A Phase II, Multicentre, Randomised, Double-blind, Parallel-Group, Placebo Controlled Study to Evaluate the Efficacy and Safety of IPN10200 as a Treatment for Cervical Dystonia in Adult Participants
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- Ipsen
- 入组人数
- 132
- 试验地点
- 87
- 主要终点
- Change from Baseline in the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) total score
研究概览
简要总结
The purpose of this study is to evaluate the efficacy and safety of the study drug, Corabotase (also known as IPN10200), and to assess how well it works when compared with placebo in treating Cervical Dystonia (CD) in adults.
CD can cause a series of abnormalities and symptoms in the head and neck that can lead to neck pain and stiffness, and headaches. CD is believed to involve deep parts within the brain that control movement, but genetic factors, environmental factors, and abnormalities in the brain may also play a role.
The usual treatment for CD includes injecting BoNT into the affected muscles, but the treatment only lasts about 3 months. Corabotase is designed to last for a longer period.
The study will consist of two periods:
- A Screening Period of up to 4 weeks (28 days) to assess whether a participant can take part in the study and requires at least one visit.
- A Treatment Period of 36 weeks.
On Day 1 of the treatment period, participants will receive either Corabotase Dose A or Dose B (additional participants may receive Corabotase Dose C) of the study drug, or placebo distributed into different muscles in the head, neck and shoulders. Participants may continue some other medications, but details need to be recorded.
There will be 10 visits to the clinic in person and one remote visits (phone call) (12 visits to the clinic for participants who receive Dose C). Participants will undergo blood samplings, urine collections, physical/neurological examinations, and clinical evaluations. Participants will also need to complete questionnaires throughout the study.
The total study duration for a participant will be up to 40 weeks (approximately 9 months).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •A clinical diagnosis of isolated Cervical Dystonia (CD) (idiopathic) characterized by dystonic symptoms localised to the head, neck, and shoulder areas with at least moderate severity at Screening and Baseline (Day 1) defined as:
- •(a) Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)-Total score ≥20
- •(b) TWSTRS-Severity subscale score ≥15
- •(c) TWSTRS-Disability subscale score ≥3
- •(d) TWSTRS-Pain subscale score ≥ 1
- •Treatment naïve or non-naïve to BoNT therapy for CD
排除标准
- •Participants presenting with a swallowing disorder of any origin which might be exacerbated by BoNT treatment, such as:
- •(a) Grade 3 or 4 on the Dysphagia Severity Scale (severe dysphagia) with swallowing difficulties and requiring a change in diet.
- •Predominant anterocollis.
- •Predominant retrocollis.
- •Traumatic torticollis or tardive torticollis.
- •Marked limitation on passive range of motion that suggests cervical contractures or structural abnormality.
研究组 & 干预措施
Group 2: Treatment Arm C
Corabotase - Dose C
干预措施: Corabotase (Biological)
Group 1: Placebo Comparator
Placebo- Group1
干预措施: Placebo (Other)
Group 1: Treatment Arm A
Corabotase - Dose A
干预措施: Corabotase (Biological)
Group 1: Treatment Arm B
Corabotase - Dose B
干预措施: Corabotase (Biological)
Group 2: Placebo Comparator
Placebo- Group 2
干预措施: Placebo (Other)
结局指标
主要结局
Change from Baseline in the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) total score
时间窗: At Week 4
The Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) is a rating scale for Cervical Dystonia (CD) consisting of three subscales: severity, disability and pain scales. The total score is the sum of each of the subscales, with a range of 0 to 85, where higher scores are indicative of greater impairment.
次要结局
- Change from Baseline in the TWSTRS total score at all other scheduled timepoints post injection until Week 36(At all timepoints post injection until Week 36.)
- Change from Baseline in the TWSTRS-Pain Subscale(At all timepoints post injection until Week 36.)
- Change from baseline in the daily Numerical Rating Scale (NRS) score(Averaged over every 7-day period until the Week 4 visit)
- Time to onset of pain reduction(From study injection to first timepoint at which at least 2-point reduction is observed in NRS score)
- Time to return of symptoms in responders (time from treatment to loss of 80% of peak treatment effect)(From randomization until Week 36)
- Change from Baseline in the TWSTRS-Disability Subscale(At all timepoints post injection until Week 36.)
- Change from Baseline in the TWSTRS-Severity Subscale(At all scheduled timepoints post injection until Week 36)
- Change from baseline in Clinical Global Impression of Severity score(At all timepoints post injection until Week 36.)
- Clinical Global Impression of Change score(At all timepoints post injection until Week 36.)
- Change from baseline in Patients' Global Impression of Severity score(At all timepoints post injection until Week 36)
- Patients' Global Impression of Change score(At all timepoints post injection until Week 36)
- Change from Baseline in the CDIP-58 total score(At all timepoints post injection until Week 36)
- Percentage of participants experiencing any Adverse Event (AEs) including treatment emergent adverse events (TEAEs), serious adverse events (SAEs), adverse event of special interest (AESI) and AE leading to treatment discontinuation(From baseline to Week 36.)
- Percentage of participants with clinically significant changes from baseline in Laboratory Parameters(At all timepoints post injection until Week 36)
- Percentage of Participants With Clinically Significant Changes from baseline in Vital Signs(At all timepoints post injection until Week 36)
- Percentage of participants with clinically significant change from baseline in focused neurological/physical examinations.(At all timepoints post injection until Week 36)
- Percentage of participants with clinically significant change from baseline in 12-lead Electrocardiogram (ECG) readings.(At all timepoints post injection until Week 36)
- Treatment-emergence of suicidal ideation/suicidal behaviour(From baseline to Week 36.)
- Percentage of participants with Binding antibodies to IPN10200(At all timepoints post injection until Week 36)
- Percentage of participants with neutralising antibodies to IPN10200(At all timepoints post injection until Week 36)