Estrogen Supplementation and Bone Health in Women With CF (STURDY)
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 75
- 试验地点
- 1
- 主要终点
- Change in lumbar spine bone mineral density assessed by dual-energy x-ray absorptiometry (DXA)
研究概览
简要总结
The goal of this study is to learn about the role of estrogen and other hormones in bone development in adolescent and young adult women with cystic fibrosis (CF). The study has two main components, an observational study and a feasibility sub-study. The main questions it aims to answer are:
- How do different forms of hormonal contraception (e.g. birth control pill or other progesterone only methods) impact bone health?
- Is a study of transdermal estradiol (estrogen skin patches) feasible in this group?
- How does transdermal estradiol impact bone health and quality of life?
Participants in the observational study will have three study visits (baseline, 6 months, and 12 months). At all visits, participants will have blood drawn and will complete questionnaires. At the baseline and 12 month visits, participants will additionally have x-rays and DXAs (bone density assessment). Between visits, participants will complete brief questionnaires. Researchers will compare various measurements over time to assess the impact of different forms of hormonal contraception.
Participants in the feasibility sub-study will apply transdermal estradiol patches once weekly for 12 months, fill out brief quality-of-life surveys in between study visits, and complete blood draws and questionnaires at four study visits (baseline, 3 months, 6 months, and 12 months). The participants will also have x-rays and DXAs performed at the baseline and 12 month visits. Researchers will examine changes in various measurements to determine the impact of transdermal estradiol.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 0 Years 至 100 Years（Child, Adult, Older Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •CF Diagnosis
- •Females who have had at least 1 menstrual cycle
- •Planning to use same formulation of estrogen supplementation (or none) for duration of study
排除标准
- •Use of anti-osteoporosis therapy including anti-resorptives such as bisphosphonates or anabolic agents such as teriparatide or denosumab
- •Conditions in which bone loss is known to be present or expected to occur, such as lactation
- •Pregnant or planning to become pregnant
- •In the opinion of the CF care team or study investigators participant should not participate in the study
- •Inability to provide informed consent/assent
- •Feasibility Sub-Study:
- •Inclusion Criteria:
- •All of above and
- •<35 years old
- •At least 2 years after first menstrual cycle
- •Symptoms of low estrogen and/or low serum estradiol levels (< 50 pg/mL)
- •Exclusion Criteria:
- •All of above and
- •Contraindications to transdermal estradiol
- •Current use of systemic estrogen (such as estrogen-containing oral contraceptive pill)
- •Previous lung or liver transplant
- •Use of chronic systemic glucocorticoids
- •Severe vitamin D deficiency (serum 25(OH)D < 6 ng/mL)
- •Conditions increasing risk of clot: history of previous deep vein thrombus or pulmonary embolism, current immobility, current peripherally inserted central catheter or mediport, known hypercoagulable condition
- •Baseline study visit falls between 1 and 8 weeks after initiation of CF transmembrane receptor (CFTR) modulator
- •Currently in pulmonary exacerbation
- •Recent antibiotic use within the past 4 weeks for an acute pulmonary exacerbation
- •Unwilling or unable to use a non-estrogen containing method of contraception such as barrier, abstinence, or progesterone-only method
研究组 & 干预措施
Transdermal Estradiol/Cyclic Progesterone
Participants will apply transdermal estradiol patches (0.1 mg/day) weekly for 12 months following baseline study visits. Participants not already using a progesterone-containing contraceptive will also take progesterone (200 mg) for 10 days per month.
干预措施: Transdermal estrogen (Drug)
Transdermal Estradiol/Cyclic Progesterone
Participants will apply transdermal estradiol patches (0.1 mg/day) weekly for 12 months following baseline study visits. Participants not already using a progesterone-containing contraceptive will also take progesterone (200 mg) for 10 days per month.
干预措施: Progesterone (Drug)
结局指标
主要结局
Change in lumbar spine bone mineral density assessed by dual-energy x-ray absorptiometry (DXA)
时间窗: Baseline and 12 months
次要结局
- Change in serum C-terminal telopeptide of type I collagen (CTX-1) levels (pg/mL)(Baseline and 12 months)
- Change in serum procollagen type I intact N-terminal propeptide (P1NP) levels (mcg/L)(Baseline and 12 months)
- Change in quality of life as assessed by Cystic Fibrosis Respiratory Symptom Diary - Chronic Respiratory Infection Symptom Score (CFRSD-CRISS) questionnaire(Baseline and 12 months)