75 Participants Needed

Estrogen Supplementation for Cystic Fibrosis

(STURDY Trial)

SM
CW
Overseen ByCynthia Wang
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 3 JurisdictionsThis treatment is already approved in other countries

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

MW

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

I meet all the criteria mentioned and am under 35 years old.
It has been over 2 years since my first period.
I have been diagnosed with cystic fibrosis.
See 3 more

Exclusion Criteria

In the opinion of the CF care team or study investigators participant should not participate in the study
Inability to provide informed consent/assent
I am on long-term steroid medication.
See 12 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

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.

12 months
3 visits (in-person)

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.

12 months
4 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • Placebo patch
  • Placebo pill
  • Progesterone
  • Transdermal estrogen
Trial OverviewThe 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.
Participant Groups
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.

Transdermal estrogen is already approved in United States, European Union, Canada for the following indications:

🇺🇸
Approved in United States as Estradiol patch for:
  • Menopause symptoms
  • Osteoporosis prevention
  • Hormone replacement therapy
🇪🇺
Approved in European Union as Estradiol patch for:
  • Menopause symptoms
  • Osteoporosis prevention
  • Hormone replacement therapy
🇨🇦
Approved in Canada as Estradiol patch for:
  • Menopause symptoms
  • Osteoporosis prevention
  • Hormone replacement therapy

Find a Clinic Near You

Who Is Running the Clinical Trial?

Johns Hopkins University

Lead Sponsor

Trials
2,366
Recruited
15,160,000+

Cystic Fibrosis Foundation

Collaborator

Trials
199
Recruited
37,800+

Findings from Research

The new matrix transdermal patch with lauric acid showed improved stability in estradiol delivery compared to traditional matrix and reservoir patches, with more consistent plasma levels in postmenopausal women.
Both the prototype and its industrial counterpart provided similar estradiol absorption, achieving higher plasma concentrations than the reference matrix patch, indicating enhanced bioavailability for hormone replacement therapy.
Estradiol pharmacokinetics after transdermal application of patches to postmenopausal women: matrix versus reservoir patches.Reginster, JY., Donazzolo, Y., Brion, N., et al.[2019]
The estradiol transdermal therapeutic system effectively delivers estradiol to maintain premenopausal levels in postmenopausal women, helping to protect against osteoporosis and reduce fracture risk with minimal dosages.
This method of delivery is well-tolerated, with local irritation being the most common side effect, and it offers similar benefits for menopausal symptoms as oral estrogens, making it a promising alternative for estrogen therapy.
Transdermal estradiol. A review of its pharmacological profile, and therapeutic potential in the prevention of postmenopausal osteoporosis.Balfour, JA., McTavish, D.[2018]
The seven-day transdermal estradiol patch significantly reduced menopausal symptoms over six months, as indicated by a decrease in the average climacteric score from 27.18 at admission to 12.18 by the end of the study.
The patch demonstrated excellent adhesion with over 94% remaining securely in place, and it was generally well tolerated, with skin irritation being the most common side effect, primarily itching.
Efficacy, safety and acceptability of a seven-day, transdermal estradiol patch for estrogen replacement therapy.Jarupanich, T., Lamlertkittikul, S., Chandeying, V.[2013]

References

Estradiol pharmacokinetics after transdermal application of patches to postmenopausal women: matrix versus reservoir patches. [2019]
Transdermal estradiol. A review of its pharmacological profile, and therapeutic potential in the prevention of postmenopausal osteoporosis. [2018]
Efficacy, safety and acceptability of a seven-day, transdermal estradiol patch for estrogen replacement therapy. [2013]
[Kinetics of a new patch for transdermal administration of 17 beta-estradiol]. [2013]
Estradiol and estrone plasma levels during application of three strengths of a 7-day estradiol transdermal patch. [2013]
Estrogen replacement therapy and the estraderm transdermal system. [2013]
Clinical experience with a 7-day estrogen patch: principles and practice. [2013]
Transdermal estradiol. A review of its pharmacodynamic and pharmacokinetic properties, and therapeutic efficacy in the treatment of menopausal complaints. [2018]