An Open Label Extension of SKY-0515 in Participants With Huntington's Disease Who Completed a Prior SKY-0515 Treatment Study
试验速览
- 阶段
- 2 期
- 状态
- Enrolling By Invitation
- 入组人数
- 500
- 试验地点
- 10
- 主要终点
- Long-term safety and tolerability of SKY-0515
研究概览
简要总结
The goal of this clinical trial is to test if the drug SKY-0515, an oral medication, can lower harmful proteins linked to Huntington's Disease (HD) and improve the symptoms of participants with HD. This study includes men and women aged 25 and older who have HD confirmed by genetic testing and meet certain requirements for physical ability and independence.
详细描述
This is an OLE study to investigate the long-term safety, tolerability, and efficacy of SKY-0515 administered orally in participants with HD. To be eligible for this OLE study, participants must have completed dosing in a prior SKY-0515 treatment study and meet eligibility criteria for this study at an approved clinical study site. The study is comprised of an enrollment visit, a treatment period, and a follow-up visit. Throughout the study, participants will be evaluated as specified in the Schedule of Activities. At the enrollment visit (which may be the same as the last treatment visit of the prior study), participants will be consented to enter the OLE study and initiate open label treatment with 9 mg tablets of SKY-0515. Open label SKY-0515 will be self-administered daily (QD).
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
- 25 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Must have completed treatment in a prior SKY-0515 treatment study and, in the opinion of the Investigator, have been compliant with the prior SKY-0515 treatment protocol study procedures.
- •Must be able and willing to meet all study requirements in the opinion of the Investigator including:
- •Participants must be able to tolerate blood draws
- •Willing and able to comply with all scheduled visits, treatment plans,laboratory tests and other study procedures
- •Women who can have children must have a negative pregnancy test before starting and use two types of birth control during the study and for 30 days after the last dose of the study drug.
- •Men must agree to use birth control during the study and for 90 days after the last dose.
排除标准
- •Have any other conditions that, in the opinion of the Investigator or Sponsor, would make the participant unsuitable for inclusion, or could interfere with the participant participating in or completing the study.
研究组 & 干预措施
9mg dose
Drug: SKY-0515 Type: Small molecule Route of Administration: Oral Dosage Frequency: Once daily Duration: until study drug is approved or study is discontinued Use: Experimental
干预措施: SKY-0515 (Drug)
结局指标
主要结局
Long-term safety and tolerability of SKY-0515
时间窗: Continuously from enrollment to study completion or approximately 36 months
To investigate the long-term safety and tolerability of SKY-0515 through the Incidence, severity, and relationship of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) (including withdrawals due to TEAEs)
次要结局
- Change From Baseline in the Z-score from the Composite Unified Huntington's Disease Rating Scale (cUHDRS)(Every 3 months for the first year, beginning with enrollment, then every six months for approximately 36 months.)
- Change from Baseline in Total Motor Score (TMS)(Every 3 months for the first year, beginning with enrollment, then every six months for approximately 36 months.)
- Change from Baseline in Total Functional Capacity (TFC)(Every 3 months for the first year, beginning with enrollment, then every six months for approximately 36 months.)
- Change from Baseline in Independence Scale Score (IS)(Every 3 months for the first year, beginning with enrollment, then every six months for approximately 36 months.)
- Change from Baseline in Symbol Digit Modalities Test (SDMT)(Every 3 months for the first year, beginning with enrollment, then every six months for approximately 36 months.)
- Change from Baseline in the Stroop Color and Word Test (SCWT)(Every 3 months for the first year, beginning with enrollment, then every six months for approximately 36 months.)