First Participant in Phase 2/3 study of Trehalose Injection in Spinocerebellar Ataxia

In the STRIDES study (NCT05490563), the first participant received a dose of 90.5 mg/mL trehalose injection (SLS-005; Seelos Therapeutics, New York, NY) for the treatment of type 3 spinocerebellar ataxia (SCA3).

"We are delighted to take part in this potentially ground-breaking and innovative method to treat SCA3, a devastating disease with no Food and Drug Administration (FDA)-approved treatment or cure", said Dr. Theresa Zesiewicz of the University of Southern Florida.

The phase 2/3 STRIDES study will have an enrollment up to 245 participants with SCA3 in a double-blind randomized placebo-controlled trial.  Participants will receive treatment with either trehalose or placebo in 1 of 2 dosage arms and will have measurements of change from baseline in the Modified Scale for Assessment and Rating of Ataxia (m-SARA) total score at week 52.  

There will also be an assessment of change from baseline in a blood-based biomarker for neurodegeneration, clinical global impression of severity,an activities of daily living score, and patient global impression of severity will also be made. Safety and tolerability will be monitored.

Trehalose is a low molecular weight disaccharide (0.342 kDa) that crosses the blood brain barrier and is thought to stabilize proteins and activate autophagy through the activation of Transcription Factor EB (TFEB). Trehalose has received a Orphan Drug Designation previously for the treatment of SCA3 from the FDA. 

Data for trehalose will be presented at ICAR on Thursday, November 3, 2022 at 4:45pm CT.