Estrogen Patches for Adolescent and Young Adult Women With Cystic Fibrosis
Study Details
Study Description
Brief Summary
The goal of this clinical trial is to learn about the role of estrogen in bone development in adolescent and young adult women with cystic fibrosis (CF) and low estrogen levels. The main questions it aims to answer are:
-
Is a study of transdermal estradiol (estrogen skin patches) feasible in this group?
-
How does transdermal estradiol impact bone health and quality of life?
Participants will apply transdermal estradiol patches (or placebo patches -- patches that look like the estradiol patches but do not contain any estradiol) once weekly for 12 months, fill out brief quality-of-life surveys twice per month, and complete blood draws and questionnaires at four study visits (baseline, 3 months, 6 months, and 12 months). The participants will also have x-rays and DXAs (bone density assessment) performed at the baseline and 12 month visits. Researchers will compare changes in various measurements between the treatment and placebo groups to determine the impact of transdermal estradiol.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 4 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Transdermal Estradiol/cyclic progesterone Participants will apply transdermal estradiol patches (0.1 mg/day) weekly for 12 months following baseline study visits. Participants not already using a progesterone-containing contraceptive will also take progesterone (200 mg) for 10 days per month. |
Drug: Transdermal estrogen
Transdermal estradiol 0.1 mg/day, applied once weekly
Other Names:
Drug: Progesterone
Progesterone 200 mg daily for 10 days/month, if participant is not on progesterone-containing contraceptive
Other Names:
|
| Placebo Comparator: Placebo transdermal estradiol/progesterone Participants will apply placebo transdermal estradiol patches weekly for 12 months following baseline study visits. Participants not already using a progesterone-containing contraceptive will also take placebo progesterone for 10 days per month. |
Drug: Placebo patch
Placebo patch, applied once weekly
Drug: Placebo pill
Placebo pill daily for 10 days/month, if participant is not on progesterone-containing contraceptive
|
Outcome Measures
Primary Outcome Measures
- Number of potential participants enrolled per month [Evaluated monthly for 2 years, or until completion of study recruitment]
Number of potential participants who complete screening visit per month.
- Rate of screen fails [Evaluated at completion of study recruitment, approximately one year]
Percentage of potential participants who fail screening.
- Study procedure completion rate [Evaluated at completion of study, approximately two years]
Percentage of participants enrolled in study who complete all study procedures.
- Acceptability as determined by participant report [Across 12 months of study participation per participant]
Acceptability during study participation by 5 point Likert scale, with a score or 3 or greater considered acceptable.
Secondary Outcome Measures
- Change in serum estradiol levels (pg/mL) [Baseline and 12 months]
- Change in serum procollagen type I intact N-terminal propeptide (P1NP) levels (mcg/L) [Baseline and 12 months]
P1NP is a marker of bone turnover.
- Change in serum C-terminal telopeptide of type I collagen (CTX-1) levels (pg/mL) [Baseline and 12 months]
CTX-1 is a marker of bone turnover.
- Change in lumbar spine bone mineral density assessed by dual-energy x-ray absorptiometry (DXA) [Baseline and 12 months]
- Change in quality of life as assessed by Cystic Fibrosis Respiratory Symptom Diary - Chronic Respiratory Infection Symptom Score (CFRSD-CRISS) questionnaire [Baseline and 12 months]
CFRSD-CRISS is a brief survey to assess quality of life of people with cystic fibrosis that will be taken by participants twice per month. The questionnaire is scored from 0-100 with higher scores indicating increased symptom severity.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
CF diagnosis
-
16-30 years old
-
At least 2 years after first menstrual cycle
-
Low serum estradiol levels (< 50 pg/mL)
Exclusion Criteria:
-
Contraindications to transdermal estradiol
-
Current use of systemic estrogen (such as estrogen-containing oral contraceptive pill)
-
Previous lung or liver transplant
-
Use of chronic systemic glucocorticoids
-
Severe vitamin D deficiency (serum 25(OH)D < 6 ng/mL)
-
Use of anti-osteoporosis therapy including anti-resorptives such as bisphosphonates or anabolic agents such as teriparatide or denosumab
-
Conditions increasing risk of clot: history of previous deep vein thrombus or pulmonary embolism, current immobility, current peripherally inserted central catheter or mediport, known hypercoagulable condition
-
Baseline study visit falls between 1 and 8 weeks after initiation of CF transmembrane receptor (CFTR) modulator
-
Currently in pulmonary exacerbation
-
Recent antibiotic use within the past 4 weeks for an acute pulmonary exacerbation
-
Pregnant, lactating, or planning to become pregnant during study
-
Unwilling or unable to use a non-estrogen containing method of contraception such as barrier, abstinence, or progesterone-only method
-
In the opinion of the CF care team or study investigators participant should not participate in the study
-
Inability to provide informed consent/assent
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Johns Hopkins | Baltimore | Maryland | United States | 21287 |
Sponsors and Collaborators
- Johns Hopkins University
- Cystic Fibrosis Foundation
Investigators
- Principal Investigator: Malinda Wu, MD, MSc, Johns Hopkins University
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- IRB00307525