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Results for “trial design”

Search indexed PubMed citations on genomics, clinical trials, systematic reviews and public health. Explore titles, authors and supplied subject terms, then open the PubMed record.

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At least 19 recordsLinked to original sources
PubMed1976

Assessment of drugs in schizophrenia. Basic trial design.

Clinical trials with major tranquilizers must take into account the clinical features of patients with schizophrenia and pharmacokinetic and pharmacodynamic properties of the drug. The objectives of the trial must be carefully defined so that appropriate selection criteria for patients, rating instruments and dosage schedules can be selected. It is useful to monitor physiological and biochemical actions of major tranquilizers as well as the clinical effects.

Antipsychotic Agents↗
PubMed1979

Gingival bleeding in an experimental clinical trial design.

An experimental clinical trial design is described in which the onset of gingival bleeding was used as an indicator of early gingivitis. Twenty-nine dental students participated in a double-blind crossover trial utilising chlorhexidine as a known plaque inhibitor. The onset of gingival bleeding was assessed in three ways, one of which yielded a statistically significant difference in favour of the active mouthrinse (P = 0.02). The method has a role in screening agents believed to be active against gingivitis.

Adult↗
PubMed1979

Clinical trial design in cancer.

The design of clinical trials in cancer is a subject which features reasonably often among FRCR (Part 1) examination questions, and as such should be of more than passing interest to oncologists. It is also a subject which is gaining in relevance since the number of trials is increasing annually due in part to the many chemotherapeutic regimes which are being proposed. This paper which is based on a lecture given in Cambridge at the Hospital Physicists' Association Annual Conference in September 1978, is intended to act as an introduction to clinical trial design. References for further reading are given and, in particular, the extensive report on randomised clinical trials to the Medical Research Council's Leukaemia Steering Committee (Peto et al., 1977, 1978) is recommended.

Double-Blind Method↗
PubMed1979

Computer simulation as a tool for clinical trial design.

Use of a computer simulation technique as a tool for optimising the design of a clinical trial is described. One of several aspects of trial design facilitated by use of the technique is the estimation of the number of patients required in the trial. The simulation tool provides the basis for structured and disciplined discussion of all the issues to be considered in designing a trial. Copies of the simulation program are available from the author.

Clinical Trials as Topic↗
PubMed2026

A translational framework for early-phase inner-ear gene therapy: clinical trial design, regulatory strategy, and ethical considerations.

PURPOSE OF REVIEW: Hereditary hearing loss has historically been approached as a diagnostic category rather than a therapeutically modifiable disease. Recent advances in molecular genetics, cochlear gene delivery, and first-in-human clinical trials are changing that. This review summarizes contemporary progress in the genetics of hearing loss, with emphasis on emerging gene-based therapies, clinical trial design, regulatory and ethical considerations, and practical implications for otolaryngologists as biologic treatment enters clinical practice. RECENT FINDINGS: Early clinical trials targeting OTOF -related DFNB9 deafness have demonstrated satisfactory safety profiles and meaningful auditory recovery, establishing the first proof-of-concept for cochlear gene therapy in humans, culminating in the April 2026 FDA approval of Otarmeni. Genetic diagnoses are increasingly informing prognosis, cochlear implant counseling, and therapeutic candidacy. Preclinical research continues to expand toward recessive, dominant, and syndromic hearing loss using gene replacement, antisense, RNA interference, and genome-editing strategies. Substantial challenges remain, including heterogeneous outcome measures, uncertain long-term efficacy, regulatory complexity, and inequitable global access. SUMMARY: The genetics of hearing loss is transitioning from a diagnostic modality to an interventional one. Widespread clinical impact will require advances in vector engineering, equitable implementation, multidisciplinary counseling, and integration with established rehabilitation pathways. For otolaryngologists, genetic literacy is becoming essential to contemporary hearing care.

Humans↗
PubMed2025

Novel approaches to clinical trial design in cancer neuroscience.

The emerging field of cancer neuroscience has revealed profound bidirectional interactions between the nervous system and cancer cells, identifying novel therapeutic vulnerabilities across diverse malignancies. This review examines the unique challenges and strategies for translating these insights into effective therapies. We propose innovative approaches to overcome these barriers through drug repurposing, enhanced biomarker development, and optimized trial designs. Repurposing neuroactive drugs with established safety profiles offers an accelerated path to clinical impact, particularly for targeting glutamatergic, adrenergic, and neurotrophic signaling pathways. Emphasizing mitigation of neurotoxicity and improved patient quality of life will be paramount moving forward. Repurposed agents that show preliminary potential for "dual use" (i.e., simultaneous toxicity mitigation and synergistic anti-tumor effects) are highlighted for special consideration. Master protocols and window-of-opportunity trials provide platforms to rapidly validate mechanisms while addressing patient-centered outcomes. By systematically addressing these foundational elements across disciplines, cancer neuroscience can translate its profound mechanistic insights into meaningful therapeutic advances for patients with treatment-resistant malignancies.

Humans↗
PubMed2026

Select Contemporary Statistical Concepts in Heart Failure Clinical Trials: Insights From the Heart Failure Collaboratory.

Evolving statistical concepts and innovative trial designs for heart failure (HF) clinical trials seek to improve the conduct, efficiency, and likelihood of meaningful evidence generation crucial for advancing therapeutic development and optimizing patient care. HF trials with conventional statistical frameworks often require large sample sizes, long follow-up times, and high cost to generate sufficient evidence. Novel statistical methodologies would be of interest if they could address these issues while retaining or enhancing the clinical relevance and reliability of results. The HFC (Heart Failure Collaboratory), comprising clinical investigators, clinicians, statisticians, patients, government representatives, payors, and industry collaborators, leads efforts to improve HF research methodologies. HFC discussions have included statistical concepts such as the estimand framework, HR drift, and analytic methods, including the win ratio and restricted mean survival time, that have not been used frequently in HF trials. The estimand framework encourages precise definition and alignment of trial objectives with trial design. The win ratio method attempts to incorporate and prioritize multiple clinically meaningful outcomes by using a hierarchy of clinical importance. The restricted mean survival time provides an alternative to the HR as a measure of therapeutic effect by quantifying the mean time gained or lost during a fixed time after randomization. This paper provides a critical review of some evolving HF trial design methodologies and statistical concepts for the HF community as discussed within the HFC. Our goal is to foster collaboration among diverse stakeholders and advance the development of effective treatments and improve patient care outcomes.

Heart Failure↗
PubMed1978

Lidocaine prophylaxis in acute myocardial infarction.

Primary ventricular fibrillation (PVF) occurs in approximately 3--10% of uncomplicated acute myocardial infarction (AMI) patients. The major reason for this variability in incidence appears to be population diferences in the time from symptom onset to admission. Other risk factors have not been consistently shown to influence the risk of PVF. Warning arrhythmias do not warn of impending PVF in from 20--80% of AMI patients; thus, antiarrhythmic therapy reserved for those with warning arrhythmias may fail to prevent PVF in a significant number of patients. Although a review of 13 controlled trials of lidocaine prophylaxis shows only two suggesting a protective effect from this drug, only one study was free of major defects in trial design. This trial showed a striking decrease in PVF incidence when lidocaine was employed. Lidocaine should probably be administered to all uncomplicated AMI patients during the first 48 hours after infarction. Its utility in preventing ventricular fibrillation in complicated AMI patients and in the very early AMI period is unclear.

Acute Disease↗
PubMed1979

The design of clinical studies to assess therapeutic efficacy in multiple sclerosis.

Poorly designed trials of therapy for multiple sclerosis (MS) can waste time and money, and may lead either to false hopes or to the overlooking of a potentially effective treatment. A well-designed trial may well develop useful scientific information even if the putative therapy fails to show any therapeutic effect. The diagnosis, clinical course, and definitions of the stages of MS are discussed as they relate to trials of therapy. The goals of such trials include favorable modification of an exacerbation, favorable modification or prevention of future exacerbations, effective treatment of the progressive stage, and improvement of function in the stable-deficit stage. There should be an orderly progression from a small preliminary trial to a modest pilot trial and, when indicated, a full trial. All types of trials require careful organization and management, appropriate selection of patients, and properly planned and recorded observations. The treatment contrast--how the new treatment will be evaluated--provides the essential structure of the trial. The hypothesis being examined, the treatment contrast, and the observations being made in the designed clinical trial will govern the form of the analysis and the nature of the interpretations. Each goal requires that specific strategies and design considerations be applied to preliminary, pilot, and full trials.

Clinical Trials as Topic↗
PubMed1977

Randomised controlled trial of treatment for mild hypertension: design and pilot trial. Report of Medical Research Council Working Party on Mild to Moderate Hypertension.

A multicentre pilot trial to assess the feasibility of undertaking a full-scale national trial of treatment for mild to moderate hypertension has been performed and is being continued. By February 1977 over 1800 patients had entered the trial and some have been under observation for three years. The results so far show that the definitive trial is administratively and scientifically feasible and ethically justified.

Adult↗
PubMed2026

Pragmatic gynecologic cancer clinical trials: statements and roadmap from the Gynecologic Cancer InterGroup Chicago Brainstorming Meeting.

Randomized controlled trials remain fundamental to evidence generation in oncology but are increasingly complex, costly, and often misaligned with real-world practice. Traditional explanatory trials, designed under ideal, controlled conditions, frequently enroll highly selected populations, limiting generalizability and underrepresenting key groups such as older adults, patients with comorbidities, and those from low- and middle-income countries. Pragmatic clinical trials offer an alternative by evaluating interventions under routine care conditions, with broader eligibility, simplified procedures, and patient-centered outcomes. To address these challenges, the Gynecologic Cancer InterGroup convened an international brainstorming meeting in May 2025 with multi-disciplinary experts, patients, and advocates to define priorities and develop a roadmap for pragmatic trials in gynecologic oncology. Key discussions emphasized embedding trial design within routine care, aligning eligibility criteria and procedures with standard practice, minimizing non-essential data collection, and prioritizing outcomes meaningful to patients, including quality of life. Innovative designs such as registry-based randomized trials, trials-within-cohorts, and cluster randomization were highlighted as feasible approaches to improve efficiency while preserving internal validity. Integration of patient-reported outcomes and real-world data was considered achievable when carefully streamlined. Major challenges identified included regulatory heterogeneity, consent complexity, data interoperability, and funding limitations, particularly in multi-national settings. Proposed solutions include simplified consent models, centralized ethics processes, hybrid funding strategies, and the responsible use of artificial intelligence to enhance patient identification, recruitment, and potential development of synthetic control arms. Patient engagement was recognized as essential to ensure relevance, feasibility, and equity. Incorporation of patient-reported outcomes was discussed as key to informing acceptance and tolerability. In summary, pragmatic trials within Gynecologic Cancer InterGroup represent a critical pathway to generate efficient, inclusive, and practice-changing evidence in gynecologic cancers across diverse health care settings.

Humans↗
PubMed1976

Evaluation of intrinsic sympathomimetic activity of beta-adrenoceptor blocking drugs in the treatment of patients with angina pectoris.

Beta-adrenoceptor blocking drugs with intrinsic sympathomimetic activity (ISA) may be less effective in the treatment of patients with angina pectoris than some others that lack this property. A review of 14 trials comparing beta-adrenoceptor blocking drug with ISA and those without ISA in angina pectoris has been made. The overall picture emerges from both acute and chronic studies using subjective and objective endpoints, that there is no striking difference in effectiveness between the two kinds of beta-adrenoceptor blocking drugs. The one exception is pindolol (a drug with ISA) which, at higher doses, has been shown to be consistently worse than propranolol (a drug without ISA). The reasons for the similarity between propranolol and other bets-blocking drugs with ISA in the trials cited are either that the trial design was defective (the trials were mainly fixed dose comparisons) or that the stimulant effects of those drugs with ISA is not of sufficient magnitude to make a difference. It is suggested that further carefully constructed clinical trials should be carried out before the second reason can be accepted.

Acute Disease↗