What is the probe design for a randomized controlled trial?
The randomized controlled trial (RCT) uses a multicenter, prospective, randomized, open-label, blinded end point study design (PROBE) [19] to compare eCBT-I with TAU. A total of 48 patients will be randomly assigned to the eCBT-I or TAU group. ...
What is a probe study?
Prospective Randomized Open Blinded End-point (PROBE) study. A 'read' is counted each time someone views a publication summary (such as the title, abstract, and list of authors), clicks on a figure, or views or downloads the full-text.
How can I get a copy of the probe study?
To read the full-text of this research, you can request a copy directly from the authors. A novel design for intervention studies is presented, the so called PROBE study (Prospective Randomized Open, Blinded End-point). This design is compared to the classical double-blind design.
What are the advantages of probe design?
This design is compared to the classical double-blind design. Among the advantages of the PROBE design are lower cost and greater similarity to standard clinical practice, which should make the results more easily applicable in routine medical care.
What is the Probe study?
The Patient Reported Outcomes, Burdens, and Experiences (PROBE} Study aims to develop a new global tool to enhance the direct patient-voice in health care decision- making. Government and private payers increasingly value data based on patient- centered outcomes research as part of the overall cost-benefit evaluation of high-cost care and treatment of diseases such as haemophilia.
What is a study initiator?
Regardless of the outcome of a study, the study initiator is dedicated to openly providing information on the trial to healthcare professionals and to the public, both at scientific congresses and in peer-reviewed journals. The study initiator will comply with all requirements for publication of study results.
How does the study initiator maintain confidentiality?
The study initiator maintains confidentiality standards by coding each subject enrolled in the study through assignment of a unique subject identification number. This means that subject names are not included in datasets that are transmitted to any study initiator's location.
Can you follow patients after they withdraw from a study?
The primary reason for withdrawal from the study should be documented on the appropriate form. Patients will not be followed for any reason after consent has been withdrawn.
Does Probe receive SAE reports?
Although it is not anticipated that the PROBE Study Team will receive reports of Adverse Events (AE) / Serious Adverse Events (SAE) within the scope of the Study, if the Investigators are made aware of an AE / SAE involving a product manufactured by a study sponsor through the conduct of the study, the Investigators commit to notify sponsor within one (1) business day of actually becoming aware of any significant product complaint, unexpected adverse event, or governmental investigation or inquiry involving a sponsor's product. Study questionnaire may not be reviewed in "real-time." It is understood the study questionnaires may be collected but not reviewed or entered into the PROBE database until the completion of an individual country sampling exercise.
What is a 24-hour probe study?
The 24-hour probe study measures the pH (acid level) in your food pipe (esophagus). It is often done along with the monitoring of nonacid and air regurgitation in a 24-hour pH/impedance probe study.
Why might I need a 24-hour probe study?
The 24-hour probe study is used to see if you have GERD. It may also be used to see if your GERD treatment is helping to reduce your stomach acid level.
What are the risks of a 24-hour probe study?
The 24-hour probe study has no real risks. There are only a few small side effects. It may not feel very good when your healthcare provider inserts the tube and when you wear it. When the tube is inserted, you may feel:
How do I get ready for a 24-hour probe study?
Your healthcare provider will explain how the 24-hour probe study is done. Be sure to ask any questions you may have.
What happens during a 24-hour probe study?
The inside of your nose will be numbed. This will make you more comfortable as the tube and sensor are inserted. Your healthcare provider will then put the tube (with multiple sensors on it) into your nose. They will push the tube down your throat while you swallow.
What happens after a 24-hour probe study?
After 24 hours, you will go back to your healthcare provider. They will take out the tube and any sensors. Your healthcare provider will check the results and compare them with your diary. They will score the results. The results will show if acid levels in your esophagus are too high.
What is the purpose of clinical trial design?
Clinical trial design is an important aspect of interventional trials that serves to optimize, ergonomise and economize the clinical trial conduct. The purpose of the clinical trial is assessment of efficacy, safety, or risk benefit ratio. Goal may be superiority, non-inferiority, or equivalence.
Why are pilot studies ineligible?
In a conventional pilot study, participants are often ineligible for analysis along with cases in future definitive studies due to concerns about selection bias, carry-over, and training effects. Where patients are few in number as in case of rare diseases, allocating them to a pilot study rather than the definitive study could be seen as a wasteful approach. In an internal pilot study, the first phase of the study is designated a “pilot phase,” and the study is continued till this sample size is achieved (definitive phase) and analysis incorporates the pilot subjects also. In contrast to external pilots, internal pilots can be large, as they do not “use up” eligible patients and do not require additional time or funds.
What is split body trial?
Randomization by body halves or paired organs (Split Body trials) – This is a scenario most often used in dermatology and ophthalmic practice where one intervention is administered to one half of the body and the comparator intervention is assigned to other half of the body.
What is the purpose of the Declaration of Helsinki?
The Declaration of Helsinki mandates the use of standard treatment as controls. Dose-comparison concurrent control – Different doses or regimens of same treatment are used as active arm and control arm in this design. The purpose is to establish a relationship between dose and efficacy/safety of the intervention.
What is controlled trial?
Controlled trials allow discrimination of the patient outcome from an outcome caused by other factors (such as natural history or observer or patient expectation). Choosing a right control at the right dose and right frequency is pivotal to trial success. The controls which can be used are:
Can effects of intervention during first period carry over into second period?
Effects of intervention during first period should not carry over into second period. In case of suspected carry over effects more complex sequences are needed which increase study duration and thus the chance of drop outs
Is immunotherapy for warts self-resolving?
In immunotherapy in warts, it is imperative to avoid an uncontrolled study. Warts can be self-resolving and hence the efficacy of immunotherapy as opposed to the self-resolution compromises the validity of the results.
When two groups are prognostically balanced, with the exception of the intervention, and an investigator observes answer
If two (or more) groups are prognostically balanced, with the exception of the intervention, and an investigator observes a difference in outcomes, a sound argument can be made attributing the difference in result to the intervention under study.
Why do clinicians use randomized controlled trials?
Clinicians, institutions, and policy makers use results from randomized controlled trials to make decisions regarding therapeutic interventions for their patients and populations. Knowing the effect the intervention has on patients in clinical trials is critical for making both individual patient as well as population-based decisions.
Does excluding patients from a randomized trial increase the risk of bias?
Although with few exceptions, excluding patients from a randomized trial will increase the risk of bias in a study.6,7Theoretically, the only way patients can be lost from a study and not increase the risk of bias is if the patients who are lost are prognostically identical to the patients who remain.
What is the Probe study?
A novel design for intervention studies is presented, the so called PROBE study (Prospective Randomized Open, Blinded End-point). This design is compared to the classical double-blind design. Among the advantages of the PROBE design are lower cost and greater similarity to standard clinical practice, which should make the results more easily applicable in routine medical care. Since end-points are evaluated by a blinded end-point committee it is obvious that there should be no difference between the two types of trials in this regard.
What is the purpose of the Net IPP trial?
The New Technologies for Intensive Prevention Programs (NET-IPP) Trial will investigate if a long-term web-based prevention program after myocardial infarction (MI) will reduce clinical events and risk factors. In a genetic sub study the impact of disclosure of genetic risk using polygenic risk scores (PRS) will be assessed.Study designPatients hospitalized for MI will be prospectively enrolled and assigned to either a 12-months web-based intensive prevention program or standard care. The web-based program will include telemetric transmission of risk factor data, e-learning and electronic contacts between a prevention assistant and the patients. The combined primary study endpoint will comprise severe adverse cardiovascular events after 2 years. Secondary endpoints will be risk factor control, adherence to medication and quality of life. In a genetic sub study genetic risk will be assessed in all patients of the web-based intensive prevention program group by PRS and patients will be randomly assigned to genetic risk disclosure vs. no disclosure. The study question will be if disclosure of genetic risk has an impact on patient motivation and cardiovascular risk factor control.Conclusions The randomized multicenter NET-IPP study will evaluate for the first time the effects of a long-term web-based prevention program after MI on clinical events and risk factor control. In a genetic sub study the impact of disclosure of genetic risk using PRS will be investigated.
What is p.Arg14del? What are its causes?
Background The p.Arg14del (c.40_42delAGA) phospholamban (PLN) pathogenic variant is a founder mutation that causes dilated cardiomyopathy (DCM) and arrhythmogenic cardiomyopathy (ACM). Carriers are at increased risk of malignant ventricular arrhythmias and heart failure, which has been ascribed to cardiac fibrosis. Importantly, cardiac fibrosis appears to be an early feature of the disease, occurring in many presymptomatic carriers before the onset of overt disease. As with most monogenic cardiomyopathies, no evidence-based treatment is available for presymptomatic carriers. Aims The PHOspholamban RElated CArdiomyopathy intervention STudy (iPHORECAST) is designed to demonstrate that pre-emptive treatment of presymptomatic PLN p.Arg14del carriers using eplerenone, a mineralocorticoid receptor antagonist with established antifibrotic effects, can reduce disease progression and postpone the onset of overt disease. Methods iPHORECAST has a multicentre, prospective, randomised, open-label, blinded endpoint (PROBE) design. Presymptomatic PLN p.Arg14del carriers are randomised to receive either 50 mg eplerenone once daily or no treatment. The primary endpoint of the study is a multiparametric assessment of disease progression including cardiac magnetic resonance parameters (left and right ventricular volumes, systolic function and fibrosis), electrocardiographic parameters (QRS voltage, ventricular ectopy), signs and/or symptoms related to DCM and ACM, and cardiovascular death. The follow-up duration is set at 3 years. Baseline results A total of 84 presymptomatic PLN p.Arg14del carriers ( n = 42 per group) were included. By design, at baseline, all participants were in New York Heart Association (NHYA) class I and had a left ventricular ejection fraction > 45% and < 2500 ventricular premature contractions during 24-hour Holter monitoring. There were no statistically significant differences between the two groups in any of the baseline characteristics. The study is currently well underway, with the last participants expected to finish in 2021. Conclusion iPHORECAST is a multicentre, prospective randomised controlled trial designed to address whether pre-emptive treatment of PLN p.Arg14del carriers with eplerenone can prevent or delay the onset of cardiomyopathy. iPHORECAST has been registered in the clinicaltrials.gov-register (number: NCT01857856).
What is PGE2 in caesarean delivery?
Introduction Labour induction in women with a previous caesarean delivery currently uses vaginal prostaglandin E2 (PGE2), which carries the risks of uterine hyperstimulation and scar rupture. We aim to compare the efficacy of mechanical labour induction using a transcervically applied Foley catheter balloon (FCB) with PGE2 in affected women attempting trial of labour after caesarean (TOLAC). Methods and analysis This single-centre non-inferiority prospective, randomised, open, blinded-endpoint study conducted at an academic maternity unit in Singapore will recruit a total of 100 women with one previous uncomplicated caesarean section and no contraindications to vaginal delivery. Eligible consented participants with term singleton pregnancies and unfavourable cervical scores (≤5) requiring labour induction undergo stratified randomisation based on parity and are assigned either FCB (n=50) or PGE2 (n=50). Treatments are applied for up to 12 hours with serial monitoring of the mother and the fetus and serial assessment for improved cervical scores. If the cervix is still unfavourable, participants are allowed a further 12 hours’ observation for cervical ripening. Active labour is initiated by amniotomy at cervical scores of ≥6. The primary outcome is the rate of change in the cervical score, and secondary outcomes include active labour within 24 hours of induction, vaginal delivery, time-to-delivery interval and uterine hyperstimulation. All analyses will be intention-to-treat. The data generated in this trial may guide a change in practice towards mechanical labour induction if this proves efficient and safer for women attempting TOLAC compared with PGE2, to improve labour management in this high-risk population. Ethics and dissemination Ethical approval is granted by the Domain Specific Review Board (Domain D) of the National Healthcare Group, Singapore. All adverse events will be reported within 24 hours of notification for assessment of causality. Data will be published and will be available for future meta-analyses. Trial registration number NCT03471858 ; Pre-results.
What is the goal of BLS training?
Background The goal for laypersons after training in basic life support (BLS) is to act effectively in an out-of-hospital cardiac arrest situation. However, it is still unclear whether BLS training targeting laypersons at workplaces is optimal or whether other effective learning activities are possible. Aim The primary aim was to evaluate whether there were other modes of BLS training that improved learning outcome as compared with a control group, i.e. standard BLS training, six months after training, and secondarily directly after training. Methods In this multi-arm trial, lay participants ( n = 2623) from workplaces were cluster randomised into 16 different BLS interventions, of which one, instructor-led and film-based BLS training, was classified as control and standard, with which the other 15 were compared. The learning outcome was the total score for practical skills in BLS calculated using the modified Cardiff Test. Results Four different training modes showed a significantly higher total score compared with standard (mean difference 2.3–2.9). The highest score was for the BLS intervention including a preparatory web-based education, instructor-led training, film-based instructions, reflective questions and a chest compression feedback device (95% CI for difference 0.9–5.0), 6 months after training. Conclusion BLS training adding several different combinations of a preparatory web-based education, reflective questions and chest compression feedback to instructor-led training and film-based instructions obtained higher modified Cardiff Test total scores 6 months after training compared with standard BLS training alone. The differences were small in magnitude and the clinical relevance of our findings needs to be further explored. Trial Registration ClinicalTrials.gov Identifier: NCT03618888. Registered August 07, 2018—Retrospectively registered, https://clinicaltrials.gov/ct2/show/NCT03618888
Why are RCTs important?
These studies are particularly valuable when confounding cannot be adequately controlled by means other than randomization and when a large study is required to assess small risks. Randomization produces unbiased estimates of effect, and a simple study design limits costs so that large studies become feasible. If either the relative risk of adverse events is small or the absolute risk is small and confounding by indication is likely, RCTs, and particularly the LST design, may be the best solution for the postmarketing evaluation of medicine safety.
