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

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Brief Title

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

Official Title

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

Brief Summary

      Background:

      Neurodegenerative disorders can lead to problems in movement or memory. Some can cause
      abnormal proteins to build up in brain cells. Researchers want to understand whether these
      diseases have related causes or risk factors.

      Objective:

      To test people with movement or thinking and memory problems to see if they are eligible for
      research studies.

      Eligibility:

      People ages 18 and older with a neurodegenerative disorder associated with accumulation of
      TDP-43 or Tau proteins

      Design:

      Participants will have a screening visit. This may take place over 2-3 days. Tests include:

      Medical history

      Physical exam

      Questions about behavior and mood

      Tests of memory, attention, concentration, and thinking

      Movement measurement. The speed at which participants can stand up from a chair, tap their
      finger and foot, and walk a short distance will be measured. Some movements will be
      videotaped. They will be videotaped while they speak and read a paragraph.

      Blood tests. This might include genetic testing.

      Lung and breathing tests

      MRI. They will lie on a table that slides into a cylinder that takes pictures of the body.
      Some participants will get a dye through IV.

      Electromyography. A thin needle will be inserted into the muscles to measure electrical
      signals.

      Nerve tests. Small electrodes on the skin record muscle and nerve activity.

      A small piece of skin may be removed.

      A skin or blood sample may be taken to create stem cells.

      Optional lumbar puncture. A needle will be inserted into the space between the bones of the
      back to collect fluid.

      If participants are not eligible for current studies, they may be contacted in the future.
    

Detailed Description

      Objectives

      The primary objective is to evaluate patients referred with a diagnosis of frontotemporal
      dementia (FTD), amyotrophic lateral sclerosis (ALS), or related adult-onset neurodegenerative
      disorders to assess patient eligibility for ongoing protocols. The secondary objective is to
      develop and maintain a registry of characterized patients and presymptopmatic carriers of
      gene mutations that cause ALS FTD spectrum disorders. 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. Presymptomatic carriers of genes known to
      cause familial FTD or ALS.

      Design

      All participants will undergo clinical tests to confirm diagnoses and to stage disease
      severity, including a standard battery of tests to measure cognitive and motor function.
      Participants may opt-in for research procedures such as phlebotomy, skin biopsy, and

      lumbar puncture to obtain biospecimens for laboratory research, and magnetic resonance
      imaging or transcranial magnetic stimulation may be used to explore biomarkers of disease.

      Outcome measures

      Clinical information will be analyzed as part of our research to identify common features and
      differences among participants.
    


Study Type

Observational


Primary Outcome

Clinical information


Condition

Frontotemporal Dementia


Study Arms / Comparison Groups

 Patients
Description:  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

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

300

Start Date

October 11, 2017

Completion Date

October 30, 2025

Primary Completion Date

October 30, 2025

Eligibility 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, primary
             progressive aphasia, semantic dementia, motor neuron disorder, amyotrophic lateral
             sclerosis, progressive bulbar palsy, corticobasal syndrome, Huntington disease or
             other related adult-onset neurodegenerative disorder OR

          -  Carry a mutation in a gene that causes familial ALS or FTD

        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.
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Justin Y Kwan, M.D., (301) 451-1229, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT03225144

Organization ID

170131

Secondary IDs

17-N-0131

Responsible Party

Sponsor

Study Sponsor

National Institute of Neurological Disorders and Stroke (NINDS)


Study Sponsor

Justin Y Kwan, M.D., Principal Investigator, National Institute of Neurological Disorders and Stroke (NINDS)


Verification Date

September 3, 2021