TAME trial: a multi-arm phase II randomised trial of four novel interventions for malnutrition enteropathy in Zambia and Zimbabwe - a study protocol
TAME trial: a multi-arm phase II randomised trial of four novel interventions for malnutrition enteropathy in Zambia and Zimbabwe - a study protocol
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DOI：
10.1136/bmjopen-2018-027548
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发表时间：
2019-11-01
期刊：
影响因子：
2.9
通讯作者：
Prendergast, Andrew
中科院分区：
文献类型：
--
作者：
Kelly, Paul;Bell, Lauren;Prendergast, Andrew
在许多国家，儿童严重急性营养不良（SAM）仍然具有令人无法接受的高死亡率，特别是当并发继发感染或代谢紊乱时。迫切需要新的治疗方法，我们已经确定了肠道粘膜愈合作为新治疗方法的潜在目标。TAME试验（治疗营养不良肠病的方法）将在一个有效的多组单盲II期设计中评估四种新的治疗方法。在赞比亚和津巴布韦的三家医院中，225名患有急性呼吸暂停综合症的儿童将在他们的住院治疗达到从稳定到增加营养摄入的过渡点后，被随机分配到其中一种治疗方法或标准治疗方法。这四种干预措施是布地奈德、牛初乳或n -乙酰氨基葡萄糖，口服或经鼻胃管给予，或特杜葡肽皮下注射。主要终点是粪便炎症标志物的综合评分，一系列次要终点包括临床和实验室终点。治疗将持续14天，主要终点的评估将在14至18天进行，最终临床评估将在28天进行。在赞比亚的一部分儿童中，内窥镜活检将用于详细评估干预措施的效果。
Introduction Severe acute malnutrition (SAM) in children in many countries still carries unacceptably high mortality, especially when complicated by secondary infection or metabolic derangements. New therapies are urgently needed and we have identified mucosal healing in the intestine as a potential target for novel treatment approaches.Methods and analysis The TAME trial (Therapeutic Approaches for Malnutrition Enteropathy) will evaluate four novel treatments in an efficient multi-arm single-blind phase II design. In three hospitals in Zambia and Zimbabwe, 225 children with SAM will be randomised to one of these treatments or to standard care, once their inpatient treatment has reached the point of transition from stabilisation to increased nutritional intake. The four interventions are budesonide, bovine colostrum or N-acetyl glucosamine given orally or via nasogastric tube, or teduglutide given by subcutaneous injection. The primary endpoint will be a composite score of faecal inflammatory markers, and a range of secondary endpoints include clinical and laboratory endpoints. Treatments will be given daily for 14 days, and evaluation of the major endpoints will be at 14 to 18 days, with a final clinical evaluation at 28 days. In a subset of children in Zambia, endoscopic biopsies will be used to evaluate the effect of interventions in detail.