Estrogen Supplementation and Bone Health in Women With CF

Cystic Fibrosis Clinical Trial

— STURDY  

Official title:

Estrogen Supplementation and Bone Health in Women With CF (STURDY)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05704036
• Other study ID #: IRB00307525
• Status: Recruiting
• Phase: Phase 4
• Start date: May 2, 2023
• Est. completion date: December 2025

Study information

• Verified date: November 2023
• Source: Johns Hopkins University
• Contact: Sara Malina
• Phone: 443-287-8997
• Email: smalina1[@]jh.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The goal of this study is to learn about the role of estrogen and other hormones in bone development in adolescent and young adult women with cystic fibrosis (CF). The study has two main components, an observational study and a feasibility sub-study. The main questions it aims to answer are: - How do different forms of hormonal contraception (e.g. birth control pill or other progesterone only methods) impact bone health? - 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 in the observational study will have three study visits (baseline, 6 months, and 12 months). At all visits, participants will have blood drawn and will complete questionnaires. At the baseline and 12 month visits, participants will additionally have x-rays and DXAs (bone density assessment). Between visits, participants will complete brief questionnaires. Researchers will compare various measurements over time to assess the impact of different forms of hormonal contraception. Participants in the feasibility sub-study 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 (baseline, 3 months, 6 months, and 12 months). The participants will also have x-rays and DXAs performed at the baseline and 12 month visits. Researchers will examine changes in various measurements to determine the impact of transdermal estradiol.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 75
• Est. completion date: December 2025
• Est. primary completion date: December 2025
• Accepts healthy volunteers: No
• Gender: Female
• Age group: 0 Years to 100 Years

Eligibility

Observational Study:

Inclusion Criteria:

• CF Diagnosis
• Females who have had at least 1 menstrual cycle
• Planning to use same formulation of estrogen supplementation (or none) for duration of study

Exclusion Criteria:

• Use of anti-osteoporosis therapy including anti-resorptives such as bisphosphonates or anabolic agents such as teriparatide or denosumab
• Conditions in which bone loss is known to be present or expected to occur, such as lactation
• Pregnant or planning to become pregnant
• In the opinion of the CF care team or study investigators participant should not participate in the study
• Inability to provide informed consent/assent Feasibility Sub-Study:

Inclusion Criteria:

• All of above and
• <35 years old
• At least 2 years after first menstrual cycle
• Symptoms of low estrogen and/or low serum estradiol levels (< 50 pg/mL)

Exclusion Criteria:

• All of above and
• 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)
• 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
• Unwilling or unable to use a non-estrogen containing method of contraception such as barrier, abstinence, or progesterone-only method

Related Conditions & MeSH terms

• Cystic Fibrosis
• Hypoestrogenism

Intervention

Drug:
• Transdermal estrogen
Transdermal estradiol 0.1 mg/day, applied once weekly
• Progesterone
Progesterone 200 mg daily for 10 days/month, if participant is not on progesterone-containing contraceptive

Locations

Country	Name	City	State
United States	Johns Hopkins	Baltimore	Maryland

Sponsors (2)

Lead Sponsor	Collaborator
Johns Hopkins University	Cystic Fibrosis Foundation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Study procedure completion rate	Percentage of participants enrolled in study who complete all study procedures.	Evaluated at completion of study, approximately two years
Other	Acceptability as determined by participant report	Acceptability during study participation by 5 point Likert scale, with a score or 3 or greater considered acceptable.	Across 12 months of study participation per participant
Primary	Change in lumbar spine bone mineral density assessed by dual-energy x-ray absorptiometry (DXA)		Baseline and 12 months
Secondary	Change in serum procollagen type I intact N-terminal propeptide (P1NP) levels (mcg/L)	P1NP is a marker of bone turnover.	Baseline and 12 months
Secondary	Change in serum C-terminal telopeptide of type I collagen (CTX-1) levels (pg/mL)	CTX-1 is a marker of bone turnover.	Baseline and 12 months
Secondary	Change in quality of life as assessed by Cystic Fibrosis Respiratory Symptom Diary - Chronic Respiratory Infection Symptom Score (CFRSD-CRISS) questionnaire	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.	Baseline and 12 months