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临床试验/NCT03616509
NCT03616509已完成4 期

Treatment With Growth Hormone in Adults With PWS, Effect on Muscular Tone Evaluated by Functional MRI, Relationship With Strength and Body Composition

Corporacion Parc Tauli1 个研究点 分布在 1 个国家目标入组 30 人开始时间: 2017年6月19日最近更新:
适应症
干预措施
相关药物

试验速览

阶段
4 期
状态
已完成
发起方
入组人数
30
试验地点
1
主要终点
Examine brain connectivity areas related with muscle tone maintenance

研究概览

简要总结

Prader-Willi syndrome (PWS) is a genetic disorder associated with growth hormone (GH) deficiency, central hypotonia and hyperphagia that leads to life-threatening obesity. Treatment with GH in adult patients is not well stablished in guidelines of Health National System (HNS). The investigators has experience in the study of brain connectivity in these patients in relation to satiety. To date, there is no evidence about the effect of GH on central hypotonia (brain areas related with muscle tone maintenance). So, the main objective is to examine these anatomical areas before and one year after GH treatment.

Methodology: Structural and functional magnetic resonance imaging to 30 PWS patients before and after GH treatment and we will compare them to a control group.

Expected results: PWS group will show abnormal functional and structural connectivity in circuitry of muscle tone maintenance that will improve after GH treatment. These favorable changes and the absence of secondary effects will help to justify the use of this treatment and its inclusion in practical clinical guidelines of HNS for the management of this syndrome in the adulthood.

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
18 Years 至 —(Adult, Older Adult)
性别
All
接受健康志愿者

入选标准

  • PWS >=18 years with growth hormone deficit
  • Signed informed consent by the patients or their legal guardian

排除标准

  • Severe obesity
  • No controled diabetes mellitus
  • No treated obstructive sleep apnea or severe obstructive sleep apnea
  • Active cancer
  • Active psychosis

研究组 & 干预措施

Placebo and Growth Hormone

Experimental

2 months on placebo followed by 12 months on GH

干预措施: Placebo (Drug)

Placebo and Growth Hormone

Experimental

2 months on placebo followed by 12 months on GH

干预措施: Growth hormone (Drug)

结局指标

主要结局

Examine brain connectivity areas related with muscle tone maintenance

时间窗: 24 months

Examine brain connectivity areas related with muscle tone maintenance making a functional MRI while the subject is making motor maneuvers, before anf after 12 months on GH treatment.

次要结局

  • Evaluation of body composition(24 months)
  • Evaluation of strength(24 months)

研究者

发起方
Corporacion Parc Tauli
申办方类型
Other
责任方
Principal Investigator
主要研究者

Olga Gimenez-Palop

Principal Investigator

Corporacion Parc Tauli

研究点 (1)

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