Trial Outcomes & Findings for Pilot Study of Denosumab in BRCA1/2 Mutation Carriers Scheduled for Risk-Reducing Salpingo-Oophorectomy (NCT NCT03382574)

NCT ID: NCT03382574

Last Updated: 2022-06-14

Results Overview

Will be assessed using immunohistochemistry (IHC). Quantitative measures of the expression of Ki67 based upon percentage of positive cells will be scored by a pathologist blinded to treatment assignment. In order to evaluate the difference of Ki67 expression between the two arms, 2-sample t-test will be considered.

Recruitment status

TERMINATED

Study phase

EARLY_PHASE1

Target enrollment

2 participants

Primary outcome timeframe

Up to 12 months

Results posted on

2022-06-14

Participant Flow

This multicenter protocol will be conducted at the following five sites: Columbia University Irving Medical Center (CUIMC), New York, NY; Weill-Cornell Medical Center, New York, NY; the Dana Farber Cancer Institute (DFCI), Boston, MA; and Tel Aviv Sourasky Medical Center and Chaim Sheba Medical Center, Tel Aviv, Israel.

2 participants consented at Tel Aviv Sourasky Medical Center, no participants randomized nor received trial intervention (Participant 1 was deemed ineligible due to elevated FSH level and Participant 2 did not start intervention due to COVID-19 lockdown restrictions).

Participant milestones

Participant milestones
Measure
Arm I (Denosumab, Risk-reducing Salpingo-oophorectomy)/Arm II (Risk-reducing Salpingo-oophorectomy)
Beginning within 3 days of menstrual cycle, patients receive denosumab SC every 4 weeks for 1-2 doses and undergo risk-reducing salpingo-oophorectomy 14-28 days after last dose. Denosumab: Given SC Salpingo-Oophorectomy: Undergo risk-reducing salpingo-oophorectomy Arm II Patients receive no treatment for 2-8 weeks and then undergo risk-reducing salpingo-oophorectomy. Salpingo-Oophorectomy: Undergo risk-reducing salpingo-oophorectomy
Overall Study
STARTED
0
Overall Study
COMPLETED
0
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pilot Study of Denosumab in BRCA1/2 Mutation Carriers Scheduled for Risk-Reducing Salpingo-Oophorectomy

Baseline characteristics by cohort

Baseline data not reported

PRIMARY outcome

Timeframe: Up to 12 months

Population: No participants were randomized, therefore no data collected.

Will be assessed using immunohistochemistry (IHC). Quantitative measures of the expression of Ki67 based upon percentage of positive cells will be scored by a pathologist blinded to treatment assignment. In order to evaluate the difference of Ki67 expression between the two arms, 2-sample t-test will be considered.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 12 months

Population: No participants were randomized, therefore no data collected.

Will be assessed using IHC. In order to evaluate the difference of Ki67 expression between the two arms, 2-sample t-test will be considered.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 12 months

Population: No participants were randomized, therefore no data collected.

Will be assessed using IHC. Including: apoptosis with cleaved caspase-3 (IHC), RANK/RANKL (IHC), estrogen receptor (ER)/progesterone receptor (PR) (IHC), CD44 and p53 (IHC), and STAT3 and pSTAT3 (IHC). Values of tissue-based biomarkers measurements such as tissue Ki67 proliferation index, serum progesterone, etc., which are continuous variables, will be summarized by descriptive statistics including mean, standard deviation, median and range. For tissue biomarkers, linear regression models will be employed to investigate the association of treatment while adjusting for possible confounders (i.e., age, race, etc.). Normality, homoscedasticity, independence of errors, and lack of multicollinearity in the covariates will be evaluated; if needed, proper transformation will be considered.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 12 months

Population: No participants were randomized, therefore no data collected.

For gene expression profiling analysis, nSolver Analysis Software (nanoString Technologies, Washington \[WA\]) will be used. Geometric mean is used for calculation of normalization factors. Student's t test is used to calculate differential expression.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to time of surgery

Population: No participants were randomized, therefore no data collected.

Values of serum biomarkers measurements such as tissue Ki67 proliferation index, serum progesterone, etc., which are continuous variables, will be summarized by descriptive statistics including mean, standard deviation, median and range.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline up to 12 months after start of intervention

Population: No participants were randomized, therefore no data collected.

2-sample t-test may be applied to evaluate any change in serum biomarkers from baseline to after intervention. To investigate the overall changes in serum biomarkers, a linear mixed model that accommodates intra-participant correlation due to repeated measurements will be utilized adjusting for any potential covariates.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 12 months after start of treatment

Population: No participants were randomized, therefore no data collected.

Categorical variables, such as adverse events, will be summarized by frequency and proportion.

Outcome measures

Outcome data not reported

Adverse Events

Arm I (Denosumab, Risk-reducing Salpingo-oophorectomy)

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Arm II (Risk-reducing Salpingo-oophorectomy)

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Powel Brown, Chair, Clinical Cancer Prevention

UT MD Anderson Cancer Center

Phone: (713) 745-3672

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place