Home Exercise for Individuals with Neurodegenerative Disease | NCT05443906

Studies
Home Exercise for Individuals with Neurodegenerative Disease

NCT05443906

Sponsor

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

Status

Recruiting

Last Update Posted

Mar 10, 2025Actual

Enrollment

30Estimated

Phase

Not Applicable

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

Home exercise

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Status Module

Record Verification Date

Mar 2025

Overall Recruitment Status or Expanded Access Status

Recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Feb 13, 2023Actual

Primary Completion Date

Jul 30, 2026Estimated

Completion Date

Jul 30, 2027Estimated

First Submitted Date

May 11, 2022

First Submission Date that Met QC Criteria

Jul 1, 2022

First Posted Date

Jul 5, 2022Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Mar 6, 2025

Last Update Posted Date

Mar 10, 2025Actual

Description Module

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not Applicable

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

30Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Home exercise	Experimental	Individually designed home exercise program	Behavioral: Home exercise

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Home exercise	Behavioral	Exercise to address limitations impacting walking	Home exercise

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

Eligibility Module

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

Accepts Healthy Volunteers

Yes

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

5 Years

Maximum Age

75 Years

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Jennifer L Keller, PT, MS	Contact	443-923-2716		keller@kennedykrieger.org
Amena Smith Fine, MD PhD	Contact	443-923-3249		finea@kennedykrieger.org

Overall Officials

Name	Affiliation	Role
Jennifer Keller, PT	Hugo W. Moser Research Institute at Kennedy Krieger, Inc.	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Kennedy Krieger Institute	Recruiting	Baltimore	Maryland	21205	United States

ID	Term
D019636	Neurodegenerative Diseases
D001259	Ataxia
C567009	Leukoencephalopathy with Brainstem and Spinal Cord Involvement and Lactate Elevation
D002524	Cerebellar Ataxia
D000326	Adrenoleukodystrophy

Ancestor Terms

ID	Term
D009422	Nervous System Diseases
D020820	Dyskinesias
D009461	Neurologic Manifestations
D012816	Signs and Symptoms
D013568	Pathological Conditions, Signs and Symptoms
D002526	Cerebellar Diseases
D001927	Brain Diseases
D002493	Central Nervous System Diseases
D020739	Brain Diseases, Metabolic, Inborn
D001928	Brain Diseases, Metabolic
D020279	Hereditary Central Nervous System Demyelinating Diseases
D056784	Leukoencephalopathies
D003711	Demyelinating Diseases
D038901	X-Linked Intellectual Disability
D008607	Intellectual Disability
D019954	Neurobehavioral Manifestations
D040181	Genetic Diseases, X-Linked
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D020271	Heredodegenerative Disorders, Nervous System
D008661	Metabolism, Inborn Errors
D018901	Peroxisomal Disorders
D008659	Metabolic Diseases
D009750	Nutritional and Metabolic Diseases
D000309	Adrenal Insufficiency
D000307	Adrenal Gland Diseases
D004700	Endocrine System Diseases