Ecopipam for Tourette Syndrome

Recruiting at 66 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

150 Participants Needed

Trial Summary

What is the purpose of this trial?

The primary objective of this study is to evaluate the long-term safety and tolerability of ecopipam tablets in children (greater than or equal to \[\>=\] 6 and less than \[\<\] 12 years of age), adolescents (\>=12 and \<18 years of age), and adults (\>=18 years of age) with Tourette's Syndrome (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.

What data supports the effectiveness of the drug Ecopipam for treating Tourette Syndrome?

Several small trials suggest that Ecopipam, a selective dopamine D1 receptor antagonist, reduces tics in Tourette Syndrome with a low risk of side effects like weight gain and movement disorders, which are common with other treatments. ^{1 2 3 4 5}

Is ecopipam safe for humans?

Ecopipam has been studied in several trials for Tourette syndrome and is generally considered to have a low risk of side effects like weight gain and movement disorders, which are common with other treatments. ^{1 2 3 4 6}

How is the drug ecopipam different from other drugs for Tourette syndrome?

Ecopipam is unique because it targets the dopamine D1 receptor, unlike most other Tourette syndrome drugs that target the D2 receptor, and it has a lower risk of causing weight gain and movement disorders. ^{1 2 3 4 5}

Eligibility Criteria

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 am between 6 and 18 years old.

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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]

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]

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]

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 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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Ecopipam for Tourette Syndrome: A Randomized Trial. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

A D1 receptor antagonist, ecopipam, for treatment of tics in Tourette syndrome. [2021]

3. United States pubmed.ncbi.nlm.nih.gov

Ecopipam, a D1 receptor antagonist, for treatment of tourette syndrome in children: A randomized, placebo-controlled crossover study. [2019]

4. United States pubmed.ncbi.nlm.nih.gov

Future therapies of Tourette syndrome. [2019]

5. Germany pubmed.ncbi.nlm.nih.gov

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

6. Italy pubmed.ncbi.nlm.nih.gov

Treatment of phonic tics in patients with Tourette's syndrome using botulinum toxin type A. [2010]