Home Exercise for Individuals with Neurodegenerative Disease

Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Status

Enrolling

Conditions

Leukoencephalopathy with Brainstem and Spinal Cord Involvement and Lactate Elevation

Leukoencephalopathy with Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder)

Adrenomyeloneuropathy

Ataxia

Neurodegenerative Diseases

LBSL

Cerebellar Ataxia

Leukodystrophy

Treatments

Behavioral: Home exercise

Study type

Interventional

Funder types

Other

Identifiers

NCT05443906

JHSOM_IRB00309985

Details and patient eligibility

About

The primary goal of this study is to address the need for targeted therapeutic interventions for impairments that impact walking in related neurodegenerative diseases.

Full description

This study will inform clinicians in quantitatively assessing baseline disease severity, selectively applying the appropriate exercise to the disease pathology, and measuring efficacy of the exercise intervention.

As part of this overall goal, the aims are to:

A. Determine feasibility of remotely supervised exercise programs. B. Optimize remote outcome measures for clinical monitoring and towards future clinical trials.

C. Measure changes in balance and walking post intervention.

Enrollment

30 estimated patients

Sex

All

Ages

5 to 75 years old

Volunteers

Accepts Healthy Volunteers

Inclusion criteria

The eligibility criteria for males is X-linked adrenoleukodystrophy as determined by biochemical determination or genetic testing.
The eligibility criteria for females is X-linked adrenoleukodystrophy as determined by biochemical determination, genetic testing, or pedigree analysis.
The Leukoenceophalopathy with brainstem and spinal cord involvement and lactate elevation inclusion criterion is a confirmed DARS2 mutation through genetic analysis.
For people with cerebellar ataxia, people with diagnoses of cerebellar damage from stroke, tumor or degeneration will be included. Those with a genetically confirmed cerebellar disorder will be asked to provide their genetic testing to note their particular type of ataxia.
We will also include patients with other neurodegenerative diseases similar to these disorders as determined by chart review and clinical exam.

Healthy Volunteers

Able to stand for 30 seconds without upper extremity support
Ambulatory (including use of a cane or a walker)
Able to walk for 2 minutes

Exclusion criteria

Other medical or psychological conditions which in the clinical judgement of the investigator would interfere with acquiring the study information or performing the exercises safely including but not limited to:

Uncontrolled hypertension, orthopedic conditions, diabetes, seizure disorder, peripheral vestibular loss, severe aphasia, dementia, pregnancy