Pilot E2 for Hypogonadal Women With CFBD

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03724955
• Date of registration: 29/10/2018
• Prospective Registration: Yes
• Primary sponsor: Emory University
• Public title: Pilot E2 for Hypogonadal Women With CFBD
• Scientific title: Pilot Study Evaluating the Effects of Estradiol for Hypogonadal Women With Cystic Fibrosis and Osteopenia
• Date of first enrolment: April 2019
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT03724955
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Outcomes Assessor).
• Phase: Phase 4

Countries of recruitment

United States

Contacts

• Name: Vin Tangpricha, MD
• Affiliation: Emory University

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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.

Exclusion Criteria:

- 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,

- 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



Age minimum: 18 Years
Age maximum: N/A
Gender: Female

Health Condition(s) or Problem(s) studied

Cystic Fibrosis Related Bone Disease

Intervention(s)

Drug: Estradiol 2 mg

Other: Placebo

Primary Outcome(s)

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]

Secondary Outcome(s)

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 percentage of FEV1 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 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]

Secondary ID(s)

IRB00107135

Source(s) of Monetary Support

Please refer to primary and secondary sponsors