Study Design and Data Analysis for Drug Therapy in Huntington Disease Patients

Section 4
4.1 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
individual’s 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).
4.2 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.
4.3 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.
4.4 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