Clinical trial NCT05443906: Home Exercise for Individuals with Neurodegenerative Disease
Official title: Study of Adapted Exercise and Mindfulness Interventions to Improve Motor Function and Sleep Quality in Individuals with Neurodegenerative Disease
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
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
Lead sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Sponsor class: OTHER
First posted: 2022-07-05
Enrollment: 30
Sex: ALL
Minimum age (years): 5
Maximum age (years): 75
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: 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 purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Change in NeuroQOL lower extremity
timeFrame: baseline 12 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Baltimore
country: United States
facility: Kennedy Krieger Institute
state: Maryland
Study officials:
affiliation: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
name: Jennifer Keller, PT
role: PRINCIPAL_INVESTIGATOR
References: