scholarly journals Development of New Antimicrobials for Urogenital Gonorrhea Therapy: Clinical Trial Design Considerations

2019 ◽  
Vol 70 (7) ◽  
pp. 1495-1500 ◽  
Author(s):  
Edward W Hook ◽  
Lori Newman ◽  
George Drusano ◽  
Scott Evans ◽  
H Hunter Handsfield ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Antimicrobial Resistance ◽  
Trial Design ◽  
Clinical Trial Design ◽  
Evaluation Process ◽  
Antibiotic Resistant ◽  
Noninferiority Trials ◽  
New Antibiotics ◽  
Public Health Challenge ◽  
New Treatments

Abstract Gonorrhea remains a major public health challenge, and current recommendations for gonorrhea treatment are threatened by evolving antimicrobial resistance and a diminished pipeline for new antibiotics. Evaluations of potential new treatments for gonorrhea currently make limited use of new understanding of the pharmacokinetic and pharmacodynamic contributors to effective therapy, the prevention of antimicrobial resistance, and newer designs for clinical trials. They are hampered by the requirement to utilize combination ceftriaxone/azithromycin therapy as the comparator regimen in noninferiority trials designed to seek an indication for gonorrhea therapy. Evolving gonococcal epidemiology and clinical trial design constraints hinder the enrollment of those populations at the greatest risk for gonorrhea (adolescents, women, and persons infected with antibiotic-resistant Neisseria gonorrhoeae). This article summarizes a recent meeting on the evaluation process for antimicrobials for urogenital gonorrhea treatment and encourages the consideration of new designs for the evaluation of gonorrhea therapy.

Randomized Clinical Trial Design for Assessing Noninferiority When Superiority Is Expected

2007 ◽  
Vol 25 (31) ◽  
pp. 5019-5023 ◽  
Author(s):  
Boris Freidlin ◽  
Edward L. Korn ◽  
Stephen L. George ◽  
Robert Gray
Keyword(s):  
Clinical Trial ◽  
Randomized Clinical Trial ◽  
Trial Design ◽  
Clinical Trial Design ◽  
Tolerability Profile ◽  
Modest Improvement ◽  
Noninferiority Trials ◽  
New Treatment ◽  
Formal Test ◽  
Hybrid Trial

The randomized clinical trial (RCT) is the gold standard for definitive evaluation of new therapies. RCTs designed to show that the therapeutic efficacy of a new therapy is not unacceptably inferior to that of standard therapy are called noninferiority trials. Traditionally, noninferiority trials have required very large sample sizes. Sometimes, a new treatment regimen with a favorable toxicity and/or tolerability profile is also expected to have some modest improvement in efficacy. In such specialized settings we describe a hybrid trial-design approach that requires a dramatically smaller sample size than that of a standard noninferiority design. This hybrid design can naturally incorporate a formal test of superiority as well as noninferiority.

scholarly journals RE-ENGINEERING ALZHEIMER CLINICAL TRIALS: GLOBAL ALZHEIMER’S PLATFORM NETWORK

2016 ◽  
pp. 1-7
Author(s):  
J. Cummings ◽  
P. Aisen ◽  
R. Barton ◽  
J. Bork ◽  
R. Doody ◽  
...  
Keyword(s):  
Alzheimer’S Disease ◽  
Alzheimer's Disease ◽  
Clinical Trial ◽  
Trial Design ◽  
Clinical Trial Design ◽  
Fund Raising ◽  
Highly Qualified ◽  
Trial Site ◽  
Trial Efficiency ◽  
New Treatments

Alzheimer’s disease (AD) drug development is costly, time-consuming, and inefficient. Trial site functions, trial design, and patient recruitment for trials all require improvement. The Global Alzheimer Platform (GAP) was initiated in response to these challenges. Four GAP work streams evolved in the US to address different trial challenges: 1) registry-to-cohort web-based recruitment; 2) clinical trial site activation and site network construction (GAP-NET); 3) adaptive proof-of-concept clinical trial design; and 4) finance and fund raising. GAP-NET proposes to establish a standardized network of continuously funded trial sites that are highly qualified to perform trials (with established clinical, biomarker, imaging capability; certified raters; sophisticated management system. GAP-NET will conduct trials for academic and biopharma industry partners using standardized instrument versions and administration. Collaboration with the Innovative Medicines Initiative (IMI) European Prevention of Alzheimer’s Disease (EPAD) program, the Canadian Consortium on Neurodegeneration in Aging (CCNA) and other similar international initiatives will allow conduct of global trials. GAP-NET aims to increase trial efficiency and quality, decrease trial redundancy, accelerate cohort development and trial recruitment, and decrease trial costs. The value proposition for sites includes stable funding and uniform training and trial execution; the value to trial sponsors is decreased trial costs, reduced time to execute trials, and enhanced data quality. The value for patients and society is the more rapid availability of new treatments for AD.

scholarly journals Clinical Trial Design, Endpoints and Regulatory Considerations in Heart Failure

2019 ◽  
Vol 17 ◽  
Author(s):  
Giuseppe Rosano
Keyword(s):  
Heart Failure ◽  
Clinical Trial ◽  
Functional Capacity ◽  
Trial Design ◽  
Clinical Trial Design ◽  
New Drugs ◽  
Regulatory Agencies ◽  
Mortality And Morbidity ◽  
Increased Risk ◽  
New Treatments

After the withdrawal of flosequinan in the early 1990’s, because of an increased risk of mortality and fatal arrhythmias, the bar for the approval of new drugs in heart failure has been raised and regulatory agencies have requested evidence for the efficacy of new treatments on mortality and morbidity end-points. However, more recently, regulatory agencies have become more open to include the assessment of functional capacity as an efficacy endpoint, at least in selected subgroups of patients. Therefore, a new therapy for the treatment of heart failure can be approved if it improves survival and/or reduces hospitalisations or if it safely improves functional capacity. This article reviews clinical trial design, trial endpoints and regulatory issues in Heart Failure trials.

Integrating radiomics into clinical trial design

2019 ◽  
Vol 63 (4) ◽  
Author(s):  
Jessica J. Waninger ◽  
Michael D. Green ◽  
Catherine Cheze Le Rest ◽  
Benjamin Rosen ◽  
Issam El Naqa
Keyword(s):  
Clinical Trial ◽  
Trial Design ◽  
Clinical Trial Design

scholarly journals X-change symposium: status and future of modern radiation oncology—from technology to biology

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Stefanie Corradini ◽  
Maximilian Niyazi ◽  
Dirk Verellen ◽  
Vincenzo Valentini ◽  
Seán Walsh ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Radiation Oncology ◽  
Trial Design ◽  
Rapid Development ◽  
Clinical Trial Design ◽  
Current Status ◽  
Treatment Modalities ◽  
Special Treatment ◽  
Image Guided Radiotherapy

AbstractFuture radiation oncology encompasses a broad spectrum of topics ranging from modern clinical trial design to treatment and imaging technology and biology. In more detail, the application of hybrid MRI devices in modern image-guided radiotherapy; the emerging field of radiomics; the role of molecular imaging using positron emission tomography and its integration into clinical routine; radiation biology with its future perspectives, the role of molecular signatures in prognostic modelling; as well as special treatment modalities such as brachytherapy or proton beam therapy are areas of rapid development. More clinically, radiation oncology will certainly find an important role in the management of oligometastasis. The treatment spectrum will also be widened by the rational integration of modern systemic targeted or immune therapies into multimodal treatment strategies. All these developments will require a concise rethinking of clinical trial design. This article reviews the current status and the potential developments in the field of radiation oncology as discussed by a panel of European and international experts sharing their vision during the “X-Change” symposium, held in July 2019 in Munich (Germany).

scholarly journals Conceptual Framework to Support Clinical Trial Optimization and End-to-End Enrollment Workflow

2019 ◽  
pp. 1-10 ◽  
Author(s):  
Neha M. Jain ◽  
Alison Culley ◽  
Teresa Knoop ◽  
Christine Micheel ◽  
Travis Osterman ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Knowledge Representation ◽  
Conceptual Framework ◽  
Trial Design ◽  
Clinical Trial Design ◽  
Prospective Clinical Trial ◽  
Future Trends ◽  
Current State ◽  
Evaluation Strategies

In this work, we present a conceptual framework to support clinical trial optimization and enrollment workflows and review the current state, limitations, and future trends in this space. This framework includes knowledge representation of clinical trials, clinical trial optimization, clinical trial design, enrollment workflows for prospective clinical trial matching, waitlist management, and, finally, evaluation strategies for assessing improvement.

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