Head to head

Acadia is said to have already commenced screening at three sites for the Phase 3 trial of trofinetide in Rett Syndrome but the trial is not yet showing on the US Clinical Trials website.

By way of comparison, GW Pharma’s Phase 3 trial of Epidiolex in Rett Syndrome was listed on ClinicalTrials.gov on 21 February, with the study start date (date on which first participant is enrolled) listed as 29 July.

GW Pharma is conducting its trials in most of the same city locations as Acadia – the only differences are that Acadia has two additional US sites (Cleveland and Phoenix) whereas GW Pharma (a British biopharma)has three additional UK-based sites. To date, the GW Pharma trial has commenced at five US sites.  

GW Pharma’s trial is in 251 patients (Acadia is planning for 180 participants). Like the trofinetide trial, the Epidiolex trial is using twice daily oral dosing of a strawberry flavoured solution and has RSBQ and CGI-I as co-primary endpoints. Patients aged 2-18 are eligible, whereas Acadia’s trial is open to patients aged 5-20 years. The expected study completion date for GW Pharma’s trial is September 2021.

In her latest blog, Rett parent and advocate, Mel Lancaster, has stated that Acadia is systematically having trial sites inspected and rolling them out as soon as possible. All issues with manufacturing-such as multi-dose storage etc., are said to have been resolved – there is enough trofinetide in four different tiered doses for every child in the trial and for the Lilac 2 extension trial. She also states that Acadia will be assisting families with the costs of trial participation.

https://clinicaltrials.gov/ct2/show/NCT03848832?cond=Rett Syndrome&phase=2&draw=2&rank=2
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