Cystic Fibrosis Related Bone DiseasePhase 4NCT03724955
Pilot E2 for Hypogonadal Women With CFBD
A Phase 4 study testing Estradiol 2 mg in people with Cystic Fibrosis Related Bone Disease, run by Emory University at 1 site in United States.
Trial summary
The study is a prospective, double blinded, placebo controlled, randomized study to evaluate the effects of daily oral estrogen supplements on bone health, sexual and reproductive health, quality of life and markers of inflammation and lung function when given to hypogonadal women with Cystic Fibrosis related Bone… Read more about this study
- Who is it for?
- Ages 18 years and older, women only
- Conditions the study is for
- Cystic Fibrosis Related Bone Disease
- What is being tested?
- Estradiol 2 mg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2019to Jun 2021
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Women only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adult and adolescent female CF patients (age ≥ 18 years),
- presenting to the CF clinic for routine follow up of cystic fibrosis,
- hypogonadal women defined as E2 level < 25,
- dual energy X-ray absorptiometry (DEXA) scan within 2 years of enrollment with T- or Z-score < -1,
- able to tolerate oral medications.
Who cannot join
- Inability to obtain or declined informed consent from the subject and/or legally authorized representative,
- Pregnancy,
- Too ill to participate in study based on investigator's or study team's opinion,
- Current use of systemic estrogen,
- History of thromboembolic event within the previous 2 years,
- History of migraines with aura,
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- Hypercoagulability including previous diagnosis of Factor V Leiden or Protein C or S deficiency,
- Current smoker,
- History of diagnosis with breast or uterine cancer,
- Current significant liver disease with cholelithiasis or cirrhosis,
- Status post lung or liver transplantation,
- Current use of systemic steroids
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Pilot Study Evaluating the Effects of Estradiol for Hypogonadal Women With Cystic Fibrosis and Osteopenia
The study is a prospective, double blinded, placebo controlled, randomized study to evaluate the effects of daily oral estrogen supplements on bone health, sexual and reproductive health, quality of life and markers of inflammation and lung function when given to hypogonadal women with Cystic Fibrosis related Bone Disease (CFBD).
Read the detailed description
Cystic fibrosis (CF) is the most common fatal heritable disease in non-Hispanic whites; CF results in frequent lung infections, chronic inflammation and progressive lung failure. With advancements in medical care, patients with CF are living longer. The median survival of women with CF born and diagnosed in 2010 is projected to be 37 years. Patients with CF are now living long enough to develop comorbidities like Cystic Fibrosis related Bone Disease (CFBD), hypogonadism manifesting as pubertal delay, premature ovarian insufficiency or hypothalamic hypogonadism. These comorbidities in women without CF can be improved by estrogen supplementation.
It is well established that treating hypogonadal (low estrogen levels) women with estrogen can improve their bone health, slow the rate of bone loss and decrease bone turnover markers. Supplemental estrogen given to hypogonadal women can improve sexual and reproductive health including reduction of perimenopausal symptoms and improvement in sexual function which can improve quality of life. There is very little research investigating the effects of estrogen treatment for hypogonadal women with CF. The purpose of this study is to test the hypothesis that daily oral estrogen supplements given to hypogonadal women with CFBD will improve their bone health, sexual and reproductive health, and quality of life and modify markers of inflammation and lung function.
- Conditions
- Cystic Fibrosis Related Bone Disease
- Being tested
- Estradiol 2 mg, Placebo
- Keywords
- Hypogonadism, Estrogen Supplement
- Medical subject headings
- Hypogonadism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Estradiol 2 mgDrug
Also called Estrace
Participants in this arm will receive Estradiol 2 mg oral daily for 6 months
PlaceboOther
The placebo group will receive placebo oral daily for 6 months
| Study group | Role | What they receive |
|---|---|---|
| Treatment Group | Experimental | The treatment group will receive Estradiol 2 mg oral daily for 6 months. Medication will be mailed to patient. All study drugs will be dispensed by the Investigational Drug Pharmacy. Receives: Drug: Estradiol 2 mg |
| Placebo Group | Placebo comparator | The placebo group will receive placebo oral daily for 6 months. Medication will be mailed to patient. Placebo will be dispensed by the Investigational Drug Pharmacy. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in serum estradiol level in picograms per milliliter (pg/mL) from baseline to 6 months among the treatment and placebo groups
Serum estradiol measures the amount of the hormone estradiol in the blood. The level is measured using a blood test that is performed at baseline/enrollment visit and at 6 months. According to Mayo Medical Laboratories, normal levels of estradiol (E2) for menstruating women range from 15 to 350 picograms per milliliter (pg/mL). The levels of estradiol among both groups are recorded and compared from baseline to 6 months.
Measured: Baseline/Enrollment visit and at 6 months
Secondary outcomes (4)
Change in Serum carboxy-terminal collagen crosslinks (CTX-1) from baseline to 6 months among the treatment and placebo groups
Serum CTX is a telopeptide that can be used as a biomarker in the serum to measure the rate of bone turnover. The CTX test measures for the presence and concentration of a crosslink peptide sequence of type I collagen, found, among other tissues, in bone. This specific peptide sequence relates to bone turnover because it is the portion that is cleaved by osteoclasts during bone resorption, and its serum levels are therefore proportional to osteoclastic activity at the time the blood sample is drawn. Although laboratory normal ranges are said to be between 50 pg/mL and 450 pg/mL, serum levels in healthy patients not taking bisphosphonates tends to be above 300 pg/mL. This is measured at baseline and at 6 months between the 2 groups and compared.
Measured: Baseline/Enrollment visit and at 6 months
change in of Serum Procollagen I Intact N-Terminal (P1NP) measured in mcg/L from baseline to 6 months among the treatment and placebo groups
Procollagen type I propeptides are derived from collagen type I, which is the most common collagen type found in mineralized bone. Procollagen type I N-terminal propeptide (P1NP) is considered the most sensitive marker of bone formation and it is particularly useful for monitoring bone formation therapies and antiresorptive therapies. It is measured through a blood test. Blood is drawn at baseline and at 6 months among the participants of both groups and compared. Reference Values for Adult male: 22-87 mcg/L; Adult female premenopausal: 19-83 mcg/L; Adult female postmenopausal: 16-96 mcg/L.
Measured: Baseline/Enrollment visit and at 6 months
Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory score from baseline to 6 months among the treatment and placebo groups
The Cystic Fibrosis Questionnaire-Revised (CFQ-R) is a validated patient-reported outcome (PRO) containing both generic scales and scales specific to cystic fibrosis (CF). The CFQ-R measures functioning in a variety of domains, including Physical Functioning, Vitality, Health Perceptions, Respiratory Symptoms, Treatment Burden, Role Functioning, Emotional Functioning, and Social Functioning. Each CFQ-R domain yields standardized scores ranging from 0 to 100; higher domain scores indicate better health-related quality of life. The study aims to look at the changes in CFQ-R-Respiratory scores among the two groups. The questionnaire is administered at baseline and at follow up visits. The change in score from Baseline to 6 months is recorded and compared.
Measured: Baseline/Enrollment visit and at 6 months
Change in percentage of FEV1 from baseline to 6 months among the treatment and placebo groups
FEV1 measures the lung functioning among the Cystic Fibrosis patients. Spirometry is used for measurement of FEV1.Spirometry is performed by deeply inhaling and forcefully exhaling into a spirometer (the device that records the various measurements of lung function). Forced expiratory volume-one second (FEV1) is a measure of how much air can be exhaled in one second following a deep inhalation. In normal people the Percentage of predicted FEV1 value is 80% or greater. 60%-69% are moderately abnormal; 35%-49% severely abnormal and less than 35% very severely abnormal. In cystic fibrosis patients it is usually less than normal. The test is administered at baseline and at follow up visits. The change in score from Baseline to 6 months is recorded and compared.
Measured: Baseline/Enrollment visit and at 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Oct 2018
- Study startApr 2019 (estimated)
- Primary completionJun 2021 (estimated)
- Study completionJun 2021 (estimated)
Why it stopped: Issues with Recruitment.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cystic Fibrosis Related Bone DiseaseStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Prospective, double-blinded, randomized, placebo-controlled, interventional
- Masking
- Double blind
- Who is masked
- Participant, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Vin Tangpricha, MD, PH.D, Emory University
- Investigators
- Vin Tangpricha, MD, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03724955
- Sponsor's protocol ID
- IRB00107135
- Status last verified
- Apr 2019
- First submitted
- 29 Oct 2018
- Last updated
- 18 Apr 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Wu M, Tirouvanziam R, Arora N, Tangpricha V. Findings from a feasibility study of estradiol for hypogonadal women with cystic fibrosis-related bone disease. Pilot Feasibility Stud. 2021 Aug 19;7(1):160. doi: 10.1186/s40814-021-00897-x.
PubMed 34412687
Study at a glance
From the registry record, last updated by the sponsor on 18 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study Evaluating the Effects of Estradiol for Hypogonadal Women With Cystic Fibrosis and Osteopenia
- ClinicalTrials.gov ID
- NCT03724955
- Acronym / sponsor ID
- IRB00107135
- Conditions
- Cystic Fibrosis Related Bone Disease
- Keywords
- Hypogonadism, Estrogen Supplement
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Issues with Recruitment.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Study start
- Apr 2019 (estimated)
- Primary completion
- Jun 2021 (estimated)
- Study completion
- Jun 2021 (estimated)
- First posted
- 30 Oct 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study is a prospective, double blinded, placebo controlled, randomized study to evaluate the effects of daily oral estrogen supplements on bone health, sexual and reproductive health, quality of life and markers of inflammation and lung function when given to hypogonadal women with Cystic Fibrosis related Bone Disease (CFBD).
Detailed description
Cystic fibrosis (CF) is the most common fatal heritable disease in non-Hispanic whites; CF results in frequent lung infections, chronic inflammation and progressive lung failure. With advancements in medical care, patients with CF are living longer. The median survival of women with CF born and diagnosed in 2010 is projected to be 37 years. Patients with CF are now living long enough to develop comorbidities like Cystic Fibrosis related Bone Disease (CFBD), hypogonadism manifesting as pubertal delay, premature ovarian insufficiency or hypothalamic hypogonadism. These comorbidities in women without CF can be improved by estrogen supplementation.
It is well established that treating hypogonadal (low estrogen levels) women with estrogen can improve their bone health, slow the rate of bone loss and decrease bone turnover markers. Supplemental estrogen given to hypogonadal women can improve sexual and reproductive health including reduction of perimenopausal symptoms and improvement in sexual function which can improve quality of life. There is very little research investigating the effects of estrogen treatment for hypogonadal women with CF. The purpose of this study is to test the hypothesis that daily oral estrogen supplements given to hypogonadal women with CFBD will improve their bone health, sexual and reproductive health, and quality of life and modify markers of inflammation and lung function.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, outcomes assessor)
- Number of arms
- 2
- Model description
- Prospective, double-blinded, randomized, placebo-controlled, interventional
| Arm | Type | Receives |
|---|---|---|
| Treatment Group | Experimental | Drug: Estradiol 2 mgThe treatment group will receive Estradiol 2 mg oral daily for 6 months. Medication will be mailed to patient. All study drugs will be dispensed by the Investigational Drug Pharmacy. |
| Placebo Group | Placebo comparator | Other: PlaceboThe placebo group will receive placebo oral daily for 6 months. Medication will be mailed to patient. Placebo will be dispensed by the Investigational Drug Pharmacy. |
Who may qualify
- Ages
- 18 years and older
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Adult and adolescent female CF patients (age ≥ 18 years),
- presenting to the CF clinic for routine follow up of cystic fibrosis,
- hypogonadal women defined as E2 level < 25,
- dual energy X-ray absorptiometry (DEXA) scan within 2 years of enrollment with T- or Z-score < -1,
- able to tolerate oral medications.
Key exclusion criteria (12 listed)
- Inability to obtain or declined informed consent from the subject and/or legally authorized representative,
- Pregnancy,
- Too ill to participate in study based on investigator's or study team's opinion,
- Current use of systemic estrogen,
- History of thromboembolic event within the previous 2 years,
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Estradiol 2 mgAlso: EstraceParticipants in this arm will receive Estradiol 2 mg oral daily for 6 months | Drug | Treatment Group |
| PlaceboThe placebo group will receive placebo oral daily for 6 months | Other | Placebo Group |
Primary outcome
Change in serum estradiol level in picograms per milliliter (pg/mL) from baseline to 6 months among the treatment and placebo groups
Time frame: Baseline/Enrollment visit and at 6 months
Serum estradiol measures the amount of the hormone estradiol in the blood. The level is measured using a blood test that is performed at baseline/enrollment visit and at 6 months. According to Mayo Medical Laboratories, normal levels of estradiol (E2) for menstruating women range from 15 to 350 picograms per milliliter (pg/mL). The levels of estradiol among both groups are recorded and compared from baseline to 6 months.
4 secondary outcomes
Change in Serum carboxy-terminal collagen crosslinks (CTX-1) from baseline to 6 months among the treatment and placebo groups
Time frame: Baseline/Enrollment visit and at 6 months
change in of Serum Procollagen I Intact N-Terminal (P1NP) measured in mcg/L from baseline to 6 months among the treatment and placebo groups
Time frame: Baseline/Enrollment visit and at 6 months
Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory score from baseline to 6 months among the treatment and placebo groups
Time frame: Baseline/Enrollment visit and at 6 months
Change in percentage of FEV1 from baseline to 6 months among the treatment and placebo groups
Time frame: Baseline/Enrollment visit and at 6 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Apr 2019.
- Derived from this study: PubMed 34412687Wu M, Tirouvanziam R, Arora N, Tangpricha V. Findings from a feasibility study of estradiol for hypogonadal women with cystic fibrosis-related bone disease. Pilot Feasibility Stud. 2021 Aug 19;7(1):160. doi: 10.1186/s40814-021-00897-x.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Vin Tangpricha, MD principal investigator
Emory University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Estradiol 2 mg in people with Cystic Fibrosis Related Bone Disease, run by Emory University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 18 Apr 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in serum estradiol level in picograms per milliliter (pg/mL) from baseline to 6 months among the treatment and placebo groups (time frame: Baseline/Enrollment visit and at 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03724955, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Apr 2019.