
• Must have a documented gene mutation in the CSF1R gene, confirmed by blood test
• Must have at least two symptoms such as cognitive or mental health problems, neurological signs, movement difficulties, or epilepsy, along with specific MRI findings
• Must have shown signs of worsening condition in the past year
• Must score at least 14 on the Montreal Cognitive Assessment (MoCA), a test measuring thinking abilities
• Must be able to walk independently or with assistance (cane, crutches), or use a wheelchair independently
• Must have a body mass index (BMI) between 17.5 and 38.0
• Women who can become pregnant and men who can father children must use effective birth control during the study and for at least 3 months afterward
• Those with cognitive difficulties must have a study partner (caregiver, family member, or friend) to help follow study requirements

Main exclusion criteria:

• Patients with a history of other types of leukoencephalopathy
• Patients outside the specified adult age range
• Patients from vulnerable populations who may not be able to give informed consent

Study focus and timeline:

The primary goal is to evaluate how safe and tolerable VGL101 is for patients with ALSP. The study lasts approximately one year and includes several stages:

1. Initial visit: Participants undergo assessments including medical history review, physical examination, blood tests, and MRI scans to confirm eligibility
2. Baseline visit: Participants receive their first dose of VGL101 via intravenous infusion, with close monitoring during and after administration
3. Regular follow-up visits: Scheduled monitoring visits include blood tests, MRI scans, and other evaluations to track progress and changes in condition
4. Medication administration: VGL101 is given at specified intervals according to the study protocol
5. Safety and tolerability assessments: Continuous monitoring of side effects and adverse reactions throughout the study
6. End of study visit: Comprehensive evaluation of health and treatment effects, with discussion of results and any necessary follow-up care

Investigational drug:

VGL101 (Iluzanebart) is an investigational medication being tested for its potential to help people with ALSP. At the molecular level, it is believed to target specific pathways involved in the disease process, although the exact mechanism is still under investigation. Participants will receive either the active treatment or a placebo to help researchers understand the medication’s effects.

Summary

There is currently one clinical trial available for patients with leukoencephalopathy, specifically targeting the rare form known as ALSP. This trial is being conducted across three European countries: France, Germany, and the Netherlands, providing access to patients in these regions. The study focuses on VGL101, an investigational drug administered intravenously, and aims to gather important safety and tolerability data over a one-year period. The trial requires participants to have a confirmed genetic mutation and documented disease progression, with comprehensive monitoring throughout the study period to ensure patient safety and collect valuable information about this potential treatment.