Trial Outcomes & Findings for Enzalutamide in Patients With Androgen Receptor Positive (AR+) Ovarian, Primary Peritoneal or Fallopian Tube Cancer and One, Two or Three Prior Therapies (NCT NCT01974765)

NCT ID: NCT01974765

Last Updated: 2023-04-28

Results Overview

Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

59 participants

Primary outcome timeframe

6 months

Results posted on

2023-04-28

Participant Flow

Participant milestones

Participant milestones
Measure	Enzalutamide After signing a screening consent, patients archival tissue will be evaluated for degree of AR positivity by AR staining. Patients with no archival tissue available will undergo a biopsy (using the modality deemed most appropriate by the patient's physician) for collection of tumor tissue for AR positivity by IHC. Only AR+ patients, defined as ≥5 % positivity by IHC, will be included. All IHC testing will be performed in the MSKCC Clinical CLIA approved laboratory. All enrolled patients will be treated with enzalutamide 160 mg by mouth QD until progression of disease (POD), unacceptable toxicity or withdrawal from study. All treatments will be administered in the outpatient setting. Enzalutamide: All enrolled patients will be treated with enzalutamide 160mg by mouth QD. Study drugs will be self-administered by patients. A cycle is 28 days.
Overall Study STARTED	59
Overall Study COMPLETED	55
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Reasons for withdrawal
Measure	Enzalutamide After signing a screening consent, patients archival tissue will be evaluated for degree of AR positivity by AR staining. Patients with no archival tissue available will undergo a biopsy (using the modality deemed most appropriate by the patient's physician) for collection of tumor tissue for AR positivity by IHC. Only AR+ patients, defined as ≥5 % positivity by IHC, will be included. All IHC testing will be performed in the MSKCC Clinical CLIA approved laboratory. All enrolled patients will be treated with enzalutamide 160 mg by mouth QD until progression of disease (POD), unacceptable toxicity or withdrawal from study. All treatments will be administered in the outpatient setting. Enzalutamide: All enrolled patients will be treated with enzalutamide 160mg by mouth QD. Study drugs will be self-administered by patients. A cycle is 28 days.
Overall Study Withdrawal by Subject	1
Overall Study Death	1
Overall Study Adverse Event	2

Baseline Characteristics

Enzalutamide in Patients With Androgen Receptor Positive (AR+) Ovarian, Primary Peritoneal or Fallopian Tube Cancer and One, Two or Three Prior Therapies

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Enzalutamide n=59 Participants After signing a screening consent, patients archival tissue will be evaluated for degree of AR positivity by AR staining. Patients with no archival tissue available will undergo a biopsy (using the modality deemed most appropriate by the patient's physician) for collection of tumor tissue for AR positivity by IHC. Only AR+ patients, defined as ≥5 % positivity by IHC, will be included. All IHC testing will be performed in the MSKCC Clinical CLIA approved laboratory. All enrolled patients will be treated with enzalutamide 160 mg by mouth QD until progression of disease (POD), unacceptable toxicity or withdrawal from study. All treatments will be administered in the outpatient setting. Enzalutamide: All enrolled patients will be treated with enzalutamide 160mg by mouth QD. Study drugs will be self-administered by patients. A cycle is 28 days.
Age, Continuous	64 years n=99 Participants
Sex: Female, Male Female	59 Participants n=99 Participants
Sex: Female, Male Male	0 Participants n=99 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=99 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	57 Participants n=99 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants
Race (NIH/OMB) Asian	5 Participants n=99 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants
Race (NIH/OMB) Black or African American	2 Participants n=99 Participants
Race (NIH/OMB) White	48 Participants n=99 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants
Race (NIH/OMB) Unknown or Not Reported	4 Participants n=99 Participants
Region of Enrollment United States	59 Participants n=99 Participants

PRIMARY outcome

Timeframe: 6 months

Outcome measures

Outcome measures
Measure	Enzalutamide n=59 Participants After signing a screening consent, patients archival tissue will be evaluated for degree of AR positivity by AR staining. Patients with no archival tissue available will undergo a biopsy (using the modality deemed most appropriate by the patient's physician) for collection of tumor tissue for AR positivity by IHC. Only AR+ patients, defined as ≥5 % positivity by IHC, will be included. All IHC testing will be performed in the MSKCC Clinical CLIA approved laboratory. All enrolled patients will be treated with enzalutamide 160 mg by mouth QD until progression of disease (POD), unacceptable toxicity or withdrawal from study. All treatments will be administered in the outpatient setting. Enzalutamide: All enrolled patients will be treated with enzalutamide 160mg by mouth QD. Study drugs will be self-administered by patients. A cycle is 28 days.
Overall Response Rate	1.7 percentage of participants with response Interval 0.2 to 100.0

PRIMARY outcome

Timeframe: 6 months

Outcome measures

Outcome measures
Measure	Enzalutamide n=59 Participants After signing a screening consent, patients archival tissue will be evaluated for degree of AR positivity by AR staining. Patients with no archival tissue available will undergo a biopsy (using the modality deemed most appropriate by the patient's physician) for collection of tumor tissue for AR positivity by IHC. Only AR+ patients, defined as ≥5 % positivity by IHC, will be included. All IHC testing will be performed in the MSKCC Clinical CLIA approved laboratory. All enrolled patients will be treated with enzalutamide 160 mg by mouth QD until progression of disease (POD), unacceptable toxicity or withdrawal from study. All treatments will be administered in the outpatient setting. Enzalutamide: All enrolled patients will be treated with enzalutamide 160mg by mouth QD. Study drugs will be self-administered by patients. A cycle is 28 days.
Partial Response Partial Response	1 Participants
Partial Response No Partial Response	58 Participants

SECONDARY outcome

Timeframe: up to 2 years

Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0. For each symptom i.e., frequency, severity, the worst score experienced by the patient will be recorded

Outcome measures

Outcome measures
Measure	Enzalutamide n=59 Participants After signing a screening consent, patients archival tissue will be evaluated for degree of AR positivity by AR staining. Patients with no archival tissue available will undergo a biopsy (using the modality deemed most appropriate by the patient's physician) for collection of tumor tissue for AR positivity by IHC. Only AR+ patients, defined as ≥5 % positivity by IHC, will be included. All IHC testing will be performed in the MSKCC Clinical CLIA approved laboratory. All enrolled patients will be treated with enzalutamide 160 mg by mouth QD until progression of disease (POD), unacceptable toxicity or withdrawal from study. All treatments will be administered in the outpatient setting. Enzalutamide: All enrolled patients will be treated with enzalutamide 160mg by mouth QD. Study drugs will be self-administered by patients. A cycle is 28 days.
Number of Participants Assessed for Treatment-Related Adverse Events	59 Participants

SECONDARY outcome

Timeframe: 2 years

Median Androgen Receptor/AR expression levels on tissue will be determined using standard immunohistochemistry methodology

Outcome measures

Outcome measures
Measure	Enzalutamide n=59 Participants After signing a screening consent, patients archival tissue will be evaluated for degree of AR positivity by AR staining. Patients with no archival tissue available will undergo a biopsy (using the modality deemed most appropriate by the patient's physician) for collection of tumor tissue for AR positivity by IHC. Only AR+ patients, defined as ≥5 % positivity by IHC, will be included. All IHC testing will be performed in the MSKCC Clinical CLIA approved laboratory. All enrolled patients will be treated with enzalutamide 160 mg by mouth QD until progression of disease (POD), unacceptable toxicity or withdrawal from study. All treatments will be administered in the outpatient setting. Enzalutamide: All enrolled patients will be treated with enzalutamide 160mg by mouth QD. Study drugs will be self-administered by patients. A cycle is 28 days.
Median Androgen Receptor/AR Expression Levels on Tissue	60 ng/dL Interval 5.0 to 99.0

Adverse Events

Enzalutamide

Serious events: 9 serious events

Other events: 14 other events

Deaths: 35 deaths

Serious adverse events

Serious adverse events
Measure	Enzalutamide n=59 participants at risk After signing a screening consent, patients archival tissue will be evaluated for degree of AR positivity by AR staining. Patients with no archival tissue available will undergo a biopsy (using the modality deemed most appropriate by the patient's physician) for collection of tumor tissue for AR positivity by IHC. Only AR+ patients, defined as ≥5 % positivity by IHC, will be included. All IHC testing will be performed in the MSKCC Clinical CLIA approved laboratory. All enrolled patients will be treated with enzalutamide 160 mg by mouth QD until progression of disease (POD), unacceptable toxicity or withdrawal from study. All treatments will be administered in the outpatient setting. Enzalutamide: All enrolled patients will be treated with enzalutamide 160mg by mouth QD. Study drugs will be self-administered by patients. A cycle is 28 days.
Infections and infestations Abdominal infection	1.7% 1/59 • 2 years
Gastrointestinal disorders Abdominal pain	3.4% 2/59 • 2 years
Cardiac disorders Cardiac arrest	1.7% 1/59 • 2 years
Gastrointestinal disorders Constipation	1.7% 1/59 • 2 years
Gastrointestinal disorders Colitis	1.7% 1/59 • 2 years
Injury, poisoning and procedural complications Hip fracture	1.7% 1/59 • 2 years
Gastrointestinal disorders Lower gastrointestinal hemorrhage	1.7% 1/59 • 2 years
Gastrointestinal disorders Small intestinal obstruction	1.7% 1/59 • 2 years

Other adverse events

Other adverse events
Measure	Enzalutamide n=59 participants at risk After signing a screening consent, patients archival tissue will be evaluated for degree of AR positivity by AR staining. Patients with no archival tissue available will undergo a biopsy (using the modality deemed most appropriate by the patient's physician) for collection of tumor tissue for AR positivity by IHC. Only AR+ patients, defined as ≥5 % positivity by IHC, will be included. All IHC testing will be performed in the MSKCC Clinical CLIA approved laboratory. All enrolled patients will be treated with enzalutamide 160 mg by mouth QD until progression of disease (POD), unacceptable toxicity or withdrawal from study. All treatments will be administered in the outpatient setting. Enzalutamide: All enrolled patients will be treated with enzalutamide 160mg by mouth QD. Study drugs will be self-administered by patients. A cycle is 28 days.
General disorders Fatigue	23.7% 14/59 • 2 years
Vascular disorders Hot flashes	20.3% 12/59 • 2 years
Gastrointestinal disorders Diarrhea	13.6% 8/59 • 2 years
Vascular disorders Hypertension	13.6% 8/59 • 2 years
Psychiatric disorders Insomnia	13.6% 8/59 • 2 years
Gastrointestinal disorders Nausea	13.6% 8/59 • 2 years
Nervous system disorders Headache	11.9% 7/59 • 2 years
Gastrointestinal disorders Abdominal Pain	10.2% 6/59 • 2 years
Musculoskeletal and connective tissue disorders Myalgia	10.2% 6/59 • 2 years
Blood and lymphatic system disorders Anemia	8.5% 5/59 • 2 years
Metabolism and nutrition disorders Anorexia	8.5% 5/59 • 2 years
Musculoskeletal and connective tissue disorders Arthralgia	8.5% 5/59 • 2 years
Musculoskeletal and connective tissue disorders Back pain	8.5% 5/59 • 2 years
Nervous system disorders Dysgeusia	8.5% 5/59 • 2 years
Skin and subcutaneous tissue disorders Rash maculo-papular	8.5% 5/59 • 2 years
Gastrointestinal disorders Constipation	6.8% 4/59 • 2 years
Nervous system disorders Memory impairment	6.8% 4/59 • 2 years
Skin and subcutaneous tissue disorders Alopecia	5.1% 3/59 • 2 years
Psychiatric disorders Anxiety	5.1% 3/59 • 2 years
Respiratory, thoracic and mediastinal disorders Cough	5.1% 3/59 • 2 years
Skin and subcutaneous tissue disorders Dry skin	5.1% 3/59 • 2 years
General disorders Malaise	5.1% 3/59 • 2 years
Respiratory, thoracic and mediastinal disorders Nasal congestion	5.1% 3/59 • 2 years
Cardiac disorders Palpitations	5.1% 3/59 • 2 years
Nervous system disorders Peripheral sensory neuropathy	5.1% 3/59 • 2 years
Nervous system disorders Dizziness	3.4% 2/59 • 2 years
Respiratory, thoracic and mediastinal disorders Dyspnea	3.4% 2/59 • 2 years
Gastrointestinal disorders Flatulence	3.4% 2/59 • 2 years
Gastrointestinal disorders Gastroesophageal reflux disease	3.4% 2/59 • 2 years
Musculoskeletal and connective tissue disorders Neck pain	3.4% 2/59 • 2 years
Renal and urinary disorders Urinary frequency	3.4% 2/59 • 2 years
Gastrointestinal disorders Vomiting	3.4% 2/59 • 2 years

Additional Information

Dr. Rachel Grisham, MD

Memorial Sloan Kettering Cancer Center

Phone: 646-888-4653

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place