Biotech Values

[DewDiligence]

Re: Telemedicine in RVNC’s clinical trials—especially ASPEN-1 for CD

…for cervical dystopia, patients can have twitches, which I assume are part of the criteria. But those wld be particularly hard to assess over video since much of the telecon SW is low frame rate. All told I’d think tele-assessment wld add significant noise to the data, thus making it much more difficult to get positive results?

In the ASPEN-1 phase-3 trial in cervical dystonia (RVNC’s most consequential in-progress trial), much of the input to the primary endpoint consists of patient-reported data:

https://www.clinicaltrials.gov/ct2/show/NCT03608397

Primary Outcome Measure:

Change from Baseline Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)-total score [ Time Frame: Week 4 and Week 6 ]

TWSTRS is used to assess the severity of cervical Dystonia and the success of its treatment. The average of the change from baseline in TWSTRS-total score at Weeks 4 and 6 will be determined. TWSTRS-total score has a minimum score of 0 and a maximum score of 85, where higher scores represent worse outcomes.

It is made up of the summation of 3 subscales: the Torticollis Severity Scale (minimum score of 0, maximum score of 35), the Disability Scale (minimum score of 0, maximum score of 30), and the Pain Scale (minimum score of 0, maximum score of 20).

p.s. “Cervical dystopia” is a world-class Freudian slip!