A Study of the Effect of Combination Lumacaftor and Ivacaftor on Markers of Hyperglycemia in Persons With Cystic Fibrosis
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 1
- 主要终点
- Change in units of insulin used over a period of 6 months to 1 year.
研究概览
简要总结
The purpose of this research study is to find out if the combined therapy lumacaftor-ivacaftor affects glycemia in patient with cystic fibrosis.
详细描述
This is a single center, open label study. Patients will have 1 visits at the Diabetes Research Center (DRC), or Clinical Research Center (CRC).
The participants will have been previously screened to make sure they are candidates for the study. These patients will be contacted prior to their first visit to discuss enrollment in the study.
At the study visit the participant will come to the CRC or DRC for a research visit. The following will occur at this study visit: informed consent; brief medical history; weight and height; vital signs and blood pressure; blood draw for DNA extraction, A1c and an extra research tube for storage. This will be scheduled at a time that is convenient to the patient.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Screening
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 18 years old or greater
- •Patients diagnosed with cystic fibrosis (CF), genotype homozygous Phe508del
- •Subject has been started on lumacaftor-ivacaftor for clinical reasons, with no contraindication for starting the drug* * Contraindications for taking drug include abnormal liver enzyme tests, renal dysfunction, pregnancy or nursing mothers
- •Exclusion Criteria
- •Does not have a HgbA1c within 1 year prior to starting medication.
- •Has not been on the combination therapy for at least 2 months
排除标准
- 未提供
研究组 & 干预措施
Lumacaftor-ivacaftor
Subjects will be monitored for glycemic changes before and after starting lumacaftor-ivacaftor.
干预措施: lumacaftor-ivacaftor (Drug)
结局指标
主要结局
Change in units of insulin used over a period of 6 months to 1 year.
时间窗: 1 year
Using chart review, the change in insulin units used per day will be calculated
Change in glycated hemoglobin (hemoglobin A1C)
时间窗: 1 year
A blood test will be used to determine the hemoglobin A1c change while on the medication.
次要结局
- Pulmonary function test (PFT) forced expiratory volume at one second (FEV1) measurements(1 year)
- Change in glycemia contingent on genetic risk score(1 year)
研究者
Jose C. Florez, MD, PhD
Chief of the Diabetes Unit, Associate Professor Harvard Medical School
Massachusetts General Hospital