Estrogen Supplementation and Bone Health in Women With CF

Trial statusRecruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexFemale
Age0-100
SponsorJohns Hopkins University

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Sequential

Treatments tested in this trial

  • Transdermal estrogen

    Drug

    Transdermal estradiol 0.1 mg/day, applied once weekly

  • Progesterone

    Drug

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

Treatment groups

75 Participants
are divided into 2 treatment groups
Group A: Observational StudyNo intervention 0 interventions
Group B: Transdermal Estradiol/Cyclic ProgesteroneExperimental treatment 2 interventions

Trial outcomes

Primary outcomes

1

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

Time frame
Baseline and 12 months

Secondary outcomes

1

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

P1NP is a marker of bone turnover.

Time frame
Baseline and 12 months
2

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

CTX-1 is a marker of bone turnover.

Time frame
Baseline and 12 months
3

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.

Time frame
Baseline and 12 months

Other outcomes

1

Study procedure completion rate

Percentage of participants enrolled in study who complete all study procedures.

Time frame
Evaluated at completion of study, approximately two years
2

Acceptability as determined by participant report

Acceptability during study participation by 5 point Likert scale, with a score or 3 or greater considered acceptable.

Time frame
Across 12 months of study participation per participant

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Johns Hopkins University

Lead sponsor

Cystic Fibrosis Foundation

Collaborator