Efficacy and safety of xaliproden in amyotrophic lateral sclerosis: results of two phase III trials

2004 ◽  
Vol 5 (2) ◽  
pp. 107-117 ◽  
Author(s):  
Vincent Meininger ◽  
Gilbert Bensimon ◽  
Walter G Bradley ◽  
Benjamin R Brooks ◽  
Patrice Douillet ◽  
...  
Keyword(s):  
Amyotrophic Lateral Sclerosis ◽  
Phase Iii ◽  
Efficacy And Safety ◽  
Two Phase ◽  
Phase Iii Trials ◽  
Lateral Sclerosis

Phase Ib study of sintilimab in combination with chemotherapy for 1L advanced or metastatic non-small cell lung cancer (NSCLC).

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. e20546-e20546 ◽  
Author(s):  
Nong Xu ◽  
Kejing Ying ◽  
Ziping Wang ◽  
Yunpeng Liu ◽  
Haiping Jiang ◽  
...  
Keyword(s):  
Locally Advanced ◽  
Primary Objective ◽  
Phase Iii ◽  
Egfr Mutations ◽  
Efficacy And Safety ◽  
Two Phase ◽  
Phase Ib ◽  
Biomarker Analysis ◽  
Phase Iii Trials ◽  
Treatment Naïve

e20546 Background: Anti-programmed death-1 antibodies (PD-1 Abs) have shown benefits in advanced NSCLC. The efficacy and safety of sintilimab, a PD-1 Ab, in combination with chemotherapy for 1L NSCLC is evaluated in this phase Ib study (NCT02937116). Methods: The study enrolled treatment-naïve unresectable locally advanced or metastatic non-squamous (nsq-) and squamous (sq-) NSCLC patients with neither EGFR mutations nor ALK rearrangements in cohort D and E respectively. Patients received sintilimab 200mg IV q3w in combination with pemetrexed 500mg/m2 and cisplatin 75mg/m2 IV q3w (4 cycles) in cohort D, or gemcitabine 1250mg/m2 D1,8 and cisplatin 75mg/m2 D1 IV q3W (6 cycles) in cohort E until disease progression, unacceptable toxicity or death. The primary objective was to evaluate the efficacy and safety of the combination. Results: As data cutoff (15 Jan 2019), 21 and 20 patients were enrolled in cohort D and E respectively. ORR in nsq- and sq-NSCLC were 68.4% (95%CI, 43.4 to 87.4) and 64.7% (95%CI, 38.3 to 85.8) respectively based on data of 19 and 17 patients with at least one radiological assessment. Median PFS was 11.4 months (95%CI, 3.1 to NA) and 6.5 months (95%CI, 5.3 to 8.0) respectively (Table). Totally 38 (92.7%) patients experienced at least one treatment emergent adverse event (TEAE). Treatment-related AEs (TRAEs) occurred in 28 (68.3%) patients. TRAE ≥grade 3 occurred in 4 (9.8%) patients. Immune related AEs occurred in 10 patients (24.4%), the most common of which were skin rash (N = 5), pneumonitis (N = 3) and hypothyroidism (N = 2). There was no AEs leading to death. The biomarker analysis was ongoing. Conclusions: The combination of sintilimab and chemotherapy showed efficacy with an acceptable safety profile in 1L nsq- and sq-NSCLC. Two phase III trials are ongoing to evaluate the combination in 1L nsq- (NCT03607539) and sq-NSCLC (NCT03629925) respectively. Clinical trial information: NCT02937116. [Table: see text]

Abstract #999926: Effect of Dosing and Titration of Osilodrostat on Efficacy and Safety in Patients With Cushing's Disease (CD): Results From Two Phase III Trials (LINC3 and LINC4)

2021 ◽  
Vol 27 (6) ◽  
pp. S112
Author(s):  
Maria Fleseriu ◽  
Richard J. Auchus ◽  
Peter J. Snyder ◽  
André Lacroix ◽  
Anthony P. Heaney ◽  
...  
Keyword(s):  
Cushing’S Disease ◽  
Phase Iii ◽  
Cushing's Disease ◽  
Efficacy And Safety ◽  
Two Phase ◽  
Phase Iii Trials

scholarly journals The Japanese Early-Stage Trial of High-Dose Methylcobalamin for Amyotrophic Lateral Sclerosis (JETALS): Protocol for a Randomized Controlled Trial (Preprint)

2018 ◽  
Author(s):  
Ryosuke Oki ◽  
Yuishin Izumi ◽  
Hiroyuki Nodera ◽  
Yasutaka Sato ◽  
Hiroshi Nokihara ◽  
...  
Keyword(s):  
Amyotrophic Lateral Sclerosis ◽  
Early Stage ◽  
Phase Iii ◽  
High Dose ◽  
Efficacy And Safety ◽  
Double Blind ◽  
One Year ◽  
Als Patients ◽  
To Receive ◽  
Lateral Sclerosis

BACKGROUND Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that affects the upper and lower motor neurons. Currently, only riluzole and edaravone are approved as drugs to treat ALS and new agents with larger effect sizes are warranted. Exploratory analyses in our previous study (study ID #E0302-J081-761) have suggested that high-dose methylcobalamin (E0302) prolonged the overall survival of ALS patients and suppressed ALS progression in patients with a disease duration of less than 12 months. OBJECTIVE This clinical trial aims to evaluate the efficacy and safety of E0302 for treatment of ALS patients within one year of onset. METHODS The Japanese early-stage trial of high-dose methylcobalamin for ALS (JETALS) is a prospective, multicenter, placebo-controlled, double-blind, randomized phase III study conducted at 24 tertiary neurology centers and is funded by the Japan Agency for Medical Research and Development. A total of 128 ALS patients within one year of onset were randomized at a 1:1 ratio to receive intramuscular injection with E0302 50 mg or placebo twice a week for 16 weeks. The primary endpoint is changes in the ALS Functional Rating Scale-Revised (ALSFRS-R) total score at 16 weeks. If patients wish to receive E0302 50 mg after the double-blind administration period, E0302 will be provided to them until March 2020 during the continuous administration period. RESULTS This study began in October 2017 and is being conducted at 24 participating institutions in Japan. The study is in progress and the patient enrollment period is scheduled to end in August 2019, with follow-up scheduled to end in March 2020. CONCLUSIONS This study is being performed to revalidate the efficacy and safety of E0302 in patients with early-stage ALS in the first year of symptom onset. If positive results are obtained, the aim is to apply for E0302 approval as a new drug for the treatment of ALS. CLINICALTRIAL ClinicalTrials.gov NCT03548311; https://clinicaltrials.gov/ct2/show/NCT03548311 (Archived by WebCite at http://www.webcitation.org/74Fw3rDzb) INTERNATIONAL REGISTERED REPOR PRR1-10.2196/12046

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