Amyotrophic Lateral Sclerosis Clinical Trial
Official title:
Investigating Complex Neurodegenerative Disorders Related to Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
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.
Status | Recruiting |
Enrollment | 300 |
Est. completion date | October 30, 2025 |
Est. primary completion date | October 30, 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | - 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 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. - Patients with tattoos above the neck or permanent make up will be excluded from undergoing 7T MRI. |
Country | Name | City | State |
---|---|---|---|
United States | National Institutes of Health Clinical Center | Bethesda | Maryland |
Lead Sponsor | Collaborator |
---|---|
National Institute of Neurological Disorders and Stroke (NINDS) |
United States,
Renton AE, Majounie E, Waite A, Simon-Sanchez J, Rollinson S, Gibbs JR, Schymick JC, Laaksovirta H, van Swieten JC, Myllykangas L, Kalimo H, Paetau A, Abramzon Y, Remes AM, Kaganovich A, Scholz SW, Duckworth J, Ding J, Harmer DW, Hernandez DG, Johnson JO, Mok K, Ryten M, Trabzuni D, Guerreiro RJ, Orrell RW, Neal J, Murray A, Pearson J, Jansen IE, Sondervan D, Seelaar H, Blake D, Young K, Halliwell N, Callister JB, Toulson G, Richardson A, Gerhard A, Snowden J, Mann D, Neary D, Nalls MA, Peuralinna T, Jansson L, Isoviita VM, Kaivorinne AL, Holtta-Vuori M, Ikonen E, Sulkava R, Benatar M, Wuu J, Chio A, Restagno G, Borghero G, Sabatelli M; ITALSGEN Consortium; Heckerman D, Rogaeva E, Zinman L, Rothstein JD, Sendtner M, Drepper C, Eichler EE, Alkan C, Abdullaev Z, Pack SD, Dutra A, Pak E, Hardy J, Singleton A, Williams NM, Heutink P, Pickering-Brown S, Morris HR, Tienari PJ, Traynor BJ. A hexanucleotide repeat expansion in C9ORF72 is the cause of chromosome 9p21-linked ALS-FTD. Neuron. 2011 Oct 20;72(2):257-68. doi: 10.1016/j.neuron.2011.09.010. Epub 2011 Sep 21. — View Citation
Snowden JS, Adams J, Harris J, Thompson JC, Rollinson S, Richardson A, Jones M, Neary D, Mann DM, Pickering-Brown S. Distinct clinical and pathological phenotypes in frontotemporal dementia associated with MAPT, PGRN and C9orf72 mutations. Amyotroph Lateral Scler Frontotemporal Degener. 2015;16(7-8):497-505. doi: 10.3109/21678421.2015.1074700. Epub 2015 Oct 16. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Clinical information | Clinical information will be analyzed as part of our research to identify common features and differences among participants. | 10/30/2025 |
Status | Clinical Trial | Phase | |
---|---|---|---|
Terminated |
NCT04428775 -
A Safety and Biomarker Study of ALZT-OP1a in Subjects With Mild-Moderate ALS Disease
|
Phase 2 | |
Recruiting |
NCT04998305 -
TJ-68 Clinical Trial in Patients With Amyotrophic Lateral Sclerosis (ALS) and Muscle Cramps
|
Phase 1/Phase 2 | |
Recruiting |
NCT05951556 -
Telehealth Implementation of Brain-Computer Interface
|
N/A | |
Terminated |
NCT04579666 -
MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS)
|
Phase 2 | |
Recruiting |
NCT04082832 -
CuATSM Compared With Placebo for Treatment of ALS/MND
|
Phase 2/Phase 3 | |
Completed |
NCT01925196 -
Natural History and Biomarkers of Amyotrophic Lateral Sclerosis and Frontotemporal Dementia Caused by the C9ORF72 Gene Mutation
|
||
Completed |
NCT02496767 -
Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices After Treatment for a Year
|
Phase 3 | |
Recruiting |
NCT04816227 -
Expression Profile Study of Macrophages From Patients Affected by ALS or Other Related Motor Impairments
|
||
Active, not recruiting |
NCT04494256 -
A Study to Assess the Safety, Tolerability, and Effect on Disease Progression of BIIB105 in Participants With Amyotrophic Lateral Sclerosis (ALS) and Participants With the ALS Ataxin-2 (ATXN2) Genetic Mutation
|
Phase 1/Phase 2 | |
Completed |
NCT03706391 -
Study of ALS Reversals 4: LifeTime Exposures
|
||
Recruiting |
NCT04882904 -
Continuous Measurement of Activity in Patients With Muscle Pathology and in Control Subjects. ActiSLA Part.
|
N/A | |
Completed |
NCT04557410 -
Open Label Study: Treatment of ALS Fatigue With PolyMVA
|
Phase 1 | |
Active, not recruiting |
NCT04948645 -
A Phase 1 Study to Investigate the Safety and Pharmacokinetics of ABBV-CLS-7262 in Patients With Amyotrophic Lateral Sclerosis
|
Phase 1 | |
Not yet recruiting |
NCT04089696 -
Validation of the "ExSpiron©" in Patients With ALS
|
N/A | |
Not yet recruiting |
NCT05860244 -
Effect of Salbutamol on Walking Capacity in Ambulatory ALS Patients
|
Phase 2 | |
Not yet recruiting |
NCT06450691 -
Modeling Amyotrophic Lateral Sclerosis With Fibroblasts
|
N/A | |
Not yet recruiting |
NCT04220190 -
RAPA-501 Therapy for ALS
|
Phase 2/Phase 3 | |
Recruiting |
NCT02917681 -
Study of Two Intrathecal Doses of Autologous Mesenchymal Stem Cells for Amyotrophic Lateral Sclerosis
|
Phase 1/Phase 2 | |
Active, not recruiting |
NCT03067857 -
Autologous Bone Marrow-Derived Stem Cell Therapy for Motor Neuron Disease
|
Phase 1/Phase 2 | |
Recruiting |
NCT02874209 -
Noninvasive Assessment of Neuronal Damage by MRI Sodium ( 23Na ) in Amyotrophic Lateral Sclerosis
|
N/A |