TERMINATED

Natural History Study in Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) is a rare, rapidly progressing, genetic, neurodegenerative disease for which no definitive treatment options and limited information on the natural history of the disease are available. The structural, genetic, and neuropathophysiological abnormalities of ALSP lead to the onset of neurologic symptoms, such as moderate to severe motor and neuropsychiatric impairments. This natural history study will collect data to contribute to the development of future novel therapies that focus on the neuropathophysiological features that underlie ALSP and that are essential to reverse, delay, or stop progression of this debilitating disorder.

Official Title

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

Quick Facts

Study Start:2021-09-13
Study Completion:2025-05-30
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:TERMINATED

Study ID

NCT05020743

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. Age 18 years or older
  2. Willing and able to provide informed consent
  3. Able to understand and follow study procedures
  4. Stable medical condition
  1. Pregnancy or breastfeeding
  2. Severe psychiatric disorders
  3. Active substance abuse
  4. Unstable medical conditions
  5. Inability to comply with study requirements

Contacts and Locations

Study Locations (Sites)

Investigative Site 4
San Francisco, California, 94158
United States
Investigative Site 5
Englewood, Colorado, 80113
United States
Investigative Site 1
Boca Raton, Florida, 33486
United States
Investigative Site 2
Jacksonville, Florida, 32224
United States
Investigative Site 11
Boston, Massachusetts, 02114
United States
Investigative Site 10
Philadelphia, Pennsylvania, 19104
United States

Collaborators and Investigators

Sponsor: Vigil Neuroscience, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-09-13
Study Completion Date2025-05-30

Study Record Updates

Study Start Date2021-09-13
Study Completion Date2025-05-30

Terms related to this study

Keywords Provided by Researchers

  • ALSP
  • CSF1R
  • leukoencephalopathy
  • Hereditary Diffuse Leukoencephalopathy with Spheroids
  • HDLS
  • CSF1R-related Leukoencephalopathy
  • Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia
  • CSF1R gene mutation
  • POLD
  • Pigmentary Orthochromatic Leukodystrophy

Additional Relevant MeSH Terms

  • ALSP