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

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ClinicalTrials.gov Identifier: NCT05020743
Recruitment Status : Recruiting
First Posted : August 25, 2021
Last Update Posted : December 3, 2021
Sponsor:
Information provided by (Responsible Party):
Vigil Neuroscience, Inc.

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

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Study Type : Observational
Estimated Enrollment : 36 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 : November 2024
Estimated Study Completion Date : November 2024


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

Asymptomatic carriers of the CSF1R gene mutation Other: No intervention
Not applicable for a Natural History Study




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



Information from the National Library of Medicine

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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 ALSP and asymptomatic carriers of CSF1R gene mutations, the causative mutation for ALSP.
Criteria

Key Inclusion Criteria for both ALSP patients and asymptomatic carriers:

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

Key Inclusion Criteria for ALSP patients, only:

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

    a. One or more 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. Findings consistent with ALSP on MRI. 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 have a cognitive or motor impairment that can affect their ability to comply with study requirements 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 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 within 12 months prior to Screening/Baseline

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
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Contact: Angela Pontius +1 857 254 4445 trials@vigilneuro.com

Locations
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United States, Florida
Investigative Site 1 Recruiting
Boca Raton, Florida, United States, 33486
Contact       trials@vigilneuro.com   
Investigative Site 2 Recruiting
Jacksonville, Florida, United States, 32224
Canada, Ontario
Investigative Site 3 Recruiting
London, Ontario, Canada, N6C 5J1
Contact       trials@vigilneuro.com   
Sponsors and Collaborators
Vigil Neuroscience, Inc.
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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: December 3, 2021
Last Verified: November 2021

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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
Additional relevant MeSH terms:
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Leukoencephalopathies
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases