Bioblast Pharma

Hope should not be rare

SCA3 clinical trial

SCA3 Phase 2 Open-Label Study

Evaluating a promising new treatment for patients with SCA3

A Phase 2, open-label clinical trial is currently being conducted with an investigational new drug known as trehalose 90 mg/mL IV solution in patients with spinocerebellar ataxia, type 3 (SCA3). The primary objective is to assess the safety and tolerability of trehalose 90 mg/mL IV solution in patients suffering from SCA3.

Although not powered for efficacy, secondary endpoints have been included to explore if trehalose 90 mg/mL IV trehalose could improve or prevent worsening of SCA3 disease markers.

This trial enrolled 14 patients in Israel who have SCA3. Each patient is receiving either a 15 g or 30 g IV dose (90 mg/mL) of trehalose for 6 to 12 months.

The efficacy of trehalose 90mg/mL IV solution for ataxia is being explored using the Scale for the Assessment and Rating of Ataxia (SARA) score, the 9 hole peg (HP) test, 25 foot walk (FW), and the Neurological Examination Score for Spinocerebellar Ataxia (NESSCA).

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