Sclérose latérale amyotrophique (SLA)
Pour plus d’informations sur les essais cliniques sur la Sclérose latérale amyotrophique, veuillez contacter Kristiana Salmon, email@example.com.
Études actuelles en recrutement
Routine MRI is normal in motor neuron diseases such as ALS. However, advanced MRI techniques can provide an objective measure of degeneration (a « biomarker ») by examining brain structure, wiring, chemistry, and function. We will develop and evaluate novel MRI techniques that could improve our understanding of ALS and provide a means to diagnose it sooner and monitor its progression. Importantly, we expect these techniques to improve how new drugs are tested, which may lead to the more rapid discovery of a treatment for ALS.
Each participant will have 3 MRI scans over a period of 8 months, along with neurological and cognitive evaluations. Study visits will take 2 – 3 hours. MRI is a safe technique that does not involve radiation.
The purpose of this study is to assess the effect of CK-2127107 versus placebo on respiratory function and other measures of skeletal muscle function in patients with ALS. Enrolled participants will be dosed with CK-2127107 150, 300, 450 mg or placebo twice daily (300, 600, 900 mg/day or placebo) for a period of 12 weeks.
Étude multicentrique, à double insu, contrôlée par placebo visant à évaluer l’efficacité potentielle et l’innocuité d’H.P. Acthar Gel dans le traitement de sujets atteints de sclérose latérale amyotrophique
This is a multicenter, multiple dose study to examine the effect of H.P. Acthar® (Acthar) on functional decline in adult subjects with amyotrophic lateral sclerosis (ALS). Approximately 213 subjects will be enrolled.
Following a screening period of up to 28 days, subjects with ALS and symptom onset (defined as first muscle weakness or dysarthria) ≤ 2 years prior to the Screening Visit will be randomized on a 2:1 basis to receive subcutaneous (SC) Acthar 0.2 mL (16 Units [U]) daily (QD) or SC matching placebo 0.2 mL QD for 36 weeks, followed by a 3 week taper.
Subjects who complete the 36 week double-blind treatment period are eligible to enter an Open Label Extension phase where all subjects will receive Acthar 0.2 mL (16 U) daily.
This study will evaluate whether prolonged oral levosimendan can preserve respiratory function more effectively than placebo, resulting in better patient functionality as measured by the ALSFRS-R scale. In this randomized, double-blind, placebo-controlled, parallel-group, multicenter study, subjects are allocated in a 2:1 ratio to receive either levosimendan (1 -2 mg daily) or placebo for 48 weeks. The primary endpoint is slow vital capacity (SVC) at 12 weeks, with the impact on patient function assessed through 48 weeks, adjusted for patient outcome, using ALSFRS-R (combined assessment of function and survival, CAFS). Other important efficacy measures include time to respiratory events, clinical global impression (CGI), assessment of dyspnea using the Borg scale and sleep scales (Pittsburgh sleep quality index and Epworth sleepiness scale). Patient safety is monitored using conventional methods including adverse events, safety laboratory tests, vital signs and 12-lead EKG. Following screening and baseline visits, patients attend the clinic at 2, 4, 8, 12, 24, 36 and 48 weeks, with telephone assessments conducted at weeks 18, 30 and 42. An end of study visit is performed 14-25 days after the last study treatment administration. The study will be monitored by an independent data and safety monitoring board. A long-term extension study will be available for patients completing the study.
Étude multicentrique de phase 1, à double insu, à répartition aléatoire, à dose unique et multiple croissante et contrôlée par placebo visant à établir l’innocuité initiale, la tolérabilité et la pharmacocinétique du GDC-0134 chez des patients atteints de sclérose latérale amyotrophique
This first-in-human, double-blind, placebo-controlled Phase I study will be conducted in participants with amyotrophic lateral sclerosis (ALS) to explore safety, tolerability, and pharmacokinetic (PK) properties of GDC-0134. It will include three components: a Single-Ascending-Dose (SAD) stage, a Multiple-Ascending-Dose (MAD) stage, and an Open-Label Safety Expansion (OSE) stage.
Études actives (ne pas recruter)
Étude de phase 1 à dose unique et multiple croissante et contrôlée par placebo visant à évaluer l’innocuité, la tolérabilité et la pharmacocinétique du BIIB067 administré à des sujets adultes atteints de sclérose latérale amyotrophique
The primary objective of the study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of BIIB067 in adults with ALS. The secondary objective is to evaluate the effects of BIIB067 on levels of superoxide dismutase 1 (SOD1) protein in the cerebrospinal fluid (CSF).
Étude de prolongation pour évaluer l’innocuité, la tolérabilité, la pharmacocinétique et l’effet à long terme sur la progression de la maladie du BIIB067 administré aux adultes atteints de la sclérose latérale amyotrophique provoquée par la mutation de la superoxyde dismutase-1 ayant déjà reçu un traitement
The primary objective of the study is to evaluate the long-term safety and tolerability of BIIB067 in participants with Amyotrophic Lateral Sclerosis Caused by Superoxide Dismutase 1 Mutation (SOD1-ALS). The secondary objective is to evaluate the pharmacokinetic (PK) profile of BIIB067 in participants with SOD1-ALS.
The purpose of this study is to assess the long-term safety and tolerability of tirasemtiv in patients with ALS.
Enrolled participants will begin dosing of tirasemtiv 125 mg twice daily (250 mg/day) for a period of 4 weeks and will titrate to their tolerated dose, the maximum dose being 250 mg twice daily (500 mg/day). This study will also compare the clinical course of patients who completed treatment with tirasemtiv in CY 4031 with those who completed treatment with placebo in CY 4031 during continued treatment of both groups with tirasemtiv during CY 4033, compare the clinical course of patients who completed treatment with tirasemtiv in CY 4031 during that study with their clinical course during continued treatment with tirasemtiv during CY 4033, and compare the clinical course of patients who completed treatment with placebo in CY 4031 during that study with their clinical course during treatment with tirasemtiv during CY 4033.