Assessment of Contraceptive Safety and Effectiveness in Cystic Fibrosis (CASE4CF)

Assessment of Contraceptive Safety and Effectiveness in Cystic Fibrosis: A Retrospective Analysis

The long-term goal is to study the safety and effectiveness of hormonal contraception for women with cystic fibrosis (CF) and contribute to national guidelines that the Cystic Fibrosis Foundation and the Centers for Disease Control and Prevention (CDC) provide to clinicians. The study objectives are to determine whether hormonal contraceptive methods improve overall pulmonary health, worsen CF-related disease or CF liver disease, or are effective against unwanted pregnancy with concomitant CF transmembrane conductance regulator (CFTR) modulator use. The hypothesis is that hormonal contraceptive methods are safe and do not worsen CF-related complications over time,improve FEV-1 when compared to non-hormonal users, and oral birth control methods with CFTR modulator use.

Study Overview

• Status: Completed
• Conditions: Cystic Fibrosis; Contraception

Detailed Description

This is a non-randomized, non-interventional, observational cohort design among reproductive-aged women (ages 18-45 years) with CF. The investigators intend to ask about 625 female participants to provide information about their types of past contraceptive use from 2008 through the most recent data available from CFFPR (2018 or 2019). Before initiating our trial among up to 625 respondents, the investigators intend to pre-test the survey questions on our self-respondent questionnaire among as many as 50 women with and without CF through CFF Community Voice, the Cystic Fibrosis Research, Inc. (CFRI), and the Cystic Fibrosis Reproductive and Sexual Health Collaborative (CFReSHC). Pre-testing the study survey questions will help ensure contraceptive, pregnancy, and other reproductive health data from respondents for the larger phase of this study are as complete and accurate as possible. Once the survey has been piloted and revised accordingly, the investigators will collect past contraceptive, pregnancy and other reproductive health data from up to 625 women at 10 different CF centers. The investigators may over-sample by 25 participants because it is possible that some participants from UW, UTSW and National Jewish may have participated an earlier published study related to contraceptive use and CFFPR. Recruiting through the CF centers will help ensure that women with a confirmed diagnosis of CF respond to the finalized survey. The investigators will link respondent self-reported contraceptive and reproductive health data with CFFPR clinical data. The investigators will ask for EMR data from the 10 different CF centers to validate DXA scan results with findings regarding osteoporosis, osteopenia, and bone fracture in the CFFPR. The investigators plan to randomly select about 10 consented subjects from each of the centers for the EMR DXA scan chart review. Because of the need to link questionnaires and clinical case report forms correctly to the same women with CF in the CFFPR clinical data, this study requests identifiable information, including the CFFPR member ID number.

• Study Type: Observational
• Enrollment (Actual): 562

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35294
      • University of Alabama at Birmingham
  • California
    • San Diego, California, United States, 92093
      • University of California - San Diego
  • Colorado
    • Denver, Colorado, United States, 80206
      • National Jewish Health
  • Maryland
    • Baltimore, Maryland, United States, 21218
      • Johns Hopkins University
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital/Harvard
  • New York
    • New York, New York, United States, 10003
      • Mount Sinai Beth Israel
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • University of Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15260
      • University of Pittsburgh
  • Texas
    • Dallas, Texas, United States, 75390
      • University of Texas Southwestern
  • Washington
    • Seattle, Washington, United States, 98105
      • University of Washington

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 45 years (Adult)
• Accepts Healthy Volunteers: Yes

• Sampling Method: Non-Probability Sample

Study Population

Women with cystic fibrosis who have not used any form of contraception, or who have used contraception for protection against pregnancy or for treatment of dysmenorrhea, pelvic pain, abnormally heavy or prolonged menstrual bleeding, endometriosis, acne or amenorrhea.

Description

Inclusion Criteria:

• Women with cystic fibrosis age 18-45 years
• Enrolled as a participant of the Cystic Fibrosis Foundation Patient Registry
• Working email address
• Can read written English

Exclusion Criteria:

• History of organ transplant
• Does not have a phone number or email address in their record
• <18 years or >45 years at enrollment

Study Plan

How is the study designed?

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort
Hormonal contraceptive users; Persons exposed to any hormonal method of birth control. This includes combined oral contraceptive pills, combined transdermal patch, combined vaginal ring, progestin-only pills, depo-medroxyacetate, levonorgestrel intrauterine system/device, hormonal subdermal implant
Non-hormonal contraceptive users; Persons exposed to any non-hormonal method of birth control. This includes male or female sterilization methods, Copper intrauterine device, internal/external condoms, diaphragm, cervical cap, withdrawal, sponge, fertility based methods, spermicide
Non-contraceptive users; Persons who did not use any method of birth control

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pulmonary exacerbations	Annual pulmonary exacerbation rate (PEx) (defined as episodes requiring intravenous antibiotic use at home or in hospital)	2010-2018

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Lung function	Absolute change in FEV1 (in liters) measurements over time.	2010-2018
Thromboembolism	Incidence of venous or arterial thromboembolism. Noted on self-respondent survey (defined as blood clot requiring anticoagulation treatment)	2010-2018
Liver dysfunction	Incidence of gallstones requiring surgery, hepatic steatosis, hepatic cirrhosis and/or increased liver enzyme test values (ALT/GGT)	2010-2018
Serum glucose trends	Incidence of insulin resistance or time to CF-related diabetes requiring insulin treatment	2010-2018
Nutritional status	BMI trends	2010-2018
Bone health	DXA scan determination of osteopenia or osteoporosis	2013-2018
Unintended pregnancy	Absolute number of self-reported unplanned pregnancies. We will also correlate date of reported unplanned pregnancy with dates of taking a CFTR modulator	2010-2018
Pseudomonas aeruginosa	Rate of conversion from non-mucoid to mucoid P. aeruginosa	2010-2018

Collaborators and Investigators

• Sponsor: University of Washington
• Investigators: Principal Investigator: Emily M Godfrey, MD, MPH, University of Washington

Study record dates

Study Major Dates

• Study Start (Actual): September 30, 2020
• Primary Completion (Actual): May 2, 2022
• Study Completion (Actual): December 6, 2022

Study Registration Dates

• First Submitted: September 23, 2020
• First Submitted That Met QC Criteria: September 23, 2020
• First Posted (Actual): September 29, 2020

Study Record Updates

• Last Update Posted (Actual): May 22, 2023
• Last Update Submitted That Met QC Criteria: May 18, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Keywords: contraception; cystic fibrosis; women; bone mineral density; birth control; CFTR modulator
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis
• Other Study ID Numbers: STUDY00008095

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No