What side effects are associated with this type of intervention?

Common treatments for Tourette Syndrome, particularly those involving dopamine receptor antagonists like Ecopipam, often have side effects including weight gain, sedation, metabolic changes, and extrapyramidal symptoms such as tardive dyskinesia. Other treatments, such as alpha-2 adrenergic agonists (e.g., clonidine and guanfacine), may cause drowsiness, fatigue, and hypotension. These side effects should be considered when choosing a treatment plan.

What prior FDA approvals exist?

The FDA has approved several medications for the treatment of Tourette Syndrome, primarily focusing on antipsychotics that target dopamine receptors. Notably, risperidone, a dopamine D2 receptor antagonist, has been used to manage tics and associated behavioral issues in Tourette Syndrome. While Ecopipam, a Dopamine D1 receptor antagonist, is currently under investigation, it represents a novel approach compared to the traditionally used D2 receptor antagonists. Other treatments include haloperidol and pimozide, which also act on dopamine receptors to alleviate symptoms.

What other types of research exist?

Promising novel alternatives to Ecopipam for Tourette Syndrome patients include: 1) VMAT2 inhibitors like valbenazine and deutetrabenazine, which have shown efficacy in reducing tics. 2) Cannabinoid-based treatments, such as nabiximols, which are being explored for their potential benefits in managing tics and associated symptoms. 3) Deep brain stimulation (DBS), a neurosurgical intervention that has shown promise in severe, treatment-refractory cases. These alternatives are currently under investigation and may offer new avenues for treatment in 2024.

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Dopamine D1 Receptor Antagonist

Ecopipam for Tourette Syndrome (D1AMOND Trial)

Phase 3

Waitlist Available

Research Sponsored by Emalex Biosciences Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

≥ 6 years of age

≥ 18 kg (~ 40 lbs.)

Timeline

Screening 14 weeks

Treatment 24 weeks

Follow Up 30 days

Awards & highlights

Pivotal Trial

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Possible side effects of ecopipam may include difficulty sleeping, restlessness, fatigue, changes in mood or behavior. Since it's being tested, there might be other unknown side effects that could emerge during the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am 6 years old or older.

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I weigh at least 40 pounds.

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I have Tourette's with both motor and vocal tics that disrupt my daily life.

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I haven't taken medication for motor or vocal tics in the last 14 days.

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I am using effective birth control during and for 30 days after the study.

Timeline

Screening ~ 14 weeks1 visit

Treatment ~ 24 weeks11 visits

Follow Up ~ 30 days2 visits

Screening ~ 14 weeks

Treatment ~ 24 weeks

Follow Up ~30 days

This trial's timeline: 14 weeks for screening, 24 weeks for treatment, and 30 days for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Yale Global Tic Severity Scale (YGTSS)

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: 1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl)Experimental Treatment1 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 PhasePlacebo Group1 Intervention

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Ecopipam Hydrochloride

2023

Completed Phase 1

~30

0 / 6Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Tourette Syndrome treatments often target the dopaminergic system due to its role in motor and behavioral symptoms. Ecopipam, a Dopamine D1 receptor antagonist, works by blocking dopamine D1 receptors, which can help reduce tics by modulating dopamine activity. Other common treatments include dopamine D2 receptor antagonists like haloperidol and risperidone, which also reduce tics by inhibiting dopamine's action at D2 receptors. These treatments are crucial for Tourette Syndrome patients as they help manage the frequency and severity of tics, improving overall quality of life and daily functioning.