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A Study to Evaluate the Effects of ABX-1431 on Patients With Tourette Syndrome

Primary Purpose

Tourette Syndrome, Chronic Motor Tic Disorder

Status

Completed

Phase

Phase 1

Locations

Germany

Study Type

Interventional

Intervention

ABX-1431

Placebo Comparator

ABX-1431

About this trial

This is an interventional basic science trial for Tourette Syndrome

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Principal Inclusion Criteria:

Patient is a male or female between the age of 18 and 65 years of age at the Screening Visit.
Patient has a diagnosis of Tourette Syndrome OR chronic motor tic disorder as defined by the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5) criteria.
Patient's Yale Global Tic Severity Scale (YGTSS) total tic sub-scale (TTS) results must be ≥ 18 (Range 0-50) at the Screening Visit.
Patients taking daily medications for symptoms of Tourette Syndrome [e.g. neuroleptics (e.g. aripiprazole, risperidone) or selective serotonin reuptake inhibitors (e.g. fluoxetine)] must be on a stable dose of medication for at least 30 days before the Screening Visit and must be expected to remain on a stable dose during this study.

Principal Exclusion Criteria:

Patient is taking potent cytochrome P450 3A4/5 inducers [e.g. carbamazepine, oxcarbazine, rifampin, St. John's Wort (Hypericum perforatum), or phenytoin]. Patient is taking strong P450 3A4/5 inhibitors including atazanavir, bocepravir, clarithromycin, grapefruit juice, indinavir, itraconazole, ketoconazole, nefazodone, nelfinavir, posaconazole, ritonavir, saquinavir, telithromycin, or voriconazole.
Patients with a diagnosis of any psychiatric comorbidity (obsessive compulsive disorder, attention deficit hyperactivity disorder) OR generalized anxiety disorder, depression or post-traumatic stress disorder that is unstable or requires alteration in therapy are excluded. Patients with a past history of psychosis or schizophrenia at any time are excluded. Patients with stable OCD or ADHD requiring no alteration in therapy may be enrolled.

Sites / Locations

Medizinische Hochschule Hannover

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Arm Label

Crossover Sequence A

Crossover Sequence B

Crossover Sequence C

Crossover Sequence D

Arm Description

Each in the fasting state: Period 1: Single-dose matching placebo Period 2: Single-dose ABX-1431

Each in the fasting state: Period 1: Single-dose ABX-1431 Period 2: Single-dose matching placebo

Each with a standard high fat meal: Period 3: Single-dose matching placebo Period 4: Single-dose ABX-1431

Each with a standard high fat meal: Period 3: Single-dose ABX-1431 Period 4: Single-dose matching placebo

Outcomes

Primary Outcome Measures

Change of rating in Modified Rush Video Scale (MRVS) over time

Change in rating of Yale Global Tic Severity Scale (YGTSS) over time

Change of rating in Adult Tic Questionnaire (ATQ) over time

Change of rating in Premonitory Urge for Tics Scale (PUTS) over time

Secondary Outcome Measures

ABX-1431 and metabolite (M55) plasma pharmacokinetics

2-AG hydrolysis in PBMC

Number and severity of adverse events (AEs), serious adverse events (SAEs), and suspected unexpected serious adverse reactions (SUSARs)

Number of patients with clinically significant change in vital signs

The following vital signs will be assessed: heart rate, blood pressure, respiratory rate, temperature

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

12-lead ECG assessments

Full Information

NCT ID

NCT03058562

First Posted

February 13, 2017

Last Updated

November 2, 2017

Sponsor

Abide Therapeutics

1. Study Identification

Unique Protocol Identification Number

NCT03058562

Brief Title

A Study to Evaluate the Effects of ABX-1431 on Patients With Tourette Syndrome

Official Title

A Randomized, Placebo-Controlled, Single-Dose Crossover Study of ABX-1431 HCl in Adult Patients With Tourette Syndrome (TS) and Chronic Motor Tic Disorder

Study Type

Interventional

2. Study Status

Record Verification Date

November 2017

Overall Recruitment Status

Completed

Study Start Date

February 1, 2017 (Actual)

Primary Completion Date

October 4, 2017 (Actual)

Study Completion Date

October 4, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Abide Therapeutics

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief Summary

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.

Detailed Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Tourette Syndrome, Chronic Motor Tic Disorder

7. Study Design

Primary Purpose

Basic Science

Study Phase

Phase 1

Interventional Study Model

Crossover Assignment

Masking

ParticipantInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

23 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Crossover Sequence A

Arm Type

Experimental

Arm Description

Each in the fasting state: Period 1: Single-dose matching placebo Period 2: Single-dose ABX-1431

Arm Title

Crossover Sequence B

Arm Type

Experimental

Arm Description

Each in the fasting state: Period 1: Single-dose ABX-1431 Period 2: Single-dose matching placebo

Arm Title

Crossover Sequence C

Arm Type

Experimental

Arm Description

Each with a standard high fat meal: Period 3: Single-dose matching placebo Period 4: Single-dose ABX-1431

Arm Title

Crossover Sequence D

Arm Type

Experimental

Arm Description

Each with a standard high fat meal: Period 3: Single-dose ABX-1431 Period 4: Single-dose matching placebo

Intervention Type

Drug

Intervention Name(s)

ABX-1431

Intervention Description

ABX-1431, capsules, 40 mg in the fasting state

Intervention Type

Drug

Intervention Name(s)

Placebo Comparator

Intervention Description

Matching Placebo

Intervention Type

Drug

Intervention Name(s)

ABX-1431

Intervention Description

ABX-1431, capsules, 20 mg with a high fat meal

Primary Outcome Measure Information:

Title

Change of rating in Modified Rush Video Scale (MRVS) over time

Time Frame

pre-dose, post-dose (4 hours, 8 hours)

Title

Change in rating of Yale Global Tic Severity Scale (YGTSS) over time

Time Frame

pre-dose, post-dose (4 hours, 8 hours)

Title

Change of rating in Adult Tic Questionnaire (ATQ) over time

Time Frame

pre-dose, post-dose (4 hours, 8 hours, 12 hours)

Title

Change of rating in Premonitory Urge for Tics Scale (PUTS) over time

Time Frame

pre-dose, post-dose (4 hours, 8 hours, 12 hours)

Secondary Outcome Measure Information:

Title

ABX-1431 and metabolite (M55) plasma pharmacokinetics

Time Frame

pre-dose, post-dose (2 hours, 4 hours, 8 hours, 24 hours)

Title

2-AG hydrolysis in PBMC

Time Frame

pre-dose, post-dose (2 hours, 4 hours, 8 hours, 24 hours)

Title

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

Title

Number of patients with clinically significant change in vital signs

Description

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)

Title

Number of patients with clinically significant change in Laboratory safety tests

Description

Time Frame

screening, pre-dose, post-dose (24 hours)

Title

12-lead ECG assessments

Time Frame

screening, pre-dose, post-dose (4 hours)

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

65 Years

Accepts Healthy Volunteers

Eligibility Criteria

Principal Inclusion Criteria: Patient is a male or female between the age of 18 and 65 years of age at the Screening Visit. Patient has a diagnosis of Tourette Syndrome OR chronic motor tic disorder as defined by the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5) criteria. Patient's Yale Global Tic Severity Scale (YGTSS) total tic sub-scale (TTS) results must be ≥ 18 (Range 0-50) at the Screening Visit. Patients taking daily medications for symptoms of Tourette Syndrome [e.g. neuroleptics (e.g. aripiprazole, risperidone) or selective serotonin reuptake inhibitors (e.g. fluoxetine)] must be on a stable dose of medication for at least 30 days before the Screening Visit and must be expected to remain on a stable dose during this study. Principal Exclusion Criteria: Patient is taking potent cytochrome P450 3A4/5 inducers [e.g. carbamazepine, oxcarbazine, rifampin, St. John's Wort (Hypericum perforatum), or phenytoin]. Patient is taking strong P450 3A4/5 inhibitors including atazanavir, bocepravir, clarithromycin, grapefruit juice, indinavir, itraconazole, ketoconazole, nefazodone, nelfinavir, posaconazole, ritonavir, saquinavir, telithromycin, or voriconazole. Patients with a diagnosis of any psychiatric comorbidity (obsessive compulsive disorder, attention deficit hyperactivity disorder) OR generalized anxiety disorder, depression or post-traumatic stress disorder that is unstable or requires alteration in therapy are excluded. Patients with a past history of psychosis or schizophrenia at any time are excluded. Patients with stable OCD or ADHD requiring no alteration in therapy may be enrolled.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Chan Beals

Organizational Affiliation

Abide Therapeutics, Inc.

Official's Role

Study Director

Facility Information:

Facility Name

Medizinische Hochschule Hannover

City

Hannover

State/Province

Niedersachsen

ZIP/Postal Code

30625

Country

Germany

12. IPD Sharing Statement

Plan to Share IPD

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A Study to Evaluate the Effects of ABX-1431 on Patients With Tourette Syndrome

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