Trial Outcomes & Findings for Ecopipam Tablets to Study Tourette's Syndrome in Children and Adolescents (NCT NCT04007991)

Last Updated: 2023-10-04

Results Overview

The YGTSS was a semi-structured clinical interview designed to measure the tic severity. This scale consisted of a tic inventory, with 5 separate rating scales to rate the severity of symptoms, and an impairment ranking. Ratings were made along 5 different dimensions on a scale of 0=none to 5=severe for motor and vocal tics, each including number, frequency, intensity, complexity, and interference. The YGTSS TTS was the summation of the severity scores of motor and vocal tics. The total tic score (TTS) ranged from 0 (none) to 50 (severe) with higher score represent more severe symptoms. A negative change from baseline indicates improvement.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

153 participants

Primary outcome timeframe

Baseline, Week 12

Results posted on

2023-10-04

Participant Flow

The study was conducted at 45 sites in 5 countries (United States, Canada, Germany, France, and Poland) from 28 June 2019 (first participant first visit) and 23 September 2021 (last participant last visit).

A total of 215 participants were screened, of which 61 participants were screen failures and 153 participants were randomized in 1:1 ratio to receive ecopipam HCl and placebo.

Participant milestones

Participant milestones
Measure	Ecopipam HCI 2 mg/kg/Day Participants received a targeted steady-state dose of 2 milligram per kilogram per day (mg/kg/day) of ecopipam HCl tablets orally, once daily during a titration and treatment period for a total of 12 weeks.	Placebo Participants received matching placebo tablets orally, once daily during a titration and treatment period for a total of 12 weeks.
Overall Study STARTED	76	77
Overall Study Safety Set	76	77
Overall Study The Modified Intention-to-Treat Set	74	75
Overall Study COMPLETED	66	71
Overall Study NOT COMPLETED	10	6

Reasons for withdrawal

Reasons for withdrawal
Measure	Ecopipam HCI 2 mg/kg/Day Participants received a targeted steady-state dose of 2 milligram per kilogram per day (mg/kg/day) of ecopipam HCl tablets orally, once daily during a titration and treatment period for a total of 12 weeks.	Placebo Participants received matching placebo tablets orally, once daily during a titration and treatment period for a total of 12 weeks.
Overall Study Withdrawal by Subject	1	1
Overall Study Physician Decision	1	1
Overall Study Non-compliance with Study Drug	0	1
Overall Study Withdrawal by Parent/Caregiver	1	2
Overall Study Adverse event, non-fatal	5	0
Overall Study Lost to Follow-up	2	1

Baseline Characteristics

Ecopipam Tablets to Study Tourette's Syndrome in Children and Adolescents

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ecopipam HCI 2 mg/kg/Day n=76 Participants Participants received a targeted steady-state dose of 2 milligram per kilogram per day (mg/kg/day) of ecopipam HCl tablets orally, once daily during a titration and treatment period for a total of 12 weeks.	Placebo n=77 Participants Participants received matching placebo tablets orally, once daily during a titration and treatment period for a total of 12 weeks.	Total n=153 Participants Total of all reporting groups
Age, Continuous	12.6 years STANDARD_DEVIATION 2.78 • n=5 Participants	12.6 years STANDARD_DEVIATION 2.63 • n=7 Participants	12.6 years STANDARD_DEVIATION 2.70 • n=5 Participants
Sex: Female, Male Female	17 Participants n=5 Participants	24 Participants n=7 Participants	41 Participants n=5 Participants
Sex: Female, Male Male	59 Participants n=5 Participants	53 Participants n=7 Participants	112 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	13 Participants n=5 Participants	11 Participants n=7 Participants	24 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	63 Participants n=5 Participants	66 Participants n=7 Participants	129 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race/Ethnicity, Customized White	66 Participants n=5 Participants	72 Participants n=7 Participants	138 Participants n=5 Participants
Race/Ethnicity, Customized Black or African American	6 Participants n=5 Participants	3 Participants n=7 Participants	9 Participants n=5 Participants
Race/Ethnicity, Customized Asian	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Race/Ethnicity, Customized American Indian or Alaska Native	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race/Ethnicity, Customized Other	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Week 12

Population: The mITT set included all randomized participants who received at least 1 dose of study drug and had at least 1 post-baseline scoring of the YGTSS.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=74 Participants Participants received a targeted steady-state dose of 2 milligram per kilogram per day (mg/kg/day) of ecopipam HCl tablets orally, once daily during a titration and treatment period for a total of 12 weeks.	Placebo n=75 Participants Participants received matching placebo tablets orally, once daily during a titration and treatment period for a total of 12 weeks.
Change From Baseline in the Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS) at Week 12	-9.87 score on a scale Standard Error 1.062	-6.42 score on a scale Standard Error 1.006

SECONDARY outcome

Timeframe: Baseline, Week 12

Population: The mITT set included all randomized subjects who received at least 1 dose of study drug and had at least 1 post-baseline scoring of the YGTSS. Here, "Overall Number of Participants Analyzed" signifies participants who were evaluable for this outcome measure.

Clinical Global Impression (CGI) scale was used to assess overall severity on a 7-point Likert scale consisted of 2 reliable and valid 7-item Likert scales used to assess severity and change in clinical symptoms. The CGI severity scale ranges from 1 = Normal, not at all ill; 2 = Borderline ill; 3 = Mildly ill; 4 = Moderately ill; 5 = Markedly ill; 6 = Severely ill; 7 = Among the most extremely ill subject. 1 = "normal, not ill at all" to 7 = "extremely ill." A negative change indicates improvement in the condition.

Outcome measures

Outcome measures
Measure	Ecopipam HCI 2 mg/kg/Day n=65 Participants Participants received a targeted steady-state dose of 2 milligram per kilogram per day (mg/kg/day) of ecopipam HCl tablets orally, once daily during a titration and treatment period for a total of 12 weeks.	Placebo n=71 Participants Participants received matching placebo tablets orally, once daily during a titration and treatment period for a total of 12 weeks.
Change From Baseline in Clinical Global Impression of Tourette Syndrome Severity (CGI-TS-S) at Week 12	-0.91 score on a scale Standard Error 0.141	-0.53 score on a scale Standard Error 0.130

Adverse Events

Ecopipam HCI 2 mg/kg/Day

Serious events: 2 serious events

Other events: 47 other events

Deaths: 0 deaths

Placebo

Serious events: 1 serious events

Other events: 38 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Ecopipam HCI 2 mg/kg/Day n=76 participants at risk Participants received a targeted steady-state dose of 2 milligram per kilogram per day (mg/kg/day) of ecopipam HCl tablets orally, once daily during a titration and treatment period for a total of 12 weeks.	Placebo n=77 participants at risk Participants received matching placebo tablets orally, once daily during a titration and treatment period for a total of 12 weeks.
Gastrointestinal disorders Vomiting	1.3% 1/76 • Screening up to 30 days after the last dose ( up to Week 16)	0.00% 0/77 • Screening up to 30 days after the last dose ( up to Week 16)
Psychiatric disorders Suicidal ideation	0.00% 0/76 • Screening up to 30 days after the last dose ( up to Week 16)	1.3% 1/77 • Screening up to 30 days after the last dose ( up to Week 16)
Infections and infestations Coronavirus infection	1.3% 1/76 • Screening up to 30 days after the last dose ( up to Week 16)	0.00% 0/77 • Screening up to 30 days after the last dose ( up to Week 16)

Other adverse events

Additional Information

Clinical contact

Emalex Biosciences, Inc.

Phone: 7150562

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee PI must consult their clinical trial agreement. In summary, PI shall have the right to publish, present or otherwise use the results for their research publication objectives, provided that such Publication does not disclose Confidential Information. PI shall submit in writing to Sponsor any material at least 90 days for review and comment. Sponsor shall advise PI of any information which is Confidential Information or which may impair Sponsor's ability to obtain patent protection.
Publication restrictions are in place

Restriction type: OTHER

Measure	Ecopipam HCI 2 mg/kg/Day n=76 participants at risk Participants received a targeted steady-state dose of 2 milligram per kilogram per day (mg/kg/day) of ecopipam HCl tablets orally, once daily during a titration and treatment period for a total of 12 weeks.	Placebo n=77 participants at risk Participants received matching placebo tablets orally, once daily during a titration and treatment period for a total of 12 weeks.
Injury, poisoning and procedural complications Contusion	0.00% 0/76 • Screening up to 30 days after the last dose ( up to Week 16)	3.9% 3/77 • Screening up to 30 days after the last dose ( up to Week 16)
Injury, poisoning and procedural complications Fall	2.6% 2/76 • Screening up to 30 days after the last dose ( up to Week 16)	0.00% 0/77 • Screening up to 30 days after the last dose ( up to Week 16)
Injury, poisoning and procedural complications Muscle strain	2.6% 2/76 • Screening up to 30 days after the last dose ( up to Week 16)	0.00% 0/77 • Screening up to 30 days after the last dose ( up to Week 16)
Injury, poisoning and procedural complications Sunburn	2.6% 2/76 • Screening up to 30 days after the last dose ( up to Week 16)	1.3% 1/77 • Screening up to 30 days after the last dose ( up to Week 16)
Nervous system disorders Headache	15.8% 12/76 • Screening up to 30 days after the last dose ( up to Week 16)	9.1% 7/77 • Screening up to 30 days after the last dose ( up to Week 16)
Nervous system disorders Somnolence	7.9% 6/76 • Screening up to 30 days after the last dose ( up to Week 16)	2.6% 2/77 • Screening up to 30 days after the last dose ( up to Week 16)
Nervous system disorders Dizziness	3.9% 3/76 • Screening up to 30 days after the last dose ( up to Week 16)	0.00% 0/77 • Screening up to 30 days after the last dose ( up to Week 16)
General disorders Fatigue	7.9% 6/76 • Screening up to 30 days after the last dose ( up to Week 16)	0.00% 0/77 • Screening up to 30 days after the last dose ( up to Week 16)
General disorders Pyrexia	1.3% 1/76 • Screening up to 30 days after the last dose ( up to Week 16)	3.9% 3/77 • Screening up to 30 days after the last dose ( up to Week 16)
Gastrointestinal disorders Nausea	5.3% 4/76 • Screening up to 30 days after the last dose ( up to Week 16)	1.3% 1/77 • Screening up to 30 days after the last dose ( up to Week 16)
Gastrointestinal disorders Abdominal pain	2.6% 2/76 • Screening up to 30 days after the last dose ( up to Week 16)	0.00% 0/77 • Screening up to 30 days after the last dose ( up to Week 16)
Gastrointestinal disorders Abdominal pain upper	1.3% 1/76 • Screening up to 30 days after the last dose ( up to Week 16)	2.6% 2/77 • Screening up to 30 days after the last dose ( up to Week 16)
Gastrointestinal disorders Diarrhea	1.3% 1/76 • Screening up to 30 days after the last dose ( up to Week 16)	3.9% 3/77 • Screening up to 30 days after the last dose ( up to Week 16)
Gastrointestinal disorders Vomiting	1.3% 1/76 • Screening up to 30 days after the last dose ( up to Week 16)	0.00% 0/77 • Screening up to 30 days after the last dose ( up to Week 16)
Respiratory, thoracic and mediastinal disorders Nasal congestion	2.6% 2/76 • Screening up to 30 days after the last dose ( up to Week 16)	0.00% 0/77 • Screening up to 30 days after the last dose ( up to Week 16)
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	2.6% 2/76 • Screening up to 30 days after the last dose ( up to Week 16)	0.00% 0/77 • Screening up to 30 days after the last dose ( up to Week 16)
Psychiatric disorders Anxiety	5.3% 4/76 • Screening up to 30 days after the last dose ( up to Week 16)	0.00% 0/77 • Screening up to 30 days after the last dose ( up to Week 16)
Psychiatric disorders Restlessness	5.3% 4/76 • Screening up to 30 days after the last dose ( up to Week 16)	0.00% 0/77 • Screening up to 30 days after the last dose ( up to Week 16)
Psychiatric disorders Insomnia	9.2% 7/76 • Screening up to 30 days after the last dose ( up to Week 16)	1.3% 1/77 • Screening up to 30 days after the last dose ( up to Week 16)
Psychiatric disorders Depressed mood	3.9% 3/76 • Screening up to 30 days after the last dose ( up to Week 16)	2.6% 2/77 • Screening up to 30 days after the last dose ( up to Week 16)
Psychiatric disorders Depression	1.3% 1/76 • Screening up to 30 days after the last dose ( up to Week 16)	2.6% 2/77 • Screening up to 30 days after the last dose ( up to Week 16)
Psychiatric disorders Irritability	3.9% 3/76 • Screening up to 30 days after the last dose ( up to Week 16)	1.3% 1/77 • Screening up to 30 days after the last dose ( up to Week 16)
Psychiatric disorders Middle insomnia	3.9% 3/76 • Screening up to 30 days after the last dose ( up to Week 16)	1.3% 1/77 • Screening up to 30 days after the last dose ( up to Week 16)
Psychiatric disorders Sleep disorder	3.9% 3/76 • Screening up to 30 days after the last dose ( up to Week 16)	1.3% 1/77 • Screening up to 30 days after the last dose ( up to Week 16)
Psychiatric disorders Tic	2.6% 2/76 • Screening up to 30 days after the last dose ( up to Week 16)	2.6% 2/77 • Screening up to 30 days after the last dose ( up to Week 16)
Musculoskeletal and connective tissue disorders Back pain	0.00% 0/76 • Screening up to 30 days after the last dose ( up to Week 16)	2.6% 2/77 • Screening up to 30 days after the last dose ( up to Week 16)
Musculoskeletal and connective tissue disorders Myalgia	2.6% 2/76 • Screening up to 30 days after the last dose ( up to Week 16)	1.3% 1/77 • Screening up to 30 days after the last dose ( up to Week 16)
Musculoskeletal and connective tissue disorders Neck pain	2.6% 2/76 • Screening up to 30 days after the last dose ( up to Week 16)	0.00% 0/77 • Screening up to 30 days after the last dose ( up to Week 16)
Infections and infestations Nasopharyngitis	6.6% 5/76 • Screening up to 30 days after the last dose ( up to Week 16)	5.2% 4/77 • Screening up to 30 days after the last dose ( up to Week 16)
Infections and infestations Coronavirus infection	1.3% 1/76 • Screening up to 30 days after the last dose ( up to Week 16)	1.3% 1/77 • Screening up to 30 days after the last dose ( up to Week 16)
Metabolism and nutrition disorders Decreased appetite	2.6% 2/76 • Screening up to 30 days after the last dose ( up to Week 16)	5.2% 4/77 • Screening up to 30 days after the last dose ( up to Week 16)