Estrogen Supplementation for Cystic Fibrosis
(STURDY Trial)
Trial Summary
What is the purpose of this trial?
This trial studies how estrogen skin patches affect bone health in young women with cystic fibrosis. Participants will use the patches regularly and undergo various health assessments. The goal is to see if this treatment can improve their bone health and quality of life. Estrogen replacement is recommended for female patients with CF-related bone disease to restore bone mineral density.
Will I have to stop taking my current medications?
The trial protocol does not specify if you must stop taking your current medications. However, if you are using systemic estrogen (like estrogen-containing birth control pills), you cannot participate in the feasibility sub-study. Also, if you are on anti-osteoporosis therapy or chronic systemic glucocorticoids, you cannot join the observational study.
What data supports the effectiveness of the drug transdermal estradiol for cystic fibrosis?
The transdermal estradiol patch is effective in maintaining stable hormone levels and has been shown to protect against bone loss in postmenopausal women, which suggests it may have potential benefits for other conditions like cystic fibrosis, although direct evidence for cystic fibrosis is not provided.12345
Is estrogen supplementation generally safe for humans?
Transdermal estrogen patches, like those containing estradiol, are generally well-tolerated in humans. The most common side effect is mild skin irritation at the application site, which occurs in about 15% of users. There is some evidence of an increased risk of breast cancer with long-term use, so more research is needed on the long-term effects.24567
How does the drug transdermal estrogen differ from other treatments for cystic fibrosis?
Transdermal estrogen is unique because it delivers estrogen through the skin, maintaining stable hormone levels without passing through the liver, which can reduce certain side effects compared to oral estrogen. This method is primarily used for hormone replacement in postmenopausal women, but its application in cystic fibrosis is novel and may offer benefits not seen with standard treatments for this condition.12568
Research Team
Malinda Wu, MD, MSc
Principal Investigator
Johns Hopkins University
Eligibility Criteria
This trial is for young women with cystic fibrosis who have low estrogen levels, are not pregnant, and agree to use non-estrogen contraception. They should be planning to maintain their current level of estrogen supplementation throughout the study and must be at least two years post their first menstrual cycle.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Observational Study
Participants will complete three study visits (baseline, 6 months, and 12 months) involving DXAs (bone density assessment), x-rays, blood draws, and questionnaires.
Feasibility Sub-study
Participants will apply transdermal estradiol patches once weekly for 12 months, fill out brief quality-of-life surveys in between study visits, and complete blood draws and questionnaires at four study visits.
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- Placebo patch
- Placebo pill
- Progesterone
- Transdermal estrogen
Transdermal estrogen is already approved in United States, European Union, Canada for the following indications:
- Menopause symptoms
- Osteoporosis prevention
- Hormone replacement therapy
- Menopause symptoms
- Osteoporosis prevention
- Hormone replacement therapy
- Menopause symptoms
- Osteoporosis prevention
- Hormone replacement therapy
Find a Clinic Near You
Who Is Running the Clinical Trial?
Johns Hopkins University
Lead Sponsor
Cystic Fibrosis Foundation
Collaborator