Estrogen Supplementation and Bone Health in Women With CF

NCT05704036 | Recruiting Phase 4 FDA Drug

• 1 other identifier: IRB00307525
• Study Type: interventional
• Target: 75
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Cystic Fibrosis; Hypoestrogenism

Keywords

Bone Mineral Density; Osteoporosis; Young Adult; Adolescent; Premenopause; Estradiol; Bone Disease; Bone Diseases, Metabolic; Hormones; Quality of Life

Outcome Measures

Primary Outcomes (1)

• Change in lumbar spine bone mineral density assessed by dual-energy x-ray absorptiometry (DXA)

  Baseline and 12 months

Secondary Outcomes (3)

• Change in serum procollagen type I intact N-terminal propeptide (P1NP) levels (mcg/L)

  Baseline and 12 months

• Change in serum C-terminal telopeptide of type I collagen (CTX-1) levels (pg/mL)

  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

Other Outcomes (2)

• Study procedure completion rate

  Evaluated at completion of study, approximately two years

• Acceptability as determined by participant report

  Across 12 months of study participation per participant

Study Arms (2)

Observational Study

NO INTERVENTION

Participants will complete three study visits (baseline, 6 months, and 12 months) involving DXAs (bone density assessment), x-rays, blood draws, and questionnaires.

Transdermal Estradiol/Cyclic Progesterone

EXPERIMENTAL

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; Drug: Progesterone

Interventions

Transdermal estrogen DRUG

Transdermal estradiol 0.1 mg/day, applied once weekly

Also known as: Transdermal estradiol, Climara

Transdermal Estradiol/Cyclic Progesterone

Progesterone DRUG

Progesterone 200 mg daily for 10 days/month, if participant is not on progesterone-containing contraceptive

Also known as: Prometrium

Transdermal Estradiol/Cyclic Progesterone

Eligibility Criteria

• Age: 0 Years - 100 Years
• Sex: female
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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:
• 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)
• 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

Sponsors & Collaborators

• Johns Hopkins University: lead
• Cystic Fibrosis Foundation: collaborator

Study Sites (1)

Johns Hopkins

Baltimore, Maryland, 21287, United States

RECRUITING

MeSH Terms

Conditions

Cystic Fibrosis; Osteoporosis; Bone Diseases; Bone Diseases, Metabolic

Interventions

Estradiol; Progesterone

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; Musculoskeletal Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Estrenes; Estranes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Estradiol Congeners; Gonadal Steroid Hormones; Gonadal Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Pregnenediones; Pregnenes; Pregnanes; Corpus Luteum Hormones; Progesterone Congeners

Study Officials

• Malinda Wu, MD, MSc

  Johns Hopkins University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Cynthia Wang

CONTACT

410-929-3056; cwang202@jh.edu

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 10, 2023
• First Posted: January 30, 2023
• Study Start: May 2, 2023
• Primary Completion (Estimated): January 1, 2027
• Study Completion (Estimated): January 1, 2027
• Last Updated: November 14, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)