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Continuous Subcutaneous Hydrocortisone Infusion Treatment for CAH

Continuous Subcutaneous Hydrocortisone Infusion Treatment for CAH
连续皮下注射氢化可的松治疗 CAH
批准号：
9550459
负责人：
Deborah Merke
金额：
$0.22万
依托单位：
EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH & HUMAN DEVELOPMENT
依托单位国家：
美国
项目类别：
财政年份：
--
资助国家：
美国
项目状态：
未结题
起止时间：
至
来源：
https://reporter.nih.gov/project-details/9550459
关键词：
Adrenal Cortex DiseasesAdrenal GlandsAdrenal gland hypofunctionAdultAldosteroneAndrogensCircadian RhythmsComorbidityCongenital adrenal hyperplasiaDataData AnalysesDexamethasoneDiseaseDrug Delivery SystemsEnrollmentFailureFutureGlucocorticoidsGrowthHormone replacement therapyHydrocortisoneInfertilityInfusion proceduresLifeMetabolic syndromeObesityOsteoporosisPatientsPhysiologicalPilot ProjectsPrednisonePumpReplacement TherapySafetySteroid 21-MonooxygenaseTestingVirilismdesignfollow-upimprovednovel strategiessubcutaneous

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中文摘要
翻译
这项研究仅限于21-羟化酶缺乏症导致的典型先天性肾上腺增生患者，但有可能显著改善其他形式肾上腺功能不全患者的生活。每一种疾病都需要终身治疗，这种新的给药方法有可能显著改善患者的生活。该试点研究已全部纳入，仅处于随访状态，数据分析正在进行中。此外，本研究将提供关于使用泵输送氢化可的松的安全性和耐受性的信息，并将产生将用于未来研究设计的数据。
英文摘要
This study is limited to patients with classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency, but has the potential to significantly improve the lives of patients with other forms of adrenal insufficiency. Each of these diseases requires life-long treatment and this novel approach to drug delivery has the potential to significantly improve patients lives. This pilot study is fully enrolled and is in follow-up only status and data analysis is underway. In addition, this study will provide information on the safety and tolerability of using a pump to deliver hydrocortisone, and will generate data that will be used in the design of future studies.
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会议论文
Modified-release Hydrocortisone Therapy as a Treatment for CAH
Study of a corticotropin releasing factor-1 receptor antagonist for the treatment of congenital adrenal hyperplasia
Novel treatment approaches: sex steroid blockade in children
  • 批准号：
    10916862
  • 项目类别：
  • 资助金额：
    $0.0万
  • 财政年份：
    --
  • 负责人：
    Deborah Merke
  • 依托单位：
    CLINICAL CENTER
Phase 2 Multi-center Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia
  • 批准号：
    10252568
  • 项目类别：
  • 资助金额：
    $0.0万
  • 财政年份：
    --
  • 负责人：
    Deborah Merke
  • 依托单位：
    CLINICAL CENTER
海外基金