A Study to Examine the Efficacy of a Therapeutic THX-110 for Tourette Syndrome

NCT03651726 | Unknown Phase 2 FDA Drug

• 1 other identifier: THX-G18-001
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 2

Brief Summary

This study evaluates the efficacy and safety of THX-110 in the management of tics and other symptoms (e.g. rage attacks, anxiety, depression, sleep difficulties) in patients with Tourette syndrome. In the first part of the study, half of the patients will receive THX-110, while the other half will receive a placebo. After completion of the first study part, patients will have the opportunity to continue into the second part of the study. In this part, all participants will receive THX-110.

Conditions

Tourette Syndrome

Outcome Measures

Primary Outcomes (1)

• Change in YGTSS-TTS

  Absolute change in YGTSS-TTS as a continuous endpoint at week 12 of the double-blind phase (visit 9) compared to baseline.

  Baseline and week 12 of the double-blind phase

Secondary Outcomes (16)

• Response to treatment according to YGTSS-TTS

  Baseline and week 12 of the double-blind phase

• YGTSS-TTS as a binary responder criterion and as a continuous endpoint at week 7 of the double-blind phase

  Baseline and week 7 of the double-blind phase

• YGTSS-GS (= YGTSS-TTS + YGTSS-impairment score)

  Baseline, week 7 and week 12 of the double-blind phase, week 19 and week 24 of the extension open label phase

• Modified Rush Video-Based Tic Rating Scale (MRVS)

  Baseline, week 7 and week 12 of the double-blind phase, week 19 and week 24 of the extension open label phase

• Clinical Global Impression-Improvement Score (CGI-I)

  Baseline, week 7 and week 12 of the double-blind phase, week 19 and week 24 of the extension open label phase

Study Arms (2)

THX-110 (dronabinol plus PEA)

EXPERIMENTAL

Double-blind phase and extension open label phase: Dose range of 2.5 mg to 10 mg dronabinol (1 to 4 capsules) plus 800 mg PEA (2 tablets) taken orally once daily; starting dose is 2.5 mg dronabinol plus 800 mg PEA. Up-titration is performed within maximal 3 weeks. The titration phase is followed by a maintenance phase of 9 weeks at stable dose.

Drug: THX-110 (dronabinol plus PEA)

Placebo

PLACEBO COMPARATOR

Double-blind phase: Range of 1 to 4 dronabinol placebo capsules plus 2 PEA placebo tablets taken orally once daily; starting dose is 1 dronabinol placebo capsule plus 2 PEA placebo tablets. Up-titration is performed within maximal 3 weeks. The titration phase is followed by a maintenance phase of 9 weeks at stable dose.

Drug: Placebo

Interventions

THX-110 (dronabinol plus PEA) DRUG

2.5 mg dronabinol capsules and 400 mg PEA tablets

Also known as: Dronabinol, Tetrahydrocannabinol, THC, Palmidrol, Palmitoylethanolamide

THX-110 (dronabinol plus PEA)

Placebo DRUG

Sesame oil pill manufactured to mimic 2.5 mg dronabinol capsules; cellulose pill manufactured to mimic 400 mg PEA tablets

Placebo

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Tourette syndrome according to DSM-5
• Male and female subjects with an age between ≥18 and \<65 years
• Total tic score (TTS) of the YGTSS \>18
• CGI-S ≥4
• Medication (and stimulation parameters for deep brain stimulation) for tics and comorbidities must be on a stable dose for at least 6 weeks before entering the study and subject must consent to maintain the stable dose during the study
• Signed written informed consent and willingness to comply with treatment and follow-up procedures
• Subjects capable of understanding the investigational nature, potential risks and benefits of the clinical study
• Women of child-bearing potential must have a negative pregnancy test (i.e., negative for urine human chorionic gonadotropin \[hCG\]) before first treatment with study medication. 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 a when used consistently and correctly), 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 without an alternative medical cause, and with a serum follicle stimulating hormone (FSH) in the post-menopausal range. Women who are surgically sterile may enter this study with historical documentation of surgical procedure (bilateral tubal ligation or bilateral oophorectomy at least 6 weeks prior screening or hysterectomy or uterine agenesis) and a negative pregnancy test.
• Male subjects must be willing to use a condom with sexual partners during this study and for a period of three months following the last administration of study medication until the follow-up visit. Male subjects must be willing to abstain from sperm donation for 3 months after the completion of this study.

You may not qualify if:

• Comorbid OCD, ADHD, depression, or anxiety disorder when unstable and/or in need of an initial adjustment for a therapy
• Presence of a comorbid psychiatric condition as developmental disability, psychotic illness or bipolar disorder
• Ongoing behavioural treatment for tics
• History of schizophrenia, seizure, psychotic, severe personality, or pervasive developmental disorder
• Current clinical diagnosis of substance abuse or dependence
• History of cannabis dependence
• Secondary and other chronic tic disorders or other significant neurological disorders
• History of severe cardiac diseases, severe cardiovascular diseases, severe renal disorders, or severe hepatic diseases and/or positive for human immunodeficiency virus (HIV), hepatitis C, or hepatitis B
• Concomitant medications have to be on stable dose since at least 6 weeks before entering the study and must be well tolerated at baseline without causing dizziness, confusion, sedation, or somnolence such as central nervous system depressants (e.g., barbiturates, benzodiazepines, ethanol, lithium, opioids, buspirone, scopolamine, antihistamines, tricyclic antidepressants, other anticholinergic agents, muscle relaxants)
• Use of cannabis or CBM in the 30-day period prior to study entry and/or positive delta-9-THC urine test at baseline
• Positive pregnancy test, i.e., positive for urine beta-hCG
• Pregnant or breast-feeding women
• Subjects who received any investigational medication or used any investigational device within 30 days prior to the first dose of study medication or is actively participating in any investigational drug or device study, or is scheduled to receive an investigational drug or to use an investigational device during the course of the study
• Subjects with a known allergy, hypersensitivity, or intolerance to the active substances and/or excipients of study medication (e.g., cannabis, cannabinoids, sesame oil)
• Any condition, which in the opinion of the investigator, would interfere with the evaluation of the study product or poses a health risk to the subject

Sponsors & Collaborators

• SciSparc: lead
• FGK Clinical Research GmbH: collaborator

Study Sites (2)

Medizinische Hochschule Hannover, Klinik für Psychiatrie, Sozialpsychiatrie und Psychotherapie

Hanover, 30625, Germany

Location

LMU Klinikum der Universität München, Klinik für Psychiatrie und Psychotherapie

Munich, 80336, Germany

Location

MeSH Terms

Conditions

Tourette Syndrome

Interventions

Dronabinol; palmidrol

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tic Disorders; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Cannabinoids; Terpenes; Hydrocarbons; Organic Chemicals

Study Officials

• Kirsten Müller-Vahl, Prof. Dr.

  Medizinische Hochschule Hannover, Klinik für Psychiatrie, Sozialpsychiatrie und Psychotherapie

  STUDY CHAIR

Central Study Contacts

Kirsten Müller-Vahl, Prof. Dr.

CONTACT

+49 511 532; mueller-vahl.kirsten@mh-hannover.de

Christoph Schindler, Prof. Dr.

CONTACT

+49 511 5350; schindler.christoph@mh-hannover.de

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Randomized, double-blind, placebo controlled, proof of concept study, followed by an open uncontrolled treatment phase

Study Record Dates

• First Submitted: August 9, 2018
• First Posted: August 29, 2018
• Study Start: August 1, 2018
• Primary Completion: November 1, 2019
• Study Completion: November 1, 2019
• Last Updated: August 29, 2018

Record last verified: 2018-08

Locations

DE (2)