Non-interventional Study of Patients With Plexiform Neurofibromas and Neurofibromatosis Type I Starting Selumetinib in Russia
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- AstraZeneca
- 入组人数
- 150
- 试验地点
- 6
- 主要终点
- Time to discontinuation of selumetinib
研究概览
简要总结
The purpose of this study is to evaluate the effectiveness, safety of selumetinib and patient's parameters of symptomatic inoperable PN NF1 in real clinical practice in Russia
详细描述
This study will be non-interventional retrospective and prospective study. Assessment of parameters will be carried out as if a patient is treated in real-life clinical setting. The patients should be enrolled into this project after evaluation of eligibility criteria by an investigator 10 clinical sites. No additional procedures besides those already used in the routine clinical practice will be applied to the patients. Study population will consist of pediatric patients aged 3 years and older with NF1 and symptomatic inoperable PN who were prescribed with selumetinib
研究设计
- 研究类型
- Observational
- 观察模型
- Case Control
- 时间视角
- Other
入排标准
- 年龄范围
- 3 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •NF1 diagnosed (according to the criteria for evaluating NF1),
- •Symptomatic inoperable PN,
- •Patients receiving therapy with selumetinib. For newly treated patients - registration on the first day of starting treatment with selumetinib,
- •Age at starting of selumetinib treatment ≥ 3 to ≤ 18 years old (included in the study or started the therapy at the age of 18 years old),
- •Patients whose parents or guardians have signed a consent form.
排除标准
- •Patients currently participating in an interventional study/clinical trial,
- •Patients are excluded if there is evidence of an optic glioma, malignant glioma, malignant peripheral nerve sheath tumor, or other cancer, requiring treatment with chemotherapy or radiation therapy,
- •Patients who receive other antitumor treatment options on optic nerve glioma, malignant glioma, malignant tumor of the peripheral nerve sheath, or other cancer. If the patient on selumetinib treatment develop optic nerve glioma, malignant glioma, malignant tumor of the peripheral nerve sheath, or other cancer, he can continue selumetinib therapy +\- other treatment options on physician decision,
- •Female patients above 12 age who are pregnant /plan to become pregnant or at breast feeding.
结局指标
主要结局
Time to discontinuation of selumetinib
时间窗: from the FSI date (21 Mar 2023) till 36 months from FSI
Best objective response on selumetinib treatment
时间窗: from the FSI date (21 Mar 2023) till 36 months from FSI
a percentage of subjects who achieved a best response of CR, PR or stable disease during treatment
Reasons for discontinuation of selumetinib
时间窗: from the FSI date (21 Mar 2023) till 36 months from FSI
Description of diagnostic algorithms for PN NF1 in Russia
时间窗: from the FSI date (21 Mar 2023) till 36 months from FSI
ORR in patients receiving selumetinib according to REiNS criteria
时间窗: from the FSI date (21 Mar 2023) till 36 months from FSI
ORR (Overall response rate) in patients receiving selumetinib according to REiNS criteria: ORR is defined as the number of patients with CR (complete response) or cPR (confirmed partial response). CR is defined as disappearance of the target lesion. PR is defined as decrease in the volume of the target PN by 20% or more compared to the baseline and is considered unconfirmed at the first detection, confirmed when observed again within 3-6 months, and durable when the response is maintained for 1 year or longer In this trial ORR could be taken from the source records of the patient retrospectively. In this case CR or cPR counted from the therapy start date of a patient, which could have been before ICF signing.
次要结局
- Safety: adverse events (date, type, severe grade, seriousness criteria, outcome);(from the FSI date (21 Mar 2023) till 36 months from FSI)
- Clinical profile of patients with PN NF1 in Russia(from the FSI date (21 Mar 2023) till 36 months from FSI)
- Clinical outcomes of selumetinib treatment(From FSI date (21 Mar 2023) till 36 months from FSI)
- Demographic profile of patients with PN NF1 in Russia(from the FSI date (21 Mar 2023) till 36 months from FSI)