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

Study Purpose:

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

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS, Primary Lateral Sclerosis (PLS), 

Study Type:

Observational Study

Study Category:

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Mary Kay Floeter, M.D., National Institute of Neurological Disorders and Stroke (NINDS)

Clinicaltrials.gov ID (11 digit #):

NCT03225144

Neals Affiliated?

No

Coordinating Center Contact Information


Carol H. Hoffman / .(JavaScript must be enabled to view this email address) / 301-451-1229
.(JavaScript must be enabled to view this email address) United States

Full Study 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

Study Sponsor:

National Institute of Neurological Disorders and Stroke (NINDS)

Participant Duration:

Estimated Enrollment:

200

Estimated Study Start Date:

09/19/2017

Estimated Study Completion Date:

01/01/2030

Posting Last Modified Date:

09/14/2017

Date Study Added to alsconsortium.org:

09/14/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    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.

  • Site Contact Information

    National Institutes of Health Clinical Center
    Robin Godwin, M.S.N. / .(JavaScript must be enabled to view this email address) / 301-451-6733
    Bethesda, Maryland 20892
    United States