Ecopipam for Tourette Syndrome

Not currently recruiting at 76 trial locations

Overseen By David JB Kim

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Emalex Biosciences Inc.

Must be taking: Ecopipam

Must not be taking: Neuroleptics, Antidepressants, Anti-anxiety

Disqualifiers: Neurological conditions, Mood disorders, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 3

Duration: 24 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and long-term effects of a new medication, ecopipam, for people with Tourette Syndrome (TS), which causes repeated movements or sounds (tics). The study aims to assess how well participants tolerate the medication over time. It targets individuals who participated in earlier related studies and experienced some benefit from this treatment. Participants must have both motor and vocal tics that interfere with daily life. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to the potential availability of a new treatment for TS.

Will I have to stop taking my current medications?

The trial requires participants to stop taking certain medications, including those for motor or vocal tics, antidepressants, anti-anxiety medications, and specific drugs like digoxin, fluoxetine, valproic acid, and bupropion, due to potential drug interactions with ecopipam.

Is there any evidence suggesting that ecopipam is likely to be safe for humans?

Research has shown that ecopipam has been tested for safety in several studies and is generally well-tolerated by patients. It works by blocking certain brain signals involved in conditions like Tourette syndrome.

In these studies, ecopipam proved to be safe, with participants experiencing only mild side effects. These effects were not serious and did not cause major problems. The trial's late phase indicates a solid understanding of its safety in humans.

Ecopipam is also under study for other conditions, which helps researchers learn more about its safety. While no treatment is without some risks, the data so far suggests that ecopipam is safe for further testing in people with Tourette syndrome. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Tourette's Syndrome?

Unlike the standard treatments for Tourette Syndrome, which often include antipsychotics or alpha-2 adrenergic agonists, ecopipam offers a novel approach by targeting dopamine D1 receptors. This mechanism of action is distinct because while most medications focus on dopamine D2 receptors, ecopipam's selective targeting of the D1 receptors could potentially reduce tics with fewer side effects. Researchers are excited because this specificity might lead to improved outcomes and better quality of life for patients, offering a fresh avenue in Tourette Syndrome management.

What evidence suggests that ecopipam might be an effective treatment for Tourette's Syndrome?

Research has shown that ecopipam, the investigational treatment in this trial, can help reduce tics in people with Tourette Syndrome (TS). One study found that ecopipam lowered the severity of tics and was safe to use. Another study noted that increasing the dose of ecopipam improved tic severity scores in children and teens. Ecopipam blocks a specific brain receptor related to movement and behavior, which may explain its benefits for TS. These findings suggest that ecopipam could be a promising option for managing Tourette Syndrome symptoms. ^{1 4 6 7 8}

Are You a Good Fit for This Trial?

This trial is for children (6+), adolescents, and adults with Tourette's Syndrome who have shown improvement on ecopipam. Participants must not have certain neurological conditions, unstable mood disorders, severe kidney or liver issues, or a recent history of substance abuse. Pregnant women and those unable to swallow tablets are excluded.

Inclusion Criteria

Participants who completed the studies EBS-101-OL-001 or PSY302A

I have been diagnosed with Tardive Dyskinesia according to DSM-5 criteria.

I completed all required visits and follow-ups for the EBS-101-TD-301 study and met the relapse criteria after 24 weeks.

Exclusion Criteria

Participants who discontinued the studies PSY-302A, EBS-101-OL-001 or EBS-101-TD-301 due to reasons such as lost to follow up, withdrawn consent, non-compliant or withdrawn by the discretion of either the site investigator or the sponsor

Pregnant or lactating women

You have been diagnosed with bipolar disorder, dementia, schizophrenia, or any other serious mental illness.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Titration

Participants undergo a 4-week titration phase to achieve a target steady-state dose of ecopipam

4 weeks

Weekly visits for dose adjustment

Treatment

Participants receive maintenance dose of ecopipam and are evaluated for safety

24 months

Monthly visits (in-person) at Months 1-12, 15, 18, 21, and 24

Follow-up

Participants are monitored for safety after the last dose of ecopipam

1 month

2 visits (in-person) at 7 and 14 days, 1 follow-up phone call at 30 days

What Are the Treatments Tested in This Trial?

Interventions

• Ecopipam

Trial Overview

The study tests the long-term safety of ecopipam tablets in treating Tourette's Syndrome across various age groups. It aims to understand how well patients tolerate this medication over an extended period.

How Is the Trial Designed?

Group I: Ecopipam 1.8 mg/kg/day Experimental Treatment 1 Intervention

Ecopipam tablets will be administered orally (PO) once daily in the evening without regard to meals at concentrations 11.2, 22.4, 33.6, 44.8, 67.2 and 89.6 milligrams (mg) containing 12.5, 25, 37.5, 50, 75 and 100 mg ecopipam HCl, respectively in 4-week titration phase to achieve a target dose of 1.8 milligram per kilogram per day (mg/kg/day) ecopipam (2 mg/kg/day ecopipam HCl). Participants will be evaluated for safety at each baseline visit and at all treatment visits up to 24 months and at follow up visits at 7 and 14 days after last dose of ecopipam.

Who Is Running the Clinical Trial?

Emalex Biosciences Inc.

Lead Sponsor

Trials

16

Recruited

1,000+

Published Research Related to This Trial

Ecopipam, a D1 receptor antagonist, significantly reduced tic scores in children and adolescents with Tourette syndrome compared to placebo, showing effectiveness at both 16 days and 30 days into treatment.

The treatment was well tolerated with minimal adverse effects, and no serious side effects like weight gain or dyskinesias were reported, suggesting it may be a safer alternative to traditional D2 receptor antagonists.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ecopipam, a D1 receptor antagonist, for treatment of tourette syndrome in children: A randomized, placebo-controlled crossover study. Gilbert, DL., Murphy, TK., Jankovic, J., et al.[2019]

Ecopipam significantly reduced tic severity in children and adolescents with Tourette syndrome compared to placebo, with a notable mean change in the Yale Global Tic Severity Score over 12 weeks (P = .01).

The treatment was well-tolerated, showing no significant weight gain or metabolic changes typically associated with antipsychotic medications, and common side effects included headache and insomnia, which were mild.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ecopipam for Tourette Syndrome: A Randomized Trial. Gilbert, DL., Dubow, JS., Cunniff, TM., et al.[2023]

Dopamine receptor antagonists are the primary treatment for Tourette's syndrome, but there is no clear consensus on which specific drug is most effective due to a lack of large controlled trials.

Tiapride is commonly used for treating tics in children in Germany, despite only one small controlled trial supporting its use, while other drugs like risperidone and pimozide appear to be more effective in adults.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[The benzamides tiapride, sulpiride, and amisulpride in treatment for Tourette's syndrome]. Müller-Vahl, KR.[2018]

In an open-label study of 18 adults with Tourette syndrome, treatment with the dopamine D1 receptor antagonist ecopipam resulted in a significant reduction in tic severity after 8 weeks, as measured by the Yale Global Tic Severity Scale (YGTSS).

While ecopipam showed promise in reducing tics, common side effects included sedation and fatigue, and further randomized controlled trials are needed to confirm its safety and efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A D1 receptor antagonist, ecopipam, for treatment of tics in Tourette syndrome. Gilbert, DL., Budman, CL., Singer, HS., et al.[2021]

New atypical antipsychotic medications are showing promise for treating Tourette syndrome, potentially offering better effectiveness and fewer side effects compared to current therapies.

Future treatments may include corticotropin-releasing factor antagonists and therapies targeting excitatory neurotransmitter systems, alongside nonpharmacologic options like immunologic and behavioral therapies, which could improve outcomes for patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Future therapies of Tourette syndrome. Chappell, PB., Scahill, LD., Leckman, JF.[2019]

In a study of 30 patients with Tourette's syndrome, 93% experienced improvement in vocal tics after receiving botulinum toxin type A (BTX-A), with 50% becoming tic-free.

The treatment had a mean response time of 5.8 days and a duration of effect lasting about 102 days, significantly improving quality of life, although 80% of patients reported hypophonia as a side effect.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Treatment of phonic tics in patients with Tourette's syndrome using botulinum toxin type A. Porta, M., Maggioni, G., Ottaviani, F., et al.[2010]

Citations

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ecopipam (PSYRX 101) News

Increasing Ecopipam Exposure Improves Yale Global Tic Severity Scale-Total Tic Scores (YGTSS-TTS) in Children and Adolescents with Tourette Syndrome (TS): ...

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A Study to Evaluate Long-term Safety of Ecopipam Tablets ...

A Study to Evaluate Long-term Safety of Ecopipam Tablets in Children, Adolescents and Adults With Tourette's Disorder. ClinicalTrials.gov ID NCT06021522.

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This Phase 3 multicenter study evaluates the maintenance of efficacy, safety and tolerability of ecopipam tablets in children, adolescents and adults in the ...

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A clinical trial of safety and tolerability for the selective ...

The D1R antagonist in this study SCH 39166 (ecopipam) has been assessed in several clinical studies and has been shown to be well tolerated and effective in ...

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Ecopipam hydrochloride (Synonyms: SCH 39166 ...

Ecopipam (SCH 39166) hydrochloride is a potent, selective and orally active antagonist of dopamine D1/D5 receptor, with Kis of 1.2 nM and 2.0 nM, respectively.

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Ecopipam

Ecopipam is a dopamine antagonist which is under development for the treatment of Lesch–Nyhan syndrome, Tourette syndrome, speech disorders, and restless ...