Ecopipam Tablets to Study Tourette’s Disorder in Children, Adolescents and Adults (D1AMOND)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Emalex Biosciences Inc., Emalex Biosciences Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

20 Jul 2023 → 4 Feb 2025

Decision date (initial)

2023-06-12

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Emalex Biosciences, Inc. United States

External identifiers

EU CT number

2023-503494-38-00

ClinicalTrials.gov

NCT05615220

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacodynamic, Therapy, Pharmacokinetic, Efficacy, Safety, Dose response

The primary objective of this study is to evaluate the maintenance of efficacy of ecopipam tablets in children (≥ 6 and < 12 years of age), adolescents (≥ 12 and < 18 years of age) with Tourette’s Disorder (TD).

Secondary objectives 1

1. The secondary objectives of this study are to evaluate the safety and tolerability of ecopipam dosed at 1.8 mg/kg/day (2 mg/kg/day ecopipam HCl) in children (≥ 6 to <12 years of age), adolescents (≥ 12 and < 18 years of age), and adults (≥ 18 years of age), to evaluate the maintenance of efficacy of ecopipam in adults (≧ 18 years of age), with TD and to characterize the pharmacokinetics (PK) of ecopipam in this patient population.

Conditions and MedDRA coding

Tourette’s Disorder

Regulatory references

Scientific advice from competent authorities

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

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 1

1. Subjects must be ≥ 6 years of age at time of screening; Subjects must weigh at least ≥18 kg (39.6 lbs.); Subjects must have TD based on the Diagnostic and Statistical Manual for Mental Disorders – 5th Edition-Text Revision (DSM-5-TR) diagnostic criteria for TD; Subjects must exhibit both motor and vocal tics that cause impairment with normal routines and have TD as their primary disorder; Subjects must have a minimum score of 20 on the YGTSS-TTS at Screening and at Baseline visits with tic symptoms causing impairment in the subject’s normal routines, including academic achievement, and/or occupational functioning, and/or social activities, and/or relationships or whose symptoms distress the subject to the extent that treatment is appropriate; 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. 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. For sites in the EU only, the subject must have received an adequate trial of non-pharmacological therapy without adequate response prior to study enrollment.

Exclusion criteria 2

1. Subjects with any unstable mood disorder (DSM-5-TR criteria) at Screening or Baseline; Subjects who have unstable medical illness or clinically significant abnormalities on laboratory tests, or ECG at Screening as determined by the Principal Investigator; Subjects with a significant risk of attempting suicide based on history, routine psychiatric status examination, investigator’s judgment, or who had an answer of ‘‘yes’’ on any question other than 1–3 (currently or within the past 30 days) on the baseline/screening version of the Columbia Suicide Severity Rating Scale (C-SSRS); Subjects with a clinical presentation at Screening or Baseline 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); Subjects who have moderate to severe renal impairment; Subjects who have hepatic impairment at Screening;
2. Subjects with a ≥25% difference in the absolute change in YGTSS-TTS between the Screening visit and the Baseline visit; Subjects with an onset of a major depressive episode in the past 6 months; Subjects who have had previous treatment with investigational medication within 4 weeks prior to Screening, oral neuroleptics within 4 weeks prior to Screening, and/or depot neuroleptics within 3 months prior to Screening (e.g., risperidone microspheres) or 6 months prior to screening (e.g., paliperidone palmitate); Subjects receiving any other medication to treat motor or vocal tics for at least 14 days prior to Baseline; Subjects receiving unstable doses or excluded medications to treat depression, anxiety or ADHD during the 4 weeks prior to Screening; ; Subjects with known hypersensitivity to any excipients of ecopipam tablets, including subjects with confirmed lactose intolerance; Subjects who have initiated new psychological therapies or deep brain stimulation within 10 weeks prior to Baseline visit.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Time from Randomization (Week 12) to Relapse in subjects ≥ 6 and < 18 years for ecopipam compared to placebo during the double-blind, R/WD period.

Secondary endpoints 1

1. Time from Randomization (Week 12) to Relapse in all subjects irrespective of age for ecopipam compared to placebo during the double-blind R/WD period.

Investigational products

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

Test 6

Placebo 1

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

Emalex Biosciences Inc.

Sponsor organisation

Emalex Biosciences Inc.

Address

1033 Skokie Boulevard Suite 600

City

Northbrook

Postcode

60062-4101

Country

United States

Locations

9 EU/EEA countries · 38 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

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 264 files

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

Application history

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