Pilot E2 for Hypogonadal Women With CFBD

NCT03724955 | Withdrawn Phase 4 FDA Drug

• 1 other identifier: IRB00107135
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief 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 Disease (CFBD).

Conditions

Cystic Fibrosis Related Bone Disease

Keywords

Hypogonadism; Estrogen Supplement

Outcome Measures

Primary Outcomes (1)

• 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.

  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

  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

  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

  Baseline/Enrollment visit and at 6 months

• Change in percentage of FEV1 from baseline to 6 months among the treatment and placebo groups

  Baseline/Enrollment visit and at 6 months

Study Arms (2)

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.

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.

Other: Placebo

Interventions

Estradiol 2 mg DRUG

Participants in this arm will receive Estradiol 2 mg oral daily for 6 months

Also known as: Estrace

Treatment Group

Placebo OTHER

The placebo group will receive placebo oral daily for 6 months

Placebo Group

Eligibility Criteria

• Age: 18 Years+
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Emory University: lead

Study Sites (1)

Emory University Hospital

Atlanta, Georgia, 30322, United States

Location

Related Publications (1)

• 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.

  PMID: 34412687 DERIVED

MeSH Terms

Conditions

Hypogonadism

Interventions

Estradiol

Condition Hierarchy (Ancestors)

Gonadal Disorders; Endocrine System Diseases

Intervention Hierarchy (Ancestors)

Estrenes; Estranes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Estradiol Congeners; Gonadal Steroid Hormones; Gonadal Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists

Study Officials

• Vin Tangpricha, MD

  Emory University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, OUTCOMES ASSESSOR
• Masking Details: Double Blinded
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Model Details: Prospective, double-blinded, randomized, placebo-controlled, interventional

Study Record Dates

• First Submitted: October 29, 2018
• First Posted: October 30, 2018
• Study Start: April 1, 2019
• Primary Completion: June 1, 2021
• Study Completion: June 1, 2021
• Last Updated: April 18, 2019

Record last verified: 2019-04

Locations

US (1)