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134 active trials for Amyotrophic Lateral Sclerosis

Trial of Theracurmin for Patients With Amyotrophic Lateral Sclerosis (ALS)

This will be a 6-month, widely inclusive, virtual, single-center, open-label pilot trial utilizing a historical control group.

Start: August 2020

Edinburgh Cognitive and Behavioural Amyotrophic Lateral Sclerosis Screen in Norway: A Prospective Cohort Study

This study evaluate use of a translated Norwegian version of the Edinburgh cognitive and behavioral amyotrophic lateral sclerosis screen (ECAS-N) as an early predictor in car-driving, working and use of advanced life-prolonging therapy.

Start: May 2017

Edinburgh Cognitive and Behavioural Amyotrophic Lateral Sclerosis Screen in Norway: A Validation Study

Cognitive impairment is present in about 30-50% of the patients with amyotrophic lateral sclerosis (ALS). Suitable screening tools are available, but none of these are evaluated in a Norwegian population.

Start: May 2017

iPSC-based Drug Repurposing for ALS Medicine (iDReAM) Study

This is a phase 1, open-label, multicenter, dose escalation study to evaluate the safety and tolerability of bosutinib to determine the maximum tolerated dose(MTD) and a recommended phase 2 dose (RP2D) of bosutinib for treatment of ALS patients. Also, efficacy will be evaluated exploratory.

Start: March 2019

MIROCALS: Modifying Immune Response and OutComes in ALS

MIROCALS is a phase II study of ld-IL-2 as a therapeutic agent for ALS. A randomized (1:1), placebo-controlled, double-blind, parallel group trial will be carried out to assess ld-IL-2 safety and clinical efficacy on survival and functional decline in newly diagnosed ALS patients treated for 18 months. Randomization will be stratified according to (i) country (n = 2 levels: UK, France) and (ii) site of onset (n= 2 levels: bulbar vs limb onset). The primary objective to evaluate the clinical efficacy and safety of the experimental drug (ld IL-2) over an 18 months period in order to establish the proof of concept (PoC) that modifying immune responses through the enhancement of regulatory T cells modifies the rate of ALS disease progression.

Start: June 2017

Investigation on the Cortical Communication (CortiCom) System

The CortiCom system consists of 510(k)-cleared components: platinum PMT subdural cortical electrode grids, a Blackrock Microsystems patient pedestal, and an external NeuroPort Neural Signal Processor. Up to two grids will be implanted in the brain, for a total channel count of up to 128 channels, for six months. In each participant, the grid(s) will be implanted over areas of cortex that encode speech and upper extremity movement.

Start: October 2021

The National Amyotrophic Lateral Sclerosis Registry

The purpose of this registry is to (A) better describe the incidence and prevalence of Amyotrophic Lateral Sclerosis (ALS) in the United States;(B) examine appropriate factors, such as environmental and occupational, that may be associated with the disease; (C) better outline key demographic factors (such as age, race or ethnicity, gender, and family history of individuals who are diagnosed with the disease) associated with the disease; and (D) better examine the connection between ALS and other motor neuron disorders that can be confused with ALS, misdiagnosed as ALS, and in some cases progress to ALS.

Start: October 2010

Exploratory Evaluation of [11C]MPC6827

This is a phase 0 study that will enable an assessment of biodistribution and estimation of absorbed dose in humans based on data collected from five healthy volunteers, which is typically the minimum number required by the FDA for first-in-human studies to assess dosimetry of a new tracer. The evaluation of the brain imaging of thirty additional subjects in the 2nd part of the study will lead to a descriptive assessment of the targeting and pharmacokinetics of MPC6827 in the brain and between normal and diseased brain.

Start: January 2021

Trial of Safety, Tolerability and Efficacy of Trametinib (SNR1611) in Patients With Amyotrophic Lateral Sclerosis (ALS)

The purpose of this study is to evaluate the safety, tolerability and efficacy of trametinib (SNR1611) in the treatment of amyotrophic lateral sclerosis.

Start: April 2020

Assessing Motor Neuron Disease Mechanisms by Threshold Tracking Transcranial Magnetic Stimulation and Magnetic Resonance Spectroscopy

Amyotrophic Lateral Sclerosis (ALS) is a motor neuron disease, which cases the death of neurons controlling the voluntary muscles. The death of motor neurons leads eventually to muscle weakness and muscle atrophy and as a consequence thereof, ALS patients die in average within three years after symptom onset due to respiratory failure. No cure for ALS is currently known, and the medical diagnosis and clinical treatment are impeded by the lack of reliable diagnostic tools for objective disease assessment, and by the limited insight in disease pathophysiology since the underlying disease mechanisms still have not been fully elucidated. An unbalance in the concentrations of GABA and glutamate, the most important inhibitory and excitatory brain metabolites, is suggested to play a role in the disease mechanisms of ALS. By applying Magnetic Resonance Spectroscopy (MRS), a magnetic resonance method which allows for quantification of brain metabolites, GABA and glutamate concentration can be quantified and thus hopefully elucidate their role in ALS disease mechanism. Threshold Tracking Transcranial Magnetic Stimulation (TT-TMS) studies carried out by a single research group have demonstrated cortical hyperexcitability (a physiology state in which neurons in the cerebral cortex are easier activated) as an early feature in ALS patients. For this reason, TT-TMS was suggested as a biomarker of ALS by the research group. However, to be able to suggest a test as a biomarker, one must show the test is reliable and reproducible. The objectives of this study are therefore: to explore the pathophysiology of ALS by investigating the interaction between neuronal networks as assessed by TT-TMS and conventional TMS and MRS, and to investigate the reliability and reproducibility of TT-TMS. The aim is to examine the utility of TT-TMS and MRS as diagnostic tools for objective detection of ALS in the early disease stage. The study will include 60 participants in total, subdivided into two groups: 30 healthy participants and 30 patients with clinical suspicion of motor neuron disease or ALS. Each participant will undergo examination with TMS and MRS, the primary outcomes will be compared between the two groups and the results from the TMS examinations and the MRS-scans will be correlated.

Start: February 2018

Rapamycin Treatment for ALS

In the last years research has pointed out potential mechanisms of pathogenesis in ALS including lack of degradation of abnormally accumulated proteins inside motor neurons, and an unbalanced function of the immune system leading to the prevalence of a neurotoxic function over neuroprotection. These two mechanisms contribute to ALS progression hence representing important therapeutic targets to modify disease expression. With a phase II clinical trial the investigators aim to study the biological response in ALS treated with Rapamycin, to obtain predictive information for a larger study. Eight Italian Centres will enroll 63 patients; treatment will be double blinded to patients and physicians, and will last 18 weeks.Follow up will be carried out for 36 months (total duration: 54 weeks).

Start: September 2017

Arimoclomol in Amyotropic Lateral Sclerosis - Open Label Extension Trial

A multicenter, non-randomized, open label trial, to assess long term safety and efficacy of Arimoclomol in subjects with Amyotrophic Lateral Sclerosis (ALS)who have completed the ORARIALS-01 trial.

Start: September 2019

A Multicenter Phase I/II Clinical Trial to Evaluate Safety of Mesenchymal Stem Cell in Patients With Amyotrophic Sclerosis Lateral

A multicenter phase I/II Clinical trial,randomized, controlled with placebo, triple blind to evaluate the safety of the intravenous administration of 3 doses of autologous mesenchymal stem cells cells from adipose tissue in patients with Amyotrophic Lateral Sclerosis (ALS).

Start: July 2014

To Determine the Maximum Tolerated Dose (MTD) of HK-001 in Healthy Volunteers

Eligible subjects will receive either different dosages of HK-001 or placebo in a 3:1 ratio in 1 of the 7 dose cohorts. After single dose administration, followed by an independent Data and Safety Monitoring Board (DSMB) meeting for safety assessments (including the available plasma pharmacokinetic profile), the subjects will be allowed to receive (Z)-BP or placebo twice a day orally at the study site for 14 consecutive days and follow up on the 28th day after the last dose administration by a site visit. The study drugs (including placebo) will be administered at the study site by following the investigator's instructions to either perform blood sampling for pharmacokinetic evaluation or maximize the treatment compliance. There will be 7 cohorts and subjects will be randomized into cohorts consisting of 8 subjects each (6 active and 2 placebo controls per cohort). Dose cohorts will be escalated sequentially from low to high dose (50 mg, BID; 100 mg, BID; 150 mg, BID; 225 mg, BID; 300 mg, BID; 400 mg, BID; 525 mg, BID) by following a modified Fibonacci sequence, and based on the decision of an independent DSMB at a set time point. Following all subjects of a cohort complete the safety and PK evaluation after receiving the last dose administration, a cohort at the next dose level will be launched if the DSMB does not identify significant safety concerns after reviewing safety data and PK profiles.

Start: February 2021

Analysis of Capillary Retinal and Papillary Vascularization in Patients With Amyotrophic Lateral Sclerosis - CAPISLA

Amyotrophic Lateral Sclerosis, or Charcot's disease, is a neurodegenerative disease affecting motor neurons. The disease affects between 5 and 10 people per 100,000 in the world, nearly 7,000 patients are affected in France. The only therapeutic treatment available to date in France is riluzole, which slows the progression of the disease. Amyotrophic Lateral Sclerosis is the first degenerative disease affecting motor neurons. However, recent evidence suggests that the impairment extends beyond motor neurons alone. Optical Coherence Tomography analyzes made it possible to highlight ophthalmologic damage in patients with Amyotrophic Lateral Sclerosis, in particular at the macula and papilla, although some results are contradictory. No angiographic Optical Coherence Tomography analysis has been performed to date in patients with Amyotrophic Lateral Sclerosis. However, in the hypothesis of microvascular involvement participating in the pathophysiology of neurodegeneration in Amyotrophic Lateral Sclerosis, these examinations could provide relevant clinical and pathophysiological data by studying the retinal microvascularization of patients with the disease.

Start: January 2021

Virtual Peer-to-peer Support Programme for Carers of MND

Background/scope There is growing recognition that family caregiving is a serious public health issue requiring supportive interventions. Family caregivers play an essential role in sustaining a stable environment enabling individuals with motor neurone disease (MND) that are technology dependent to live at home. The family caregivers can experi¬ence exceptional burden and significant decline in psychological wellbeing due to MND's rapid and pro¬gressive nature with profoundly debilitating effects and intensive support needs. Dependence on assistive technology adds an additional level of complexity to family caregiving due to the need to learn how to operate and troubleshoot medical devices, train other caregivers, and negotiate appointments with new specialties within the healthcare system. Despite the recognized impact of caregiving for individuals with MND, data are scarce as to effective interventions that provide direct practical and psychosocial supports. Difficulty accessing support may increase psychological distress. As the burden of caring increases due to disease progression and increasing technology dependence, access to existing informal support networks may diminish. Online peer support using virtual modalities is a flexible and low cost form of support. Peers, people who have experienced the same health problem and have similar characteristics as support recipients, can be a key source of emotional, informational, and affirmational support. Peer support improves psychological well-being of caregivers of people with conditions such as dementia, cancer, and brain injury. Although peer support programmes for family caregivers of people with MND exist, data as to their efficacy are limited. Therefore, we have developed an online peer support programme, completed beta and usability testing and now propose to test the effect on caregiver psychological wellbeing and caregiver burden. Aim/research question(s) Overall aim: to determine the efficacy of a 12-week online peer support programme on family caregiver psychological health and caregiver burden. Primary research question: What is the effect of the online peer support programme on psychological distress measured using the Hospital Anxiety and Depression Scale (HADS)? Secondary research questions: What is the effect on positive affect, caregiver burden, caregiving mastery, caregiving personal gain, and coping? How do participants use the programme (fidelity and reach)? What is the perceived usability and acceptability? Methods The investigators will conduct a parallel group randomised controlled trial with participants allocated to 12-week access to the online peer support programme or a usual care control group. The investigators will enrol family caregivers of an individual with MND who is referred for consideration or receiving any of the following assisted ventilation cough assist gastroscopy and enteral feeding i.e., entering King's clinical staging Stage 4A: nutritional support; or Stage 4B: respiratory support [51]: The 12-week peer-to-peer support programme entails: audio, video, or text private messaging; synchronous weekly chat; asynchronous discussion forum; and informational resources. The investigators will collect demographic and caregiving data including the Caregiver Assistance Scale and Caregiving Impact Scale, and caregiver measures (HADS, Positive and Negative Affect Schedule, Zarit Burden Interview, Pearlin Mastery Scale, Personal Gain Scale, Brief COPE) at baseline and programme completion. The investigators will download use of online peer support programme features, assess usability, and conduct semi-structured interviews to explore acceptability using the Theoretical Framework of Acceptability. To test for a medium size effect (d=0.5), at 5% level of significance (2-sided) with power 80%, 64 participants are required in each arm (128 total). Adjusting for 20% attrition requires 154 participants. Proposed findings The proposed study will demonstrate the effect of a online peer support programme on psychological distress, positive affect, caregiving burden, mastery, personal gain and coping. Data on programme fidelity will enable the investigators to objectively assess acceptability and interpret study results. Data on usability and acceptability will inform future scalability of the online peer support programme outside of the trial both nationally and internationally, and to other family caregiver populations.

Start: June 2021

Outcome Measures and Biomarkers in a Cohort of Spinal Muscular Atrophy Type III/ IV Patients

The "SMOB" project intends to contribute to fill the gap with reliable and operational outcome measures for type III and IV SMA. In analysing the reliability in imaging (spinal and muscular), electrophysiology analysis (MUNIX), and evaluate the evolution of respiratory function for 50 patients' cohort. The investigators would also take the opportunity to collect biologic samples in order to investigate genetic markers and to assess quality of life of patients by QoL-gNMD questionnaire. The investigators aim to build a database that will allow us to evaluate the effectiveness of a new therapy for adult SMA patients by studying the natural history of the disease. The investigators have distributed the various expertise in Work Package where several centers are involved. This study is original in that it evaluates the parameters of qMRI and MUNIX in correlation with blood biomarkers. To our knowledge, there are no quantitative MRI (spinal and muscular) biomarkers and/or electrophysiological (MUNIX technique) highlighted for tracking the progression of the adult form of SMA type III and IV. This pilot study would allow identification of predictive markers of the disease progression, and to have validated, sensitive to change and relevant measurement tools that could be used as endpoints in future therapeutic trials.

Start: January 2021

HEALEY ALS Platform Trial - Regimen D Pridopidine

The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. Regimen D will evaluate the safety and efficacy of a single study drug, pridopidine, in participants with ALS.

Start: December 2020

HERV-K Suppression Using Antiretroviral Therapy in Volunteers With Amyotrophic Lateral Sclerosis (ALS)

Background: - Some people with Amyotrophic Lateral Sclerosis (ALS) have a high level of the virus HERV-K in their blood. Researchers do not think this virus causes ALS. But they don t know why some people with ALS have a high level of it. They want to know if HERV-K can be suppressed by drugs that are used to treat HIV infection. Objectives: - To learn how drugs usually taken for HIV infection affect people with Amyotrophic Lateral Sclerosis (ALS). Eligibility: - Adults at least 18 years old with ALS and high levels of HERV-K but no HIV. Design: Interested participants can contact the study team and, if eligible, the study team will arrange for a screening blood draw to determine the HERV-K level. Participants with a high HERV-K level will be screened with medical history, physical exam, questionnaires, nerve conduction test, lumbar puncture, and blood and breathing tests. After screening, participants will start taking the 4 study drugs. Participants will have up to 12 study visits over a period of 72 weeks. After starting study drugs, they will have study visits at Weeks 1 and 4 and then every 4 weeks until Week 28. They will be asked how they are feeling and have an exam and blood drawn. At 3 visits, they will have tests of nerve conduction, breathing, and their ALS symptoms. At Week 24, they will stop taking the study drugs and have a repeat lumbar puncture. After the Week 48 visit, their participation is finished.

Start: April 2019

Outcomes Mandate National Integration With Cannabis as Medicine for Prevention and Treatment of COVID-19

This will be a multistate, multicenter clinical study to determine the efficacy and safety of medical cannabis for a wide variety of chronic medical conditions.

Start: December 2018