Use of Metformin as a Fetal Hemoglobin Inducer in Patients With Hemoglobinopathies
试验速览
- 阶段
- 早期 1 期
- 状态
- 已完成
- 入组人数
- 37
- 试验地点
- 2
- 主要终点
- Change in Fetal Hemoglobin (HbF) Percentage (SCA) or Change in Total Hemoglobin (Hb)
研究概览
简要总结
The purpose of this study is to determine whether metformin is effective in the treatment for sickle cell anemia (SCA).
详细描述
This is a dose escalation, pilot study for subjects with sickle cell anemia (SCA) disease to determine if metformin has a beneficial effect on the treatment and quality of life of SCA patients.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 10 Years 至 60 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of sickle cell anemia
- •Age greater than or equal to 10 and less than or equal to 60 years of age.
- •If on hydroxyurea, fetal hemoglobin less than 20% at a stable dose (mg/kg) determined by the primary hematology provider over at least four months.
- •Creatinine less than or equal to 1.4 mg/dL and estimated glomerular filtration rate greater than 45 ml/min/1.73 m2
- •Liver function tests (specifically ALT and conjugated bilirubin) less than or equal to 4 times upper limits of normal.
排除标准
- •Failure to meet inclusion criteria
- •Simple or chronic red blood cell transfusion therapy in the last 3 months OR a HbA level greater than 5% in SCA patients
- •Refusal to use medically effective birth control if female and sexually active.
- •If on hydroxyurea, not at stable dose of hydroxyurea for a minimum of 4 months (temporary exclusion).
- •Creatinine greater than 1.4mg/dL
- •Liver function tests (ALT and conjugated bilirubin) greater than 4 times upper limits of normal.
研究组 & 干预措施
Group A: Hydroxyurea + Metformin
Subjects who are currently taking Hydroxyurea as part of standard of care and have sickle cell anemia.
干预措施: Metformin (Drug)
Group A: Hydroxyurea + Metformin
Subjects who are currently taking Hydroxyurea as part of standard of care and have sickle cell anemia.
干预措施: Questionnaires (Behavioral)
Group B: Metformin (Group B has closed to enrollment)
Subjects who are not taking Hydroxyurea as part of standard of care and have sickle cell anemia.
干预措施: Metformin (Drug)
Group B: Metformin (Group B has closed to enrollment)
Subjects who are not taking Hydroxyurea as part of standard of care and have sickle cell anemia.
干预措施: Questionnaires (Behavioral)
结局指标
主要结局
Change in Fetal Hemoglobin (HbF) Percentage (SCA) or Change in Total Hemoglobin (Hb)
时间窗: 1 Year
Change in HbF percentage (%) or total Hb will be assessed by comparing baseline values to on treatment values per subject and will be summarized.
次要结局
- Variability of Hemoglobin Response(1 Year)
- Impact on Quality of Life(1 Year)
- Evaluation of RNA Sequencing(1 Year)
- Change in Laboratory Values(1 Year)
研究者
Titilope Fasipe
Assistant Professor
Baylor College of Medicine