Assessment of Drug Therapy for Huntington Disease: Study Design and Treatment Protocols

This presentation outlines the study design, objectives, treatment protocols, and data analysis for the assessment of drug therapy for Huntington Disease. The main objective is to assess the effectiveness and safety of PBT2 and Beraxotene on improving motor function and neuron survivability. The study will adopt a randomized double-blinded placebo-controlled design, with patients randomly assigned to receive placebo, low dose, or high dose therapy. The primary outcomes include drug therapy feasibility, effectiveness, and safety, as well as changes in total functional capacity. Data analysis will involve descriptive statistics and covariance analysis. The study will be conducted based on ethical principles for medical research ethics.

Study design
Presentation

Objectives of study
• Main objective is assessment of drug of PBT2
and Beraxotene on improvement of the motor
function and neuron survivability
• Primary outcomes;
– Drug therapy feasibility
– Effectiveness and safety of drug therapy
– Change from baseline to end of 3 years evaluation
on total functional capacity

• Primary evaluation based on recruitment,
adherence and retention rates
• Secondary otucomes measure of motor
function assessed using UHDRS TMS
• Specific assessment on dysarthria, tongue
protrusion , bradykinesia and luria rigidity of
arms functionality
• Cognitive assessment will be assessed using
verbal fluency on tests using MMSE

Data analysis
• Descriptive data will be used to asses on the
evaluation, eligibility , recruitment and
retention rates at 95% confidence levels
• Covariance analysis will be used to control for
age factor, UHDRS, gender and baseline
assessments
• Primary tolerability will be assessed using
frequency and occurrence of adverse effects
and lab results

Treatment protocols
• One year recruitment period
• Patients suffering from Hunnington disease.
• Screening of the participants will be done
using screening log
• Blinding will be conducted by blinded
assessors
• Incidence of unbinding will be recorded

• Intervention group involve a control group
who will be given placebo
• Group two will be given a low dose drug while
group three will be given high dose drug
• Assessment based on administration of drugs
on weekly basis and follow up visits done on
monthly basis while consultation will take
place for three years

Study design
• 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
• Study will be done based on ethical principles
for medical research ethics

1200
Screened
1000 enrolled
and
randomised
200 declined
330 assigned
placebo
335 assigned low
dose therapy
335 assigned
high dose
therapy

Randomization
• Randomization will be performed during the
baseline assessment
• Group assignment will be relayed to the
respective site coordinators
• Randomization ratio will be used in the ration
of 1:1 on eligible participants
• 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
• 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 for equal
treatment
END