A Long-term Extension Study to Evaluate the Safety and Tolerability of Daily Oral LUM-201 in Children With Growth Hormone Deficiency (GHD)
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 发起方
- Lumos Pharma
- 入组人数
- 150
- 主要终点
- To evaluate the long-term safety and tolerability of LUM-201
研究概览
简要总结
This is a Multi-national Trial. The Goal of the Trial is to Offer Subjects Who Complete 12 Months in the LUM-201-10 Phase 3 Trial up to an Additional 36 Months of Treatment of LUM-201 While Evaluating Safety and Tolerability of LUM-201.
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
- 4 Years 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Parent/caregiver must be willing to provide written informed consent, and the subject must sign the assent, as applicable.
- •Subject must have successfully completed 12 months of participation in the LUM-201 Phase 3 GHD trial, and be eligible for continuation of treatment, pending all other enrollment criteria are met.
- •Subject who is sexually active must use an acceptable form of contraception.
- •Subject must be eligible for the Day 1 visit as confirmed by the Investigator.
排除标准
- •Subject has a medical or genetic condition that, in the opinion of the Investigator and/or MMs, adds unwarranted risk to use of LUM-
- •Pregnancy.
- •Subject has planned or is receiving current long-term treatment with medications known to prolong the QT interval or act as substrates, inducers, or inhibitors of the cytochrome system cytochrome P450 type 3A4 that metabolizes LUM-201 (see Appendix 6 for list of example medications). Subjects receiving shorter-term (two weeks or less) treatment with these medications should be evaluated on case-by-case basis by the Investigator in consultation with the MMs.
研究组 & 干预措施
LUM-201
干预措施: LUM-201 (Drug)
结局指标
主要结局
To evaluate the long-term safety and tolerability of LUM-201
时间窗: Day 1 - Month 36
* The number of subjects with at least 1 (serious) treatment-related adverse event (TEAE). * The number of subjects with at least 1 suspected unexpected serious adverse reaction. * The number of subjects with clinically significant abnormalities related to trial laboratory tests or electrocardiograms (ECGs). * The number of subjects with at least 1 adverse event of special interest (AESI). * Annual change from baseline in body weight standard deviation score (SDS). * Annual change from baseline in body mass index (BMI) SDS.
次要结局
- To evaluate the long-term effect of LUM-201 on growth.(Day 1 - Month 36)
- To evaluate the long-term effect of LUM-201 on pharmacodynamic (PD) markers.(Day 1 - Month 36)