Menu Menu
 

Medical Professionals Research Information

Screening, Genes and QuestionnairesResearch brain

Gene Collection

  • A Multi-center gene collection sample and questionnaire
  • Southeast Center of Excellence Participating: All Centers within Region, headed by University of Florida and the TSAICG
    • The aim of this study is to find a gene that causes Tourette Syndrome
    • Tourette Syndrome Association International Consortium for Genetics (TSAICG) is a group of clinicians and scientists who have been working together, trying to find genes that cause Tourette Syndrome (TS). In the last few years, we have identified some chromosomes that may hold TS genes. Now, we want to follow up those findings and identify the genes that cause TS. In order to do this, we need to collect information about tics and other symptoms and DNA from thousands of people with TS. Finding the genes that cause TS is a key step to improving diagnosis and treatment for people with TS
    • Special Considerations: If appropriate and chosen for study, then patient will be provided with swab materials needed to complete at home
  • Contact information for Study: www.findtsgene.org
    • East Coast: Cornelia Illmann, PhD (1-877-883-9350)
    • West Coast: Jenny Zhong, B.A. (1-877-992-9950)

Screening Tool for Tics

  • Screen of Behavior and Movements in a Clinic Population:
  •  Southeast Center of Excellence Participating: University of South Florida, Rothman Center
    • The aim of this study is to develop a screening measure for tics in children and adolescents.
      • Inclusion:
        • Males or females aged 4 to 17 years of age
      • Exclusion:
        • None
  • Contact Information for Study:Alexis Wolfe (727-767-7238)

Genetics and Epidemiology Study

Medication Trials

Ecopipam

  • A multicenter, double-blind, placebo-controlled, randomized, cross-over study to assess the efficacy and safety of ecopipam in children ages 7-17 with TS (PSY 302):
  • Southeast Center of Excellence Participating: University of South Florida, Rothman Center
    •  To evaluate an experimental drug called ecopipam in children ages 7-17 with Tourette’s syndrome. Participants are in the trial for 3-4 months with visits every 2 weeks to monitor safety and efficacy. An Open-Label Extension of the study has recently been approved to provide participants who responded well to Ecopipam a year’s worth of study drug with visits every 3 months to check on efficacy and safety.
      • Inclusion:
        • Subjects must exhibit both motor and vocal tics.
        • Subjects must be age (≥ 7 to ≤ 18 years of age)
        • Subjects must weigh ≥ 45 lbs
        • Adolescent females of childbearing potential who are sexually active must be using effective contraception (i.e., oral contraceptives, intrauterine device, double barrier method of condom and spermicide) and agree to continue use of contraception for the duration of their participation in the study. They must also agree to use contraception for 30 days after their last dose of study drug.
        • Sexually active male subjects must use a barrier method of contraception during the study and agree to continue the use of male contraception for at least 30 days after the last dose of study drug.
      • Exclusion:
        • Subjects who have unstable medical illness or clinically significant abnormalities on laboratory tests, or ECG at Screening.
        • Subjects with a major depressive episode in the past 2 years
        • Subjects with a history of attempted suicide
        • Subjects with clinically significant suicidality (based on C-SSRS scale)
        • Subjects with a first-degree relative with a major depressive episode that resulted in any psychiatric hospitalization, or attempted/ completed suicide with the exception of a hospitalization for post-partum depression.
        • Subjects with a history of seizures (excluding febrile seizures that occurred >2 years in the past)
        • Subjects with a myocardial infarction within 6 months.
        • Girls who are currently pregnant or lactating.
        • Subjects who have a need for medication (other than ecopipam) with possible effects on TS symptoms (i.e., lithium, psychostimulants)
        • Subjects who have a need for medications which would have unfavorable interactions with ecopipam, e.g., dopamine antagonists or agonists [including buproprion], tetrabenazine, or monoamine oxidase inhibitors.
        • Subjects with a lifetime history of significant psychiatric disorder(s) as rated using the American Psychiatric Association DSM-5 Cross-Cutting Symptom Measures rating scale.
        • Subjects with current or recent (past 3 months) DSM-IV substance abuse or dependence (with the exception of nicotine).
        • Subjects with positive urine drug screen (cocaine, amphetamine, methamphetamine, tetrahydrocannabinol (THC), benzodiazepines, barbiturates, phencyclidine (PCP), opiates) at Screening. Subjects with urine positive only for benzodiazepines and/or marijuana (i.e., a user but not an abuser as based on DSM-IV criteria) may be eligible.
        • Subjects who have had previous treatment with ecopipam .
        • Subjects who have had treatment with:
          • investigational medication within 3 months of starting study
          • depot neuroleptics within 3 months of starting study
          • other psycho tropics with possible effects on TS symptoms (i.e., lithium, tetrabenazine) within 2 weeks prior to Screening.
          • oral neuroleptics within 4 weeks
          • selective serotonin reuptake inhibitors unless the dosage has been stable for a minimum of 4 weeks prior to study start and not prescribed to relieve the neurological signs of TS
  • Contact Information for Study: Alexis Wolfe (727-767-7238)

T-Force

  • A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of NBI-98854 in Pediatric Subjects with Tourette Syndrome:
  • Southeast Center of Excellence Participating: University of South Florida, Rothman Center
    • The aim of the T-Force Green study is to evaluate the efficacy of two active doses of Valbenazine administered once daily in pediatric subjects with TS, to assess the safety and tolerability of repeated daily doses, and to evaluate plasma exposure of Valbenazine and its metabolite following repeated daily doses. The duration of the study is 11 weeks total with 6 weeks of active medication or placebo.
      • Inclusion:
        • Be male or female, aged 6 to 17 years, inclusive.
        • Be in good general health, as determined by medical history, physical examination, clinical laboratory assessments, and 12-lead ECG.
        • Have a Diagnostic and Statistical Manual of Mental Disorders diagnosis of Tourette Syndrome (DSM-IV or -V).
        • Have TS symptoms that impair school, occupational, and/or social function.
        • If medications are being used to treat TS symptoms and/or associated conditions (ie, TS spectrum diagnoses), subjects must be on stable doses of these medications for a minimum of 30 days before baseline (Day -1), and the medication regimen is expected to remain stable throughout the study period. (The use of tetrabenazine to treat TS symptoms is prohibited.)
        • Subjects of childbearing potential who do not practice total abstinence must be instructed on the proper use of barrier methods of contraception and agree to use hormonal or two forms of nonhormonal contraception (dual contraception) consistently from screening until 30 days after the last dose of study drug. Acceptable methods of contraception include the following:
          • Condom with spermicide (cream, spray, foam, gel, suppository, or polymer film).
          • Diaphragm with spermicide (with or without condom).
          • Cervical cap with spermicide (with or without condom).
          • Vaginal sponge impregnated with spermicide used with condom.
          • Intrauterine device (IUD).
          • Hormonal contraception taken for at least 3 months prior to screening.
        • Subjects who practice total abstinence from sexual intercourse as the preferred lifestyle are not required to use contraception (periodic abstinence is not acceptable).
        • Female subjects of childbearing potential must have a negative serum β-human chorionic gonadotropin (β-hCG) pregnancy test at screening and negative urine pregnancy test at baseline (Day -1).
        • Have a body mass index (BMI) greater than or equal to the 5th percentile, but less than the 95th percentile of his/her age- and gender-matched BMI percentile at screening. (BMI is defined as the subject’s weight in kilograms divided by the square of the subject’s height in meters).
        • Adolescent subjects (12 to 17 years of age) must have a negative urine drug screen (negative for amphetamines, barbiturates, benzodiazepine, phencyclidine, cocaine, opiates, or cannabinoids) at screening and baseline (Day -1). Subjects who are on stable doses of prescribed and supervised (not prn) benzodiazepines, opiates, or psychostimulants (for subjects with comorbid ADHD) are allowed to participate in the study.
        • Adolescent subjects (12 to 17 years of age) must have a negative alcohol breath test at screening and baseline (Day -1).
        • Be willing and able to adhere to the study regimen and study procedures described in the protocol and informed consent/assent forms, including all requirements at the study center and return for the follow-up visit.
      • Exclusion:
        • Have an unstable medical condition or chronic disease (including history of neurological, hepatic, renal, cardiovascular, gastrointestinal, pulmonary, or endocrine disease), or malignancy that could confound interpretation of study outcome.
        • Had a medically significant illness within 30 days of screening.
        • Excessive use of tobacco and/or nicotine-containing products (based on the investigator’s assessment) within 30 days of screening.
        • Have a history of substance (drug) dependence or substance or alcohol abuse within the 3 months before baseline (Study Day -1), as defined in the DSM-IV or -V.
        • Are currently pregnant or lactating.
        • Have a known history of neuroleptic malignant syndrome.
        • Have a known history of long QT syndrome or cardiac arrhythmia.
        • Have aspartate transaminase (AST), alanine transaminase (ALT), gamma glutamyl transferase (GGT), total bilirubin, or serum creatinine levels greater than the ULN at screening.
        • Have a hematologic malignancy or solid tumor diagnosed within 3 years prior to screening, with the exception of localized skin cancer or carcinoma in situ of the cervix.
        • Have a positive human immunodeficiency virus antibody (HIV-Ab) test result, hepatitis B surface antigen (HBsAg) test result or hepatitis C virus antibody (HCV-ab) test result at screening.
        • Have received an investigational drug within 30 days before screening or plan to use an investigational drug (other than NBI-98854) during the study.
        • Have a blood loss ≥250 mL or donated blood within 56 days or donated plasma within 7 days of baseline (Day -1).
        • Have initiated Comprehensive Behavioral Intervention for Tics (CBIT) during the screening period or at baseline (Day -1) or plan to initiate CBIT during the study.
        • Self-report consumption of more than 6 caffeine-containing beverages a day within the last month before baseline (Day -1).
        • Have a significant risk of suicidal or violent behavior. Subjects with any lifetime suicidal behavior or suicidal ideation of type 4 (active suicidal ideation with some intent to act, without specific plan) or type 5 (active suicidal ideation with specific plan and intent) in the past 6 months before screening based on the Columbia Suicide Severity Rating Scale (C-SSRS) Children’s Version should be excluded.
        • Have ingested foods containing poppy seeds within 7 days before screening and baseline (Day -1).
        • Have an allergy, hypersensitivity or intolerance to tetrabenazine
        • Have had previous experience with the study drug
        • Have a history of or suspected poor compliance in clinical research studies.
  • Contact Information for Study: Alexis Wolfe (727-767-7238)

T-Forward

  • A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of NBI-98854 in Adult Subjects with Tourette Syndrome (T-Forward):
  • Southeast Center of Excellence Participating: University of South Florida, Rothman Center and the University of Florida
    • The aim is to evaluate the safety, tolerability, pharmacokinetics, and efficacy of 2 active doses of NBI-98854 within adults aged 18 – 64 years. The duration of the trial is 8 weeks of treatment and a follow up 2 weeks post treatment.
      • Inclusion:
        • Be male or female, aged 18 to 64 years, inclusive.
        • Be in good general health, as determined by medical history, physical examination, clinical laboratory assessments, and 12-lead ECG.
        • Have a DSM-IV or -V diagnosis of TS.
        • Have TS symptoms that impair school, occupational, and/or social function.
        • Subjects with TS spectrum diagnoses (eg, obsessive-compulsive disorder [OCD], Attention-Deficit Hyperactivity Disorder [ADHD]) must have a stable psychiatric status as clinically determined by the investigator at screening and baseline.
        • If medications are being used to treat TS symptoms and/or TS spectrum diagnoses, subjects must be on stable doses of these medications for a minimum of 30 days before baseline (Day -1), and the medication regimen is expected to remain stable throughout the study period. The use of dopamine antagonists (eg, pimozide, haloperidol, aripiprazole, risperidone, clozapine, olanzapine, ziprasidone) and/or tetrabenazine to treat TS symptoms is prohibited. Other non-dopaminergic tic suppression therapy (eg, clonidine, guanfacine) is allowed during the study period as long as the dose regimen has been stable for a minimum of 30 days before baseline (Day -1).
        • Subjects with stable medical conditions requiring medications that are not prohibited per protocol must be on stable doses of these medications for a minimum of 30 days before baseline (Day -1), and the medication regimen is expected to remain stable throughout the study period.
        • Subjects of childbearing potential who do not practice total abstinence must be instructed on the proper use of barrier methods of contraception and agree to use hormonal contraception or two forms of nonhormonal contraception (dual contraception) consistently from screening until 30 days after the last dose of study drug. Acceptable methods of contraception include the following:
          • Condom with spermicide (cream, spray, foam, gel, suppository, or polymer film).
          • Diaphragm with spermicide (with or without condom).
          • Cervical cap with spermicide (with or without condom).
          • Vaginal sponge impregnated with spermicide used with condom.
          • Intrauterine device (IUD).
          • Hormonal contraception taken for at least 3 months prior to screening.
            • The following subjects are not required to use contraception:
              • Subjects who practice total abstinence from sexual intercourse as the preferred lifestyle are not required to use contraception (periodic abstinence is not acceptable).
              • Female subjects with partners or male subjects who had been vasectomized at least 3 months prior to screening.
              • Female subjects who have been postmenopausal for at least 1 year prior to screening.
              • Female subjects who are surgically sterile (ie, bilateral oophorectomy, hysterectomy or bilateral tubal ligation) at least 3 months prior to screening.
            • Female subjects of childbearing potential must have a negative serum β-human chorionic gonadotropin (β-hCG) pregnancy test at screening and negative urine pregnancy test at baseline (Day -1).
            • Have a body mass index (BMI) of 18 to 40 kg/m2 (inclusive) at screening. (BMI is defined as the subject’s weight in kg divided by the square of the subject’s height in meters.)
            • Subjects must have a negative urine drug screen (negative for amphetamines, barbiturates, benzodiazepine, phencyclidine, cocaine, opiates, or cannabinoids) at screening and baseline (Day -1). Subjects who are on stable doses of prescribed and supervised (not prn) benzodiazepines, opiates, or psychostimulants (for subjects with comorbid ADHD) are allowed to participate in the study. Subjects with a positive urine drug screen for cannabinoids are eligible for participation if the use is for medicinal purposes and there is no indication of cannabinoid abuse.
            • Subjects must have a negative alcohol breath test at screening and baseline (Day -1).
            • Be willing to provide authorization for access to personal health information in conjunction with US Health Insurance Portability and Accountability Act (HIPAA).
            • Be willing and able to adhere to the study regimen and study procedures described in the protocol and informed consent forms, including all requirements at the study center and return for the final study day.
          • Exclusion:
            • Have an unstable medical condition or chronic disease (including history of neurological, hepatic, renal, cardiovascular, gastrointestinal, pulmonary, or endocrine disease), or malignancy that could confound interpretation of study outcome.
            • Had a medically significant illness within 30 days of screening.
            • Excessive use of tobacco and/or nicotine-containing products (based on the investigator’s assessment) within 30 days of screening.
            • Have a history of substance (drug) dependence or abuse within the 3 months before baseline (Day -1), as defined in the DSM-IV (Substance Dependence or Abuse) or DSM-V (Substance Use Disorder).
            • Are currently pregnant or lactating.
            • Have a known history of neuroleptic malignant syndrome.
            • Have a known history of long QT syndrome or cardiac tachy-arrhythmia.
            • Have a screening or Day -1 average triplicate ECG QT interval corrected for heart rate using corrected QT interval using Fridericia’s formula (QTcF) of >450 msec (males) or >470 msec (females) or the presence of any clinically significant cardiac abnormality.
            • Have a hematologic malignancy or solid tumor diagnosed within 3 years prior to screening, with the exception of localized skin cancer or carcinoma in situ of the cervix.
            • Have biochemistry or hematology not within the laboratory’s reference range and deemed by the investigator to be clinically significant at screening.
            • Have a positive human immunodeficiency virus antibody (HIV-Ab) test result, hepatitis B surface antigen (HBsAg) test result or hepatitis C virus antibody (HCV-Ab) test result at screening.
            • Have received an investigational drug within 30 days before screening or plan to use an investigational drug (other than NBI-98854) during the study.
            • Have initiated Comprehensive Behavioral Intervention for Tics (CBIT) during the screening period or at baseline (Day -1) or plan to initiate CBIT during the study.
            • Have a blood loss ≥550 mL or donated blood within 30 days before baseline (Day -1).
            • Have a significant risk of suicidal or violent behavior. Subjects with any suicidal behavior or suicidal ideation of type 4 (active suicidal ideation with some intent to act, without specific plan) or type 5 (active suicidal ideation with specific plan and intent) within the past year before screening based on the Columbia Suicide Severity Rating Scale (C-SSRS) should be excluded.
            • Have ingested foods containing poppy seeds within 7 days before screening and baseline (Day -1).
            • Have an allergy, hypersensitivity, or intolerance to tetrabenazine.
            • Have a history of or suspected poor compliance in clinical research studies.
    • Contact Information for Study: Alexis Wolfe (727-767-7238) at the Rothman Center
    • Contact Information for Study: Heather Simpson, MOT, OTR/L (352-294-5385; simph@shands.ufl.edu) at the University of Florida

Other Studies

Handwriting and Tourette Syndrome

  • A multicenter graphomotor assessment completed by rehab professionals to assess handwriting quality and efficiency in children and adolescents with TS.
  • Southeast Center of Excellence Participating: University of Alabama-Birmingham/Children’s of Alabama and University of Florida