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Tolerability and efficacy of the monoaminergic stabilizer (−)-OSU6162 (PNU-96391A) in Huntington’s disease: a double-blind cross-over study

  • Angelica Kloberg (a1), Radu Constantinescu (a2), Marie Karin Lena Nilsson (a3), Maria Lizzie Carlsson (a3), Arvid Carlsson (a3), Jan Wahlström (a4) and Sara Haghighi (a5)...



To evaluate the safety (primary objective) and efficacy (secondary objective) of (−)-OSU6162 in Huntington’s disease (HD).


In a double-blind, cross-over trial, patients with HD were randomly assigned to start treatment on either (−)-OSU6162 or placebo. After 4 weeks, those patients who initially received active drug were switched to placebo for another 4 weeks, and vice versa. During the first week the (−)-OSU6162 dose was 15 mg twice daily, during the second week 30 mg twice daily, and during the last 2 weeks 45 mg twice daily. Motor, cognitive, mental and social functions were rated by the clinical investigator or by self-assessment, using established rating scales.


Fifteen patients fulfilling inclusion and exclusion criteria completed the study. (−)-OSU6162 was well tolerated by all patients and no adverse effects were observed. (−)-OSU6162 treatment significantly improved the Short Form 36 Vitality score, mainly due to an improvement of the individual item ‘worn-out’ (VT3). In addition, an improvement of depressive symptoms was found using Beck Depression Inventory. In contrast to a general trend of improvement in several non-motor variables only small and non-significant differences between (−)-OSU6162 and placebo were found regarding motor functions.


(−)-OSU6162 offers promise for the treatment of HD, as a drug with good tolerability, capable of improving the patients’ experienced non-motor functions such as energy and mood and thus alleviating symptoms of great importance for their quality of life.


Corresponding author

Dr. Angélica Kloberg, PhD, A Carlsson Research AB, Sahlgrenska Science Park, Medicinaregatan 8A, vån 2, lab SE 413 46 Gothenburg, Sweden. Tel: +46-31-7411892; E-mail:


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Tolerability and efficacy of the monoaminergic stabilizer (−)-OSU6162 (PNU-96391A) in Huntington’s disease: a double-blind cross-over study

  • Angelica Kloberg (a1), Radu Constantinescu (a2), Marie Karin Lena Nilsson (a3), Maria Lizzie Carlsson (a3), Arvid Carlsson (a3), Jan Wahlström (a4) and Sara Haghighi (a5)...


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