Tourette SyndromePhase 2NCT03625453
Study of ABX-1431 in Adult Patients With Tourette Syndrome or Chronic Motor Tic Disorder
A Phase 2 study testing ABX-1431 in people with Tourette Syndrome and Motor Tic Disorder, run by Abide Therapeutics at 8 sites in 3 countries.
Trial summary
Two-part study consisting of a double-blind, randomized, placebo-controlled, study at two target dose levels (Part 1) and an open-label, non-randomized study (Part 2) to determine the efficacy of ABX-1431 in treating adult patients with Tourette syndrome or Chronic Motor Tic Disorder as measured by the change from… Read more about this study
- Who is it for?
- Ages 18 to 64 years
- Conditions the study is for
- Tourette Syndrome, Motor Tic Disorder
- What is being tested?
- ABX-1431
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 50 participants
- Where
- 8 sitesin 3 countries
- Study dates
- 15 Oct 2018to 20 Jan 2020
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 64 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Patient is a male or female ≥ 18 to 64 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 YGTSS-TTS results must be ≥ 22 (range 0-50) at the Baseline/Randomization Visit.
- At the Baseline/Randomization Visit patients must be taking a stable drug regimen for tics and comorbidities for 30 days and must be expected to remain on a stable drug regimen during this study. Patients receiving no medication for TS may participate. Patients who have recently discontinued medication for tics must have discontinued them for at least 30 days. For neuroleptic drugs (e.g., risperidone, aripiprazole), the minimum discontinuation period is 30 days prior to the Baseline/Randomization Visit. For injectable depot neuroleptic drugs the minimum discontinuation period is one dosage cycle plus 14 days. (See Inclusion 'f' for discontinuation period of cannabinoid medication in patients using these products for tics.)
- Patient is legally competent, has been informed of the nature and scope of relevance for the study, voluntarily agrees to participation, agrees to the study restrictions, and has signed the informed consent form (ICF) approved by the Ethics Review Committee (ERC).
- Patients using cannabinoid medications for their tics must discontinue their use at least 14 days prior to the Baseline/Randomization Visit. The investigator and patient must be confident that these patients will not require these medications for the duration of the study until 14 days after the last dose of study medication. Examples of cannabinoids include cannabis in any form, nabilone or Δ9-tetrahydrocannabinol (THC)-containing medications such as nabiximols (Sativex®) or dronabinol. The use of recreational cannabinoids during this study is not permitted.
- Female patients of child-bearing potential must have a negative pregnancy test [serum or urine human chorionic gonadotropin (hCG)] at the Screening Visit and other indicated visits. They must practice a highly effective, reliable, and medically approved contraceptive regimen during the study (e.g., theoretical failure rate less than 1% per year as described in the 2014 Heads of Medicines Agencies: Clinical Trials Facilitation Group report on contraception in clinical trials, which include oral or parenteral or implanted hormonal contraception, vaginal ring releasing hormonal contraception (e.g., Nuvaring, intrauterine device, or intrauterine system). Post-menopausal women may enter this study. Post-menopausal women are defined as those without menses in the past 12 months, and with a serum follicle stimulating hormone (FSH) in the post-menopausal range. Women who are surgically sterile may enter this study with written documentation of the surgical procedure.
- Male patients must be willing to use a condom with sexual partners during this study until 14 days after the last dose of study medication. Male patients must be willing to abstain from sperm donation for 3 months after the completion of this study.
Who cannot join
- Patient is taking strong inducers or inhibitors of cytochrome P450 (CYP)3A4/5 or CYP2C9. Examples of strong CYP3A4/5 inducers include carbamazepine, efavirenz, nevirapine, barbiturates, pioglitazone, modafinil, enzalutamide, oxcarbazepine, rifampicin, St. John's Wort (Hypericum perforatum), and phenytoin. Examples of potent CYP3A4/5 inhibitors include atazanavir, boceprevir, clarithromycin, grapefruit juice, indinavir, itraconazole, ketoconazole, nefazodone, suboxone, nelfinavir, posaconazole, ritonavir, saquinavir, telithromycin, and voriconazole. Examples of CYP2C9 strong inducers/inhibitors include carbamazepine, enzalutamide, fluconazole, valproic acid, phenobarbital, nevirapine, rifampicin, and St. John's Wort.
- Patient has evidence of alcohol abuse or dependence, as defined by the DSM-5 criteria, with two or more of the 11 criteria at the Screening Visit or within 1 year before the Screening Visit.
- Patient has evidence of drug or chemical abuse (except nicotine), as defined by the DSM-5 criteria, at the Screening Visit or within 1 year before the Screening Visit. Patient has evidence of marijuana or cannabis dependency or has been treated for cannabis dependency, as defined by DSM-5 criteria, at the Screening Visit or within 1 year before the Screening Visit. (Patients who are prescribed stimulants should not be assessed for Stimulant Use Disorder. Patients who use cannabinoid-based medicine (e.g., cannabis in any form, nabilone, or THC-containing medications) for treatment of Tourette Syndrome with the investigator's knowledge should not be assessed for Cannabis Use Disorder, but these patients must comply with abstinence requirements for this study (See Inclusion 'f')). Patients who have a positive urine drug screen at the Screening Visit for drugs other than those allowed, as indicated in this criterion, are excluded.
- Patient is unwilling to comply with study restrictions including abstinence from cannabis and alcohol from the Baseline/Randomization Visit until the follow-up telephone call 14 days after the last dose of study medication.
- Patients receiving ongoing psychological therapy for tics such as Habit Reversal Training or Comprehensive Behavioral Intervention for Tics are excluded. Patients who have completed behavioral therapy for tics at least 30 days before the Baseline/Randomization Visit may participate.
- Patient is a lactating or pregnant female, or a female who intends to become pregnant within 90 days following the last dose of study medication.
Show all 23Show fewer
- Patient has one or more of the following laboratory results at the Screening Visit:
- Aspartate transaminase (AST) > 3 x upper limit of normal (ULN)
- Alanine transaminase (ALT) > 3 x ULN
- Total bilirubin > 2 x ULN (unless due to Gilbert's syndrome)
- Patient has an estimated creatinine clearance less than 60 mL/minute at the Screening Visit. Creatinine clearance (Clcr) is estimated by the Cockcroft-Gault (C-G) equation from a spot serum creatinine (mg/dL) determination using the following formula:
- CLcr (mL/min) = [140 - age (years)] × weight (kg) / 72 × serum creatinine (mg/dL)] × [0.85 for female patients]
- Patient has a serum albumin level below the laboratory normal range at the Screening Visit, and the physician cannot rule out hepatic insufficiency based on consideration of clinical symptoms, clinical signs, and other laboratory tests. (The physician may decide that a low value of a serum albumin is clinically insignificant or the test may be repeated.)
- Patient has symptomatic chronic Hepatitis B and/or Hepatitis C Virus infection. Asymptomatic seropositive individuals without clinical or clinical laboratory manifestations of hepatitis may be enrolled.
- Patient has a clinically significant abnormality on the ECG at the Screening Visit.
- Patients with a history of cancer will be excluded, with two exceptions: Patients with a history of squamous or basal cell carcinoma of the skin treated with documented success of curative therapy > 3 months may be enrolled. Patients with cancer and in remission with no treatment for at least 2 years prior to the Screening Visit may be enrolled.
- Patient has Tourette Syndrome or Chronic Motor Tic Disorder and also has mental retardation, autism spectrum disorder (ASD), dystonia, or post-traumatic stress disorder (PTSD). Mental retardation may be defined by medical history. (If the patient's available medical history is unclear, the investigator may use any validated screening test such as the Wechsler Abbreviated Scale of Intelligence (WASI-II); an intelligence quotient < 80 (well below average) is an exclusion). ASD and PTSD may be determined by medical history. (If the patient's available medical history is unclear, the investigator may use the diagnostic criteria for ASD and PTSD, as defined by the DSM-5.)
- Patient has confounding medical conditions such as active infection, primary or acquired immunodeficiency (HIV testing is not mandated), uncontrolled diabetes (i.e., known Type 1 or 2 diabetes with most recent HbA1c level > 9.0% or has not had an HbA1c measurement within past 12 months), clinically significant cardiac disease (i.e., significant cardiac conduction abnormalities, uncontrolled hypertension, myocardial infarction, cardiac arrhythmia or unstable angina < 6 months to enrollment, New York Heart Association (NYHA) Grade II or greater congestive heart failure, serious cardiac arrhythmia requiring medication, Grade II or greater peripheral vascular disease, and history of cerebrovascular accident within 6 months), clinically significant renal disease (e.g., acute kidney injury (e.g., hematuria, oligouria, or an acute rise in serum creatinine of ≥ 0.5 mg / dL (≥ 44 umol/L)) or chronic kidney disease defined by estimated glomerular filtration rate < 60 ml / min or oligouria), or any suspected hepatic insufficiency. (See Exclusions 'g' 'h' and 'i' for laboratory test exclusion limits.) Physicians should rely on the medical history, physical examination, and protocol specified laboratory tests to exclude individuals with these conditions.
- Patient has a diagnosis of any psychiatric comorbidity such obsessive compulsive disorder, attention deficit hyperactivity disorder, anxiety disorder, and/or depression and has undergone a change in therapy in the 30 days before the Baseline/Randomization Visit or is in need of a change in treatment. Patients with stable obsessive compulsive disorder or attention deficit hyperactivity disorder or depression requiring no alteration in therapy may be enrolled. Patients with a past history of psychosis or schizophrenia at any time are excluded.
- Patient has a history of suicidal ideation with intent to act or a plan to act, or a suicide attempt in the last 3 years preceding the Baseline/Randomization Visit.
- Patient has participated in an investigational study for medications with the last visit within 1 month for a non-biologic agent and 90 days for a biologic agent of the Baseline/Randomization Visit. The total volume of blood donated in the past 56 days may not exceed 450 mL.
- Patient is an employee of the investigative site or sponsor or a close relative of an employee of the investigative site.
- Patient has intolerance or hypersensitivity to the investigational medicinal product, ABX-1431 or the excipients. The excipients used in the capsule formulation are microcrystalline cellulose, croscarmellose sodium, magnesium stearate (vegetable origin), and gelatin capsule shells. The capsule shells are comprised of gelatin, red iron oxide, and titanium dioxide.
Trial locations and contacts
8 sites in 3 countries.
8 sites in 3 countries
Germany5 sites
Charité, Universitätsmedizin Berlin, Klinik für Neurologie mit Experimenteller Neurologie
Berlin
Uniklinik Köln, Klinik für Psychiatrie und Psychotherapie
Cologne
Medizinische Hochschule Hannover (MHH), Klinik für Psychiatrie, Sozialpsychiatrie und Psychotherapie
Hanover
Universität zu Lübeck, CBBM / Institut für Neurogenetik
Lübeck
LMU Klinikum der Universität München, Klinik für Psychiatrie und Psychotherapie
München
Spain2 sites
Complejo Hospitalario Gregorio Marañón
Madrid
Complejo Hospitalario Regional Virgen Del Rocío
Seville, Andalusia
About this study
Official title: A Randomized, Placebo-Controlled Study of ABX-1431 in Adult Patients With Tourette Syndrome or Chronic Motor Tic Disorder
Two-part study consisting of a double-blind, randomized, placebo-controlled, study at two target dose levels (Part 1) and an open-label, non-randomized study (Part 2) to determine the efficacy of ABX-1431 in treating adult patients with Tourette syndrome or Chronic Motor Tic Disorder as measured by the change from baseline in Total Tic Score of the Yale Global Tic Severity Scale (YGTSS-TTS) compared with placebo.
Read the detailed description
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).
- Conditions
- Tourette Syndrome, Motor Tic Disorder
- Being tested
- ABX-1431, Placebo
- Keywords
- Tourette Syndrome, Chronic Motor Tic Disorder
- Medical subject headings
- Tourette Syndrome, Tic Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ABX-1431Drug
Part 1: 8 weeks with daily administration; Patients who choose to enter Part 2: additional 4 weeks with daily administration
PlaceboDrug
Part 1: 8 weeks with daily administration
| Study group | Role | What they receive |
|---|---|---|
| ABX-1431 | Experimental | Oral use of hard capsule (10 milligrams), maximum dose per day: 40 milligrams Receives: Drug: ABX-1431 |
| Placebo | Placebo comparator | Oral use of hard capsule Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in Total Tic Score of the Yale Global Tic Severity Scale (YGTSS-TTS) compared with placebo
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.
Measured: Day 56 (40 mg ABX-1431 per day) and Day 28 (20 mg ABX-1431 per day)
Secondary outcomes (7)
Adult Tic Questionnaire (ATQ)
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.
Measured: Part 1: days 0, 14, 28, 42, 56; Part 2: days 0, 14, 28
Premonitory Urge for Tics Scale (PUTS)
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.
Measured: Part 1: days 0, 14, 28, 42, 56; Part 2: days 0, 14, 28
Clinical Global Impressions Scale for Improvement (CGII)
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.
Measured: Part 1: days 14, 28, 42, 56; Part 2: days 14, 28
AE occurrence
All Adverse Events (AE) occurring during the clinical trial will be registered, documented and evaluated.
Measured: Throughout the study: Part 1: day - 30 to day 70; Part 2: day 0 to day 42
SAE occurrence
All Serious Adverse Events (SAE) occurring during the clinical trial will be registered, documented and evaluated.
Measured: Throughout the study: Part 1: day - 30 to day 70; Part 2: day 0 to day 42
SUSAR occurrence
All Serious Unexpected Serious Adverse Reactions (SUSAR) occurring during the clinical trial will be registered, documented and evaluated.
Measured: Throughout the study: Part 1: day - 30 to day 70; Part 2: day 0 to day 42
Discontinuations due to AE occurrence
All discontinuations due to (Adverse Events) AE occurring during the clinical trial will be evaluated.
Measured: Throughout the study: Part 1: day - 30 to day 70; Part 2: day 0 to day 42
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Aug 2018
- Study start15 Oct 2018
- Primary completion20 Jan 2020
- Study completion20 Jan 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Tourette SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Randomized, placebo-controlled
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- Abide Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- FGK Clinical Research GmbH, BASi (Bioanalytical Systems, Inc.), CogState Ltd.
- Responsible party
- Sponsor
- Investigators
- Chan Beals, M.D., Ph.D., Study director, Abide Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03625453
- Sponsor's protocol ID
- ABX-1431 PN018
- Other IDs
- 2018-000100-41, 2018
- Status last verified
- Apr 2020
- First submitted
- 8 Aug 2018
- Last updated
- 8 Apr 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Muller-Vahl KR, Fremer C, Beals C, Ivkovic J, Loft H, Schindler C. Monoacylglycerol Lipase Inhibition in Tourette Syndrome: A 12-Week, Randomized, Controlled Study. Mov Disord. 2021 Oct;36(10):2413-2418. doi: 10.1002/mds.28681. Epub 2021 Jun 12.
PubMed 34117788
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Placebo-Controlled Study of ABX-1431 in Adult Patients With Tourette Syndrome or Chronic Motor Tic Disorder
- ClinicalTrials.gov ID
- NCT03625453
- Acronym / sponsor ID
- ABX-1431 PN018
- Conditions
- Tourette Syndrome, Motor Tic Disorder
- Keywords
- Tourette Syndrome, Chronic Motor Tic Disorder
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- Abide TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- FGK Clinical Research GmbH, BASi (Bioanalytical Systems, Inc.), CogState Ltd.
- Study start
- 15 Oct 2018 (actual)
- Primary completion
- 20 Jan 2020 (actual)
- Study completion
- 20 Jan 2020 (actual)
- First posted
- 10 Aug 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Two-part study consisting of a double-blind, randomized, placebo-controlled, study at two target dose levels (Part 1) and an open-label, non-randomized study (Part 2) to determine the efficacy of ABX-1431 in treating adult patients with Tourette syndrome or Chronic Motor Tic Disorder as measured by the change from baseline in Total Tic Score of the Yale Global Tic Severity Scale (YGTSS-TTS) compared with placebo.
Detailed description
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
- Model description
- Randomized, placebo-controlled
| Arm | Type | Receives |
|---|---|---|
| ABX-1431 | Experimental | Drug: ABX-1431Oral use of hard capsule (10 milligrams), maximum dose per day: 40 milligrams |
| Placebo | Placebo comparator | Drug: PlaceboOral use of hard capsule |
Who may qualify
- Ages
- 18 to 64 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (8 listed)
- Patient is a male or female ≥ 18 to 64 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 YGTSS-TTS results must be ≥ 22 (range 0-50) at the Baseline/Randomization Visit.
- At the Baseline/Randomization Visit patients must be taking a stable drug regimen for tics and comorbidities for 30 days and must be expected to remain on a stable drug regimen during this study. Patients receiving no medication for TS may participate. Patients who have recently discontinued medication for tics must have discontinued them for at least 30 days. For neuroleptic drugs (e.g., risperidone, aripiprazole), the minimum discontinuation period is 30 days prior to the Baseline/Randomization Visit. For injectable depot neuroleptic drugs the minimum discontinuation period is one dosage cycle plus 14 days. (See Inclusion 'f' for discontinuation period of cannabinoid medication in patients using these products for tics.)
- Patient is legally competent, has been informed of the nature and scope of relevance for the study, voluntarily agrees to participation, agrees to the study restrictions, and has signed the informed consent form (ICF) approved by the Ethics Review Committee (ERC).
Key exclusion criteria (23 listed)
- Patient is taking strong inducers or inhibitors of cytochrome P450 (CYP)3A4/5 or CYP2C9. Examples of strong CYP3A4/5 inducers include carbamazepine, efavirenz, nevirapine, barbiturates, pioglitazone, modafinil, enzalutamide, oxcarbazepine, rifampicin, St. John's Wort (Hypericum perforatum), and phenytoin. Examples of potent CYP3A4/5 inhibitors include atazanavir, boceprevir, clarithromycin, grapefruit juice, indinavir, itraconazole, ketoconazole, nefazodone, suboxone, nelfinavir, posaconazole, ritonavir, saquinavir, telithromycin, and voriconazole. Examples of CYP2C9 strong inducers/inhibitors include carbamazepine, enzalutamide, fluconazole, valproic acid, phenobarbital, nevirapine, rifampicin, and St. John's Wort.
- Patient has evidence of alcohol abuse or dependence, as defined by the DSM-5 criteria, with two or more of the 11 criteria at the Screening Visit or within 1 year before the Screening Visit.
- Patient has evidence of drug or chemical abuse (except nicotine), as defined by the DSM-5 criteria, at the Screening Visit or within 1 year before the Screening Visit. Patient has evidence of marijuana or cannabis dependency or has been treated for cannabis dependency, as defined by DSM-5 criteria, at the Screening Visit or within 1 year before the Screening Visit. (Patients who are prescribed stimulants should not be assessed for Stimulant Use Disorder. Patients who use cannabinoid-based medicine (e.g., cannabis in any form, nabilone, or THC-containing medications) for treatment of Tourette Syndrome with the investigator's knowledge should not be assessed for Cannabis Use Disorder, but these patients must comply with abstinence requirements for this study (See Inclusion 'f')). Patients who have a positive urine drug screen at the Screening Visit for drugs other than those allowed, as indicated in this criterion, are excluded.
- Patient is unwilling to comply with study restrictions including abstinence from cannabis and alcohol from the Baseline/Randomization Visit until the follow-up telephone call 14 days after the last dose of study medication.
- Patients receiving ongoing psychological therapy for tics such as Habit Reversal Training or Comprehensive Behavioral Intervention for Tics are excluded. Patients who have completed behavioral therapy for tics at least 30 days before the Baseline/Randomization Visit may participate.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ABX-1431Part 1: 8 weeks with daily administration; Patients who choose to enter Part 2: additional 4 weeks with daily administration | Drug | ABX-1431 |
| PlaceboPart 1: 8 weeks with daily administration | Drug | Placebo |
Primary outcome
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.
7 secondary outcomes
Adult Tic Questionnaire (ATQ)
Time frame: Part 1: days 0, 14, 28, 42, 56; Part 2: days 0, 14, 28
Premonitory Urge for Tics Scale (PUTS)
Time frame: Part 1: days 0, 14, 28, 42, 56; Part 2: days 0, 14, 28
Clinical Global Impressions Scale for Improvement (CGII)
Time frame: Part 1: days 14, 28, 42, 56; Part 2: days 14, 28
AE occurrence
Time frame: Throughout the study: Part 1: day - 30 to day 70; Part 2: day 0 to day 42
SAE occurrence
Time frame: Throughout the study: Part 1: day - 30 to day 70; Part 2: day 0 to day 42
SUSAR occurrence
Time frame: Throughout the study: Part 1: day - 30 to day 70; Part 2: day 0 to day 42
Discontinuations due to AE occurrence
Time frame: Throughout the study: Part 1: day - 30 to day 70; Part 2: day 0 to day 42
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Apr 2020.
- Derived from this study: PubMed 34117788Muller-Vahl KR, Fremer C, Beals C, Ivkovic J, Loft H, Schindler C. Monoacylglycerol Lipase Inhibition in Tourette Syndrome: A 12-Week, Randomized, Controlled Study. Mov Disord. 2021 Oct;36(10):2413-2418. doi: 10.1002/mds.28681. Epub 2021 Jun 12.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Chan Beals, M.D., Ph.D. study director
Abide Therapeutics, Inc.
8 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ABX-1431 in people with Tourette Syndrome and Motor Tic Disorder, run by Abide Therapeutics at 8 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in Germany, Spain, Poland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03625453, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Apr 2020.