Gender Disparity and Hormones in Cystic Fibrosis

Sponsor

University of Texas Southwestern Medical Center (Other)

Overall Status

Completed

CT.gov ID

NCT02036879

Collaborator

(none)

Enrollment

Locations

Arms

53.9

Actual Duration (Months)

27.5

Patients Per Site

0.5

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The objective of this study is to investigate the impact of hormones on lung disease in Cystic Fibrosis (CF) patients. Due to improved therapies, CF patients are living longer and healthier lives than they did 20 years ago. However, females have been shown to have a survival disadvantage. The median life expectancy is 33 in women and 37 in men with CF. The hypothesis is that estrogen and/or progesterone negatively impact lung health in CF. Therefore, understanding the impact of sex hormones (including the use of birth control pills) on the disease process is increasingly important. The purpose of this study is to determine if lung function, respiratory symptoms, or various markers of lung health change during different phases of the natural ovulatory cycle in order to understand if estrogen or progesterone hormones are impacting the disease relative to fluctuations in men with stable testosterone levels. The research objectives of this project are to:

Determine if lung function, respiratory symptoms, or various markers of lung health change during different hormonal phases of the ovulatory cycle in women.
Determine if men change lung function, respiratory symptoms, or various markers of lung health over time.
Determine if oral contraceptive pills in women stabilize fluctuations in symptoms and improve lung health.

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis (CF)	Drug: Loestrin (norethindrone acetate and ethinyl estradiol)	Early Phase 1

Study Design

Study Type:

Interventional

Actual Enrollment :

55 participants

Allocation:

Non-Randomized

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Basic Science

Official Title:

Gender Disparity and Hormones in Cystic Fibrosis

Actual Study Start Date :

Feb 1, 2014

Actual Primary Completion Date :

Aug 1, 2018

Actual Study Completion Date :

Aug 1, 2018

Arms and Interventions

Arm	Intervention/Treatment
No Intervention: Main study The main study is an observational study. All women will be followed for one menstrual cycle (or approximately one month) observationally off of any hormone supplementation. They will have 3 study visits corresponding to their menstrual cycle phases (menses, ovulation, and luteal). Women participating in the main study may participate in the optional interventional sub-study. Men participating in this study will be followed for 1 month observationally. They will have 3 study visits that correlate with the female arm of this study.
Experimental: Loestrin Optional Substudy Women participating in the main study may participate in the optional sub-study. Following a negative urine pregnancy test, women will be started on once daily oral Loestrin (1.5 mg norethindrone + 0.03 mg ethyl estradiol). They will be followed for two months on this agent and have 2 additional study visits.	Drug: Loestrin (norethindrone acetate and ethinyl estradiol) This is an optional substudy that females participating in the main study can choose to participate in. Loestrin, an oral contraceptive or birth control pill, will be prescribed and taken daily for approximately 2 months.

Arm

Intervention/Treatment

No Intervention: Main study

The main study is an observational study. All women will be followed for one menstrual cycle (or approximately one month) observationally off of any hormone supplementation. They will have 3 study visits corresponding to their menstrual cycle phases (menses, ovulation, and luteal). Women participating in the main study may participate in the optional interventional sub-study. Men participating in this study will be followed for 1 month observationally. They will have 3 study visits that correlate with the female arm of this study.

Experimental: Loestrin Optional Substudy

Women participating in the main study may participate in the optional sub-study. Following a negative urine pregnancy test, women will be started on once daily oral Loestrin (1.5 mg norethindrone + 0.03 mg ethyl estradiol). They will be followed for two months on this agent and have 2 additional study visits.

Drug: Loestrin (norethindrone acetate and ethinyl estradiol)

This is an optional substudy that females participating in the main study can choose to participate in. Loestrin, an oral contraceptive or birth control pill, will be prescribed and taken daily for approximately 2 months.

Outcome Measures

Primary Outcome Measures

Change in percent predicted forced expiratory volume in 1 second (FEV1) [Through Visit 5, if applicable (or approximately 3 months)]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Male and female cystic fibrosis patients
Must be greater than or equal to 18 years of age.
All subjects must understand and sign the informed consent.
Subjects must have the ability to read and write in English.
Female subjects starting this study must be willing to use a double barrier method of birth control (such as condom or diaphragm) used with a spermicide (a substance that kills sperm), while participating in the study.

Exclusion Criteria for Main Study:

Women who are pregnant, breast feeding, or who have had an oophorectomy.
Women who have received a hormone contraceptive injection (such as Depo Provera) within the last 3 months.

Exclusion Criteria for Substudy:

Men
Women who have a history of breast cancer, abnormal vaginal bleeding, liver disease, coronary artery disease, cerebrovascular disease, uncontrolled hypertension, diabetes mellitus with vascular disease, or have had a stroke, heart attack, or blood clot within the past year, due to a possible reaction to Loestrin.
Women who currently smoke cigarettes, due to the increased risk of serious cardiovascular events with Loestrin use.