Long Term Study of Ecopipam Tablets in Children, Adolescents and Adults with Tourette’s Disorder

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Emalex Biosciences Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

30 Apr 2024 → ongoing

Decision date (initial)

2023-08-07

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Emalex Biosciences, Inc., United States

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Safety, Others, Efficacy

The primary objective of this study is to evaluate the long-term safety and tolerability of ecopipam tablets in children (≥ 6 and < 12 years of age), adolescents (≥ 12 and < 18 years of age), and adults (≥ 18 years of age) with TD who are eligible following their participation in studies EBS-101-TD-301, EBS-101-OL-001, or PSY-302A.

Secondary objectives 1

1. The secondary objective of this study is to assess the durability of treatment effect in the same patient population described above.

Conditions and MedDRA coding

Tourette's Disorder

Regulatory references

Scientific advice from competent authorities

Federal Institute For Drugs And Medical Devices, Medicines And Healthcare Products Regulatory Agency, Food And Drug Administration

EMA paediatric investigation plan (PIP)

EMEA-002564-PIP01-19

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

1. Subjects enrolling from the EBS-101-TD-301 completed all visits through Week 24 and days 7 and 14 safety follow-up; OR met relapse criteria, as defined in the EBS-101-TD-301 study, during the double-blind R/WD period and have completed the Early Termination visit, Day 7, and Day 14 Follow Up visits. Subjects enrolling from studies EBS-101-OL-001 or PSY302A who completed the respective studies; In the opinion of the Investigator the subject must have had clinical benefit from ecopipam and would benefit from continued participation; Female participants of childbearing potential must agree to use a highly effective method of contraception (i.e., pregnancy rate of less than 1% per year) during the study and for 30 days after the discontinuation of the IMP. Adequate contraceptive methods include combined hormonal contraception associated with inhibition of ovulation (oral, intravaginal, transdermal), progestogen-only hormonal contraception with inhibition of ovulation (oral, injectable, implantable), intrauterine devices (IUDs), intrauterine hormone-releasing system (IUS), true sexual abstinence (when this is in line with the preferred and usual lifestyle of the participant), bilateral tubal occlusion, vasectomized male partner or a female participant who is not of childbearing potential.
2. Female participants and female partners of male study participants using a hormonal contraceptive must also use a barrier method (i.e., condom or occlusive cap [diaphragm or cervical/vault caps]) and should have been stable on their hormonal contraceptive treatment for at least 4 weeks prior to Screening; Sexually active male subjects must use a highly effective method of contraception during the study and agree to continue the use of highly effective contraception for at least 30 days after the last dose of study drug; If <18 years of age, both of the subject’s parents or legal guardian, based on country and/or local laws, must sign a written informed consent and subject must sign a written informed assent according to the requirements of the site’s IRB/EC. If ≥18 years of age, subjects must sign a written informed consent according to the requirements of the site’s IRB/EC: Subjects must have TD based on Diagnostic and Statistical Manual for Mental Disorders – 5th Edition (DSM-5-TR diagnostic criteria) for TD.

Exclusion criteria 4

1. Any subject who was either lost to follow up, withdrawn consent, is non-compliant with study procedures, or study drug or otherwise withdrawn by either the site investigator or the sponsor at their discretion from PSY-302A, EBS-101-OL-001 or EBS-101-TD-301 studies; Subjects with a clinical presentation and/or history consistent with another neurologic condition that may have had accompanying abnormal movements (e.g., Huntington’s disease, Parkinson’s disease, Wilson’s disease, stroke, Restless Legs Syndrome); Any unstable mood disorder (DSM-5-TR criteria) at time of Screening or Baseline; Subjects who have unstable medical illness or clinically significant abnormalities on laboratory tests or ECG at Screening or Baseline as determined by the Principal Investigator. Subjects who have moderate to severe renal impairment at Screening; Subjects who have hepatic impairment at Screening; Adult subjects with PHQ-9 score > 10 at Screening or Baseline; Subjects with a history of neuroleptic malignant syndrome;
2. Subjects who have had previous treatment with investigational medication within 4 weeks prior to Screening for subjects who completed studies EBS-101-OL-001 or PSY-302A; Oral neuroleptics within 4 weeks prior to Screening; depot neuroleptics within 3 months prior to Baseline (e.g., risperidone microspheres) or 6 months prior to Baseline (e.g., paliperidone palmitate) for those subjects who completed EBS-101-OL-001 and PSY-302A; Subjects receiving other medications to treat motor or vocal tics except for baclofen, botulinum toxin, clonazepam, guanfacine, and topiramate would need those discontinued for at least 14 days prior to Baseline; Subjects receiving anti-depressant, anti-anxiety or ADHD medications unless the dosage has been stable for a minimum of 4 weeks prior to Baseline, unless otherwise excluded; Subjects who have required deep brain stimulation treatment ; Subjects with an onset of a major depressive episode in the past 6 months; Subject with a significant risk of attempting suicide based on history (suicide attempt in past 1 year or who have had 2 or more lifetime suicide attempts), or who had an answer of ‘‘yes’’ to either questions 4 or 5 (currently or within the past 30 days) on the baseline/screening version of the C-SSRS; Subjects with a history of seizures (excluding febrile seizures that occurred > 2 years prior to Baseline);
3. Subjects with a myocardial infarction within 6 months from Screening; Female subjects who are currently pregnant or lactating or planning to become pregnant during the course of the study; Subjects who have a need for medications which would have unfavorable interactions with ecopipam, e.g., CYP2D6 substrates with a narrow therapeutic window (e.g., digoxin), and drug with similar mechanism of action to ecopipam such as dopamine antagonists (e.g. neuroleptics), and VMAT2 inhibitors (e.g. tetrabenazine). Refer to Section 22 and 23 for allowable and prohibited medications; Subjects with current or recent (past 3 months) DSM-5 substance use disorder (with the exception of nicotine); Subjects with positive urine drug screen for cocaine, amphetamine, benzodiazepines, barbiturates, phencyclidine (PCP) or opiates at Baseline, except those receiving stable, prescribed treatment conditions unless specifically prohibited;
4. Subjects with a lifetime history of bipolar disorder type I or II, dementia, schizophrenia, or any other psychotic disorder; Subjects unable to swallow tablets; Subjects with a known hypersensitivity to any of ecopipam’s excipients, including subjects with confirmed lactose intolerance: Subjects who are employed by the sponsor, vendors working on the study, study site personnel or their family members; Siblings or family members of any current subject participating in the study; Subjects deprived of liberty by administrative or judicial decision, patients under court protection, guardianship, curatorship or family guardianship; Any subject who has required medical management with weight loss medication: Any subject who in the opinion of the investigator is not a suitable candidate for the study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. There are not any endpoints for the study since it is an Open Label study

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 6

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Emalex Biosciences Inc.

Sponsor organisation

Emalex Biosciences Inc.

Address

330 North Wabash Avenue Suite 3500

City

Chicago

Postcode

60611-3603

Country

United States

Scientific contact point

Organisation

Emalex Biosciences Inc.

Contact name

Frederick E. Munschauer

Public contact point

Organisation

Emalex Biosciences Inc.

Contact name

Frederick E. Munschauer

Third parties 4

Locations

8 EU/EEA countries · 36 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 218 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

17 submissions — initial application plus substantial / non-substantial modifications