Find ALS Research Opportunities

Background: Amyotrophic lateral sclerosis type 4 (ALS4) is an inherited motor neuron disease. People with ALS4 have a change in the amount of RNA and DNA that bind together. This binding of RNA with DNA forms units called R-loops. Researchers want to learn how R-loops are related to ALS4. To do this, they will study people with inherited neurological conditions that may affect R-loop levels. These include ALS4, progressive external opthalmoplegia with mitochondrial deletions (PEOB2), Aicardi-Goutieres syndrome (AGS), and ataxia and oculomotor apraxia type 2 (AOA2). Objective: To learn how the binding of RNA with DNA (R-loops) is related to neurological disease. Eligibility: People age 5 and older with ALS4, PEOB2, AGS, and AOA2. Healthy relatives and nonrelatives are also needed. Design: Participants may be screened with a review of x-rays and other medical records. Healthy relative and nonrelative participants will have 1 visit. All other participants will have 4 visits over 3 years. At visits, participants will undergo some or all of the following: Medical history Physical exam Tests of muscle strength and volume and physical function Blood tests Pregnancy test (for some females) Skin biopsy of forearm Magnetic resonance imaging (MRI) Dual x-ray absorptiometry (DEXA). Some tests are optional. The MRI uses a magnetic field and radio waves to take pictures. Participants will lie on a table that slides in and out of the scanner. The scanner makes noise. They will get earplugs. The DEXA scan uses x-rays to take pictures. MRI and DEXA will be used to measure muscle, fat, and lean body mass. ...

Registration

ClinicalTrials.gov

Study category

Observational, Biomarker, Genetic, Presymptomatic, Natural history, Biospecimen, Imaging

Study type

Observational study

Sponsor / center

National Institute of Neurological Disorders and Stroke (NINDS)

Intervention(s)

Not stated in the official study record.

Gene / variant

Any / Not gene-specific

Disease status

Presymptomatic gene carrier, Healthy control, Diagnosed ALS

Age

5 Years to 120 Years

Disease duration

Not stated in the official study record.

Genetic-testing requirement

Not stated in the official study record.

Research center

Not stated in the official study record.

Locations

1

States available

Maryland

Remote participation

Not stated in the official study record.

Last ClinicalTrials.gov update

09-30-2026

RAPA-501-ALS is a phase 2/3 expansion cohort study of RAPA-501 autologous hybrid TREG/Th2 cells in patients living with amyotrophic lateral sclerosis (pwALS).

Registration

ClinicalTrials.gov

Study category

Interventional, Drug trial

Study type

Interventional study

Sponsor / center

Rapa Therapeutics LLC

Intervention(s)

RAPA-501 Autologous T stem cells

Gene / variant

Any / Not gene-specific

Disease status

Familial / genetic ALS, Diagnosed ALS

Age

18 Years to Not stated in the official study record.

Disease duration

≤ 12 months

Genetic-testing requirement

Not stated in the official study record.

Research center

Not stated in the official study record.

Locations

1

States available

Massachusetts

Remote participation

Partial / hybrid

Last ClinicalTrials.gov update

09-29-2026

The goal of this observational study is to develop new ways to detect early changes associated with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease. The study will examine whether changes in breathing, magnetic resonance imaging (MRI) of the brainstem and spinal cord, and blood markers can identify early signs of ALS before major physical changes occur. The main questions the study aims to answer are: * Can changes in automatic breathing help identify early ALS-related changes? * Can breathing measures distinguish people with ALS from people without ALS? * Do people with genetic risk for ALS who show changes in breathing also have changes in the brainstem and spinal cord or higher levels of blood markers associated with nerve damage? Participants will complete breathing and muscle function tests, an MRI scan of the brainstem and spinal cord, and blood testing. The study will include people with ALS, people with genetic risk for ALS who do not yet have symptoms, and healthy adults for comparison.

Registration

ClinicalTrials.gov

Study category

Observational, Biomarker, Genetic, Presymptomatic, Imaging

Study type

Observational study

Sponsor / center

Thomas Jefferson University

Intervention(s)

Not stated in the official study record.

Gene / variant

C9orf72

Disease status

Presymptomatic gene carrier, Healthy control, Diagnosed ALS

Age

18 Years to Not stated in the official study record.

Disease duration

Not stated in the official study record.

Genetic-testing requirement

Not stated in the official study record.

Research center

Not stated in the official study record.

Locations

1

States available

Pennsylvania

Remote participation

Not stated in the official study record.

Last ClinicalTrials.gov update

09-28-2026

Background: \- Electrical impedance myography (EIM) is a new technique being studied to see if it is helpful in evaluating muscle disorders and nerve disorders. EIM looks at how a mild, painless electrical current travels through muscles. Researchers want to gain experience in using the EIM device. They will collect information on the results of using it on people with and without nerve and muscle diseases, and compare that with information from other standard tests. First, they will test the device on healthy people. Then they will test people with a variety of neuromuscular diseases. Because the test is noninvasive and not painful, researchers will test both children and adults. Objectives: \- To gain experience using the EIM muscle testing device. Eligibility: * Healthy volunteers at least 2 years old. * Individuals at least 2 years old who have neuromuscular disease. Design: * Participants will be screened with a medical history and physical exam. * Participants will have one 2-3 hour clinic visit. Researchers may request follow-up visits. * Participants will be tested with the EIM device. The device and small electrodes will be placed on their skin. An electric current will pass through the device, but the participants will not feel this. * Participants may have an ultrasound test. A gel will be put on their skin, and a device will be moved over the skin. * Participants may have a nerve test. Electrodes will be placed on their skin, and they will feel a small shock. * Participants may have a test where a thin needle is inserted in their muscle.

Registration

ClinicalTrials.gov

Study category

Interventional, Device, Genetic, Imaging

Study type

Interventional study

Sponsor / center

National Institute of Neurological Disorders and Stroke (NINDS)

Intervention(s)

EIM testing, Nerve & muscle ultrasonography, Nerve conduction studies, Electromyography (EMG)

Gene / variant

Any / Not gene-specific

Disease status

Healthy control, Diagnosed ALS

Age

2 Years to 110 Years

Disease duration

Not stated in the official study record.

Genetic-testing requirement

Not stated in the official study record.

Research center

Not stated in the official study record.

Locations

1

States available

Maryland

Remote participation

Not stated in the official study record.

Last ClinicalTrials.gov update

09-28-2026

This study is designed to learn more about the natural history of inherited neurological disorders and the role of heredity in their development. It will examine the genetics, symptoms, disease progression, treatment, and psychological and behavioral impact of diseases in the following categories: hereditary peripheral neuropathies; hereditary myopathies; muscular dystrophies; hereditary motor neuron disorders; mitochondrial myopathies; hereditary neurocognitive disorders; inherited neurological disorders without known diagnosis; and others. Many of these diseases, which affect the brain, spinal cord, muscles, and nerves, are rare and poorly understood. Children and adults of all ages with various inherited neurological disorders may be eligible for this study. Participants will undergo a detailed medical and family history, and a family tree will be drawn. They will also have a physical and neurological examination that may include blood test and urine tests, an EEG (brain wave recordings), psychological tests, and speech and language and rehabilitation evaluations. A blood sample or skin biopsy may be taken for genetic testing. Depending on the individual patient's symptoms, imaging tests such as X-rays, CT or MRI scans and muscle and nerve testing may also be done. Information from this study may provide a better understanding of the genetic underpinnings of these disorders, contributing to improved diagnosis, treatment, and genetic counseling, and perhaps leading to additional studies in these areas.

Registration

ClinicalTrials.gov

Study category

Observational, Genetic, Presymptomatic, Natural history, Biospecimen, Imaging

Study type

Observational study

Sponsor / center

National Institute of Neurological Disorders and Stroke (NINDS)

Intervention(s)

Not stated in the official study record.

Gene / variant

Other specified ALS gene

Disease status

Presymptomatic gene carrier, Diagnosed ALS

Age

2 Years to 120 Years

Disease duration

Not stated in the official study record.

Genetic-testing requirement

Not stated in the official study record.

Research center

Not stated in the official study record.

Locations

2

States available

Maryland

Remote participation

Not stated in the official study record.

Last ClinicalTrials.gov update

09-28-2026

The purpose of this 2-part study is to assess the safety and efficacy of CORT113176 (dazucorilant) in patients with Amyotrophic Lateral Sclerosis (ALS).

Registration

ClinicalTrials.gov

Study category

Interventional, Drug trial, Genetic

Study type

Interventional study

Sponsor / center

Corcept Therapeutics

Intervention(s)

Dazucorilant 300 mg, Dazucorilant 150 mg, Placebo, Dazucorilant

Gene / variant

SOD1

Disease status

Familial / genetic ALS, Diagnosed ALS

Age

18 Years to Not stated in the official study record.

Disease duration

Not stated in the official study record.

Genetic-testing requirement

Yes

Research center

Not stated in the official study record.

Locations

35

States available

Arizona, California, New Jersey, New York

Remote participation

Not stated in the official study record.

Last ClinicalTrials.gov update

09-24-2026

This study is testing whether giving people with ALS specially matched immune cells called B cells from a related donor is safe. Up to 10 participants will each receive two B cell infusions at least 60 days apart. The main goals are to watch closely for side effects, see whether the participant's immune system rejects the donor cells, and determine whether this treatment should be studied further in people with ALS. The first three participants will receive the infusions in the hospital with extra safety spacing between participants; if those infusions appear safe, the remaining participants may receive them in an outpatient setting.

Registration

ClinicalTrials.gov

Study category

Interventional, Drug trial

Study type

Interventional study

Sponsor / center

Mark Poznansky

Intervention(s)

B cell infusion

Gene / variant

Any / Not gene-specific

Disease status

Diagnosed ALS

Age

18 Years to 70 Years

Disease duration

> 36 months accepted

Genetic-testing requirement

Not stated in the official study record.

Research center

Not stated in the official study record.

Locations

1

States available

Massachusetts

Remote participation

Not stated in the official study record.

Last ClinicalTrials.gov update

09-24-2026

According to the World Health Organization (WHO) definition, a rare disease is a disease affecting fewer than 6.5 people per 10,000; in China, it generally refers to diseases with a prevalence of less than 1/10,000 (i.e., with a total of no more than 140,000 patients). Such diseases are mostly hereditary or congenital in origin. Patients with rare diseases often present with complex symptoms, most of which are neurological. Although a large number of rare diseases have been identified, the small number of patients with each disease and the diversity of symptoms make early diagnosis difficult and render large-sample clinical trials challenging. Consequently, most rare diseases lack curative treatments; available therapies are of limited efficacy or prohibitive cost, ultimately leading to severe disability or death and imposing a substantial socioeconomic burden. This study establishes a registry for motor neuron diseases (MND), spinocerebellar ataxias (SCAs), hereditary muscular dystrophy (HMD), and hereditary spastic paraplegia (HSP). Owing to the low incidence, complex clinical diagnosis, unclear pathogenic mechanisms, and strong genetic heterogeneity, epidemiological data on rare motor neuron diseases are limited, further increasing the difficulty of biomarker screening and targeted therapeutic development. Therefore, there is an urgent need to establish a comprehensive registry and to obtain reliable evidence on risk factors and early diagnosis through research. This study aims to establish a registry of rare neurological diseases in the southern Anhui Province and to build a high-quality biobank of human biological resources.

Registration

ClinicalTrials.gov

Study category

Observational, Biomarker, Genetic, Biospecimen, ALS/FTD

Study type

Observational study

Sponsor / center

First Affiliated Hospital of Wannan Medical College

Intervention(s)

Not stated in the official study record.

Gene / variant

Any / Not gene-specific

Disease status

ALS-FTD where applicable, Diagnosed ALS

Age

Not stated in the official study record. to Not stated in the official study record.

Disease duration

Not stated in the official study record.

Genetic-testing requirement

Not stated in the official study record.

Research center

Not stated in the official study record.

Locations

1

States available

Not stated in the official study record.

Remote participation

Not stated in the official study record.

Last ClinicalTrials.gov update

09-24-2026

The primary objective of this dose-finding study is to evaluate the safety, tolerability and pharmacodynamics of single dose of INS1202 via IT administration in participants ≥ 18 to \<80 years of age with ALS who carry superoxide dismutase type 1 (SOD1) mutations or harbor no known ALS-related genetic mutation.

Registration

ClinicalTrials.gov

Study category

Interventional, Drug trial, Genetic

Study type

Interventional study

Sponsor / center

Insmed Gene Therapy LLC

Intervention(s)

INS1202

Gene / variant

SOD1

Disease status

Familial / genetic ALS, Sporadic ALS, Diagnosed ALS

Age

18 Years to 79 Years

Disease duration

> 36 months accepted

Genetic-testing requirement

Not stated in the official study record.

Research center

Not stated in the official study record.

Locations

7

States available

California, Maryland, Massachusetts, Missouri, Ohio, Pennsylvania

Remote participation

Not stated in the official study record.

Last ClinicalTrials.gov update

09-23-2026

The investigators will develop and evaluate controllers for a powered neck exoskeleton in patients with amyotrophic lateral sclerosis (ALS) to understand how to allow adequate personalization and function and achieves satisfaction from users. In this small device feasibility study, participants will be enrolled from the ALS clinic at the University of Utah Hospital. After obtaining written consent, participants will perform tasks while using the neck exoskeleton with the controllers. Tasks include computerized tracking tasks and simulated activities of daily living. Breaks will be added between tasks to avoid fatigue. Head-neck kinematics, gaze data, controller preferences, and survey responses will be recorded.

Registration

ClinicalTrials.gov

Study category

Interventional, Device, Prevention

Study type

Interventional study

Sponsor / center

University of Utah

Intervention(s)

neck exoskeleton controller

Gene / variant

Any / Not gene-specific

Disease status

Diagnosed ALS

Age

18 Years to Not stated in the official study record.

Disease duration

Not stated in the official study record.

Genetic-testing requirement

Not stated in the official study record.

Research center

Not stated in the official study record.

Locations

1

States available

Utah

Remote participation

Not stated in the official study record.

Last ClinicalTrials.gov update

09-23-2026

Study OLMP is a master protocol that will support a collection of individual sub studies that share key design components. Participants from the originator study OWAA (NCT07100119) will be assigned to the appropriate study treatment group: Sporadic Amyotrophic Lateral Sclerosis OL01 (NCT07571174). The studies aim to evaluate the safety and tolerability of different treatments in participants with Amyotrophic Lateral Sclerosis (ALS) that will last at least 96 weeks.

Registration

ClinicalTrials.gov

Study category

Interventional, Drug trial

Study type

Interventional study

Sponsor / center

Eli Lilly and Company

Intervention(s)

LY4256984

Gene / variant

Any / Not gene-specific

Disease status

Diagnosed ALS

Age

18 Years to 80 Years

Disease duration

Not stated in the official study record.

Genetic-testing requirement

Not stated in the official study record.

Research center

Not stated in the official study record.

Locations

11

States available

Not stated in the official study record.

Remote participation

Not stated in the official study record.

Last ClinicalTrials.gov update

09-22-2026

ALT001 is an allogeneic unrelated human umbilical cord mesenchymal stem cell-derived protein complex under investigation for the treatment of amyotrophic lateral sclerosis (ALS). This is a multicenter, randomized, double-blind, placebo-controlled, Phase 2 dose-ranging study in patients with ALS. Approximately 150 participants will be randomized 1:1:1 to receive ALT001 1.0 μg/kg, ALT001 2.0 μg/kg, or matching placebo by intravenous infusion during the 24-week double-blind (DB) treatment period. During the OLE period, the active treatment groups will continue to receive their original dose under blinded conditions, whereas the placebo group will undergo re-randomization in a 1:1 ratio to receive 1.0 or 2.0 μg/kg ALT001. The primary objective is to evaluate the dose-response relationship and efficacy of ALT001 at 1.0 and 2.0 μg/kg and to determine the recommended dose for subsequent studies. Secondary objectives include assessment of biomarkers related to efficacy and exploration of changes in cytokines, immune function, proteomics, and immunogenicity (anti-drug antibodies, ADA), as well as evaluation of the safety of ALT001.

Registration

ClinicalTrials.gov

Study category

Interventional, Drug trial, Biomarker

Study type

Interventional study

Sponsor / center

Darwin Origin (Hubei) Biopharmaceutical Co., Ltd.

Intervention(s)

ALT001 1.0 μg/kg, ALT001 2.0 μg/kg, Placebo

Gene / variant

Other specified ALS gene

Disease status

Sporadic ALS, Diagnosed ALS

Age

18 Years to 75 Years

Disease duration

≤ 12 months

Genetic-testing requirement

Not stated in the official study record.

Research center

Not stated in the official study record.

Locations

1

States available

Not stated in the official study record.

Remote participation

Not stated in the official study record.

Last ClinicalTrials.gov update

09-22-2026

The goal of this clinical trial is to learn if the drug pridopidine works to treat amyotrophic lateral sclerosis in adults. It will also help to learn about the safety of pridopidine. The main question it aims to answer is: Does pridopidine slow disease progression of ALS? Researchers will compare pridopidine to a placebo (a look-alike substance that contains no drug) to see if pridopidine works to treat ALS. Participants will: Take pridopidine or a placebo by mouth every day for 48 weeks. Afterwards, all participants will take pridopidine for another 48 weeks. Visit the clinic once every 1-3 months for checkups and tests

Registration

ClinicalTrials.gov

Study category

Interventional, Drug trial, Genetic, Presymptomatic

Study type

Interventional study

Sponsor / center

Prilenia

Intervention(s)

Pridopidine, Placebo

Gene / variant

C9orf72, SOD1

Disease status

Presymptomatic gene carrier, Diagnosed ALS

Age

18 Years to 80 Years

Disease duration

≤ 18 months

Genetic-testing requirement

Not stated in the official study record.

Research center

Emory ALS Center

Locations

56

States available

Arizona, California, Colorado, Florida, Georgia, Illinois, Kansas, Maryland, Massachusetts, Missouri, Nebraska, New York, Pennsylvania, Texas, Washington

Remote participation

Not stated in the official study record.

Last ClinicalTrials.gov update

09-22-2026

Center-specific information: Emory lists oral medication, vital capacity greater than 60%, disease onset fewer than 18 months before screening, ability to swallow oral medication, and a 48-week double-blind period with an optional open-label extension.

The main purpose of this study is to assess the long-term safety and tolerability of LY4256984 in participants with Amyotrophic Lateral Sclerosis (ALS). This study is a long-term extension of study J6I-MC-OWAA (NCT07100119) and is part of the OLMP (NCT07571200) master protocol that will last approximately 96 weeks.

Registration

ClinicalTrials.gov

Study category

Interventional, Drug trial

Study type

Interventional study

Sponsor / center

Eli Lilly and Company

Intervention(s)

LY4256984

Gene / variant

Any / Not gene-specific

Disease status

Diagnosed ALS

Age

18 Years to 80 Years

Disease duration

Not stated in the official study record.

Genetic-testing requirement

Not stated in the official study record.

Research center

Not stated in the official study record.

Locations

11

States available

Not stated in the official study record.

Remote participation

Not stated in the official study record.

Last ClinicalTrials.gov update

09-22-2026

The goal of this clinical trial is to learn if drug CB03-154 works to treat ALS in adults. It will also learn about the safety of drug CB03-154. The main questions it aims to answer are: * Does drug CB03-154 have an effect on delaying disease progression, improving function, and prolonging survival in adult ALS patients? * What medical problems do patients have when taking drug CB03-154? Researchers will compare drug CB03-154 to a placebo (a look-alike substance that contains no drug) to see if drug CB03-154 works to treat ALS. Participants (adult ALS patients) will: * Take drug CB03-154 or a placebo every day for 39 weeks (an additional 39 weeks would be required if entering the open-label extension phase). * Visit the clinic approximately every 2-3 months for checkups and tests, and there is also telephone follow-up in between. * Keep a diary of daily medication (CB03-154 or other concomitant medications), and if there are any unplanned medications, the reason (disease or symptoms) also need be recorded.

Registration

ClinicalTrials.gov

Study category

Interventional, Drug trial, ALS/FTD, Imaging

Study type

Interventional study

Sponsor / center

Shanghai Zhimeng Biopharma, Inc.

Intervention(s)

Test drug CB03-154 5mg group, Test drug CB03-154 10mg group, Test drug CB03-154 15mg group, Placebo Group

Gene / variant

Other specified ALS gene

Disease status

ALS-FTD where applicable, Diagnosed ALS

Age

18 Years to 65 Years

Disease duration

≤ 12 months

Genetic-testing requirement

Not stated in the official study record.

Research center

Not stated in the official study record.

Locations

1

States available

Not stated in the official study record.

Remote participation

Not stated in the official study record.

Last ClinicalTrials.gov update

09-22-2026

The goal of this clinical trial is to learn whether cyclophosphamide (CTX) combined with standard treatment can slow disease progression in adults with amyotrophic lateral sclerosis (ALS). It will also evaluate the safety and tolerability of CTX. The main questions it aims to answer are: 1. Does CTX combined with standard treatment reduce the decline in ALS Functional Rating Scale-Revised (ALSFRS-R) scores over 48 weeks compared with standard treatment alone? 2. What medical problems and side effects do participants have while receiving CTX? 3. Are markers of neuroinflammation and immune activity, including TSPO-PET, neurofilament light chain (NfL), upper motor neuron burden, electrophysiological measures, immune cell profiles, and autoantibodies, associated with treatment response? Researchers will compare CTX combined with standard treatment with standard treatment alone to see whether CTX can slow the progression of ALS. Participants will: 1. Be randomly assigned to receive CTX plus standard treatment or standard treatment alone 2. Receive CTX treatment for up to 36 weeks if assigned to the CTX group 3. Visit the study center regularly for clinical assessments, blood tests, lung function tests, electrophysiological tests, and other safety evaluations 4. Complete assessments of physical function, muscle strength, respiratory function, and quality of life 5. Undergo biomarker assessments, including TSPO-PET imaging and NfL testing 6. Be followed for 48 weeks during the main study period and for up to 96 weeks for long-term outcomes and safety

Registration

ClinicalTrials.gov

Study category

Interventional, Drug trial, Biomarker, Genetic, Imaging

Study type

Interventional study

Sponsor / center

Huashan Hospital

Intervention(s)

Cyclophosphamide, Riluzole

Gene / variant

SOD1

Disease status

Familial / genetic ALS, Diagnosed ALS

Age

18 Years to 75 Years

Disease duration

≤ 24 months

Genetic-testing requirement

Not stated in the official study record.

Research center

Not stated in the official study record.

Locations

0

States available

Not stated in the official study record.

Remote participation

Not stated in the official study record.

Last ClinicalTrials.gov update

09-22-2026

Amyotrophic lateral sclerosis (ALS) is a serious rapidly progressive disease of the nervous system. The average survival from the time of diagnosis is two to three years. The patient physical and psychological sufferings in ALS are immense, and apart from Riluzole, there is no effective treatment. Care of advanced ALS have an estimated cost of 4-8 million NOK per year. Perhaps the most challenging topic of ALS care is the decision to extend ventilation support into the stages of disease that require treatment both during day and night. In these cases, treatment is clearly life-sustaining and although quality of life may be maintained, the burden of caregiving imposed upon family or health care workers is huge, regardless of tracheostomy (TIV) or non-invasive (NIV) modality. The present study is a longitudinal questionnaire study in Norway measuring overall quality of life, health-related quality of life, and disease-specific quality of life in ALS patients, partners and children before and after the introduction of life sustaining ventilation support. The investigators aim to increase the knowledge on how life-sustaining ventilation support with NIV or TIV affects the quality of life in ALS patients, life partners and children. The results from the study may provide crucial information for clinicians and patients on one of the most difficult ethical issues of ALS treatment. The investigators anticipate that this information will facilitate a shared decision making processes, weighing benefits and disadvantages in a wider perspective.

Registration

ClinicalTrials.gov

Study category

Interventional, Device

Study type

Interventional study

Sponsor / center

Haukeland University Hospital

Intervention(s)

Long term mechanical ventilation support, No long term mechanical ventilation support

Gene / variant

Any / Not gene-specific

Disease status

Diagnosed ALS

Age

8 Years to Not stated in the official study record.

Disease duration

Not stated in the official study record.

Genetic-testing requirement

Not stated in the official study record.

Research center

Not stated in the official study record.

Locations

9

States available

Not stated in the official study record.

Remote participation

Not stated in the official study record.

Last ClinicalTrials.gov update

09-21-2026

The primary objective is to demonstrate, in a population of chronic neuromuscular disease the non-inferiority of a rehabilitation treatment integrated with robotic and/or technological devices compared to traditional rehabilitation treatment in the level of fatigue. The main question it aims to answer is: Are high-tech rehabilitation interventions, including robotic systems, virtual reality, and stabilometric platforms, not inferior to traditional rehabilitation methods in improving balance, motor function, fatigue levels, sarcopenia, cognitive engagement, and overall quality of life in patients with chronic neuromuscular diseases (NMDs)? Researchers will compare a robotic treatment group, that consists in an high-tech rehabilitation, with a control group, that will receive the traditional rehabilitative treatment.

Registration

ClinicalTrials.gov

Study category

Interventional, Drug trial, Biomarker

Study type

Interventional study

Sponsor / center

Istituti Clinici Scientifici Maugeri SpA

Intervention(s)

High-tech rehabilitative treatment, Rehabilitation

Gene / variant

Any / Not gene-specific

Disease status

Diagnosed ALS

Age

18 Years to 80 Years

Disease duration

Not stated in the official study record.

Genetic-testing requirement

Not stated in the official study record.

Research center

Not stated in the official study record.

Locations

7

States available

Not stated in the official study record.

Remote participation

Not stated in the official study record.

Last ClinicalTrials.gov update

09-21-2026

Amyotrophic lateral sclerosis (ALS) is a serious rapidly progressive disease of the nervous system. The mean survival from the time of diagnosis is 2.5 years. Apart from Riluzole, there is no effective treatment. Care of advanced ALS will have a cost of 4-8 million NOK (Norwegian kroner) per year. Research i.a. from the investigators department has shown that increased activity in histone deacetylation enzymes (sirtuins) together with increased access to Nicotinamide Adenine Dinucleotide (NAD) can delay disease progression. Nicotinamide riboside (NR) can increase cells' access to NAD and Pterostilbene will stimulate sirtuins. The investigators want to study whether combination therapy with NR and Pterostilbene can inhibit neurodegeneration in ALS and thereby delay disease development, increase survival and improve quality of life in ALS. In the NO-ALS extension study the investigators will follow the patients who completed the original NO-ALS study. Objectives are to evaluate adverse events and give patients possibility of compassionate use, and secondarily to see if the combination of NR and pterostilbene (EH301) will decrease progression of motor symptoms and loss of vital capacity, and increase survival time in patients with ALS.

Registration

ClinicalTrials.gov

Study category

Interventional, Drug trial, ALS/FTD

Study type

Interventional study

Sponsor / center

Haukeland University Hospital

Intervention(s)

EH301 (Nicotinamide Riboside/Pterostilbene)

Gene / variant

Any / Not gene-specific

Disease status

ALS-FTD where applicable, Diagnosed ALS

Age

35 Years to Not stated in the official study record.

Disease duration

Not stated in the official study record.

Genetic-testing requirement

Not stated in the official study record.

Research center

Not stated in the official study record.

Locations

17

States available

Not stated in the official study record.

Remote participation

Not stated in the official study record.

Last ClinicalTrials.gov update

09-21-2026

The objective of the present study is to assess the efficacy of tSMS in ALS patients. This will be achieved by monitoring: * levels of NF-L and other potential innovative biomarkers, * clinical progression, trough ALSFRS-R. After at least three-month follow-up, participants will be recruited to undergo biemispheric tSMS for two daily sessions of 120 minutes each, at home, for 12 months. Together with clinical status, which will be evalueted each three months, blood and urine samples will be collected before the start of the tSMS administration (M0) and during the treatment (M3, M6, M9, M12), to detect potential theranostic biomarkers. In a subgroup of patients, ad additional blood and urine sample will be collected 3 months before M0 (M-3). Moreover, cortical excitability will be tested through transcranial magnetic stimulation (TMS) before and after the tSMS stimulation period.

Registration

ClinicalTrials.gov

Study category

Interventional, Device, Biomarker

Study type

Interventional study

Sponsor / center

Campus Bio-Medico University

Intervention(s)

Transcranial magnetic stimulation (tSMS)

Gene / variant

Any / Not gene-specific

Disease status

Diagnosed ALS

Age

18 Years to Not stated in the official study record.

Disease duration

Not stated in the official study record.

Genetic-testing requirement

Not stated in the official study record.

Research center

Not stated in the official study record.

Locations

1

States available

Not stated in the official study record.

Remote participation

Not stated in the official study record.

Last ClinicalTrials.gov update

09-18-2026