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Investigating Complex Neurodegenerative Disorders Related to Amyotrophic Lateral Sclerosis and Frontotemporal Dementia

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ClinicalTrials.gov Identifier: NCT03225144
Recruitment Status : Recruiting
First Posted : July 21, 2017
Last Update Posted : July 11, 2018
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute of Neurological Disorders and Stroke (NINDS) )

Brief Summary:

Objectives

The primary objective is to screen patients who are referred with a diagnosis of frontotemporal dementia, motor neuron disorder, or related adult-onset neurodegenerative disorder to assess patient eligibility for ongoing protocols. The secondary objective is to develop and maintain a registry of characterized patients who may be eligible for future trials. An exploratory objective is to obtain biospecimens from clinically characterized patients to carry out laboratory-based studies aimed at understanding the molecular pathways and genetic overlap between these neurodegenerative disorders.

Study population

Adults referred with clinical diagnoses of frontotemporal dementia, motor neuron disorder, or related adult-onset neurodegenerative disorder.

Design

Participants will all undergo diagnostic screening tests, a standard battery of tests to measure cognitive and motor function, and blood for clinical testing and research. Additional diagnostic and research testing, including magnetic resonance imaging, electromyography, neuropsychological testing, lumbar puncture, and skin biopsy will be obtained in selected patients.

Outcome measures

Patients will be screened for eligibility for current research protocol or entered into a registry future research protocols


Condition or disease
Frontotemporal Lobar Degeneration Amytrophic Lateral Sclerosis Progressive Supranuclear Palsy

Detailed Description:

Objectives

The primary objective is to screen patients who are referred with a diagnosis of frontotemporal dementia, motor neuron disorder, or related adult-onset neurodegenerative disorder to assess patient eligibility for ongoing protocols. The secondary objective is to develop and maintain a registry of characterized patients who may be eligible for future trials. An exploratory objective is to obtain biospecimens from clinically characterized patients to carry out laboratory-based studies aimed at understanding the molecular pathways and genetic overlap between these neurodegenerative disorders.

Study population

Adults referred with clinical diagnoses of frontotemporal dementia, motor neuron disorder, or related adult-onset neurodegenerative disorder.

Design

Participants will all undergo diagnostic screening tests, a standard battery of tests to measure cognitive and motor function, and blood for clinical testing and research. Additional diagnostic and research testing, including magnetic resonance imaging, electromyography, neuropsychological testing, lumbar puncture, and skin biopsy will be obtained in selected patients.

Outcome measures

Patients will be screened for eligibility for current research protocol or entered into a registry future research protocols


Study Type : Observational
Estimated Enrollment : 200 participants
Observational Model: Cohort
Time Perspective: Cross-Sectional
Official Title: Investigating Complex Neurodegenerative Disorders Related to Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
Actual Study Start Date : October 11, 2017
Estimated Primary Completion Date : June 30, 2022
Estimated Study Completion Date : June 30, 2022


Group/Cohort
Patients
patients who are referred with a diagnosis of frontotemporal dementia, motor neuron disorder, or related adult-onset neurodegenerative disorder to assess patient eligibility for ongoing protocols



Primary Outcome Measures :
  1. eligibility for research protocols [ Time Frame: ongoing ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
screen patients who are referred with a diagnosis of frontotemporal dementia, motor neuron disorder, or related adult-onset neurodegenerative disorder to assess patient eligibility for ongoing protocols
Criteria
  • INCLUSION CRITERIA:

Patients will be included if they

  • Are age 18 or older
  • Have been given a diagnosis by a neurologist of frontotemporal dementia, frontotemporal lobar degeneration, primary progressive aphasia, semantic dementia, motor neuron disorder, amyotrophic lateral sclerosis, primary lateral sclerosis, progressive bulbar palsy, corticobasal syndrome, Huntington disease or other related adult-onset neurodegenerative Disorder

EXCLUSION CRITERIA:

Patients will be excluded if they

  • have other major neurological or medical diseases that may cause progressive weakness or cognitive dysfunction, such as structural brain or spinal cord disease, metabolic diseases, paraneoplastic syndromes, infectious diseases, peripheral neuropathy or radiculopathy or other significant neurological abnormalities.
  • Have an unstable medical condition that, in the opinion of the investigators, makes participation unsafe
  • require daytime ventilator support at the time of study entry
  • are unable to travel to NIH
  • Patients with pacemakers or other implanted electrical devices, brain stimulators, dental implants, aneurysm clips (metal clips on the wall of a large artery), metallic prostheses (including metal pins and rods, heart valves, and cochlear implants), permanent eyeliner, implanted delivery pumps, or shrapnel fragments, metal fragments in the eye) will not be excluded but will not undergo magnetic resonance imaging.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03225144


Contacts
Contact: Carol H Hoffman (301) 451-1229 carol.hoffman@nih.gov

Locations
United States, Maryland
National Institutes of Health Clinical Center Recruiting
Bethesda, Maryland, United States, 20892
Contact: For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)    800-411-1222 ext TTY8664111010    prpl@cc.nih.gov   
Contact: Robin Godwin, M.S.N.    (301) 451-6733    robin.godwin@nih.gov   
Sponsors and Collaborators
National Institute of Neurological Disorders and Stroke (NINDS)
Investigators
Principal Investigator: Mary Kay Floeter, M.D. National Institute of Neurological Disorders and Stroke (NINDS)

Additional Information:
Responsible Party: National Institute of Neurological Disorders and Stroke (NINDS)
ClinicalTrials.gov Identifier: NCT03225144     History of Changes
Other Study ID Numbers: 170131
17-N-0131
First Posted: July 21, 2017    Key Record Dates
Last Update Posted: July 11, 2018
Last Verified: July 3, 2018

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by National Institutes of Health Clinical Center (CC) ( National Institute of Neurological Disorders and Stroke (NINDS) ):
Motor Neuron Disease
Frontotemporal Dementia
Corticobasal Syndrome

Additional relevant MeSH terms:
Frontotemporal Dementia
Pick Disease of the Brain
Sclerosis
Dementia
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Aphasia, Primary Progressive
Supranuclear Palsy, Progressive
Frontotemporal Lobar Degeneration
Neurodegenerative Diseases
Pathologic Processes
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurocognitive Disorders
Mental Disorders
Neuromuscular Diseases
Spinal Cord Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Aphasia
Speech Disorders
Language Disorders
Communication Disorders
Neurobehavioral Manifestations
Neurologic Manifestations
Signs and Symptoms
Basal Ganglia Diseases
Movement Disorders