Ruxolitinib for Premalignant Breast Disease

NCT02928978 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: H-38855
• Study Type: interventional
• Target: 84
• Locations: 1 country
• Sites: 8

Brief Summary

This study is evaluating how ruxolitinib affects premalignant breast cells. One half of the study participants will receive ruxolitinib for approximately 15 days, and the other half will receive a placebo (sugar pill) for approximately 15 days. Once study participants have completed their ruxolitinib or placebo, participants will undergo surgery to remove the premalignant breast tissue.

Conditions

Ductal Carcinoma In Situ; Atypical Lobular Hyperplasia; Atypical Ductal Hyperplasia; Lobular Carcinoma In Situ

Keywords

Premalignant Breast Disease; ALH; ADH; DCIS; LCIS; Breast Cancer Prevention; Ruxolitinib

Outcome Measures

Primary Outcomes (1)

• Change in Apoptosis

  The number of premalignant breast cells in apoptosis at the time of diagnosis will be compared to the number of cells in apoptosis following treatment with 15 (+/- 5) days of ruxolitinib or placebo.

  15 days (+/- 5 days)

Secondary Outcomes (1)

• Proliferation/Apoptosis Biomarkers

  15 days (+/- 5 days)

Study Arms (2)

Ruxolitinib

EXPERIMENTAL

Participants will receive ruxolitinib 20 mg by mouth twice daily for 15 days (+/- 5 days). Ruxolitinib will be supplied as four, 5 mg tablets.

Drug: Ruxolitinib

Placebo

PLACEBO COMPARATOR

Participants will receive a placebo (sugar pill) that is designed to mimic ruxolitinib. The placebo will be supplied as four, 5 mg tablets. Participants assigned to this arm will take four, 5 mg tablets by mouth twice daily for 15 days (+/- 5 days).

Drug: Placebo (for Ruxolitinib)

Interventions

Ruxolitinib DRUG

tablet (taken by mouth)

Also known as: Jakafi, INCB018424

Ruxolitinib

Placebo (for Ruxolitinib) DRUG

tablet (taken by mouth)

Placebo

Eligibility Criteria

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

You may qualify if:

• Have a breast biopsy showing ADH (atypical ductal hyperplasia), ALH (atypical lobular hyperplasia), LCIS (lobular carcinoma in situ), or DCIS (ductal carcinoma in situ) requiring surgical excision. Microinvasive disease is allowed.
• NOTE: Tissue from the diagnostic biopsy must be accessible/available for research correlates (i.e., a tissue block or \~10 unstained slides). Due to the nature of the study, fewer slides may be accepted with prior permission from the Protocol Chair if there is insufficient tissue.
• Women and men age 18 and older.
• Adequate hematologic and organ function, defined as follows:
• Absolute neutrophil count ≥ 1500/mm3
• Hemoglobin ≥ 9.0 g/dL
• Platelet levels \>200 x 109/L
• Total bilirubin ≤ 1.5 x institutional upper limit of normal (ULN)
• AST/ALT ≤ 2.5 x institutional ULN
• Alkaline phosphatase ≤ 5 x institutional ULN
• Creatinine clearance \> 50 mL/min as calculated by the Cockcroft-Gault method
• Willing to not use concomitant strong CYP3A4 inhibitors as this could interfere with the metabolism of ruxolitinib (i.e azole antifungals, clarithromycin, conivaptan, grapefruit juice, mibefradil, nefazodone, protease inhibitors, telithromycin).
• Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation.
• If the patient undergoes germline genetic testing, the results must be received prior to randomization, as the results may affect the surgical approach and, in turn, the date of surgical excision.
• Patient understands the study regimen, its requirements, risks, and discomforts, and is able and willing to sign a written informed consent document.

You may not qualify if:

• Treatment with selective estrogen receptor modulators (SERMs) or aromatase inhibitors for breast cancer prevention within 1 year prior to starting study treatment.
• Treatment with any other investigational agents within 30 days of starting study treatment.
• Current diagnosis of invasive breast cancer (current microinvasive disease is allowed), or previous history of invasive breast cancer diagnosed within the last 5 years.
• NOTE: If previous history of ER+ invasive breast cancer diagnosed \> 5 years ago, patient must be off endocrine therapy for at least 1 year prior to starting study treatment.
• Uncontrolled intercurrent illness including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, end stage renal disease (ESRD), or psychiatric illness/social situations that would limit compliance with study requirements.
• Women who are pregnant or nursing.
• HIV-positive participants on combination antiretroviral therapy are ineligible because of the potential for pharmacokinetic interactions with ruxolitinib.
• Prior or current treatment with a JAK inhibitor, for any indication.
• Known active Hepatitis B or C.

Sponsors & Collaborators

• Julie Nangia: lead
• Incyte Corporation: collaborator
• Translational Breast Cancer Research Consortium: collaborator

Study Sites (8)

University of Alabama at Birmingham

Birmingham, Alabama, 35294, United States

Location

Indiana University Melvin and Bren Simon Cancer Center

Indianapolis, Indiana, 46202, United States

Location

Montefiore Medical Center

The Bronx, New York, 10461, United States

Location

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

Location

Vanderbilt-Ingram Cancer Center

Nashville, Tennessee, 37232, United States

Location

University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Harris Health System - Smith Clinic

Houston, Texas, 77054, United States

Location

O'Quinn Medical Tower - McNair Campus; Dan L Duncan Comprehensive Cancer Center

Houston, Texas, 77054, United States

Location

MeSH Terms

Conditions

Carcinoma, Intraductal, Noninfiltrating; Breast Carcinoma In Situ

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Carcinoma in Situ; Neoplasms, Ductal, Lobular, and Medullary; Breast Neoplasms; Neoplasms by Site; Breast Diseases; Skin Diseases; Skin and Connective Tissue Diseases

Study Officials

• Julie Nangia, M.D.

  Baylor College of Medicine

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: October 7, 2016
• First Posted: October 10, 2016
• Study Start: May 13, 2018
• Primary Completion: August 29, 2024
• Study Completion: September 18, 2024
• Last Updated: April 13, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (8)