Ecopipam for Tourette Syndrome

(D1AMOND Trial)

No longer recruiting at 120 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests ecopipam tablets to determine their effectiveness in helping people with Tourette's Disorder (TD) manage tics. Researchers aim to assess the treatment's long-term efficacy and safety for both children and adults. Participants will initially take the actual medication, then possibly switch to a placebo (a pill with no active drug) to compare results. The trial seeks individuals with Tourette's who experience both motor and vocal tics that disrupt daily life and are not currently on other tic medications. As a Phase 3 trial, this treatment represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking therapy for Tourette's Disorder.

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.

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

Research has shown that ecopipam hydrochloride is generally well-tolerated by people with Tourette's Syndrome. In one study, participants took ecopipam for a year, and no new or unexpected side effects emerged, indicating likely safety. Another study found that ecopipam effectively reduced tics in children and teenagers without causing significant mental health issues. Overall, ecopipam appears safe, but it's important to note that these studies involved only a small number of participants. ^{1 2 3 4 5}

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

Unlike the standard treatments for Tourette Syndrome, which often include antipsychotics or behavioral therapy, ecopipam offers a unique approach by targeting dopamine D1 receptors, rather than the more commonly targeted D2 receptors. This distinct mechanism is important because it may reduce tics without the side effects commonly associated with current medications, such as weight gain or sedation. Researchers are excited about ecopipam because it holds the potential to provide a more effective and tolerable treatment option for individuals with Tourette Syndrome.

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

Research has shown that ecopipam, which participants in this trial may receive, can help reduce tics in people with Tourette Syndrome (TS). One study found that only 41.9% of patients taking ecopipam experienced a return of symptoms, compared to 68.1% of those taking a placebo. This indicates that ecopipam can help keep symptoms under control. Long-term use of ecopipam has also lessened symptoms and improved quality of life. Ecopipam is considered safe and shows promise for managing TS symptoms, especially in children and teenagers. ^{1 5 6 7 8}

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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 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]

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]

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39730854/

Safety and Efficacy of Ecopipam in Patients with Tourette ... Ecopipam is effective in reducing the severity of tics in subjects with TS and has a good safety profile. However, only a limited number of ...

2. emalexbiosciences.com emalexbiosciences.com/news/emalex-biosciences-lead-candidate-meets-primary-and-secondary-endpoints-in-phase-3-tourette-syndrome-study/

Emalex Biosciences' Lead Candidate Meets Primary and ... The study found 41.9% of subjects randomized to ecopipam relapsed and 68.1% of subjects randomized to placebo relapsed. The results were ...

3. neurologylive.com neurologylive.com/view/ecopipam-efficacy-reduces-relapse-phase-3-tourette-syndrome

Ecopipam Maintains Efficacy and Reduces Relapse Risk ... New research highlights ecopipam's effectiveness in reducing Tourette syndrome symptoms and relapse risk in children, offering a promising ...

4. psychiatrictimes.com psychiatrictimes.com/view/new-positive-12-month-extension-data-ecopipam-for-tourette-syndrome-in-pediatric-patients

Ecopipam for Tourette Syndrome in Pediatric Patients Ecopipam for Tourette syndrome (TS) was found to be safe and effective in a 12-month, open-label extension (OLE) of a phase 2b study.

5. movementdisorders.onlinelibrary.wiley.com movementdisorders.onlinelibrary.wiley.com/doi/full/10.1002/mdc3.70091

Safety and Effect of 12‐Month Ecopipam Treatment in ... Compared to baseline, significantly reduced TS symptom severity and improved QOL were observed in children and adolescents. Tourette syndrome ( ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT06021522

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.

7. neurology.org neurology.org/doi/10.1212/WNL.0000000000204698

Safety and Tolerability of Ecopipam in Tourette Syndrome ... In the D1AMOND trial of children and adolescents with TS, ecopipam significantly reduced the YGTSS-TTS while not affecting measures of common psychiatric ...

8. emalexbiosciences.com emalexbiosciences.com/news/emalex-biosciences-releases-results-of-12-month-open-label-extension-study-for-tourette-syndrome-candidate/

Emalex Biosciences Releases Results of 12-Month Open ... An open-label extension study of 121 pediatric patients with Tourette syndrome evaluated the safety profile of the investigational drug ecopipam for up to 12- ...