Pilot Study Evaluating the Effects of Estradiol for Hypogonadal Women With Cystic Fibrosis and Osteopenia
试验速览
- 阶段
- 4 期
- 状态
- 撤回
- 试验地点
- 1
- 主要终点
- Change in serum estradiol level in picograms per milliliter (pg/mL) from baseline to 6 months among the treatment and placebo groups
研究概览
简要总结
The study is a prospective, double blinded, placebo controlled, randomized study to evaluate the effects of daily oral estrogen supplements on bone health, sexual and reproductive health, quality of life and markers of inflammation and lung function when given to hypogonadal women with Cystic Fibrosis related Bone Disease (CFBD).
详细描述
Cystic fibrosis (CF) is the most common fatal heritable disease in non-Hispanic whites; CF results in frequent lung infections, chronic inflammation and progressive lung failure. With advancements in medical care, patients with CF are living longer. The median survival of women with CF born and diagnosed in 2010 is projected to be 37 years. Patients with CF are now living long enough to develop comorbidities like Cystic Fibrosis related Bone Disease (CFBD), hypogonadism manifesting as pubertal delay, premature ovarian insufficiency or hypothalamic hypogonadism. These comorbidities in women without CF can be improved by estrogen supplementation.
It is well established that treating hypogonadal (low estrogen levels) women with estrogen can improve their bone health, slow the rate of bone loss and decrease bone turnover markers. Supplemental estrogen given to hypogonadal women can improve sexual and reproductive health including reduction of perimenopausal symptoms and improvement in sexual function which can improve quality of life. There is very little research investigating the effects of estrogen treatment for hypogonadal women with CF. The purpose of this study is to test the hypothesis that daily oral estrogen supplements given to hypogonadal women with CFBD will improve their bone health, sexual and reproductive health, and quality of life and modify markers of inflammation and lung function.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Outcomes Assessor)
盲法说明
Double Blinded
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Adult and adolescent female CF patients (age ≥ 18 years),
- •presenting to the CF clinic for routine follow up of cystic fibrosis,
- •hypogonadal women defined as E2 level < 25,
- •dual energy X-ray absorptiometry (DEXA) scan within 2 years of enrollment with T- or Z-score < -1,
- •able to tolerate oral medications.
排除标准
- •Inability to obtain or declined informed consent from the subject and/or legally authorized representative,
- •Pregnancy,
- •Too ill to participate in study based on investigator's or study team's opinion,
- •Current use of systemic estrogen,
- •History of thromboembolic event within the previous 2 years,
- •History of migraines with aura,
- •Hypercoagulability including previous diagnosis of Factor V Leiden or Protein C or S deficiency,
- •Current smoker,
- •History of diagnosis with breast or uterine cancer,
- •Current significant liver disease with cholelithiasis or cirrhosis,
- •Status post lung or liver transplantation,
- •Current use of systemic steroids
研究组 & 干预措施
Treatment Group
The treatment group will receive Estradiol 2 mg oral daily for 6 months. Medication will be mailed to patient. All study drugs will be dispensed by the Investigational Drug Pharmacy.
干预措施: Estradiol 2 mg (Drug)
Placebo Group
The placebo group will receive placebo oral daily for 6 months. Medication will be mailed to patient. Placebo will be dispensed by the Investigational Drug Pharmacy.
干预措施: Placebo (Other)
结局指标
主要结局
Change in serum estradiol level in picograms per milliliter (pg/mL) from baseline to 6 months among the treatment and placebo groups
时间窗: Baseline/Enrollment visit and at 6 months
Serum estradiol measures the amount of the hormone estradiol in the blood. The level is measured using a blood test that is performed at baseline/enrollment visit and at 6 months. According to Mayo Medical Laboratories, normal levels of estradiol (E2) for menstruating women range from 15 to 350 picograms per milliliter (pg/mL). The levels of estradiol among both groups are recorded and compared from baseline to 6 months.
次要结局
- Change in percentage of FEV1 from baseline to 6 months among the treatment and placebo groups(Baseline/Enrollment visit and at 6 months)
- Change in Serum carboxy-terminal collagen crosslinks (CTX-1) from baseline to 6 months among the treatment and placebo groups(Baseline/Enrollment visit and at 6 months)
- change in of Serum Procollagen I Intact N-Terminal (P1NP) measured in mcg/L from baseline to 6 months among the treatment and placebo groups(Baseline/Enrollment visit and at 6 months)
- Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory score from baseline to 6 months among the treatment and placebo groups(Baseline/Enrollment visit and at 6 months)
研究者
Vin Tangpricha, MD, PH.D
Professor
Emory University