Tourette SyndromePhase 1NCT03058562
A Study to Evaluate the Effects of ABX-1431 on Patients With Tourette Syndrome
A Phase 1 study testing ABX-1431 in people with Tourette Syndrome and Chronic Motor Tic Disorder, run by Abide Therapeutics at 1 site in Germany.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Tourette Syndrome, Chronic 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
- 23 participants
- Where
- 1 siteGermany
- Study dates
- 1 Feb 2017to 4 Oct 2017
- Phase
- Phase 1
- 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 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Trial locations and contacts
1 site in 1 country.
About this study
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
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.
Read the 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.
- Conditions
- Tourette Syndrome, Chronic Motor Tic Disorder
- Being tested
- ABX-1431, Placebo Comparator
- Medical subject headings
- Tourette Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ABX-1431Drug
ABX-1431, capsules, 40 mg in the fasting state
Placebo ComparatorDrug
Matching Placebo
ABX-1431Drug
ABX-1431, capsules, 20 mg with a high fat meal
| Study group | Role | What they receive |
|---|---|---|
| Crossover Sequence A | Experimental | Each in the fasting state:
Period 1: Single-dose matching placebo
Period 2: Single-dose ABX-1431 Receives: Drug: ABX-1431; Drug: Placebo Comparator |
| Crossover Sequence B | Experimental | Each in the fasting state:
Period 1: Single-dose ABX-1431
Period 2: Single-dose matching placebo Receives: Drug: ABX-1431; Drug: Placebo Comparator |
| Crossover Sequence C | Experimental | Each with a standard high fat meal:
Period 3: Single-dose matching placebo
Period 4: Single-dose ABX-1431 Receives: Drug: Placebo Comparator; Drug: ABX-1431 |
| Crossover Sequence D | Experimental | Each with a standard high fat meal:
Period 3: Single-dose ABX-1431
Period 4: Single-dose matching placebo Receives: Drug: Placebo Comparator; Drug: ABX-1431 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change of rating in Modified Rush Video Scale (MRVS) over time
Measured: pre-dose, post-dose (4 hours, 8 hours)
Change in rating of Yale Global Tic Severity Scale (YGTSS) over time
Measured: pre-dose, post-dose (4 hours, 8 hours)
Change of rating in Adult Tic Questionnaire (ATQ) over time
Measured: pre-dose, post-dose (4 hours, 8 hours, 12 hours)
Change of rating in Premonitory Urge for Tics Scale (PUTS) over time
Measured: pre-dose, post-dose (4 hours, 8 hours, 12 hours)
Secondary outcomes (6)
ABX-1431 and metabolite (M55) plasma pharmacokinetics
Measured: pre-dose, post-dose (2 hours, 4 hours, 8 hours, 24 hours)
2-AG hydrolysis in PBMC
Measured: 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)
Measured: 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
Measured: 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
Measured: screening, pre-dose, post-dose (24 hours)
12-lead ECG assessments
Measured: screening, pre-dose, post-dose (4 hours)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Feb 2017
- Study start1 Feb 2017
- Primary completion4 Oct 2017
- Study completion4 Oct 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 1
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- Abide Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Chan Beals, Study director, Abide Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03058562
- Sponsor's protocol ID
- ABX-1431_PN015
- Status last verified
- Nov 2017
- First submitted
- 13 Feb 2017
- Last updated
- 6 Nov 2017
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03058562
- Acronym / sponsor ID
- ABX-1431_PN015
- Conditions
- Tourette Syndrome, Chronic Motor Tic Disorder
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- Abide TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 1 Feb 2017 (actual)
- Primary completion
- 4 Oct 2017 (actual)
- Study completion
- 4 Oct 2017 (actual)
- First posted
- 23 Feb 2017 (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.
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.
Study design
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Crossover Sequence A | Experimental | Drug: ABX-1431, Drug: Placebo ComparatorEach in the fasting state: Period 1: Single-dose matching placebo Period 2: Single-dose ABX-1431 |
| Crossover Sequence B | Experimental | Drug: ABX-1431, Drug: Placebo ComparatorEach in the fasting state: Period 1: Single-dose ABX-1431 Period 2: Single-dose matching placebo |
| Crossover Sequence C | Experimental | Drug: Placebo Comparator, Drug: ABX-1431Each with a standard high fat meal: Period 3: Single-dose matching placebo Period 4: Single-dose ABX-1431 |
| Crossover Sequence D | Experimental | Drug: Placebo Comparator, Drug: ABX-1431Each with a standard high fat meal: Period 3: Single-dose ABX-1431 Period 4: Single-dose matching placebo |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
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-1431ABX-1431, capsules, 40 mg in the fasting state | Drug | Crossover Sequence A, Crossover Sequence B |
| Placebo ComparatorMatching Placebo | Drug | Crossover Sequence A, Crossover Sequence B, Crossover Sequence C, Crossover Sequence D |
| ABX-1431ABX-1431, capsules, 20 mg with a high fat meal | Drug | Crossover Sequence C, Crossover Sequence D |
Primary outcome
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)
6 secondary outcomes
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
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
Time frame: screening, pre-dose, post-dose (24 hours)
12-lead ECG assessments
Time frame: screening, pre-dose, post-dose (4 hours)
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 6 Nov 2017.
- Individual participant dataSharing planned: No
Related trials for Tourette Syndrome
Not yet recruiting
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Recruiting
Deep Brain Stimulation Therapy in Movement Disorders
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Exploring the Role of Neuroactive Steroids in Tourette Syndrome
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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 study director
Abide Therapeutics, Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ABX-1431 in people with Tourette Syndrome and Chronic Motor Tic Disorder, run by Abide Therapeutics at 1 site in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 6 Nov 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. 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. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 of rating in Modified Rush Video Scale (MRVS) over time (time frame: pre-dose, post-dose (4 hours, 8 hours)). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 6 Nov 2017.