Study of ABX-1431 in Adult Patients With Tourette Syndrome or Chronic Motor Tic Disorder
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|ClinicalTrials.gov Identifier: NCT03625453|
Recruitment Status : Completed
First Posted : August 10, 2018
Last Update Posted : April 8, 2020
|Condition or disease||Intervention/treatment||Phase|
|Tourette Syndrome Motor Tic Disorder||Drug: ABX-1431 Drug: Placebo||Phase 2|
ABX-1431 is a potent and selective, orally available, irreversible inhibitor of monoacylglycerol lipase, a metabolic enzyme that regulates the activity of the endogenous cannabinoid (endocannabinoid) system. It is being developed as a potential first-in-class compound for the treatment of Tourette Syndrome.
This study will assess the safety, tolerability, and effect on tics ABX-1431 in adults with Tourette Syndrome or chronic motor tic disorder in an 8-week study. It is a two-part study. Part 1 is a double-blind, randomized, placebo-controlled study of ABX-1431 at two target dose levels. Part 2 is an optional, open-label, non-randomized study of ABX-1431.
Patients will participate in the main study (Part 1) for approximately 10 to 14 weeks (up to 30-day screening period; 56-day treatment period; 14-day follow-up period). For patients who choose to participate in Part 2, there is a period of up to 4 weeks between the last study visit in Part 1 and first study visit in Part 2. Patients who choose to enter Part 2 will be treated with open-label ABX-1431 for an additional 6 weeks (28-day treatment period; 14-day follow-up period).
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||50 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||Randomized, placebo-controlled|
|Masking:||Double (Participant, Investigator)|
|Official Title:||A Randomized, Placebo-Controlled Study of ABX-1431 in Adult Patients With Tourette Syndrome or Chronic Motor Tic Disorder|
|Actual Study Start Date :||October 15, 2018|
|Actual Primary Completion Date :||January 20, 2020|
|Actual Study Completion Date :||January 20, 2020|
Oral use of hard capsule (10 milligrams), maximum dose per day: 40 milligrams
Part 1: 8 weeks with daily administration; Patients who choose to enter Part 2: additional 4 weeks with daily administration
Placebo Comparator: Placebo
Oral use of hard capsule
Part 1: 8 weeks with daily administration
- Change from baseline in Total Tic Score of the Yale Global Tic Severity Scale (YGTSS-TTS) compared with placebo [ Time Frame: Day 56 (40 mg ABX-1431 per day) and Day 28 (20 mg ABX-1431 per day) ]The Yale Global Tourette Severity Scale (YGTSS) is a clinician-completed rating scale to capture TS symptom severity. The number, frequency, intensity, complexity, and interference of both motor and vocal tics are rated on an ordinal scale between 0 and 5, with higher scores indicating greater severity. Motor and vocal tics subscales are reported (0 - 25) and then summed for a total tic score (TTS) (0 - 50). The TTS has been used as the registration endpoint for pharmacological therapies. The YGTSS has an Impairment subscale (0 - 50) which is then added for a total global score of 0 - 100.
- Adult Tic Questionnaire (ATQ) [ Time Frame: Part 1: days 0, 14, 28, 42, 56; Part 2: days 0, 14, 28 ]The Adult Tic Questionnaire (ATQ) is a tic self-rating scale. The ATQ records the occurrence, frequency and intensity for any of 27 endorsed common motor and vocal tics. The occurrence is being indicated as 1 for a present tic or 0 if no tic has been experienced. Frequency is measured on a scale from 1 (weekly frequency or less) to 4 (constant tics, almost all the time during the day). Intensity is being rated on a scale of 1 - 4 with 1 representing mild tics and 4 strong tics. The ATQ will principally be analyzed by summing the product of the intensity (1-4) and frequency (1-4) to determine the severity (2-8) across all endorsed tics. For clarity, the number of each endorsed tic and the total of intensity and frequency across all tics may be separately analyzed by mixed-model repeated-measures. The clinical relevance of changes in the ATQ may also be presented by the change in the average intensity and frequency score.
- Premonitory Urge for Tics Scale (PUTS) [ Time Frame: Part 1: days 0, 14, 28, 42, 56; Part 2: days 0, 14, 28 ]The Premonitory Urge for Tics Scale (PUTS) is a patient self-assessment of the intensity of agreement of several statements describing the unpleasant qualities of premonitory feelings preceding tics. Each statement is being rated on a scale from 1 (not at all) to 4 (very much). The total score is being calculated by summing the single scores. The range of the total score is between 9, corresponding to a minimal intensity of premonitory urges for tics and 36, an extremely high intensity with probable severe impairment.
- Clinical Global Impressions Scale for Improvement (CGII) [ Time Frame: Part 1: days 14, 28, 42, 56; Part 2: days 14, 28 ]The Clinical Global Impression of Improvement (CGI-I) is a 7-point ordinal, clinician-rated scale used to assess patients' overall improvement in disease status relative to their condition at baseline. A rating of 1 indicates a very much improved condition, compared to the subject's prior condition, while a rating of 7 means a very much worse condition respectively.
- AE occurrence [ Time Frame: Throughout the study: Part 1: day - 30 to day 70; Part 2: day 0 to day 42 ]All Adverse Events (AE) occurring during the clinical trial will be registered, documented and evaluated.
- SAE occurrence [ Time Frame: Throughout the study: Part 1: day - 30 to day 70; Part 2: day 0 to day 42 ]All Serious Adverse Events (SAE) occurring during the clinical trial will be registered, documented and evaluated.
- SUSAR occurrence [ Time Frame: Throughout the study: Part 1: day - 30 to day 70; Part 2: day 0 to day 42 ]All Serious Unexpected Serious Adverse Reactions (SUSAR) occurring during the clinical trial will be registered, documented and evaluated.
- Discontinuations due to AE occurrence [ Time Frame: Throughout the study: Part 1: day - 30 to day 70; Part 2: day 0 to day 42 ]All discontinuations due to (Adverse Events) AE occurring during the clinical trial will be evaluated.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03625453
|Charité, Universitätsmedizin Berlin, Klinik für Neurologie mit Experimenteller Neurologie|
|Medizinische Hochschule Hannover (MHH), Klinik für Psychiatrie, Sozialpsychiatrie und Psychotherapie|
|Uniklinik Köln, Klinik für Psychiatrie und Psychotherapie|
|Universität zu Lübeck, CBBM / Institut für Neurogenetik|
|LMU Klinikum der Universität München, Klinik für Psychiatrie und Psychotherapie|
|Centrum Medyczne Damiana Holding Sp zo.o.|
|Complejo Hospitalario Regional Virgen Del Rocío|
|Sevilla, Andalucía, Spain|
|Complejo Hospitalario Gregorio Marañón|
|Study Director:||Chan Beals, M.D., Ph.D.||Abide Therapeutics, Inc.|