Progress in clinical trial preparation and preclinical studies
A strategy and plan for securing approval from the regulatory authorities for clinical trials and, if successful, the candidate drug, has progressed over the quarter. Consultants specialising in working with the regulators to get such approvals have now been contractually retained and initial discussions had.
To assist with management and administration of the whole initiative, a hierarchical map has been created defining both the major and the sub-components of the plan. Nine major streams have been identified with 52 sub-components, each sub- component representing a significant project, or set of projects. This map constitutes the basis for detailed planning, which is well underway in some parts of some of the nine streams. The nine major streams are:
- biomarker investigations
- regulatory approvals
- trial participants
- clinical trial
- dose modelling
- candidate drug
- market development
- program management
Opportunities for applying for grant funding for the clinical trial are being regularly reviewed and the most suitable ones further investigated. Currently one application is pending decision and announcement by the Federal Minister for Health, which is expected in the near future.
Blood based biomarker study
The process of collecting patient blood samples and Spastic Paraplegia Rating Scale scores is ongoing, with ten patient samples having been collected.
Ethics protocol amendments have now been approved. HSP patients from the Royal North Shore Hospital Neurogenetics clinic, who have previously consented to being contacted regarding relevant research will be invited to participate via phone call. This approval will expedite the sample collection process.
In the mean time, the biomarker is being tested in patient fibroblast cells, a resource available in Professor Carolyn Sue’s laboratory at the Kolling Institute, Royal North Shore Hospital. Patient fibroblast cells, like blood, are a source of easily accessible non-neuronal patient samples.
The work is currently in progress and is being carried out by clinical trial team member Dr. Sue-Faye Siow as part of her PhD research.
An update on the second blood biomarker study in Germany that has been underway for more than 12 months was not available at time of publication.
Smartphone app study
Six HSPers from the south-east Queensland area participated in the second round of testing in mid-November for a smartphone app designed to quantify changes in movement and mobility, potentially constituting an important measure of drug effectiveness in a clinical trial.
The engineering team at Griffith University, Nathan, will now analyse the data, including a comparison with the the data collected in the first round in February on the four HSPers who participated in both rounds.
Dose modelling study
Associate Prof Michelle Hill recently advised that the development and validation of a reliable, repeatable test or assay to measure the minute concentration of the target compound is proving challenging and needs more time than originally scheduled.
Once the assay is validated, the measurement of target compound concentrations in the brain and spinal cord tissue of the experimental mice can go ahead.
Following that, the data will be analysed and interpreted with the desired result being the knowledge needed to determine what oral drug dose and formulation will achieve the target concentration in the upper motor neurons of people with HSP.
The projected date for completion of the study is now mid-February.