Ecopipam for Tourette Syndrome

(D1AMOND Trial)

Recruiting at 110 trial locations

Overseen By Meredith M Miller

Age: Any Age

Sex: Any

Travel: May Be Covered

Trial Phase: Phase 3

Sponsor: Emalex Biosciences Inc.

Must not be taking: Tic medications, Anxiety drugs

Disqualifiers: Mood disorders, Renal insufficiency, others

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: Unknown

216 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial tests ecopipam tablets in people with Tourette's Disorder. The medication aims to reduce tics by blocking certain brain signals. Ecopipam is being tested for its safety and tic-reducing activity in adults with Tourette syndrome.

Will I have to stop taking my current medications?

Yes, you will need to stop taking any medications used to treat motor or vocal tics at least 14 days before starting the trial.

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

Several small trials suggest that ecopipam, a selective dopamine 1 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 medications. ^{1 2 3 4 5}

Is ecopipam safe for humans?

Ecopipam has been studied in several trials for Tourette syndrome, showing a low risk of side effects like weight gain and movement disorders, which are common with other treatments. ^{1 2 3 6 7}

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

Ecopipam is unique because it targets the dopamine D1 receptor, unlike other drugs for Tourette syndrome that target the D2 receptor. This difference may lead to fewer side effects like weight gain and movement disorders, which are common with other treatments. ^{1 2 3 8 9}

Eligibility Criteria

This trial is for children, adolescents, and adults with Tourette's Disorder who weigh at least 40 lbs and have a significant number of tics. Participants must not be on tic medications for two weeks before the study starts and should use effective contraception if sexually active.

Inclusion Criteria

I am 6 years old or older.

I weigh at least 40 pounds.

Minimum score of 20 on the YGTSS-R Total Tic Score

Exclusion Criteria

You are currently pregnant or breastfeeding.

You have a high risk of harming yourself.

My medication doses for anxiety, depression, or ADHD are stable.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

Titration

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

4 weeks

Maintenance

Participants continue on an open-label maintenance phase to assess the stability of the treatment

8 weeks

Randomized-Withdrawal

Participants are randomized to either continue ecopipam or switch to placebo to evaluate maintenance of efficacy

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Ecopipam Hydrochloride

Trial Overview The study tests ecopipam tablets to see if they help maintain control over tics in people with Tourette's. It starts with an open phase where everyone gets the drug, followed by a random phase where half get ecopipam and half get a placebo without knowing which one they're taking.

Participant Groups

Group I: 1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl) Experimental Treatment 1 Intervention

Ecopipam 11.2, 22.4, 33.6, 44.8, 67.2 and 89.6 mg tablets (containing 12.5, 25, 37.5, 50, 75 and 100 mg ecopipam HCl, respectively); 1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl) target dose; oral administration daily in evenings.

Group II: Placebo during R/WD Phase Placebo Group 1 Intervention

Matching Placebo tablets during R/WD period taken orally in the evening.

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]

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]

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 10-week study involving 11 children and adolescents with Tourette's disorder, aripiprazole significantly reduced tic severity, with most participants showing marked improvement in their symptoms.

The treatment was generally safe and well-tolerated, although some participants experienced side effects like increased appetite, weight gain, and mild extrapyramidal symptoms, indicating the need for further controlled trials to confirm its efficacy and safety.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Aripiprazole in children and adolescents with Tourette's disorder: an open-label safety and tolerability study. Lyon, GJ., Samar, S., Jummani, R., et al.[2021]

A review of six randomized controlled trials involving 528 children and adolescents with Tourette's syndrome found that aripiprazole (ARI) has similar efficacy to other treatments like tiapride (TIA) and haloperidol (HAL) for controlling tic symptoms.

Importantly, ARI was associated with significantly fewer extrapyramidal symptoms compared to HAL, making it a safer alternative treatment option for managing Tourette's syndrome.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Aripiprazole for Tourette's syndrome: a systematic review and meta-analysis. Zheng, W., Li, XB., Xiang, YQ., et al.[2022]

In a retrospective study of 28 patients with idiopathic hemifacial spasm, treatment with tiapride combined with horn of saiga tatarica resulted in an 89.29% effective rate, with 25 out of 28 patients showing significant reduction in spasm after 3 to 12 months.

The treatment was found to be effective and safe, but the authors emphasize the need for further blinded trials and long-term follow-up to establish it as a standard medical treatment for hemifacial spasm.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Tiapride with the horn of saiga tatarica in the treatment of hemifacial spasm]. Zhang, H., Cheng, S.[2013]

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

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

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

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

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

Future therapies of Tourette syndrome. [2019]

5. New Zealand pubmed.ncbi.nlm.nih.gov

Current Approaches and New Developments in the Pharmacological Management of Tourette Syndrome. [2019]

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

Aripiprazole in children and adolescents with Tourette's disorder: an open-label safety and tolerability study. [2021]

7. England pubmed.ncbi.nlm.nih.gov

Aripiprazole for Tourette's syndrome: a systematic review and meta-analysis. [2022]

8. Russia (Federation) pubmed.ncbi.nlm.nih.gov

[Treatment of Tourette syndrome]. [2013]

9. China pubmed.ncbi.nlm.nih.gov

[Tiapride with the horn of saiga tatarica in the treatment of hemifacial spasm]. [2013]