Pilot E2 for Hypogonadal Women With CFBD

Emory University

Status and phase: Withdrawn; Phase 4

Conditions: Cystic Fibrosis Related Bone Disease

Treatments: Drug: Estradiol 2 mg; Other: Placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT03724955

IRB00107135

Details and patient eligibility

About

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).

Full description

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.

Sex: Female

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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.

Exclusion criteria

• 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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0 participants in 2 patient groups, including a placebo group

Treatment Group

Experimental group

Description:

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.

Treatment:

Drug: Estradiol 2 mg

Placebo Group

Placebo Comparator group

Description:

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.

Treatment:

Other: Placebo