What side effects are associated with this type of intervention?

Common treatments for Cystic Fibrosis (CF) include estrogen replacement therapy, which can inhibit Ca2+ signaling and airway surface liquid volume homeostasis, potentially worsening lung function. Anti-estrogens like tamoxifen may counteract these effects but carry risks such as endometrial cancer and thromboembolic events. Inhaled antibiotics like tobramycin and aztreonam can cause bronchial constriction and nephrotoxicity. CFTR modulators like ivacaftor may improve pancreatic function but have limited efficacy in older patients and can cause liver enzyme elevations.

What prior FDA approvals exist?

The FDA has approved several treatments for cystic fibrosis, primarily focusing on CFTR modulators that target the underlying genetic defect. These include ivacaftor, lumacaftor/ivacaftor, tezacaftor/ivacaftor, and elexacaftor/tezacaftor/ivacaftor. These drugs work by improving the function of the defective CFTR protein, thereby enhancing chloride transport and improving clinical outcomes. While the context does not provide specific information on FDA approvals for estrogen replacement therapy in CF, it does mention ongoing studies investigating the impact of transdermal estradiol on bone health and quality of life in women with CF.

What other types of research exist?

Promising alternatives to transdermal estradiol for cystic fibrosis patients in 2024 include hormonal contraceptives (e.g., birth control pills or progesterone-only methods) and nonhormonal agents such as newer antidepressants. These alternatives can potentially impact bone health and quality of life positively.

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Hormone Therapy

Estrogen Supplementation for Cystic Fibrosis (STURDY Trial)

Q: What is the mechanism of action of this treatment?

Common treatments for Cystic Fibrosis (CF) include CFTR modulators, inhaled hypertonic saline, DNase, and antibiotics. CFTR modulators, such as ivacaftor and lumacaftor, target the defective CFTR protein to improve its function, thereby enhancing chloride transport and reducing mucus buildup. Inhaled hypertonic saline helps to hydrate and clear thick mucus from the airways, while DNase breaks down DNA in the mucus to reduce its viscosity. Antibiotics are used to treat and prevent lung infections. These treatments are crucial for CF patients as they address the underlying issues of mucus clearance and infection control, improving lung function and quality of life. Transdermal estradiol, being studied for its impact on bone health, may also influence mucus clearance by modulating estrogen levels, which have been shown to affect airway surface liquid volume and calcium signaling in CF patients.

Phase 4

Recruiting

Led By Malinda Wu, MD, MSc

Research Sponsored by Johns Hopkins University

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

All of above and <35 years old

CF Diagnosis

Must not have

Use of chronic systemic glucocorticoids

Currently in pulmonary exacerbation

Timeline

Screening 3 weeks

Treatment Varies

Follow Up evaluated at completion of study, approximately two years

Awards & highlights

Drug Has Already Been Approved

No Placebo-Only Group

Pivotal Trial

Summary

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.

Who is the study for?

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.

What is being tested?

The study examines how hormonal contraception affects bone health in CF patients and tests the feasibility of transdermal estradiol (estrogen skin patches). Participants will undergo blood tests, questionnaires, x-rays, and DXAs over a year to monitor changes in bone health.

What are the potential side effects?

Potential side effects may include skin irritation from patches, hormone-related changes such as mood swings or weight gain. Estrogen can also increase the risk of blood clots; however specific side effects related to this trial are not detailed.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I meet all the criteria mentioned and am under 35 years old.

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I have been diagnosed with cystic fibrosis.

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I plan to keep my estrogen supplementation the same throughout the study.

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I have had at least one menstrual cycle.

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I have symptoms of low estrogen or my blood test shows low estrogen levels.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am on long-term steroid medication.

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I am currently experiencing a worsening of my lung condition.

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I cannot use skin patches that deliver estrogen.

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I have a history or condition that increases my risk of blood clots.

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I am currently taking estrogen or estrogen-containing pills.

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I have had a lung or liver transplant.

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I have taken antibiotics for a lung issue in the last 4 weeks.

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I cannot or will not use non-estrogen birth control methods.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ evaluated at completion of study, approximately two years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~evaluated at completion of study, approximately two years

This trial's timeline: 3 weeks for screening, Varies for treatment, and evaluated at completion of study, approximately two years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Change in quality of life as assessed by Cystic Fibrosis Respiratory Symptom Diary - Chronic Respiratory Infection Symptom Score (CFRSD-CRISS) questionnaire

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

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

Other study objectives

Acceptability as determined by participant report

Study procedure completion rate

Awards & Highlights

Drug Has Already Been Approved

The FDA has already approved this drug, and is just seeking more data.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: Transdermal Estradiol/Cyclic ProgesteroneExperimental Treatment2 Interventions

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.

Group II: Observational StudyActive Control1 Intervention

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Progesterone

2013

Completed Phase 4

~4510

0 / 13Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Cystic Fibrosis (CF) include CFTR modulators, inhaled hypertonic saline, DNase, and antibiotics. CFTR modulators, such as ivacaftor and lumacaftor, target the defective CFTR protein to improve its function, thereby enhancing chloride transport and reducing mucus buildup. Inhaled hypertonic saline helps to hydrate and clear thick mucus from the airways, while DNase breaks down DNA in the mucus to reduce its viscosity. Antibiotics are used to treat and prevent lung infections. These treatments are crucial for CF patients as they address the underlying issues of mucus clearance and infection control, improving lung function and quality of life. Transdermal estradiol, being studied for its impact on bone health, may also influence mucus clearance by modulating estrogen levels, which have been shown to affect airway surface liquid volume and calcium signaling in CF patients.

From pipeline to patient: new developments in cystic fibrosis therapeutics.Targeting the Root Cause of Cystic Fibrosis.17beta-Estradiol inhibits Ca2+-dependent homeostasis of airway surface liquid volume in human cystic fibrosis airway epithelia.