Home Exercise for Individuals with Neurodegenerative Disease

Not Applicable

Recruiting

• Conditions: Neurodegenerative Diseases; Leukodystrophy; Ataxia; LBSL; Leukoencephalopathy with Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder); Leukoencephalopathy with Brainstem and Spinal Cord Involvement and Lactate Elevation; Cerebellar Ataxia; Adrenomyeloneuropathy
• Interventions: Behavioral: Home exercise

• Registration Number: NCT05443906
• Lead Sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Brief Summary

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

Detailed 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.

Study Timeline

• Study First Submitted: 2022-05-11
• Study First Submitted QC: 2022-07-01
• Study First Posted: 2022-07-05
• Study Start: 2023-02-13
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-06
• Last Update Posted: 2025-03-10
• Primary Completion: 2026-07-30
• Study Completion: 2027-07-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 30

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Home exercise	Home exercise	Individually designed home exercise program

Primary Outcome Measures

Name	Time	Method
Change in NeuroQOL lower extremity	baseline 12 weeks	Patient reported outcome

Trial Locations

Locations (1)

Kennedy Krieger Institute; 🇺🇸 Baltimore, Maryland, United States