Pre-pubertal Low Dose Transdermal Estradiol in Turner Syndrome
2026年8月24日 更新者：Children's National Research Institute
Ultra-low Dose Transdermal Estradiol Therapy in Prepubertal Girls With Turner Syndrome and Primary Ovarian Insufficiency.
This is a research study to find out if treatment with transdermal estradiol patches started between 8 to 11.5 years is safe in girls with Turner syndrome who have ovarian failure and to see whether it may improve performance on two tests of working speed and short-term memory.
研究概览
地位
尚未招聘
详细说明
The investigators hypothesize that ultra-low dose TDE will improve neurocognitive measures without a negative impact on growth and pubertal maturation in the peripubertal period. To test this hypothesis, investigators will conduct a prospective single center pilot trial and recruit up to 15 girls with TS and POI (FSH>10 in the past year), ages 8 to <11.5 years who will receive ultra-low dose TDE [defined as 1/8th patch of 0.025mg/24h applied weekly (delivering 3.125mcg/24h)] for 6 months to address the following objectives.
Primary: Study the effect of ultra-low dose TDE therapy on processing speed and working memory. The investigators will administer the Pattern Comparison Processing Speed Test as well as the List Sorting Working Memory test NIH toolbox EnglishV3 at baseline, and 6-months of TDE treatment.
Secondary: Assess change in pubertal status, FSH, LH and estradiol levels with ultra-low dose TDE therapy. Height, weight measurements, assessment of Tanner staging for breast development, laboratory measurements of ultrasensitive estradiol, FSH, LH, lipid profile at baseline and 6-months of TDE treatment will be completed
研究类型
介入性
注册 (估计的)
15
阶段
- 阶段2
联系人和位置
本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。
学习联系方式
- 姓名：Roopa Kanakatti Shankar, MBBS, MS
- 电话号码：202-476-2121
- 邮箱：roopa.shankar@childrensnational.org
研究联系人备份
- 姓名：Kimberly Pitner, MSHS, BSN, RN
- 电话号码：202-476-1403
- 邮箱：kboucher@childrensnational.org
学习地点
-
-
District of Columbia
-
Washington D.C.、District of Columbia、美国、20010
- Children's National Hospital
-
接触：
- Kimberly Pitner, MSHS, BSN, RN
- 电话号码：202-476-1403
- 邮箱：kboucher@childrensnational.org
-
首席研究员：
- Roopa Kanakatti Shankar, MBBS, MS
-
-
参与标准
研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
- 孩子
接受健康志愿者
不
描述
Inclusion Criteria:
- Provision of signed and dated informed consent form
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Female, aged 8 years up to 11 y and 180 days with karyotype confirmed Turner syndrome
- FSH result> 10miu/mL in the past 12 months. Since variability in FSH is not uncommon, a single measure of FSH >10mIU/mL in the past calendar year will be deemed sufficient for inclusion into the study even if repeat measures may be under 10mIU/mL.
- Ability to take the transdermal estradiol medication and be willing to adhere to the treatment regimen
- Ability to complete the primary endpoint testing in English. Non-English speakers may be recruited if the participant is capable of performing the English version of the tests.
Exclusion Criteria:
- Spontaneous thelarche with breast Tanner stage 3 or more, spontaneous menarche or history of abnormal uterine bleeding.
- Previous estrogen exposure
- Cognitive impairment is severe enough to prevent assessment of the primary outcome measures.
- Untreated hypothyroidism or celiac disease: Individuals may be recruited if on treatment and stable medication dose in past 3 months.
- Subjects known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia
- History of any type of malignancy
- History of a known clotting disorder/deep venous thrombosis
- Any clinically significant abnormality as determined by the principal investigator.
学习计划
本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。
研究是如何设计的？
设计细节
- 主要用途：治疗
- 分配：不适用
- 介入模型：单组作业
- 屏蔽：无（打开标签）
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
|
实验性的：Estradiol Treatment
Patients opt to receive 6 months of transdermal ultra low dose estradiol treatment
|
Estradiol transdermal patch 0.025mg/24 hours- 1/8th fractionated patch will be applied and changed weekly for 6 months
|
研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Pattern Comparison processing speed test
大体时间：6 months
|
Changes in the Pattern Comparison processing speed test (PC) at 6 months on the NIH toolbox EnglishV3 compared to baseline.
Pattern Comparison processing speed test (PC) is a measure of the speed of information processing.
Participants see a pair of simple pictures on the iPad screen and must quickly discern whether the pictures are the same or different.
The total raw score is the number of items answered correctly during the 80 seconds of presentation time (Max 133 items).
The raw score is converted to a age-adjusted normative scores.
The Median Response Time Per Item is also available.
|
6 months
|
|
List Sorting Working Memory (LSWM) test
大体时间：6 months
|
Changes in the List sorting working memory test on the NIH toolbox EnglishV3 at 6 months compared to baseline.
The List Sorting Working Memory test measures a participant's working memory, the ability to hold information in a short-term buffer and then manipulate the information.
Pictures and written names of different foods and animals are displayed with accompanying audio-recorded and visual prompts, and the participant is asked to say the items back in size order from smallest to largest, first within a single dimension and then on two dimensions.
The raw score (up to 24 Max) is then converted to age-adjusted normative scores.
|
6 months
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Breast Tanner stage
大体时间：6 months
|
Changes in breast sexual maturity rating (Tanner staging) on examination over 6 months with ultra-low dose estradiol treatment
|
6 months
|
|
Pubertal hormone changes
大体时间：6 months
|
Changes in serum estradiol (measured in pg/mL), after 6 months of ultra-low dose estradiol treatment compared to baseline.
|
6 months
|
|
LH
大体时间：6 months
|
Luteinizing hormone (LH) (measured in U/L) over 6 months of estradiol therapy
|
6 months
|
|
FSH
大体时间：6 months
|
Change in Follicle stimulating hormone (FSH) measured in U/L over 6 months of estradiol therapy
|
6 months
|
其他结果措施
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Height velocity
大体时间：6 months
|
Change in annualized height velocity (in cm/year) over 6 months of treatment with ultra-low dose estradiol.
This will be calculated based on height measurements (in cm) at baseline and 6 months.
|
6 months
|
|
Lipid profile
大体时间：6 months
|
Changes in lipid profile (total cholesterol, triglycerides, high density lipoproteins, low density lipoproteins) over 6 months of treatment with ultra-low dose estradiol will be assessed.
|
6 months
|
合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始 (估计的)
2026年9月1日
初级完成 (估计的)
2027年6月1日
研究完成 (估计的)
2027年6月1日
研究注册日期
首次提交
2026年8月5日
首先提交符合 QC 标准的
2026年8月24日
首次发布 (实际的)
2026年8月25日
研究记录更新
最后更新发布 (实际的)
2026年8月25日
上次提交的符合 QC 标准的更新
2026年8月24日
最后验证
2026年8月1日
更多信息
与本研究相关的术语
其他相关的 MeSH 术语
其他研究编号
- STUDY00002034
计划个人参与者数据 (IPD)
计划共享个人参与者数据 (IPD)？
不
IPD 计划说明
Patient Privacy and on account of being a pilot trial
药物和器械信息、研究文件
研究美国 FDA 监管的药品
是的
研究美国 FDA 监管的设备产品
不
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