Gender Disparity and Hormones in Cystic Fibrosis

NCT02036879 | Completed Early Phase 1 FDA Drug

• 1 other identifier: STU 072013-079
• Study Type: interventional
• Target: 55
• Locations: 1 country
• Sites: 2

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.

Conditions

Cystic Fibrosis (CF)

Keywords

Cystic Fibrosis; Loestrin; Hormone

Outcome Measures

Primary Outcomes (1)

• Change in percent predicted forced expiratory volume in 1 second (FEV1)

  Through Visit 5, if applicable (or approximately 3 months)

Study Arms (2)

Main study

NO INTERVENTION

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.

Loestrin Optional Substudy

EXPERIMENTAL

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)

Interventions

Loestrin (norethindrone acetate and ethinyl estradiol) DRUG

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.

Loestrin Optional Substudy

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• University of Texas Southwestern Medical Center: lead

Study Sites (2)

Children's Medical Center

Dallas, Texas, 75207, United States

Location

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

norethindrone acetate, ethinyl estradiol, ferrous fumarate drug combination; Norethindrone Acetate; Ethinyl Estradiol

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

Norethindrone; Norpregnenes; Norpregnanes; Norsteroids; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Norpregnatrienes; Estrogenic Steroids, Alkylated; Estradiol Congeners; Gonadal Steroid Hormones; Gonadal Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists

Study Officials

• Raksha Jain, MD, MSCI

  University of Texas Southwestern Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: December 26, 2013
• First Posted: January 15, 2014
• Study Start: February 1, 2014
• Primary Completion: August 1, 2018
• Study Completion: August 1, 2018
• Last Updated: August 31, 2020

Record last verified: 2020-08

Locations

US (2)