A Double-blind, Placebo-controlled, Phase IIb, Multi-center, Ten-week Prospective Study to Evaluate the Efficacy and Safety of NOE-105 in Adult Male Patients With Childhood Onset Fluency Disorder (Orpheus)
概览
- 阶段
- 2 期
- 干预措施
- NOE-105
- 疾病 / 适应症
- Childhood-Onset Fluency Disorder
- 发起方
- Noema Pharma AG
- 入组人数
- 75
- 试验地点
- 9
- 主要终点
- Change From Baseline to End of 6 Weeks in Total MLGSSS
- 状态
- 已完成
- 最后更新
- 2个月前
概览
简要总结
This study is designed to evaluate the effectiveness of NOE-105 on speech fluency without the known antipsychotic-induced side effects of commonly used treatments for childhood onset fluency disorder (COFD).
详细描述
NOE-105 is an investigational selective PDE10A inhibitor with a potential therapeutic effect for the treatment of COFD. In this study adult male patients may be randomized to a double-blind, placebo-controlled, parallel group treatment with NOE-105 or placebo once daily. The study is designed to find the maximum tolerated dose of NOE-105 and thereafter, to maintain the participants at this dose until they have completed a total of 10 weeks treatment period. Following up to 10 weeks of treatment, participants will visit the study site for a follow-up visit within 28 (± 7) days of the date of the last dose of study treatment.
研究者
入排标准
入选标准
- •Patients must be 18 to 55 years of age inclusive, at the time of signing the informed consent.
- •Patients who satisfy DSM-5 criteria for childhood onset fluency disorder and are suitable for pharmacotherapy.
- •Have a history of stuttering for more than or equal to ≥ 2 years with onset consistent to developmental in nature before age 8 years.
- •Patient reported global stuttering experience as "moderate" at screening and baseline.
- •Patients must discontinue all medications used to treat stuttering for at least 14 days prior to receiving study treatment. With the exception of antipsychotic therapies (see exclusion criterion #11), other psychotropic drugs will be allowed provided they have been stable for at least 14 days prior to receiving study treatment and are expected to remain stable for the duration of the study.
- •BMI within the range 19 to 35 kg/m2 (inclusive).
- •Male Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- •Male patients must use a condom during the treatment period and until the end of relevant systemic exposure in the male participant, plus a further 90-day period. In addition, for a non-pregnant WOCBP partner.
- •Capable of giving signed informed consent
- •Able to read and write in English
排除标准
- •Stuttering is related to a known neurological cause eg, stroke, etc.
- •Low IQ in the opinion of the investigator.
- •Patients with uncontrolled seizure disorders.
- •A history of severe traumatic brain injury or stroke.
- •Patients who are, in the investigator's opinion, at imminent risk of suicide.
- •Known to have tested positive for human immunodeficiency virus.
- •Known DSM-5 diagnosis of substance abuse or dependence.
- •Unstable medical illness or clinically significant abnormalities on screening tests/exams.
- •Any unstable medical conditions or are currently ill (eg, congenital heart disease, arrhythmia or cancer), which, in the investigator's judgment, will put them at a risk of major adverse event during this trial, are expected to progress during the study, or will interfere with safety and efficacy assessments.
- •Initiation of new behavioral therapies for stuttering within 10 weeks prior to baseline.
研究组 & 干预措施
Active
Escalating doses of NOE-105 capsules
干预措施: NOE-105
Placebo
Escalating doses of matching placebo
干预措施: Placebo
结局指标
主要结局
Change From Baseline to End of 6 Weeks in Total MLGSSS
时间窗: 43 days
MLGSSS refers to Maguire-Leal-Garibaldi Self-rated Stuttering Scale. The scale includes ten questions that are assessed on a 0 to 10 scale with 0 being no impact and 10 being highest impact. The total score ranges from 0 to 100 with the higher the score the worse the stuttering.
次要结局
- Change From Baseline in Sheehan Disability Scale(Up to 71 days)
- Change From Baseline to End Point in Clinician-rated Stuttering Severity Instrument-4(Up to 71 days)