An Open Label Expanded Access Program Intended to Provide Treatment With Ecopipam (EBS-101) to Children, Adolescents, and Adults in the U.S. With Tourette's Disorder
试验速览
- 阶段
- 不适用
- 状态
- Available
- 试验地点
- 26
研究概览
简要总结
The goal of this expanded access study is to learn about the long-term effects of Ecopipam in participants with Tourette's Disorder (TD). The main question it aims to answer is:
- Is ecopipam safe and well-tolerated in participants equal and over the age of 6 to 50 with TD?
- Assess quality of life measures and to provide access to ecopipam while NDA is being prepared and submitted for review for marketing approval
Eligible participants with TD will take ecopipam with guidance from their treating physician. In addition to the standard of care from the treating physician, treatment with ecopipam will continue until ecopipam becomes commercially available in the United States or the program is terminated.
While in the EAP program, participants will be asked regarding how safe and tolerable ecopipam is to the participant. Several questionnaires will be collected regarding participants experiences while taking ecopipam during monthly visits for the first year and every three months thereafter until the conclusion of the study.
详细描述
The main purpose of this study is to further study the safety and tolerability of ecopipam in children, adolescents, and adults with Tourette's Disorder (TD).
The secondary objectives of this program are to assess patient-reported outcome measures including quality of life (QOL) and to provide access to ecopipam while a U.S. New Drug Application (NDA) is being prepared and submitted for review for marketing approval in the U.S.
This is an open label expanded access program for ecopipam (EBS-101) intended to collect safety information and physician-administered psychiatric scales and patient-reported outcomes in children, adolescents, and adults in the U.S. with TD who are unable to take currently approved medications for TD (aripiprazole, haloperidol, or pimozide) or any other D2 receptor antagonists (D2rA) due to treatment failure, intolerability, safety or lack of access.
At the Screening visit, participants will provide informed consent [adult participants or children/adolescent Legally Acceptable Representative (LAR)] and assent (children/adolescents) and be assessed against the inclusion/exclusion criteria for participation in the study. Demographic information, disease comorbidities, and concomitant medications will be documented, vital signs and a physical examination will be performed, blood and urine samples will be collected for safety laboratories, an electrocardiogram (ECG) will be performed, lifetime history for suicidality will be assessed using the Columbia Suicidality Severity Rating Scale (C-SSRS), and age group validated scales to assess anxiety and depression will be performed. The participant and in the case of pediatric participants their LAR will also be asked to complete scales to assess the severity of their disease and quality of life.
Based on satisfying Screening visit criteria, participants will receive a telephone call to confirm Enrollment (Day 1). Visit dates will be defined from date of first dose. Once Enrollment is confirmed, eligible participants will be registered with a specialty pharmacy who will dispense ecopipam. Participants will be gradually titrated over a period of 2-4 weeks depending on their weight to achieve a target steady-state dose of 1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl).
研究设计
- 研究类型
- Expanded Access
入排标准
- 年龄范围
- 6 Years 至 50 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •to be eligible for participation in the Study:
- •Age 6 to 50 years old inclusive at the time of informed consent/assent.
- •Participant must sign/date the most current Informed Consent Form (ICF). For minor participants the LAR must/sign date the most current consent and the participant must sign/date the most current age-appropriate assent.
- •In the opinion of the principal investigator (PI), the participant or in the case of a minor their LAR is capable of understanding and complying with protocol and program requirements, oral drug administration and care instructions.
- •Weight ≥18 kg (39.6 lbs.).
- •In the opinion of the PI, the participant meets diagnostic criteria for Tourette's Syndrome (TS) per Diagnostic and Statistical manual for Mental Disorders - 5th Edition (DSM-5-TR diagnostic criteria for TD).
- •The participant must have been treated with an FDA-approved treatment for TD (aripiprazole, haloperidol, or pimozide) or any other D2r antagonist (D2rA) and have experienced treatment failure, tolerability or safety issues as per LAR, participant or prescribing clinician or not have access to any of these approved medications.
- •Females of child-bearing potential must have a negative urine pregnancy test at Screening.
- •Females of child-bearing potential who are sexually active or become sexually active must be using double barrier method (condom and IUD, condom and spermicide, etc.) in addition to any oral contraceptive they may be using and agree to continue use of double barrier contraception for the duration of their participation in the study and for 30 days after their last dose of study drug. Females of non-childbearing potential are exempt from this criterion.
- •Non-Childbearing potential is defined by:
- •Post menopausal defined as >= 2 years amenorrheic, or,
- •Bilateral salpingo-oopherectomy, or
- •Hysterectomy, or
- •Pre-menarche
- •Sexually active male participants must use a barrier method of contraception during the study and agree to continue the use of male contraception for at least 30 days after the last dose of study drug.
- •Male participants must agree to not donate sperm and female participants not to donate eggs for at least 30 days after the last dose of study drug.
- •Diagnosis and Main Criteria for Inclusion:
- •Inclusion Criteria
- •Participants must fulfill all of the following inclusion criteria to be eligible for participation in the Study:
- •Age 6 to 50 years old inclusive at the time of informed consent/assent.
- •Participant must sign/date the most current Informed Consent Form (ICF). For minor participants the LAR must/sign date the most current consent and the participant must sign/date the most current age-appropriate assent.
- •In the opinion of the principal investigator (PI), the participant or in the case of a minor their LAR is capable of understanding and complying with protocol and program requirements, oral drug administration and care instructions.
- •Weight ≥18 kg (39.6 lbs.).
- •In the opinion of the PI, the participant meets diagnostic criteria for Tourette's Syndrome (TS) per Diagnostic and Statistical manual for Mental Disorders - 5th Edition (DSM-5-TR diagnostic criteria for TD).
- •The participant must have been treated with an FDA-approved treatment for TD (aripiprazole, haloperidol, or pimozide) or any other D2r antagonist (D2rA) and have experienced treatment failure, tolerability or safety issues as per LAR, participant or prescribing clinician or not have access to any of these approved medications.
- •Females of child-bearing potential must have a negative urine pregnancy test at Screening.
- •Females of child-bearing potential who are sexually active or become sexually active must be using double barrier method (condom and IUD, condom and spermicide, etc.) in addition to any oral contraceptive they may be using and agree to continue use of double barrier contraception for the duration of their participation in the study and for 30 days after their last dose of study drug. Females of non-childbearing potential are exempt from this criterion.
- •Non-Childbearing potential is defined by:
- •Post menopausal defined as >= 2 years amenorrheic, or,
- •Bilateral salpingo-oopherectomy, or
- •Hysterectomy, or
- •Pre-menarche
- •Sexually active male participants must use a barrier method of contraception during the study and agree to continue the use of male contraception for at least 30 days after the last dose of study drug.
- •Male participants must agree to not donate sperm and female participants not to donate eggs for at least 30 days after the last dose of study drug.
- •Participants cannot be enrolled in the study if they meet any of the following criteria.
- •Clinically meaningful abnormalities on laboratory tests at Screening as determined by the PI.
- •Specific laboratory findings at enrollment
- •T. Bili >= 2.5 x ULN for age (Participants with a history of Gilbert's Disorder must have prior documentation of the disorder.)
- •ALT >= 2.5 x ULN for age
- •AST >= 2.5 x ULN for age
- •Positive urine drug screen for cocaine, amphetamine, benzodiazepines, barbiturates, phencyclidine (PCP), or opiates at Screening, except those receiving stable, prescribed treatment for attention deficit/hyperactivity disorder (ADHD) or a positive for benzodiazepine prescribed (either routine or as needed) for any anxiety disorder or anxiety associated with a depressive disorder
- •Severe renal impairment or end stage renal disease as defined by eGFR < 60 ml/min/1.73m2
- •Any of the following cardiovascular:
- •ECG with a QTcF > 450 for males or > 470 for females at screening.
- •A history or finding on the screening ECG of any significant arrhythmia in the opinion of the principal investigator
- •A known family history of QT prolongation syndrome with sudden death
- •Current or anticipated need for medications known to prolong the QT interval
- •A lifetime history of any of the following disorders:
- •Myocardial Infarction
- •Cancer (excluding Basal Cell Carcinoma or Cervical Cancer in situ)
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排除标准
- 未提供