This is the classic website, which will be retired eventually. Please visit the modernized ClinicalTrials.gov instead.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 8 for:    ALSP
Previous Study | Return to List | Next Study

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05020743
Recruitment Status : Recruiting
First Posted : August 25, 2021
Last Update Posted : January 3, 2024
Sponsor:
Information provided by (Responsible Party):
Vigil Neuroscience, Inc.

Study Description
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information
Brief Summary:
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.

Condition or disease Intervention/treatment
ALSP Other: No intervention

Study Design
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
Study Type : Observational
Estimated Enrollment : 60 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Natural History Study of Patients With Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia (ALSP)
Actual Study Start Date : September 13, 2021
Estimated Primary Completion Date : June 2027
Estimated Study Completion Date : June 2027

Resource links provided by the National Library of Medicine


Groups and Cohorts
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Group/Cohort Intervention/treatment
Patients with ALSP Other: No intervention
Not applicable for a Natural History Study
Patients with Prodromal ALSP Other: No intervention
Not applicable for a Natural History Study


Outcome Measures
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :
  1. Magnetic Resonance Imaging (MRI) [ Time Frame: Months 6, 12, 18, 24, 30, and 36 Months ]
    Change from Baseline in structural and volumetric MRI


Eligibility Criteria
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with Definitive ALSP and Prodromal ALSP.
Criteria

Key Inclusion Criteria for both Definitive ALSP patients and Prodromal ALSP patients:

1. Subjects who have documentation of a gene mutation in the CSF1R gene

Key Inclusion Criteria for Definitive ALSP patients, only:

  1. Subjects who fulfill both of the following criteria (a and b):

    a. More than two findings of clinical signs or symptoms in the following categories: i. Cognitive impairment or psychiatric problem ii. Pyramidal signs on neurological examination iii. Extrapyramidal signs, such as rigidity, tremor, abnormal gait, or bradykinesia iv. Epilepsy b. MRI findings consistent with ALSP: specifically, bilateral cerebral white matter lesions with or without thinning of the corpus callosum NOTE: Subjects with other causes of leukoencephalopathy, including vascular dementia, multiple sclerosis, or leukodystrophy (e.g., adrenoleukodystrophy, Krabbe disease, metachromatic leukodystrophy), will be excluded.

  2. Subjects who, in the investigator's opinion, have demonstrated clinical progression of their ALSP within the past year.
  3. Subjects who meet the criteria for definitive ALSP must have a designated caregiver who spends at least 4 hours per week with them. The caregiver must be able and willing to assist the subject in complying with the study requirements, be able to provide information during study visits, and be willing to sign a caregiver ICF.

Key Inclusion Criteria for Prodromal ALSP patients, only:

1. MRI findings consistent with ALSP: specifically, bilateral cerebral white matter lesions with or without thinning of the corpus callosum. Prodromal subjects may have none or up to and including 2 ALSP-related clinical signs or symptoms (i.e., they do not meet the clinical criteria outlined in the Definitive ALSP as "more than two").

Key Exclusion Criteria (for all study participants):

  1. Subjects with any neurological or psychiatric diseases that can produce cognitive, motor, or behavioral impairment similar to ALSP, including, but not limited to, Alzheimer's disease, frontotemporal dementia, ALS, stroke, Huntington disease, multiple sclerosis, Parkinson's disease, and Down syndrome, or with active alcohol/drug abuse
  2. Subjects who are unable to undergo MRI
  3. Subjects with any condition or situation that, in the opinion of the investigator or sponsor medical personnel, may place the subject at significant risk, confound the study results, or interfere significantly with the subject's participation in the study.
  4. Subjects who have previously undergone HSCT or plan to undergo HSCT within 12 months of the Screening/Baseline visit.
Contacts and Locations
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05020743


Contacts
Layout table for location contacts
Contact: Vigil Clinical Trials +1 857 254 4445 trials@vigilneuro.com

Locations
Layout table for location information
United States, California
Investigative Site 4 Recruiting
San Francisco, California, United States, 94158
Contact       trials@vigilneuro.com   
United States, Colorado
Investigative Site 5 Recruiting
Englewood, Colorado, United States, 80113
Contact       trials@vigilneuro.com   
United States, Florida
Investigative Site 1 Active, not recruiting
Boca Raton, Florida, United States, 33486
Investigative Site 2 Recruiting
Jacksonville, Florida, United States, 32224
Contact       trials@vigilneuro.com   
United States, Massachusetts
Investigative Site 11 Recruiting
Boston, Massachusetts, United States, 02114
Contact: trials@vigilneuro.com         
United States, Pennsylvania
Investigative Site 10 Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact       trials@vigilneuro.com   
Canada, Ontario
Investigative Site 3 Recruiting
London, Ontario, Canada, N6C 5J1
Contact       trials@vigilneuro.com   
Germany
Investigative Site 8 Recruiting
Leipzig, Germany, 04103
Contact       trials@vigilneuro.com   
Investigative Site 9 Recruiting
Tübingen, Germany, 72076
Contact       trials@vigilneuro.com   
Netherlands
Investigative Site 6 Recruiting
Amsterdam, Netherlands, 1105 AZ
Contact       trials@vigilneuro.com   
United Kingdom
Investigative Site 7 Recruiting
London, United Kingdom, WC1N 3BG
Contact       trials@vigilneuro.com   
Sponsors and Collaborators
Vigil Neuroscience, Inc.
More Information
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information
Layout table for additonal information
Responsible Party: Vigil Neuroscience, Inc.
ClinicalTrials.gov Identifier: NCT05020743    
Other Study ID Numbers: VGL101-01.002
First Posted: August 25, 2021    Key Record Dates
Last Update Posted: January 3, 2024
Last Verified: January 2024

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Vigil Neuroscience, Inc.:
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:
Layout table for MeSH terms
Leukoencephalopathies
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases