Biopharmaceutical company Dimerix ASX: DXB) reports that the first paediatric patient for its ACTION3 trial has been recruited at a site in Manchester, UK.
This represents a significant milestone being achieved in recruiting paediatric patients aged 12-17 years old into ACTION3 to support potential marketing approval in this population.
The dose of DMX-200 to be used in paediatric patients aged 12-17 years participating in the trial will be the same dose as provided to adults in the trial (120 mg twice daily).
This determination was based on reviewing the aggregate interim safety and pharmacokinetic data, including simulations in paediatric patients (12-17 years old), from the adult cohort of the ACTION3 Phase 3 trial taken at the first interim analysis point in March 2024 where the IDMC noted “the safety margin [of DMX-200] should allow [the ACTION3 Phase 3] clinical study to proceed in this paediatric population using adult doses”.
Approximately 19 of the planned >170 study sites are specialist paediatric kidney centres across the UK, USA, Mexico and Argentina, and have been selected to recruit paediatric patients (12-17 years old) with FSGS into the ACTION3 study.
Patients in this paediatric cohort of the blinded ACTION3 Phase 3 clinical trial will also be randomised (i.e. unknowingly placed on either placebo or DMX-200) and will be followed for a period of two years.
Should the paediatric cohort aged 12-17 years old be successful, and DMX-200 be approved in adults, it may allow Dimerix to expand its approval for DMX-200 and market DMX-200 to paediatric in key territories, including the US and Europe.
FSGS is one of the leading causes of kidney failure in children, with 20% of all presentations of Nephrotic Syndrome in paediatric patients caused by FSGS.
The DMX-200 paediatric development plan aims to collect sufficient data for potential marketing approval in children over one year old in accordance with the FDA and EMA expectations and with key components including:
o Paediatric patients from 12 to 17 years of age to be included in current ACTION3 study in patients with FSGS – in line with US FDA6 and European EMA advice.
o In silico modelling, simulation and extrapolation of paediatric data from ACTION3 will be undertaken in order to support a confirmatory small open-label study in children from 1 to 11 years of age
“The recruitment of the first paediatric patient for ACTION3 is highly significant, being the first step towards providing a potential new treatment for children with FSGS. The study is entering a very exciting phase as we open the last remaining study sites and work towards full recruitment later this year, as well as the blinded interim analysis anticipated in August 2025.” Chief Medical Officer, Dr David Fuller, said.
