Investigating Complex Neurodegenerative Disorders Related to Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03225144|
Recruitment Status : Recruiting
First Posted : July 21, 2017
Last Update Posted : July 27, 2020
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.
To test people with movement or thinking and memory problems to see if they are eligible for research studies.
People ages 18 and older with a neurodegenerative disorder associated with accumulation of TDP-43 or Tau proteins
Participants will have a screening visit. This may take place over 2-3 days. Tests include:
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.
|Condition or disease|
|Frontotemporal Lobar Degeneration Amytrophic Lateral Sclerosis Progressive Supranuclear Palsy|
The primary objective is to evaluate patients referred with a diagnosis of frontotemporal dementia (FTD), amyotrophic lateral sclerosis (ALS), 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 and presymptopmatic carriers of gene mutations that cause ALS or FTD. 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.
Adults referred with clinical diagnoses of frontotemporal dementia, motor neuron disorder, or related adult-onset neurodegenerative disorder. Presymptomatic carriers of genes that cause familial FTD or ALS.
Participants will all 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.
Clinical information will be analyzed as part of our research to identify common features and differences among participants.
|Study Type :||Observational|
|Estimated Enrollment :||200 participants|
|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 :||October 30, 2025|
|Estimated Study Completion Date :||October 30, 2025|
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
- eligibility for protocols [ Time Frame: 10/30/2025 ]The determination of whether patients have a diagnosis that is eligible for inclusion in other protocols is the only outcome.
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
|Contact: Carol H Hoffman||(301) firstname.lastname@example.org|
|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 email@example.com|
|Contact: Jennifer Farren (301) 496-5526 firstname.lastname@example.org|
|Principal Investigator:||Justin Y Kwan, M.D.||National Institute of Neurological Disorders and Stroke (NINDS)|