Combination Of Drug Of PBT2 And Beraxotene †MyAssignmenthelp.com

Question: Discuss about the Combination Of Drug Of PBT2 And Beraxotene. Answer: Objectives The primary objective is the combination of drug of PBT2 and Beraxotene being an improvement in the motor function and neuron survivability. The primary outcomes of this study would be the feasibility assessment, effectiveness and safety of the drug therapy. Safety factor would be accessed through review of weekly health diaries and falls diaries among the participants. This will include prior knowledge of falls, changes in medications and hospital admissions. Diaries for recording would be provided to participants during baseline assessment and returned monthly basis. Another primary outcome of the study would be the measure of completion of assigned medication to assess its tolerability. Further the primary objective of this study is to detremeine the influcen of the drugs therapy compared to the placebo effect. Also primary outcome of interest will be change from the baseline assessment to end of three years gaining total functional motor capacity. This will seek establishment on the impact progression on the change on the total functional capacity using UHDRS between baseline and three years. The primary objective of evaluation is the recruitment, adherence rates and retention of patients. The recruitment phase will be assessed under the use of site recruitment logs. The retention rate of participants will be measured using the percentages of individuals who will have completed the intervention. Adherence rates will be assessed using the percentages of individuals sessions which have been completed. In this case successful intervention rate will be defined as at least 75% of the supervised sessions and 75% of other unsupervised sessions, (Harrison et al., 2013). Secondary outcome measure of motor function will be assessed using modified motor score, a subset of UHDRS TMS, this has been chosen due to the specific focus of the voluntary impairments. This component will assess dysarthria, tongue protrusion , bradykinesia and luria rigidity of arms. UHDRS is an analytical tool to assess motor function, behavioural aspects, behavioural and functional ability. Outcomes in this case will involve motor score, behavioural frequency, functional ability assessment and independent scale. Cognitive assessment will be assessed using verbal fluency on tests. The MMSE being a common validated tool, will be used for general purpose of cognitive assessment, in this case lower score will signify impairment, (Busse et al., 2013). Data Analysis Descriptive data will be used to asses on the evaluation, eligibility , recruitment and retention rates at 95% confidence levels. Graphical illustrations will be used to check the distribution associated with data outcome. The primary and secondary analyses will be made comparable with among interventions and control groups. Covariance analysis will be used to control for age factor, UHDRS, gender and baseline assessments. Analysis of the outcomes will be assessed on the presumption of intention to treat basis. Primary outcome measure will be measured on the change from the baseline information to the end of the study period. This will be effected using the hunnington motor scale on the UHDRS. Further safety measures and tolerability and adverse effects will be assessed based on the assigned dosage. The primary tolerability will be assessed using frequency and occurrence of adverse effects and lab results. The efficacy measures will be measured based on the changes on baseline and monthly basis on UHDRS Mmse outcomes will be compared on the treatment groups using repeated measures and covariance of analysis as mentioned above. Analysis of the secondary outcome measures will be assessed on the UHDRS tools further demographic characteristics such as medical history of the patients. Treatment protocols The recruitment period will be January 2017 to December 2017. Patients suffering from Huntington disease will be enrolled in the study. Patients receiving clinical care and attending assessments will be given trial information. Screening of the participants will be done using screening log which records of number of people who have been approached on the trial and eligibility. Blinding will be conducted by blinded assessors. Site coordinators will be requested not to make any disclosure on allocation of assessors. In order to manage confounding factors, incidence of unbinding will be recorded. Intervention group involve a control group who will be given placebo to imitate the drug. Group two will be given a low dose drug while group three will be given high dose drug. This will measure the level for drug effectiveness on managing Huntington disease among the elderly. The three group of participants will entail administration of group one being categorised as control group getting no treatment. Group tow will be given lose drug therapy and group three will be given high drug therapy treatment therapy. These doses will be administered on weekly basis and follow up visits done on monthly basis while consultation will take place for three years. The consultation phase will entail physician assessments using the UHDRS to measure the severity of the disease. Study design This study will adopt a randomised double blinded placebo controlled study. Patients will be randomly assigned into the three groups whom they will receive placebo, low dose of 10mg or high dose of 100 mg drug. The study will take a period of 3 years of consultation and medication follow-ups on monthly basis. The effects of study intervention will be compared to that of the control treatment and assigned to the groups stated. The patients allocated the control group will be given a placebo treatment. The use of placebo effects on clinical trials has been used to justify on no treatment as no standards treatment is available. The study groups will be constructed to be truly comparable. This will be done through standardization. This can be done through intake of study medication and methods of assessment to be clearly identified. Double blinding study design ensures that biasness is eliminated through blinding as another form of randomization. In double blinding neither of the study participants will be privy of the of the treatment plans being assigned. Thus with this study the physicians will be blinded so as to ensure that the objectivity of the study is ensured. Blinding of physicians is crucial in ensuring that factors such as attitude could not affect the reliability of taking medicines, compliance and treatment response. This study will be done based on ethical principles for medical research which involve human subjects as adopted by the general assembly of world medical association of 1964. Further the study will be conducted in accordance with international conference on harmonization on best clinical practices and application university ethical guidelines. Randomization Randomization in cli9nical studies are beneficial in that it reduces bias and offers fair treatment of prognosis and not based on individual prognostic patients characteristics. Further in this study randomization will reduce the effects of confounding factors. In this study randomisation of the study participants will be done the three balanced groups by offering treatment and having control group. In RCT designs patients are often randomised and assigned different treatment groups. This is to ensure that confounding factors are equally divided among the different groups in the study. Randomization of study participants trial randomization will be performed during the baseline assessment. Group assignment will be relayed to the respective site coordinators, who will be able to communicate and inform the participant on the complete ion of the baseline survey. Randomization ratio will be used in the ration of 1:1 on eligible participants. This will be aided by computer generated block randomization plan. Minimization procedure will be effective as a way of achieving balance amongst the groups. Variables which will be used are age, site, gender and UHDRS TMS. A masked randomization code will be utilised so as to hide exposure and privy knowledge of the study. These confounding factors must be controlled as they can affect the patient treatment phase. Thus randomization in this case will be of essence in ensuring that statistical analysis of covariance is done. Participants will be stratified in form of gender so as to ensure an equal number of male and female participants. Equal balancing of the groups will be done so as to ensure that there is equal treatment of participants in control and experimental group. References Harrison, D.J., Busse, M., Openshaw, R., Rosser, A.E., Dunnett, S.B. and Brooks, S.P., 2013. Exercise attenuates neuropathology and has greater benefit on cognitive than motor deficits in the R6/1 Huntington's disease mouse model. Experimental neurology, 248, pp.457-469. Busse, M., Quinn, L., Debono, K., Jones, K., Collett, J., Playle, R., Kelly, M., Simpson, S., Backx, K., Wasley, D. and Dawes, H., 2013. A randomized feasibility study of a 12-week community-based exercise program for people with Huntington's disease. Journal of Neurologic Physical Therapy, 37(4), pp.149-158.