An Open-Label Extension Study to Assess the Long-Term Safety and Tolerability of ZYN002 Administered as a Transdermal Gel to Children, Adolescents, and Young Adults With Fragile X Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 476
- 试验地点
- 52
- 主要终点
- To evaluate the long-term safety and tolerability of ZYN002 administered as a transdermal gel formulation.
研究概览
简要总结
ZYN002 is a pharmaceutically manufactured Cannabidiol that is developed as a clear gel that can be applied to the skin (called transdermal delivery).
The gel will be applied to clean, dry, intact skin of the shoulders and/or upper arms.
Participants from the ZYN2-CL-016 and ZYN2-CL-033 studies who meet the inclusion criteria and none of the exclusion criteria for study ZYN2-CL-017 are eligible.
Parents/caregivers will apply the study gel twice daily for the 52-week treatment period.
详细描述
This is an open-label extension, multiple-center study, to assess the long-term safety and tolerability of cannabidiol administered as ZYN002, a transdermal gel, for the treatment of child and adolescent patients with Fragile X Syndrome (FXS). Male and female patients with FXS will be treated for up to 72 months. Up to 450 male and female patients, ages 3 to 18 years will be enrolled.
Parents/caregivers will apply the study gel twice daily for the 52-week treatment period.
Participants from study ZYN-CL-016 who weigh less than or equal to 35 kg, will receive 1 sachet of ZYN002, applied every 12 hours (± 2 hours).
Participants from study ZYN-CL-016 who weigh more than 35 kg will receive 2 sachets of ZYN002, applied every 12 hours (± 2 hours).
Participants from study ZYN-CL-033 who weigh less than or equal to 30 kg, will receive 1 sachet of ZYN002, applied every 12 hours (± 2 hours).
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
- 3 Years 至 29 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participated in study ZYN2-CL-016 or Study ZYN2-CL-
- •Patients and parents/caregivers agree to abide by all study restrictions and comply with all study procedures.
- •Patients and parents/caregivers must be adequately informed of the nature, risks of the study, and give written informed consent prior to enrollment in ZYN2-CL-
- •In the Investigator's opinion, the patients and parents/caregivers are reliable and are willing and able to comply with all protocol requirements and procedures.
- •Females of childbearing potential must have a negative pregnancy test at all designated visits
排除标准
- •Patient is receiving any investigational drugs (not ZYN002) or using any experimental devices.
- •Patient has an ongoing serious adverse event (SAE) or has experienced a SAE in ZYN2-CL-016 or ZYN2-CL-033, which in the opinion of the Investigator, should exclude them from participation.
- •Females who are pregnant, nursing, or planning a pregnancy; females of childbearing potential and male patients with a partner of childbearing potential who are unwilling or unable to use an acceptable method of contraception for the duration of therapy and for three months after the last dose of trial drug.
- •Patients who have alanine aminotransferase (ALT), aspartate aminotransferase (AST), or total bilirubin levels ≥ 2 times the upper limit of normal (ULN) or has alkaline phosphatase levels ≥ 3 times the ULN as determined from patient safety laboratories.
研究组 & 干预措施
ZYN002
ZYN002 - cannabidiol Transdermal Gel
干预措施: ZYN002 - Cannabidiol Transdermal Gel (Drug)
结局指标
主要结局
To evaluate the long-term safety and tolerability of ZYN002 administered as a transdermal gel formulation.
时间窗: Up to 96 month.
Safety assessment will include collection of any treatment emergent adverse events
次要结局
- To evaluate the long-term efficacy of ZYN002 in the treatment of symptoms of FXS.(Change from baseline to end of treatment)