Cystic Fibrosis, Hypoestrogenism
Conditions
Keywords
Bone Mineral Density, Osteoporosis, Young Adult, Adolescent, Premenopause, Estradiol, Bone Disease, Bone Diseases, Metabolic, Hormones, Quality of Life
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.
Interventions
DRUGProgesterone
Progesterone 200 mg daily for 10 days/month, if participant is not on progesterone-containing contraceptive
Transdermal estradiol 0.1 mg/day, applied once weekly
Sponsors
Cystic Fibrosis Foundation
Johns Hopkins University
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
FEMALE
Age
0 Years to 100 Years
Healthy volunteers
No
Inclusion criteria
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)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in lumbar spine bone mineral density assessed by dual-energy x-ray absorptiometry (DXA) | Baseline and 12 months |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in serum procollagen type I intact N-terminal propeptide (P1NP) levels (mcg/L) | Baseline and 12 months | P1NP is a marker of bone turnover. |
| Change in serum C-terminal telopeptide of type I collagen (CTX-1) levels (pg/mL) | Baseline and 12 months | CTX-1 is a marker of bone turnover. |
| 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 | 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. |
Other
| Measure | Time frame | Description |
|---|---|---|
| Study procedure completion rate | Evaluated at completion of study, approximately two years | Percentage of participants enrolled in study who complete all study procedures. |
| Acceptability as determined by participant report | Across 12 months of study participation per participant | Acceptability during study participation by 5 point Likert scale, with a score or 3 or greater considered acceptable. |
Countries
United States
Contacts
Primary ContactCynthia Wang
Outcome results
None listed