Search results for " Phase III as Topic"
showing 10 items of 41 documents
Intraspinal stem cell transplantation for amyotrophic lateral sclerosis: Ready for efficacy clinical trials?
2016
Intraspinal stem cell (SC) transplantation represents a new therapeutic approach for amyotrophic lateral sclerosis (ALS) clinical trials. There are considerable difficulties in designing future efficacy trials, some related to the field of ALS and some that are specific to SCs or the mode of delivery. In October 2015, the most controversial points on SC transplantation were addressed during an international workshop intended to bring together international SC and ALS researchers in a public discussion on a topic for which expertise is limited. During the meeting, a discussion was started on the basic structure of the ideal clinical trial testing the efficacy and safety of SC transplantation…
Frequency and prognostic impact of ALK amplifications and mutations in the European Neuroblastoma Study Group (SIOPEN) high-risk neuroblastoma trial …
2021
Purpose: In neuroblastoma (NB), the ALK receptor tyrosine kinase can be constitutively activated through activating point mutations or genomic amplification. We studied ALK genetic alterations in high-risk (HR) patients on the HR-NBL1/SIOPEN trial to determine their frequency, correlation with clinical parameters, and prognostic impact. Materials and methods: Diagnostic tumor samples were available from 1,092 HR-NBL1/SIOPEN patients to determine ALK amplification status (n = 330), ALK mutational profile (n = 191), or both (n = 571). Results: Genomic ALK amplification (ALKa) was detected in 4.5% of cases (41 out of 901), all except one with MYCN amplification (MNA). ALKa was associated with …
Clinical pharmacokinetics and pharmacodynamics of ramucirumab in the treatment of colorectal cancer
2016
Colorectal cancer is the third most common cancer worldwide. The prognosis of colorectal cancer patients still remains dismal and half of them will develop metastatic disease. Angiogenesis plays an essential role in colorectal tumorigenesis, and the VEGF pathway is one of the targets that has been validated up to now. The use of antiangiogenics along with chemotherapy has become an accepted standard for colorectal cancer.This review discusses the efficacy and safety profile of ramucirumab, a fully human immunoglobulin G1 monoclonal antibody against the vascular endothelial growth factor receptor-2 (VEGFR-2), for the treatment of second-line metastatic colorectal cancer upon progression to f…
The role of chemotherapy in localized and locally advanced rectal cancer: A systematic revision
2017
Curative treatment of rectal cancer depends on an optimal surgical resection, with the addition of neoadjuvant radiotherapy (RT) with or without concomitant chemotherapy (ChT) in more advanced tumors. The role of adjuvant ChT is controversial and a more intensified neoadjuvant approach with the addition of ChT before or after RT, or even as single modality, is currently being explored in trials. A systematic review selecting randomised phase II and III trials on the role of ChT in localized rectal cancer was performed. Data show that neoadjuvant ChRT improves locoregional control in resected rectal cancer. Short-course RT (SCRT) could give similar outcomes to ChRT. The addition of oxaliplat…
Treatment of HER2 positive advanced breast cancer with T-DM1: A review of the literature
2014
Abstract Background Trastuzumab emtansine (T-DM1), a new agent developed for the treatment of HER2-positive breast cancer, is an antibody-drug conjugate with a complex compound obtained by the conjugation of trastuzumab, a stable thioether linker, and the potent cytotoxic drug maytansine-derivate(DM1), which inhibits cell division and induces cell death. Field of study PubMed database, ESMO, ASCO, San Antonio Breast Cancer Symposium Meeting abstracts and clinicaltrials.gov were searched using the terms “Anti-HER2 treatment breast cancer and trastuzumab emtansine (T-DM1) “; papers considered relevant for the aim of this review were selected. Findings/results The phase I trials have determine…
The hard road to data interpretation: 3 or 6 months of adjuvant chemotherapy for patients with stage III colon cancer?
2019
Background Six months of adjuvant oxaliplatin-based chemotherapy is standard for patients with stage III colon cancer following surgery. However, oxaliplatin is associated with peripheral neurotoxicity which worsens over treatment duration. Consequently, a shorter treatment duration, if equally effective, would be extremely beneficial. A pooled analysis of data for 12 834 stage III colon cancer patients, from six randomised phase III trials of adjuvant therapy, the International Duration Evaluation of Adjuvant chemotherapy study, was carried out and the results presented at the ASCO Annual Meeting 2017. To clarify the potential impact of these results on clinical practice, ESMO decided to s…
Outcomes of single versus double hormone receptor–positive breast cancer. A GEICAM/9906 sub-study
2018
Abstract Background Retrospective data suggest better outcomes for patients with double hormonal receptor (oestrogen [ER] and progesterone receptor [PgR])–positive (dHR+) early breast cancer, compared with single hormonal receptor–positive, sHR+, (ER+/PgR– or ER–/PgR+) disease. Here, we evaluate the classification according to intrinsic subtypes and clinical outcomes of sHR+ versus dHR+ in HER2-negative breast cancer patients enrolled in GEICAM/9906 study ( NCT00129922 ). Methods Archival tumours were retrieved retrospectively for the analysis of ER, PgR and HER2 status and classified into intrinsic subtypes using the PAM50 gene expression assay. Disease-free survival (DFS) and overall surv…
Construction of quality of life change patterns: example in oncology in a phase III therapeutic trial (FFCD 0307)
2015
Objective Quality of life data in cancerology are often difficult to summarize due to missing data and difficulty to analyze the pattern of evolution in different groups of patients. The aim of this work was to apply a new methodology to construct Quality of Life (QoL) change patterns within patients included in a clinical trial comparing to regimen of treatment in locally advanced eosogastric cancer. Materials and methods In this trial, QoL was assessed every 2 months by self-reported EORTC QLQ-C30 questionnaire. Physical dimension scores were analyzed. After multiple imputation of missing data, 27 statistical measures aiming to describe the variation of QoL measures among follow-up were c…
"Baseline physical functioning status of metastatic colorectal cancer patients predicts the overall survival but not the activity of a front-line oxa…
2010
BACKGROUND: No differences in response rate (RR), progression-free survival (PFS), overall survival (OS) and quality of life (QoL) were seen in patients randomly treated with biweekly oxaliplatin plus either fluorouracil/folinic acid or capecitabine. METHODS: We investigated the independent effect of baseline clinical characteristics and physical functioning (PF) domain on RR, PFS, and OS in 310 patients who completed the EORTC QLQ-C30 questionnaire. Multivariate analyses stratified by treatment were performed. An exploratory analysis was done by grouping patients with a PF score superior or equal to the highest quartile (n = 111), included between the highest and the lowest quartiles (n = …
Effects of enzyme replacement therapy on growth in patients with mucopolysaccharidosis type II
2010
Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is an X-linked, recessive, lysosomal storage disorder caused by deficiency of iduronate-2-sulfatase. It has multisystemic involvement, with manifestations in the brain, upper respiratory tract, heart, abdomen, joints and bones. Bone involvement leads to decreased growth velocity and short stature in nearly all patients. A therapeutic option for patients with MPS II is enzyme replacement therapy (ERT) with idursulfase (Elaprase®). We compared annual growth rates before and during ERT in 18 patients from Mainz, Germany, and Manchester, UK. Group 1 included nine patients who started ERT before 10 years of age; group 2 contained nine patie…