Overcoming challenges in conducting early phase breast cancer prevention trials: Bazedoxifene and conjugated estrogens vs waitlist control

Contemp Clin Trials. 2024 Nov:146:107697. doi: 10.1016/j.cct.2024.107697. Epub 2024 Sep 17.

Abstract

Background: The combination of bazedoxifene 20 mg (BZA) and conjugated estrogens 0.45 mg (CE) marketed as Duavee® is approved for vasomotor symptom relief and osteoporosis prevention. Our pilot study suggested it had potential breast cancer risk reduction, and we proposed a multisite Phase IIB primary prevention trial assessing change in breast imaging and tissue risk biomarkers. By the time funding was acquired in February 2021, Duavee® was unavailable with an uncertain return date. A redesign was needed to salvage the study.

Methods: The basic trial design was minimally altered. Women age 45-64 at elevated risk for breast cancer with vasomotor symptoms and no menses for at least 2 months have mammography, phlebotomy, and benign breast tissue sampling before and after 6 months of intervention. However, instead of Duavee® (single pill) vs placebo, women are randomized to 6 months of BZA + CE vs Waitlist. Those initially randomized to Waitlist can receive BZA + CE after 6 months. The primary endpoint is between arm difference in change in a fully automated measure of mammographic density with blood and tissue-based secondary endpoints.

Outcomes: Accrual initiation was delayed due to contractual difficulties surrounding BZA importation during COVID-19 and deploying a fully automated method (Volpara®) to assess the primary endpoint. To accommodate this delay, a mid-grant no cost extension along with amended eligibility requirements were employed. 61/120 participants needed were entered in the initial 27 months of accrual and 37 months of funding. Despite a late start, accrual is likely to be completed within the funding period.

Keywords: Breast cancer prevention; Duavee; Fibroglandular volume; Random periareolar fine needle aspiration.

Publication types

  • Clinical Trial Protocol

MeSH terms

  • Breast Density / drug effects
  • Breast Neoplasms* / prevention & control
  • Estrogens, Conjugated (USP)* / administration & dosage
  • Estrogens, Conjugated (USP)* / therapeutic use
  • Female
  • Humans
  • Indoles / administration & dosage
  • Indoles / therapeutic use
  • Mammography / economics
  • Mammography / methods
  • Middle Aged
  • Primary Prevention / methods
  • Randomized Controlled Trials as Topic*
  • Research Design
  • Selective Estrogen Receptor Modulators* / administration & dosage
  • Selective Estrogen Receptor Modulators* / therapeutic use
  • Waiting Lists

Substances

  • bazedoxifene
  • Estrogens, Conjugated (USP)
  • Indoles
  • Selective Estrogen Receptor Modulators