View clinical trials related to Amyotrophic Lateral Sclerosis.
Filter by:This registry study aims to collect data on the provision of assistive devices, medicines, and other healthcare measures, such as ventilation therapy and nutrition support, in patients with Amyotrophic lateral sclerosis (ALS), Spinal muscular atrophy (SMA) and other neurological disorders. The data collected should describe the clinical practice, meaning real-world evidence and patient-reported outcomes.
Patients with sporadic ALS (sALS), which refers to those without a family history of ALS, are typically not subjected to genetic investigations as part of their standard care. Therefore, their mutation status is often unknown. Even patients with familial ALS (fALS), who have a known family history of ALS, are not regularly screened for genetic mutations. This project aims to study a large group of ALS patients, examining their family history, clinical characteristics, healthcare measures, and genetic variants in ALS's most commonly mutated genes: SOD1, C9orf72, FUS, and TARDBP. Examining genetically distinct ALS cohorts is significant, as understanding the relationship between genotype and disease progression is essential in determining the therapeutic potential of future genetic therapies.
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 G will evaluate the safety and efficacy of a single study drug, DNL343, in participants with ALS.
The median survival of patients with amyotrophic lateral sclerosis (ALS) is 3 to 5 years and mortality is mainly related to respiratory failure. Non-invasive ventilation (NIV) and multidisciplinary management improve the quality of life and survival of patients. However, patients have mobility difficulties related to the progressive worsening of functional disabilities. The research team hypothesize that the use of a multimodal digital platform, including in particular telemonitoring of NIV and teleconsultation, will slow down the evolution of the disabilities of patients with ALS and improve their quality of life.
This observational study will use new smartwatch technology to continuously and remotely monitor the health of ALS patients and healthy controls over time. This information will be used to develop digital biomarkers for ALS.
The purpose of this investigation is to see if outpatient initiation of noninvasive home mechanical ventilation combined with closed telemonitoring and follow-up in patients with amyotrophic lateral sclerosis is non-inferior to initiation during admission to the hospital The primary hypothesis is that outpatient intiation of noninvasive home mechanical ventilation combined with closed telemonitoring and follow-up is non-inferior to initiation during hospitalization in patients with amyotrophic lateral sclerosis.
The study aims to evaluate the effect of mechanical insufflator-exsufflator on the respiratory functions of Amyotrophic Lateral Sclerosis (ALS) patients evaluated via peak expiratory flow on cough (PEFC) measurements. The evolution of their PEFC is monitored to see if the curative management can have a positive impact on the latter.
This is a Phase 1, cross-over, 2-part study for pharmacokinetic (PK) assessment of SAR443820 when co-administered with cytochrome P450 3A4 (CYP3A4) inhibitors (erythromycin ethyl succinate (EES) in Part A and possibly itraconazole in Part B). In Part A, the objective is to assess the effects of repeated administration of EES as CYP3A4 inhibitor, on the PK profile of a single oral dose of SAR443820 tablet in healthy male and female participants. In Part B, the objective is to assess the effects of repeated administration of itraconazole on the PK profile of a single oral dose of SAR443820 capsule in healthy male participants. Part A includes a screening period, Period 1 (SAR443820), a wash-out period and Period 2 (SAR443820 + EES). Part B includes a screening period, Period 1 (SAR443820), a wash-out period and Period 2 (SAR443820 + itraconazole). The washout period between single SAR443820 administration in Period 1 and the start of dosing with EES (Part A) or itraconazole (Part B) in Period 2 is at least 4 days. The study duration is approximately 7 weeks for each Part A and Part B. The treatment duration is: - For SAR443820 (both Part A and Part B): 1 day in each Period; single dose of SAR443830 on Period 1 (P1)-Day 1 and on Period 2 (P2)-Day 6 for each Part. - For EES (Part A): 9 days of treatment in Period 2 with P2-Day 1 starting at least 4 days after P1-Day 1. - For itraconazole (Part B): the treatment duration lasts 11 days in Period 2 and it is fixed once the results of Part A are issued, P2-Day 1 starting at least 4 days after P1-Day 1.
Part 1: This is an open label, balanced randomized, single dose, 2-sequence, 2-period (period 1 and period 2), 2-treatment crossover (between Treatment A and Treatment B for Part 1), study part to determine the relative bioavailability of SAR443820 in tablet formulation versus capsule formulation under fasted conditions. Two treatments are as follows: - Treatment A: SAR443820 - tablet formulation in fasted condition - Treatment B: SAR443820 - capsule formulation in fasted condition Each administration will be a single dose of SAR443820 separated by a wash out of at least 5 days. Part 2: This is an open-label, balanced randomized, single dose, 2-sequence, 2-period (period 1 and period 2), 2-treatment crossover (between Treatment C and Treatment D for Part 2) study part to perform a preliminary assessment of the effect of a high-fat meal on pharmacokinetic parameters of single dose of SAR443820 in tablet formulation. Two treatments are as follows: - Treatment C: SAR443820 - tablet formulation in fasted condition - Treatment D: SAR443820 - tablet formulation in fed condition Each administration will be a single dose of SAR443820 separated by a wash out of at least 5 days. Participants are not allowed to participate in more than one part of the study. In both Parts 1 and 2, the assessment of pharmacokinetic, safety and tolerability are performed at each treatment period at baseline (prior single dosing) up to 48-hour postdosing in healthy adult male and female participants.
This is a Phase 1, single-center study conducted in 2 parts: Part 1a, single ascending dose (SAD-TDU16519): Double-blind, randomized, placebo-controlled sequential ascending single oral doses including up to 6 cohorts. Each cohort will include 8 participants (6 receiving SAR443820 and 2 placebo). Part 1b (TDU16519): - Open label, single SAR443820 dose in one or two separated cohort(s) for SAR443820 measurements in CSF and in plasma. Part 2, multiple ascending dose (MAD -TDR16520): Double-blind, randomized, placebo-controlled, sequential ascending repeated oral doses for 14 days, including up to 4 cohorts. Each cohort will include 10 participants (8 receiving SAR443820 and 2 placebo).