Home Exercise for Individuals with Neurodegenerative Disease

NCT ID: NCT05443906

Last Updated: 2025-03-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

NA

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-02-13

Study Completion Date

2027-07-30

Brief Summary

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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

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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.

Conditions

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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

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Pre-test post-test design. Based on pre-test assessments participants will be instructed in an individualized exercise program. Participants will participate in 18 hours of remotely supervised and home practice exercises.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Home exercise

Individually designed home exercise program

Group Type EXPERIMENTAL

Home exercise

Intervention Type BEHAVIORAL

Exercise to address limitations impacting walking

Interventions

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Home exercise

Exercise to address limitations impacting walking

Intervention Type BEHAVIORAL

Eligibility Criteria

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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
Minimum Eligible Age

5 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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

OTHER

Sponsor Role lead

Responsible Party

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jennifer keller

Physical Therapist

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Jennifer Keller, PT

Role: PRINCIPAL_INVESTIGATOR

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

Locations

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Kennedy Krieger Institute

Baltimore, Maryland, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Jennifer L Keller, PT, MS

Role: CONTACT

443-923-2716

Amena Smith Fine, MD PhD

Role: CONTACT

443-923-3249

Facility Contacts

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Amena Smith Fine, MD, PhD

Role: primary

443-923-3249

Jennifer Keller, PT

Role: backup

4439232716

Other Identifiers

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JHSOM_IRB00309985

Identifier Type: -

Identifier Source: org_study_id

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