Efficacy and safety of the dopaminergic stabilizer Pridopidine (ACR16) in patients with Huntington's disease.
OBJECTIVE
To evaluate the efficacy and safety of the dopaminergic stabilizer pridopidine (ACR16) in patients with Huntington's disease (HD).


METHODS
In a randomized, double-blind, placebo-controlled, 4-week trial, patients with HD received pridopidine (50 mg/d, n = 28) or placebo (n = 30). The primary outcome measure was the change from baseline in weighted cognitive score, assessed by cognitive tests (Symbol Digit Modalities, verbal fluency, and Stroop tests). Secondary outcome measures included changes in the Unified Huntington's Disease Rating Scale, Hospital Anxiety and Depression Scale, Leeds Sleep Evaluation Questionnaire, Reitan Trail-Making Test A, and Clinical Global Impression of Change. Safety assessments were also performed.


RESULTS
There was no significant difference between pridopidine and placebo in the change from baseline of the weighted cognitive score. However, secondary measures such as affective symptoms showed trends toward improvement, and there was significant improvement in voluntary motor symptoms compared with placebo (P < 0.05). Pridopidine was well tolerated, with a safety profile similar to placebo.


CONCLUSIONS
Pridopidine shows promise as a treatment for some of the symptoms of HD. In this small-scale study, the most notable effect was improvement in voluntary motor symptoms. Larger, longer-term trials are warranted.
Lundin
Anders
A
Prima Psychiatry, Danderyd Hospital, Stockholm, Sweden. anderslundin1@gmail.com
Dietrichs
Espen
E
Haghighi
Sara
S
Göller
Marie-Louise
ML
Heiberg
Arvid
A
Loutfi
Ghada
G
Widner
Håkan
H
Wiktorin
Klas
K
Wiklund
Leif
L
Svenningsson
Anders
A
Sonesson
Clas
C
Waters
Nicholas
N
Waters
Susanna
S
Tedroff
Joakim
J
D000342:Affective Symptoms / Q000188:drug therapy / Q000523:psychology; D001007:Anxiety / Q000188:drug therapy / Q000523:psychology; D003071:Cognition / Q000187:drug effects / Q000502:physiology; D004298:Dopamine / Q000378:metabolism*; D018492:Dopamine Antagonists / Q000627:therapeutic use*; D065127:Dopamine D2 Receptor Antagonists; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D006816:Huntington Disease / Q000188:drug therapy* / Q000523:psychology; D008297:Male; D008875:Middle Aged; D010880:Piperidines / Q000627:therapeutic use*; D017448:Receptors, Dopamine D2 / Q000378:metabolism; D012720:Severity of Illness Index; D016896:Treatment Outcome
D018492:Dopamine Antagonists; D065127:Dopamine D2 Receptor Antagonists; D010880:Piperidines; D017448:Receptors, Dopamine D2; C483720:pridopidine; D004298:Dopamine