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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Crossover Sequence A | Experimental | Each in the fasting state: Period 1: Single-dose matching placebo Period 2: Single-dose ABX-1431 |
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| Crossover Sequence B | Experimental | Each in the fasting state: Period 1: Single-dose ABX-1431 Period 2: Single-dose matching placebo |
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| Crossover Sequence C | Experimental | Each with a standard high fat meal: Period 3: Single-dose matching placebo Period 4: Single-dose ABX-1431 |
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| Crossover Sequence D | Experimental | Each with a standard high fat meal: Period 3: Single-dose ABX-1431 Period 4: Single-dose matching placebo |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ABX-1431 | Drug | ABX-1431, capsules, 40 mg in the fasting state |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change of rating in Modified Rush Video Scale (MRVS) over time | pre-dose, post-dose (4 hours, 8 hours) | |
| Change in rating of Yale Global Tic Severity Scale (YGTSS) over time | pre-dose, post-dose (4 hours, 8 hours) | |
| Change of rating in Adult Tic Questionnaire (ATQ) over time | pre-dose, post-dose (4 hours, 8 hours, 12 hours) | |
| Change of rating in Premonitory Urge for Tics Scale (PUTS) over time | pre-dose, post-dose (4 hours, 8 hours, 12 hours) |
| Measure | Description | Time Frame |
|---|---|---|
| ABX-1431 and metabolite (M55) plasma pharmacokinetics | pre-dose, post-dose (2 hours, 4 hours, 8 hours, 24 hours) | |
| 2-AG hydrolysis in PBMC | pre-dose, post-dose (2 hours, 4 hours, 8 hours, 24 hours) |
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Principal Inclusion Criteria:
Principal Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Chan Beals | Abide Therapeutics, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Medizinische Hochschule Hannover | Hanover | Lower Saxony | 30625 | Germany |
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| ID | Term |
|---|---|
| D005879 | Tourette Syndrome |
| ID | Term |
|---|---|
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
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| ID | Term |
|---|---|
| C000634455 | ABX-1431 |
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| Placebo Comparator | Drug | Matching Placebo |
|
| ABX-1431 | Drug | ABX-1431, capsules, 20 mg with a high fat meal |
|
| Number and severity of adverse events (AEs), serious adverse events (SAEs), and suspected unexpected serious adverse reactions (SUSARs) | 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 | screening, pre-dose, post-dose (2 hours, 4 hours, 8 hours, 24 hours) |
| Number of patients with clinically significant change in Laboratory safety tests | 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 | screening, pre-dose, post-dose (24 hours) |
| 12-lead ECG assessments | screening, pre-dose, post-dose (4 hours) |
| D013981 | Tic Disorders |
| D009069 | Movement Disorders |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D065886 | Neurodevelopmental Disorders |
| D001523 | Mental Disorders |