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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：
8736955
负责人：
Deborah Merke
金额：
$0.85万
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/8736955
关键词：
Adrenal Cortex DiseasesAdrenal GlandsAdrenal gland hypofunctionAdultAldosteroneAndrogensChildhoodCircadian RhythmsComorbidityCongenital adrenal hyperplasiaDataDexamethasoneDiseaseDrug Delivery SystemsEnrollmentFailureFutureGlucocorticoidsGrowthHormone replacement therapyHydrocortisoneInfertilityInfusion proceduresLifeMetabolic syndromeObesityOsteoporosisPatientsPhysiologicalPilot ProjectsPrednisonePumpRecruitment ActivityReplacement TherapySafetySteroid 21-MonooxygenaseTestingVirilismdesignimprovednovel 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。这项新批准的试点研究已招募了一名患者，并正在积极招募。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，并将产生用于未来儿科研究设计的数据。
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 newly approved pilot study has enrolled one patient and is actively recruiting. 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 pediatric 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