Home Exercise for Individuals with Neurodegenerative Disease

Study of Adapted Exercise and Mindfulness Interventions to Improve Motor Function and Sleep Quality in Individuals with Neurodegenerative Disease

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

Study Overview

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

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 Type: Interventional
• Enrollment (Estimated): 30
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Amena Smith Fine, MD PhD; Phone Number: 443-923-3249; Email: finea@kennedykrieger.org
• Study Contact Backup: Name: Jennifer L Keller, PT, MS; Phone Number: 443-923-2716; Email: keller@kennedykrieger.org

Study Locations

• United States
  • Maryland
    • Baltimore, Maryland, United States, 21205
      • Recruiting
      • Kennedy Krieger Institute
      • Contact: Jennifer Keller, PT; Phone Number: 4439232716; Email: keller@kennedykrieger.org
      • Contact: Amena Smith Fine, MD, PhD; Phone Number: 443-923-3249; Email: finea@kennedykrieger.org

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years to 75 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: Yes

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Home exercise; Individually designed home exercise program	Behavioral: Home exercise; Exercise to address limitations impacting walking

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in NeuroQOL lower extremity	Patient reported outcome	baseline 12 weeks

Collaborators and Investigators

• Sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
• Investigators: Principal Investigator: Jennifer Keller, PT, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Publications and helpful links

General Publications

• Keller JL, Fritz N, Chiang CC, Jiang A, Thompson T, Cornet N, Newsome SD, Calabresi PA, Zackowski K. Adapted Resistance Training Improves Strength in Eight Weeks in Individuals with Multiple Sclerosis. J Vis Exp. 2016 Jan 29;(107):e53449. doi: 10.3791/53449.

Study record dates

Study Major Dates

• Study Start (Actual): February 13, 2023
• Primary Completion (Estimated): July 30, 2026
• Study Completion (Estimated): July 30, 2027

Study Registration Dates

• First Submitted: May 11, 2022
• First Submitted That Met QC Criteria: July 1, 2022
• First Posted (Actual): July 5, 2022

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: March 6, 2025
• Last Verified: March 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neurologic Manifestations; Endocrine System Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Neurobehavioral Manifestations; Demyelinating Diseases; Heredodegenerative Disorders, Nervous System; Adrenal Gland Diseases; Mental Retardation, X-Linked; Intellectual Disability; Genetic Diseases, X-Linked; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Dyskinesias; Hereditary Central Nervous System Demyelinating Diseases; Cerebellar Diseases; Adrenal Insufficiency; Peroxisomal Disorders; Neurodegenerative Diseases; Leukoencephalopathies; Ataxia; Cerebellar Ataxia; Adrenoleukodystrophy
• Other Study ID Numbers: JHSOM_IRB00309985

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No