scholarly journals ACTR-27. EVOLUTION OF THE NEUROSURGEON’S ROLE IN CLINICAL TRIALS FOR GBM: A SYSTEMATIC OVERVIEW OF THE CLINICALTRIALS.GOV DATABASE

2019 ◽  
Vol 21 (Supplement_6) ◽  
pp. vi18-vi19
Author(s):  
Alireza Mansouri ◽  
Michelle Beyn ◽  
Aditya Pancholi ◽  
Clement Chow ◽  
Alexandre Boutet ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Phase I ◽  
Primary Outcome ◽  
High Volume ◽  
Local Delivery ◽  
Local Drug Delivery ◽  
Industry Funding ◽  
Novel Device ◽  
The Usa

Abstract BACKGROUND The therapeutic challenge of glioblastoma (GBM) has catalyzed the active pursuit of a high volume of clinical trials to evaluate novel interventions for this deadly disease. Our enhanced understanding of the biology of GBMs has translated into an evolution of the role of the neurosurgeon as well. In this study, we have evaluated the current landscape of surgical clinical trials in GBM to characterize this evolution, describe current trends, and identify potential gaps in methodological features. METHODS The ClinicalTrials.gov database was searched for surgical/procedural trials in individuals with GBM on May 14th, 2019 without date limitations. Demographics, specific intervention (e.g. technical, device use, or local drug delivery), phase, sample size, and main outcome measures were abstracted. RESULTS 270 of 2140 trials (12.6%) were identified as procedural. The majority were based in the USA (189/270, 70%), single-center (188/270, 70%), and not randomized by design (215/270, 80%). Primary and recurrent GBMs were evenly addressed. Industry-funding supported 106/270 (39%) of studies. The leading test intervention was local delivery of therapeutics (44.8%), followed by use of novel devices (33%). Surgical technique/procedures comprised 20%. Early Phase designs predominated (177/270, 65.5%) but 67 (24.8%) did not report Phase. The greatest surge in new registrations over the last decade was seen in Phase I trials. The top primary outcome was safety/tolerability/ feasibility (107/270, 39%), followed by survival (62/270, 22%); 35 (12.6%) did not report a primary outcome. Approximately 15% of studies were terminated, withdrawn or suspended. Only 6 records were associated with reported results. CONCLUSIONS In GBMs, procedural interventions comprise a notable proportion of trials. Local delivery of therapeutics and novel device applications, predominantly through Phase I designs, represent the evolved role of the neurosurgeon in neuro-oncology. Improved documentation of design, outcomes, and reporting of results are needed to better inform the field and increase efficiency.

scholarly journals Race and ethnicity representation in clinical trials: findings from a literature review of Phase I oncology trials

2021 ◽  
Author(s):  
D Ross Camidge ◽  
Haeseong Park ◽  
Karen E Smoyer ◽  
Ira Jacobs ◽  
Lauren J Lee ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Phase I ◽  
Predominantly White ◽  
Race And Ethnicity ◽  
Phase I Trials ◽  
Minimal Representation ◽  
Phase I Clinical Trials ◽  
Demographic Representation ◽  
The Usa ◽  
Race Ethnicity

Aim: To provide an assessment of published literature on the demographic representation in Phase I trials of biopharmaceutical oncology agents. Materials & methods: We conducted a rapid evidence assessment to identify demographic representation reported in Phase I clinical trials for biopharmaceutical oncology agents published in 2019. Results: Globally, the population was predominantly White/Caucasian (62.2%). In the USA, the distribution was heavily skewed toward White/Caucasian (84.2%), with minimal representation of Blacks/African–Americans (7.3%), Asians (3.4%), Hispanics/Latinos (2.8%) or other race/ethnicity groups. Conclusion: Our data highlight that Phase I oncology trials do not reflect the population at large, which may perpetuate health disparities. Further research is needed to understand and address barriers to participation, particularly among under-represented groups

The role of an initial single-blind placebo period in phase I clinical trials

1994 ◽  
Vol 8 (2) ◽  
pp. 185-187 ◽  
Author(s):  
P. Nony ◽  
JP Boissel ◽  
P. Girard ◽  
L. Lion ◽  
MC Haugh ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Phase I ◽  
Phase I Clinical Trials ◽  
Placebo Period ◽  
Single Blind

scholarly journals Rethinking Remdesivir for COVID-19: A Bayesian Reanalysis of Trial Findings

2020 ◽  
Author(s):  
Sarahanne Miranda Field ◽  
Joyce M. Hoek ◽  
Ymkje Anna de Vries ◽  
Maximilian Linde ◽  
Merle-Marie Pittelkow ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Mortality Rate ◽  
Clinical Improvement ◽  
Primary Outcome ◽  
Secondary Outcome ◽  
Moderate Evidence ◽  
The World ◽  
The Usa ◽  
Regulatory Bodies ◽  
Take All

Following testing in clinical trials, the use of remdesivir for treatment of COVID-19 has been authorized for use in parts of the world, including the USA and Europe. These early authorizations were largely based on results from two clinical trials. A third study published by Wang et al. was deemed inconclusive. We demonstrate the utility of Bayesian reanalyses in the context of non-significant results like the Wang et al. trial. Results of a reanalysis of the three trials show ambiguous evidence for the primary outcome of clinical improvement and moderate evidence against efficacy of remdesivir for the secondary outcome of mortality rate. We recommend that regulatory bodies take all available evidence into account for endorsement decisions.

scholarly journals Rethinking remdesivir for COVID-19: A Bayesian reanalysis of trial findings

PLoS ONE ◽  
2021 ◽  
Vol 16 (7) ◽  
pp. e0255093
Author(s):  
Joyce M. Hoek ◽  
Sarahanne M. Field ◽  
Ymkje Anna de Vries ◽  
Maximilian Linde ◽  
Merle-Marie Pittelkow ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Primary Outcome ◽  
Secondary Outcome ◽  
Marketing Approval ◽  
Significant Finding ◽  
Efficacy Data ◽  
Bayesian Hypothesis Testing ◽  
The Usa ◽  
Regulatory Bodies ◽  
Take All

Background Following testing in clinical trials, the use of remdesivir for treatment of COVID-19 has been authorized for use in parts of the world, including the USA and Europe. Early authorizations were largely based on results from two clinical trials. A third study published by Wang et al. was underpowered and deemed inconclusive. Although regulators have shown an interest in interpreting the Wang et al. study, under a frequentist framework it is difficult to determine if the non-significant finding was caused by a lack of power or by the absence of an effect. Bayesian hypothesis testing does allow for quantification of evidence in favor of the absence of an effect. Findings Results of our Bayesian reanalysis of the three trials show ambiguous evidence for the primary outcome of clinical improvement and moderate evidence against the secondary outcome of decreased mortality rate. Additional analyses of three studies published after initial marketing approval support these findings. Conclusions We recommend that regulatory bodies take all available evidence into account for endorsement decisions. A Bayesian approach can be beneficial, in particular in case of statistically non-significant results. This is especially pressing when limited clinical efficacy data is available.

scholarly journals The role of genomic profiling in adolescents and young adults (AYAs) with advanced cancer participating in phase I clinical trials

2018 ◽  
Vol 95 ◽  
pp. 20-29 ◽  
Author(s):  
Terri Patricia McVeigh ◽  
Raghav Sundar ◽  
Nikolaos Diamantis ◽  
Stan B. Kaye ◽  
Udai Banerji ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Young Adults ◽  
Phase I ◽  
Advanced Cancer ◽  
Adolescents And Young Adults ◽  
Genomic Profiling ◽  
Phase I Clinical Trials
Sign in / Sign up

Export Citation Format

Share Document