Evaluation of Efficacy and Safety of Empagliflozin in Treatment of Neutropenia in Patients With Glycogenosis Ib
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Empaglifozin safety and tolerability measured by occurrence of adverse reactions
研究概览
简要总结
Treatment of neutropenia of Glycogenosis type 1b patients with empagliflozin
详细描述
Symptoms of glycogen storage disease type Ib (GSD Ib) include - among others - hypoglycemia, hepatomegaly and neutropenia with concomitant neutrophil dysfunction, which results in recurrent bacterial and fungal infections, and inflammatory bowel disease.
At present filgrastim is the only available drug to treat neutropenia in GSD Ib patients; it stimulates neutrophil production, but doesn't restore their function. Part of GSD Ib patients doesn't respond to filgrastim treatment. The latest research results showed, that neutropenia and neutrophil dysfunction in GSD Ib patients are results of extensive accumulation of 1,5-anhydroglucitol-phosphate. Empagliflozin, a SGLT2 inhibitor, inhibits renal glucose and 1,5-anhydroglucitol reabsorption and is an effective and safe method of treatment of neutropenia in this group of patients. Empagliflozin (Jardiance®) is a drug, which is registered in Poland to treat type II diabetes in adults. The aim of our study is to evaluate the efficacy and safety of neutropenia in patients with GSD Ib with empagliflozin (Jardiance®).
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
- 4 Weeks 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Minimum age 4 weeks old female Or Male
- •GSD1b confirmed by genetic analysis with neutropenia and/or reduced respiratory burst
- •Informed consent signed by the parents/assigns, and the recipient (>13 years old)
排除标准
- •Risk of non-compliance
- •Chronic renal diseases (eGFR < 60 ml/min/1,73 m2)
- •Active urinary tract infection (temporal criterion, up to recovery)
- •Participation in another clinical trial (minimum 6 months from the end of participation until the date of signing the Informed Consent Form)
- •Participation in therapeutic experiment, in addition to the experimental treatment with empagliflozin (minimum 12 months from the end of participation until the date of signing the Informed Consent Form)
- •Pregnancy, breastfeeding
- •Allergy to Empagliflozin
- •Lack of informed consent
研究组 & 干预措施
oral administration of Empagliflozin
干预措施: Empagliflozin (Drug)
结局指标
主要结局
Empaglifozin safety and tolerability measured by occurrence of adverse reactions
时间窗: 2 years
Empaglifozin saftey and tolerability measured by occurrence of adverse reactions
次要结局
- Efficacy of neutropenia treatment measured as percentage of the patients(2 years)
- Dosis change/withdrawal of filgrastrim(2 years)
- Degree of metabolic compensation(2 years)
研究者
Dariusz Rokicki
Pricipal Investigator
Children's Memorial Health Institute, Poland