A Phase 3, Long-Term, Open-Label and Single-Arm Study of MYOBLOC® in the Treatment of Troublesome Sialorrhea in Adult Subjects
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 187
- 试验地点
- 35
- 主要终点
- Occurrence of Adverse Events of Special Interest (AESI)
研究概览
简要总结
Multicenter, open-label, outpatient study of the safety and effectiveness of repeated doses of MYOBLOC over a 1-year duration in adult subjects with troublesome sialorrhea.
详细描述
Multicenter, open-label, outpatient study of the safety and effectiveness of repeated doses of MYOBLOC over a 1-year duration in adult subjects with troublesome sialorrhea. The primary goal is to determine the long-term safety and tolerability of MYOBLOC (administered intraglandularly as single total dose of 3,500 Units) treatments every 13 weeks over a maximum possible duration of 1 year in adult subjects with troublesome sialorrhea. The secondary goal is to assess the magnitude of therapeutic response of MYOBLOC (administered intraglandularly as a single total dose of 3,500 Units) using effectiveness assessments performed at intervals after treatments every 13 weeks over a maximum possible duration of 1 year.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Seeking treatment for troublesome sialorrhea for at least 3 months that is occurring secondary to any disorder or related to any cause, including, but not limited to, Parkinson's Disease (PD), adult cerebral palsy, amyotrophic lateral sclerosis (ALS), stroke, traumatic brain injury, oral cancer, and side effects of other medications.
- •Able to read and provide written informed consent before enrollment into the study, or the subject's caregiver (Legally Authorized Representative) can provide written informed consent.
- •Male or female, 18 to 85 years of age (inclusive).
- •Minimum unstimulated salivary flow rate of 0.2 g/min at screening
- •Minimum Investigator's Drooling Frequency and Severity Scale (DFSS) score of 4 at screening.
- •Ability and availability to participate in the study for up to 1 year (ALS subjects: ability and availability to participate in the study for at least 6 months), based on overall health of the subject and disease prognosis
排除标准
- •A moderate to high risk of aspiration will exclude participation in this study. Subjects whose risk of aspiration are judged by the Investigator to be satisfactorily controlled by placement of PEG tube or G-tube for nutritional support are eligible to participate.
- •Respiratory forced vital capacity (FVC) of <20% of predicted
- •Prior botulinum toxin type A or B treatment in the salivary gland(s) identified for treatment in this study within 24 weeks before screening. Prior botulinum toxin type A or B treatment into other anatomical regions not selected for treatment in this study is not exclusionary, but must have occurred at least 12 weeks before screening
- •Subjects should be excluded if, in the Investigator's opinion, the subject failed to respond to previous treatment with botulinum toxin. Subjects should not receive nor have any plans for receiving any botulinum toxin treatment, other than the study drug (MYOBLOC), during the entire course of the study (from the point the informed consent is signed until subject's participation is complete).
- •Concomitant use, or exposure within 5 half-lives of screening, of aminoglycoside antibiotics, curare-like agents, or other agents that interfere with neuromuscular function
- •Prior salivary gland surgery
- •Current treatment or treatment at any time during the study with Coumadin® (warfarin) or similar anti-coagulant medications. Anti-platelet medications are not specifically exclusionary
- •Evidence of any clinically significant neurologic disease
- •Pregnancy or lactation
- •Anticipated or scheduled surgery during the study period. A PEG tube/G tube may be placed for nutritional support at any time during the study and will not exclude the subject from continued study participation.
- •Major surgery (requiring general anesthesia, except PEG tube/G tube placement ) within the previous 6 months before screening.
- •Current infection at the sialorrhea treatment injection site(s)
- •History of drug or alcohol abuse currently or within the previous 6 months
- •Participation in another clinical drug, device, or biological agent study within 30 days of screening or while participating in this study
研究组 & 干预措施
MYOBLOC Injection
After a screening period (up to 21 days), subjects who satisfy all eligibility criteria may receive single dose injections over the course of 1 year.
干预措施: MYOBLOC (Drug)
结局指标
主要结局
Occurrence of Adverse Events of Special Interest (AESI)
时间窗: Visits at Weeks 4 and 13 combined for each TS; including Visit at Week 8 for TS1
Treatment Session (TS) AESI includes: Aspiration, Aspirational pneumonia, Choking and Dysphagia.
Occurrence, Seriousness, Severity, and Causality Assessment of Treatment Emergent Adverse Events (TEAE)
时间窗: Visits at Weeks 4 and 13 combined for each TS; including Visit at Week 8 for TS1
TEAEs Related to Study Medication include TEAEs classified as Possibly, Probably and Definitely Related. Treatment Session (TS)
Occurrence of Dental Adverse Events
时间窗: Visits at Weeks 4 and 13 combined for each TS; including Visit at Week 8 for TS1
Treatment Session (TS)
Columbia Suicide Rating Scale (C-SSRS): Suicidal Ideation, Behavior Scores and Intensity Scores
时间窗: Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13
Participants Analyzed does not match the Participant Flow because one participant discontinued prior to the first post-injection visit. Per protocol participants were only required to complete two treatment sessions with a maximum of 4 treatment sessions allowed. All subjects received 3500 U doses except for 13 subjects who had decreased doses at Treatment Session 2 and/or 3 (2500 U to 3000 U).
次要结局
- Change From Baseline in Drooling Impact Score (DIS)(Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13)
- Change From Baseline in Patient Global Impression of Severity (PGI-S)(Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13)
- Change From Baseline in Unstimulated Salivary Flow Rate (USFR): Treatment Sessions 1-4(Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13)
- Change From Baseline in Drooling Frequency and Severity Scale Performed by Trained Assessor (DFSS-I)(Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13)
- Change From Baseline in Clinical Global Impression of Severity (CGI-S)(Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13)
- Clinical Global Impression of Change (CGI-C)(TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13)
- Change From Baseline in Drooling Frequency and Severity Scale Performed by Subject (DFSS-S)(Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13)
- Patient Global Impression of Change (PGI-C)(TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13)