Developing an Aggregator Mechanism for Late-Stage Clinical Trials of Neglected Disease Product Candidates

2020 ◽  
Author(s):  
Gavin Yamey ◽  
Marco Schäferhoff ◽  
Mary H. Moran ◽  
Mohamed Mustafa Diab ◽  
Kaci Kennedy McDade ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Late Stage ◽  
Neglected Disease

Utilization and reach of the Fight Colorectal Cancer Late Stage MSS CRC Clinical Trial Finder.

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. 3561-3561
Author(s):  
Reese Garcia ◽  
Andrea Dwyer ◽  
Sharyn Worrall ◽  
Christopher Ryan Heery ◽  
Dustin A. Deming ◽  
...  
Keyword(s):  
Colorectal Cancer ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Late Stage ◽  
Stage Iv ◽  
The United States ◽  
Web Based ◽  
The United Kingdom ◽  
Patient Feedback ◽  
Selection Of

3561 Background: Colorectal cancer (CRC) remains one of the most lethal cancer killers worldwide. Recently, research has shown great strides in the treatment of MSI-H mCRC using immunotherapy, however, these treatments have not been effective in MSS patients, who make up a majority of CRC cases. Due to numerous barriers, clinical trial enrollment numbers remain as low as 9% of the eligible populations, despite the reliance of many late stage CRC patients on clinical trials for treatment. Perhaps greatest of these barriers is the lack of meaningful patient-facing clinical trial matching, making advances in MSS mCRC IO clinical research extremely slow. Methods: In May 2017, Fight Colorectal Cancer (FightCRC) launched its web-based trial finder, The Late Stage MSS Trial Finder (TF) with the late Dr. Tom Marsilije, a stage IV CRC patient and researcher, and Flatiron Health. The TF is a publicly available immunotherapy-based repository of clinical trials. An algorithm automatically codes for a subset of trials from ClinicalTrials.gov to be uploaded into the tool, and trained FightCRC advocates follow a strategic logic flow to prioritize trials of highest potential benefit and lowest risk for patients. Results: Between 30 and 100 trials are uploaded into the TF for curation each week. A total of 378 trials have been indexed in the TF to date. In February 2019, a mobile application was introduced. From May 2017 to January 2019, the tool has seen > 15,000 users, yielding 26,000 searches in 105 countries; primarily from the United States, China, the United Kingdom, Canada, and France. On average, users navigate to 2.5 pages and spend > 2.5 minutes per use. Providers are using this as a tool to find clinical trials and to discuss these options in real time. CRC patient feedback confirmed the platform functionality. Conclusions: The Trial Finder is a unique tool for MSS mCRC patients pursuing clinical trials. The success of the tool may be attributed to patient focused selection of therapies that show promise. The FightCRC late stage MSS CRC trial finder is being widely utilized, in diverse settings. With our patient curators and Medical Advisory Board, FightCRC will improve the search features and outcome tracking with user feedback. The goal for the TF is to address key barriers to patient entry into clinical trials and promote patient-provider discussions to inform decision making.

The association of adaptive early phase study design and late phase study results in oncology using clinicaltrials.gov data.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e14078-e14078
Author(s):  
Donna Elise Levy ◽  
Srikanta Banerjee ◽  
Fred K Tabung
Keyword(s):  
Clinical Trials ◽  
Study Design ◽  
Late Stage ◽  
Early Phase ◽  
Late Phase ◽  
Adaptive Methods ◽  
Positive Outcome ◽  
Cancer Type ◽  
Traditional Methods ◽  
Phase Study

e14078 Background: Traditional methods such as the 3+3 design developed in the 1940s continue to be used in the majority of early phase oncology studies. Researchers have found that the traditional methods identify the appropriate dose level only 30% of the time. Patients are also exposed to sub-therapeutic doses due to the conservative methods. With these limitations on traditional design options, innovative methods need to be developed, adopted and assessed. Methods: This quantitative study assessed the association of the design methods (adaptive versus traditional) used for early phase oncology studies (adaptive versus traditional) and the outcome of late stage clinical trials. Differences by cancer type and by drug classification were also assessed. A sample of studies for this analysis was extracted from the National Institute of Health Clinical registry and results database. The data used for the analysis was extracted by the Clinical Trials Transformation Initiative (CTTI) Aggregate Analysis of ClinicalTrials.gov (AACT). Results: When assessing study design and outcome, there were lower odds of a positive outcome when adaptive methods were used though this association was not statistically significant (OR [95% highest posterior density (HPD)]:0.66 [0.20, 1.21]). Among the different drug types, using adaptive compared to traditional methods was associated with significantly higher odds of a positive outcome for taxanes, OR: 2.75, 95% HPD: 1.01, 5.16) and other, OR: 3.23, 95% HPD: 1.58, 5.46) but no association among studies of monoclonal antibodies or protein kinase inhibitors. There were no significant associations between early phase study design and outcome in late phase studies by cancer type (lung, breast, other). Conclusions: While results associated with the use of adaptive methods were not significant, further research should be conducted using all completed oncology clinical trials in the database to more precisely determine the relationship between adaptive study design in early phase oncology studies and outcomes in late stage studies. In addition, improvements in traditional versus adaptive design capture in the ClinicalTrials.gov database needs to be considered.

scholarly journals Metastatic Urachal Carcinoma Treated With Several Different Combined Regimens: A Case Report

2021 ◽  
Vol 11 ◽  
Author(s):  
Han Zheng ◽  
Wei Song ◽  
Xiemin Feng ◽  
Hong Zhao
Keyword(s):  
Elderly Patient ◽  
Monoclonal Antibody ◽  
Clinical Trials ◽  
Case Report ◽  
Late Stage ◽  
Pulmonary Metastases ◽  
Urachal Carcinoma ◽  
First Report ◽  
Rare Cancers ◽  
Therapeutic Decisions

Urachal carcinoma is a rare bladder malignance. This study presents a case of an elderly patient with urachal carcinoma who was found to have pulmonary metastases 1 year after 5 recurrent resections. The patient was treated with up to 7 different chemotherapy regimens, including a VEGF monoclonal antibody and anti-PD-1 antibody. This is the first report of PD-1 antibody being used in patients with urachus, although the disease progressed after only four cycles of the application. The patient’s disease was controlled by the FOLFIRI combined with the VEGF monoclonal antibody regimen. The most prominent issues at present are the difficulty of obtaining drugs for rare cancers and the lack of late-stage clinical trials to guide therapeutic decisions.

scholarly journals Value-Generating Exploratory Trials in Neurodegenerative Dementias

Neurology ◽  
2021 ◽  
pp. 10.1212/WNL.0000000000011774
Author(s):  
Lauren G. Friedman ◽  
Nicholas McKeehan ◽  
Yuko Hara ◽  
Jeffrey L. Cummings ◽  
Dawn C. Matthews ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Late Stage ◽  
Development Program ◽  
Clinical Development ◽  
Advisory Panel ◽  
Phase 2 ◽  
Proof Of Concept ◽  
Dose Selection ◽  
Clinical Development Program ◽  
Neurodegenerative Dementias

Drug development for Alzheimer's disease and other neurodegenerative dementias, including frontotemporal dementia, has experienced a long history of phase 2 and phase 3 clinical trials that failed to show efficacy of investigational drugs. Despite differences in clinical and behavioral characteristics, these disorders have shared pathologies and common challenges in designing early-phase trials that are predictive of late-stage success. Here, we discuss exploratory clinical trials in neurodegenerative dementias. These are generally phase 1b or phase 2a trials that are designed to assess pharmacologic effects and rely on biomarker outcomes, with shorter treatment durations and fewer patients than traditional phase 2 studies. Exploratory trials can establish go/no-go decision points, support proof-of-concept and dose selection, and terminate drugs that fail to show target engagement with suitable exposure and acceptable safety profiles. Early failure saves valuable resources including opportunity costs. This is especially important for programs in academia and small biotechnology companies but may be applied to high-risk projects in large pharmaceutical companies to achieve proof-of-concept more rapidly at lower costs than traditional approaches. Exploratory studies in a staged clinical development program may provide promising data to warrant the substantial resources needed to advance compounds through late-stage development. To optimize the design and application of exploratory trials, the Alzheimer's Drug Discovery Foundation and the Association for Frontotemporal Degeneration convened an advisory panel to provide recommendations on outcome measures and statistical considerations for these types of studies and study designs that can improve efficiency in clinical development.

scholarly journals Clinical trials and late-stage drug development for Alzheimer's disease: an appraisal from 1984 to 2014

2014 ◽  
Vol 275 (3) ◽  
pp. 251-283 ◽  
Author(s):  
L. S. Schneider ◽  
F. Mangialasche ◽  
N. Andreasen ◽  
H. Feldman ◽  
E. Giacobini ◽  
...  
Keyword(s):  
Alzheimer’S Disease ◽  
Alzheimer's Disease ◽  
Clinical Trials ◽  
Drug Development ◽  
Late Stage
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