A Natural History Study of Patients with Adult-Onset | Clinical Research Studies Listing

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

Recruiting

18 years - 100 years

All

Phase N/A

50 participants needed

1 Location

Brief description of study

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.

Detailed description of study

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.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

Conditions: ALSP,leukoencephalopathy,axonal spheroids and pigmented glia
Age: 18 years - 100 years
Gender: All

Inclusion

For Definitive ALSP patients, only:

Subjects who fulfill both of the following criteria (1 and 2)

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
MRI findings consistent with ALSP: specifically, bilateral cerebral white matter lesions with or without thinning of the corpus callosum

Clinical progression of their ALSP within the past year
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

For Prodromal ALSP patients, only:

MRI findings consistent with ALSP. Prodromal subjects may have none or up to 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").

Exclusion

Any neurological or psychiatric diseases that can produce cognitive, motor, or behavioral impairment similar to ALSP
Unable to undergo MRI

Updated on 01 Aug 2024. Study ID: 21-0721

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