Back to resultsEstrogen Patches for Adolescent and Young Adult Women With Cystic Fibrosis
Primary Purpose
Cystic Fibrosis, Hypoestrogenism
Status
Recruiting
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
Transdermal estrogen
Placebo patch
Progesterone
Placebo pill
Sponsored by
About this trial
This is an interventional treatment trial for Cystic Fibrosis focused on measuring Bone Mineral Density, Osteoporosis, Young Adult, Adolescent, Premenopause, Estradiol, Bone Disease, Bone Diseases, Metabolic, Hormones, Quality of Life
Eligibility Criteria
Inclusion Criteria: CF diagnosis 16-30 years old At least 2 years after first menstrual cycle Low serum estradiol levels (< 50 pg/mL) Exclusion Criteria: 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 Severe vitamin D deficiency (serum 25(OH)D < 6 ng/mL) Use of anti-osteoporosis therapy including anti-resorptives such as bisphosphonates or anabolic agents such as teriparatide or denosumab Conditions increasing risk of clot: history of previous deep vein thrombus or pulmonary embolism, current immobility, current peripherally inserted central catheter or mediport, known hypercoagulable condition Baseline study visit falls between 1 and 8 weeks after initiation of CF transmembrane receptor (CFTR) modulator Currently in pulmonary exacerbation Recent antibiotic use within the past 4 weeks for an acute pulmonary exacerbation Pregnant, lactating, or planning to become pregnant during study Unwilling or unable to use a non-estrogen containing method of contraception such as barrier, abstinence, or progesterone-only method In the opinion of the CF care team or study investigators participant should not participate in the study Inability to provide informed consent/assent
Sites / Locations
- Johns HopkinsRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Transdermal Estradiol/cyclic progesterone
Placebo transdermal estradiol/progesterone
Arm Description
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.
Participants will apply placebo transdermal estradiol patches weekly for 12 months following baseline study visits. Participants not already using a progesterone-containing contraceptive will also take placebo progesterone for 10 days per month.
Outcomes
Primary Outcome Measures
Number of potential participants enrolled per month
Number of potential participants who complete screening visit per month.
Rate of screen fails
Percentage of potential participants who fail screening.
Study procedure completion rate
Percentage of participants enrolled in study who complete all study procedures.
Acceptability as determined by participant report
Acceptability during study participation by 5 point Likert scale, with a score or 3 or greater considered acceptable.
Secondary Outcome Measures
Change in serum estradiol levels (pg/mL)
Change in serum procollagen type I intact N-terminal propeptide (P1NP) levels (mcg/L)
P1NP is a marker of bone turnover.
Change in serum C-terminal telopeptide of type I collagen (CTX-1) levels (pg/mL)
CTX-1 is a marker of bone turnover.
Change in lumbar spine bone mineral density assessed by dual-energy x-ray absorptiometry (DXA)
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.
Full Information
NCT ID
NCT05704036
First Posted
January 10, 2023
Last Updated
June 9, 2023
Sponsor
Johns Hopkins University
Collaborators
Cystic Fibrosis Foundation
1. Study Identification
Unique Protocol Identification Number
NCT05704036
Brief Title
Estrogen Patches for Adolescent and Young Adult Women With Cystic Fibrosis
Official Title
Transdermal Estradiol for Adolescent and Young Adult Women With CF
Study Type
Interventional
2. Study Status
Record Verification Date
April 2023
Overall Recruitment Status
Recruiting
Study Start Date
May 2, 2023 (Actual)
Primary Completion Date
December 2024 (Anticipated)
Study Completion Date
December 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Johns Hopkins University
Collaborators
Cystic Fibrosis Foundation
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
The goal of this clinical trial is to learn about the role of estrogen in bone development in adolescent and young adult women with cystic fibrosis (CF) and low estrogen levels. The main questions it aims to answer are:
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 will apply transdermal estradiol patches (or placebo patches -- patches that look like the estradiol patches but do not contain any estradiol) once weekly for 12 months, fill out brief quality-of-life surveys twice per month, 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 (bone density assessment) performed at the baseline and 12 month visits. Researchers will compare changes in various measurements between the treatment and placebo groups to determine the impact of transdermal estradiol.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis, Hypoestrogenism
Keywords
Bone Mineral Density, Osteoporosis, Young Adult, Adolescent, Premenopause, Estradiol, Bone Disease, Bone Diseases, Metabolic, Hormones, Quality of Life
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
75 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Transdermal Estradiol/cyclic progesterone
Arm Type
Experimental
Arm Description
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.
Arm Title
Placebo transdermal estradiol/progesterone
Arm Type
Placebo Comparator
Arm Description
Participants will apply placebo transdermal estradiol patches weekly for 12 months following baseline study visits. Participants not already using a progesterone-containing contraceptive will also take placebo progesterone for 10 days per month.
Intervention Type
Drug
Intervention Name(s)
Transdermal estrogen
Other Intervention Name(s)
Transdermal estradiol, Climara
Intervention Description
Transdermal estradiol 0.1 mg/day, applied once weekly
Intervention Type
Drug
Intervention Name(s)
Placebo patch
Intervention Description
Placebo patch, applied once weekly
Intervention Type
Drug
Intervention Name(s)
Progesterone
Other Intervention Name(s)
Prometrium
Intervention Description
Progesterone 200 mg daily for 10 days/month, if participant is not on progesterone-containing contraceptive
Intervention Type
Drug
Intervention Name(s)
Placebo pill
Intervention Description
Placebo pill daily for 10 days/month, if participant is not on progesterone-containing contraceptive
Primary Outcome Measure Information:
Title
Number of potential participants enrolled per month
Description
Number of potential participants who complete screening visit per month.
Time Frame
Evaluated monthly for 2 years, or until completion of study recruitment
Title
Rate of screen fails
Description
Percentage of potential participants who fail screening.
Time Frame
Evaluated at completion of study recruitment, approximately one year
Title
Study procedure completion rate
Description
Percentage of participants enrolled in study who complete all study procedures.
Time Frame
Evaluated at completion of study, approximately two years
Title
Acceptability as determined by participant report
Description
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
Secondary Outcome Measure Information:
Title
Change in serum estradiol levels (pg/mL)
Time Frame
Baseline and 12 months
Title
Change in serum procollagen type I intact N-terminal propeptide (P1NP) levels (mcg/L)
Description
P1NP is a marker of bone turnover.
Time Frame
Baseline and 12 months
Title
Change in serum C-terminal telopeptide of type I collagen (CTX-1) levels (pg/mL)
Description
CTX-1 is a marker of bone turnover.
Time Frame
Baseline and 12 months
Title
Change in lumbar spine bone mineral density assessed by dual-energy x-ray absorptiometry (DXA)
Time Frame
Baseline and 12 months
Title
Change in quality of life as assessed by Cystic Fibrosis Respiratory Symptom Diary - Chronic Respiratory Infection Symptom Score (CFRSD-CRISS) questionnaire
Description
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
10. Eligibility
Sex
Female
Minimum Age & Unit of Time
16 Years
Maximum Age & Unit of Time
30 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
CF diagnosis
16-30 years old
At least 2 years after first menstrual cycle
Low serum estradiol levels (< 50 pg/mL)
Exclusion Criteria:
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
Severe vitamin D deficiency (serum 25(OH)D < 6 ng/mL)
Use of anti-osteoporosis therapy including anti-resorptives such as bisphosphonates or anabolic agents such as teriparatide or denosumab
Conditions increasing risk of clot: history of previous deep vein thrombus or pulmonary embolism, current immobility, current peripherally inserted central catheter or mediport, known hypercoagulable condition
Baseline study visit falls between 1 and 8 weeks after initiation of CF transmembrane receptor (CFTR) modulator
Currently in pulmonary exacerbation
Recent antibiotic use within the past 4 weeks for an acute pulmonary exacerbation
Pregnant, lactating, or planning to become pregnant during study
Unwilling or unable to use a non-estrogen containing method of contraception such as barrier, abstinence, or progesterone-only method
In the opinion of the CF care team or study investigators participant should not participate in the study
Inability to provide informed consent/assent
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Sara Malina
Phone
443-287-8997
Email
smalina1@jh.edu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Malinda Wu, MD, MSc
Organizational Affiliation
Johns Hopkins University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Johns Hopkins
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sara Malina
Phone
443-287-8997
Email
smalina1@jh.edu
First Name & Middle Initial & Last Name & Degree
Malinda Wu, MD, MSc
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Estrogen Patches for Adolescent and Young Adult Women With Cystic Fibrosis
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