The study will investigate the effects and the safety of a single-dose of ABX-1431 HCl on tics and other symptoms of Tourette Syndrome. During part 1 (periods 1 and 2) each patient will receive study drug once and placebo once. Patients who complete part 1 with adequate clinical safety will be offered the option to participate in part 2 (periods 3 and 4) and again willl receive study drug once and placebo once where, in contrast to part 1, administration will take place with a standard high fat meal.
This is a single dose, double-blind, randomized, placebo-controlled, cross-over study. This study will assess the single dose effects of ABX-1431 HCl on tics and other symptoms of Tourette Syndrome. All patients will undergo a screening visit for enrollment criteria. Eligible patients will be treated with a single dose of ABX-1431 HCl or placebo followed by efficacy, safety and pharmacokinetics assessments. After a washout period of 1-3 weeks, patients will undergo identical procedures with the other treatment. Only patients who complete the first part of the study with adequate clinical safety will be offered the option to participate in an additional two period crossover, where ABX-1431 HCl or placebo is taken with a standard high fat meal. Again, efficacy, safety and pharmacokinetics assessments will be done. After a washout period of 1-3 weeks, patients will undergo identical procedures with the other treatment. This study will enroll 20 patients with a diagnosis of Tourette Syndrome OR chronic motor tic disorder.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
BASIC_SCIENCE
Masking
TRIPLE
Enrollment
23
Medizinische Hochschule Hannover
Hanover, Lower Saxony, Germany
Change of rating in Modified Rush Video Scale (MRVS) over time
Time frame: pre-dose, post-dose (4 hours, 8 hours)
Change in rating of Yale Global Tic Severity Scale (YGTSS) over time
Time frame: pre-dose, post-dose (4 hours, 8 hours)
Change of rating in Adult Tic Questionnaire (ATQ) over time
Time frame: pre-dose, post-dose (4 hours, 8 hours, 12 hours)
Change of rating in Premonitory Urge for Tics Scale (PUTS) over time
Time frame: pre-dose, post-dose (4 hours, 8 hours, 12 hours)
ABX-1431 and metabolite (M55) plasma pharmacokinetics
Time frame: pre-dose, post-dose (2 hours, 4 hours, 8 hours, 24 hours)
2-AG hydrolysis in PBMC
Time frame: pre-dose, post-dose (2 hours, 4 hours, 8 hours, 24 hours)
Number and severity of adverse events (AEs), serious adverse events (SAEs), and suspected unexpected serious adverse reactions (SUSARs)
Time frame: screening, pre-dose, post-dose (0 hours, 2 hours, 4 hours, 8 hours, 12 hours, 24 hours), follow-up
Number of patients with clinically significant change in vital signs
The following vital signs will be assessed: heart rate, blood pressure, respiratory rate, temperature
Time frame: screening, pre-dose, post-dose (2 hours, 4 hours, 8 hours, 24 hours)
Number of patients with clinically significant change in Laboratory safety tests
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The following laboratory safety tests will be assessed: Alanine aminotransferase (ALT), Aspartate aminotransferase (AST), Albumin, Alkaline phosphatase, Bicarbonate, Calcium, Chloride, Cholesterol, Creatinine, Glucose, Potassium, Sodium, Bilirubin, total (bilirubin to be fractionated if total bilirubin is elevated), Blood urea nitrogen (BUN)/Urea, hemoglobin, hematocrit, red blood cell (RBC) count, platelet count, white blood cell (WBC) count (total and differential count), polymorphonuclear leukocytes (neutrophils), lymphocytes, eosinophils, monocytes, basophils; Urinalysis: pH, specific gravity, glucose, ketones, leukocyte, esterase, nitrites, occult blood, protein
Time frame: screening, pre-dose, post-dose (24 hours)
12-lead ECG assessments
Time frame: screening, pre-dose, post-dose (4 hours)