Trial Outcomes & Findings for Management of Atypical Endometrial Hyperplasia and Endometrial Carcinoma Using Megestrol Acetate (NCT NCT00483327)

Last Updated: 2018-04-04

Results Overview

Patients are evaluated every 12 weeks while on treatment. The response is evaluated by endometrial biopsy or dilation and curettage (D\&C)/hysteroscopy. Complete response (CR) is defined as endometrial sampling is read as normal or proliferative endometrium. Partial response (PR) is defined as the biopsy sample has changed on the endometrial evaluation scale by at least one level towards normal. Stable disease (SD) is defined as no change in pathology between the index and follow-up sample. Progressive disease (PD) is defined the follow-up sample has changed towards neoplasia on the endometrial evaluation scale by at least one level or imaging is concerning for myometrial invasion or extrauterine disease such that conservative management is no longer medically appropriate.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

31 participants

Primary outcome timeframe

up to 24 months

Results posted on

2018-04-04

Participant Flow

From May 2007 to April 2012, total 31 patients were recruited to the study from New York University medical center and its affiliated hospitals.

One patient withdrew before the start of the treatment; ony 30 patients started the treatment.

Participant milestones

Participant milestones
Measure	Megestrol Acetate 80 mg (2 tablets) orally at breakfast, 80 mg at dinner for at least 12 weeks and up to 2 years.
Overall Study STARTED	30
Overall Study COMPLETED	20
Overall Study NOT COMPLETED	10

Reasons for withdrawal

Reasons for withdrawal
Measure	Megestrol Acetate 80 mg (2 tablets) orally at breakfast, 80 mg at dinner for at least 12 weeks and up to 2 years.
Overall Study Adverse Event	1
Overall Study Withdrawal by Subject	3
Overall Study Lost to Follow-up	4
Overall Study Patient Non Compliance	2

Baseline Characteristics

Management of Atypical Endometrial Hyperplasia and Endometrial Carcinoma Using Megestrol Acetate

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Megestrol Acetate n=31 Participants 80 mg (2 tablets) orally at breakfast, 80 mg at dinner for at least 12 weeks and up to 2 years.
Age, Customized 18-24 years	1 participants n=99 Participants
Age, Customized 25-34 years	12 participants n=99 Participants
Age, Customized 35-44 years	12 participants n=99 Participants
Age, Customized 45-54 years	4 participants n=99 Participants
Age, Customized 55-64 yeras	2 participants n=99 Participants
Sex: Female, Male Female	31 Participants n=99 Participants
Sex: Female, Male Male	0 Participants n=99 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants
Race (NIH/OMB) Asian	6 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	4 Participants n=99 Participants
Race (NIH/OMB) White	18 Participants n=99 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=99 Participants
Region of Enrollment United States	31 participants n=99 Participants
Histological Diagnosis Atypical endometrial hyperplasia	20 participants n=99 Participants
Histological Diagnosis FIGO Grade 1 endometrioid carcinoma	9 participants n=99 Participants
Histological Diagnosis FIGO Grade 2 endometrioid carcinoma	2 participants n=99 Participants

PRIMARY outcome

Timeframe: up to 24 months

Population: Patient who were able to complete at least one full course (12 weeks) of treatment

Outcome measures

Outcome measures
Measure	Megestrol Acetate n=30 Participants 80 mg (2 tablets) orally at breakfast, 80 mg at dinner for at least 12 weeks and up to 2 years.	Grade 3 80 mg (2 tablets) orally at breakfast, 80 mg at dinner for at least 12 weeks and up to 2 years.
Best Pathologic Responses Pathologic CR	17 participants	—
Best Pathologic Responses Unconfirmed CR	4 participants	—
Best Pathologic Responses PR	6 participants	—
Best Pathologic Responses SD	1 participants	—
Best Pathologic Responses PD	2 participants	—

SECONDARY outcome

Timeframe: up to 36 months

Population: Any patient with at least one dose of treatment.

Patients with adverse events (AEs) which were possibly, probably, or definitely related to the treatment. AEs were evaluated according to Common Terminology Criteria for Adverse Events (CTCAE) 3.

Outcome measures

Outcome measures
Measure	Megestrol Acetate n=30 Participants 80 mg (2 tablets) orally at breakfast, 80 mg at dinner for at least 12 weeks and up to 2 years.	Grade 3 n=30 Participants 80 mg (2 tablets) orally at breakfast, 80 mg at dinner for at least 12 weeks and up to 2 years.
Toxicity and Tolerability Mood alterations	4 participants	0 participants
Toxicity and Tolerability Headache	5 participants	2 participants
Toxicity and Tolerability Thromboembolic event	0 participants	1 participants
Toxicity and Tolerability Carpal tunnel syndrome	1 participants	0 participants
Toxicity and Tolerability Depression	3 participants	0 participants
Toxicity and Tolerability Bloating	4 participants	0 participants
Toxicity and Tolerability Weight gain	9 participants	0 participants
Toxicity and Tolerability Weakness	1 participants	0 participants
Toxicity and Tolerability Vaginal Spotting	6 participants	0 participants
Toxicity and Tolerability Vaginal Pain	1 participants	0 participants
Toxicity and Tolerability Nausea	6 participants	0 participants
Toxicity and Tolerability Insomnia	4 participants	0 participants
Toxicity and Tolerability Fatigue	6 participants	0 participants
Toxicity and Tolerability Abdominal Pain	2 participants	0 participants
Toxicity and Tolerability Constipation	3 participants	0 participants
Toxicity and Tolerability Increased Appetite	4 participants	0 participants

SECONDARY outcome

Timeframe: up to 4 years

Population: The original PI for this study is no longer at our institution. Additionally, co-investigator has stated that this data was not collected and therefore not analyzed. This information is not available for reporting as it does not exist.

For each patient, assessed every 12 weeks during treatment and every 6 months during follow-up.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 3 years after the treatment for each patient

Population: Only 7 participants in the trial pursued pregnancy.

Outcome measures

Outcome measures
Measure	Megestrol Acetate n=7 Participants 80 mg (2 tablets) orally at breakfast, 80 mg at dinner for at least 12 weeks and up to 2 years.	Grade 3 80 mg (2 tablets) orally at breakfast, 80 mg at dinner for at least 12 weeks and up to 2 years.
Number of Women Who Became Pregnant	3 participants	—

Adverse Events

Megestrol Acetate

Serious events: 1 serious events

Other events: 29 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Megestrol Acetate n=30 participants at risk 80 mg (2 tablets) orally at breakfast, 80 mg at dinner for at least 12 weeks and up to 2 years.
Vascular disorders Thrombosis/thrombus/embolism	3.3% 1/30 All the adverse events are reported here regardless of attribution.

Other adverse events

Other adverse events
Measure	Megestrol Acetate n=30 participants at risk 80 mg (2 tablets) orally at breakfast, 80 mg at dinner for at least 12 weeks and up to 2 years.
Immune system disorders Allergic reaction/hypersensitivity (including drug fever)	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Gastrointestinal disorders Anorexia	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Nervous system disorders Confusion	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Gastrointestinal disorders Constipation	10.0% 3/30 All the adverse events are reported here regardless of attribution.
General disorders Constitutional Symptoms - Other: thirst	10.0% 3/30 All the adverse events are reported here regardless of attribution.
Renal and urinary disorders Cystitis	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Skin and subcutaneous tissue disorders Dermatology/Skin - Other: skin peeling	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Gastrointestinal disorders Diarrhea	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Gastrointestinal disorders Distension/bloating, abdominal	16.7% 5/30 All the adverse events are reported here regardless of attribution.
Nervous system disorders Dizziness	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Skin and subcutaneous tissue disorders Dry skin	6.7% 2/30 All the adverse events are reported here regardless of attribution.
Gastrointestinal disorders Dysphagia (difficulty swallowing)	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Respiratory, thoracic and mediastinal disorders Dyspnea (shortness of breath)	13.3% 4/30 All the adverse events are reported here regardless of attribution.
Blood and lymphatic system disorders Edema: head and neck:	3.3% 1/30 All the adverse events are reported here regardless of attribution.
General disorders Fatigue (asthenia, lethargy, malaise)	23.3% 7/30 All the adverse events are reported here regardless of attribution.
General disorders Fever (in the absence of neutropenia, where neutropenia is defined as ANC <1.0 x 10e9/L)	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Gastrointestinal disorders Flatulence	3.3% 1/30 All the adverse events are reported here regardless of attribution.
General disorders Flu-like syndrome	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Skin and subcutaneous tissue disorders Flushing	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Gastrointestinal disorders Gastrointestinal - Other: Increased Appetite	13.3% 4/30 All the adverse events are reported here regardless of attribution.
Metabolism and nutrition disorders Glucose, serum-low (hypoglycemia)	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Reproductive system and breast disorders Hemorrhage, GU: Vagina	56.7% 17/30 All the adverse events are reported here regardless of attribution.
Injury, poisoning and procedural complications Hemorrhage/bleeding associated with surgery, intra-operative or postoperative	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Endocrine disorders Hot flashes/flushes	10.0% 3/30 All the adverse events are reported here regardless of attribution.
Cardiac disorders Hypertension	6.7% 2/30 All the adverse events are reported here regardless of attribution.
Cardiac disorders Hypotension	3.3% 1/30 All the adverse events are reported here regardless of attribution.
General disorders Insomnia	13.3% 4/30 All the adverse events are reported here regardless of attribution.
Reproductive system and breast disorders Irregular menses (change from baseline)	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Reproductive system and breast disorders Libido	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Nervous system disorders Memory impairment	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Nervous system disorders Mood alteration: Agitation	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Nervous system disorders Mood alteration: Anxiety	10.0% 3/30 All the adverse events are reported here regardless of attribution.
Nervous system disorders Mood alteration: Depression	16.7% 5/30 All the adverse events are reported here regardless of attribution.
Musculoskeletal and connective tissue disorders Muscle weakness, generalized or specific area (not due to neuropathy): Whole body/generalized	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Musculoskeletal and connective tissue disorders Musculoskeletal/Soft Tissue - Other: Spasm	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Gastrointestinal disorders Nausea	23.3% 7/30 All the adverse events are reported here regardless of attribution.
Nervous system disorders Neuropathy: cranial: CN V Motor-jaw muscles; Sensory-facial	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Nervous system disorders Neuropathy: sensory	10.0% 3/30 All the adverse events are reported here regardless of attribution.
General disorders Odor (patient odor)	3.3% 1/30 All the adverse events are reported here regardless of attribution.
General disorders Pain - Other: side of body	6.7% 2/30 All the adverse events are reported here regardless of attribution.
Gastrointestinal disorders Pain: Abdomen NOS	23.3% 7/30 All the adverse events are reported here regardless of attribution.
Musculoskeletal and connective tissue disorders Pain: Back	10.0% 3/30 All the adverse events are reported here regardless of attribution.
Respiratory, thoracic and mediastinal disorders Pain: Chest/thorax NOS	6.7% 2/30 All the adverse events are reported here regardless of attribution.
Musculoskeletal and connective tissue disorders Pain: Extremity-limb	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Nervous system disorders Pain: Head/headache	23.3% 7/30 All the adverse events are reported here regardless of attribution.
Musculoskeletal and connective tissue disorders Pain: Muscle	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Nervous system disorders Pain: Neuralgia/peripheral nerve	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Reproductive system and breast disorders Pain: Pelvis	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Respiratory, thoracic and mediastinal disorders Pain: Pleura	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Respiratory, thoracic and mediastinal disorders Pain: Throat/pharynx/larynx	6.7% 2/30 All the adverse events are reported here regardless of attribution.
Reproductive system and breast disorders Pain: Vagina	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Metabolism and nutrition disorders Proteinuria	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Skin and subcutaneous tissue disorders Pruritus/itching	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Skin and subcutaneous tissue disorders Rash/desquamation	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Skin and subcutaneous tissue disorders Rash: acne/acneiform	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Renal and urinary disorders Renal/Genitourinary - Other: Burning With Urination	3.3% 1/30 All the adverse events are reported here regardless of attribution.
General disorders Rigors/chills	3.3% 1/30 All the adverse events are reported here regardless of attribution.
General disorders Sweating (diaphoresis)	6.7% 2/30 All the adverse events are reported here regardless of attribution.
Renal and urinary disorders Urinary frequency/urgency	13.3% 4/30 All the adverse events are reported here regardless of attribution.
Immune system disorders Urticaria (hives, welts, wheals)	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Reproductive system and breast disorders Vaginal discharge (non-infectious)	6.7% 2/30 All the adverse events are reported here regardless of attribution.
Gastrointestinal disorders Vomiting	3.3% 1/30 All the adverse events are reported here regardless of attribution.
General disorders Weight gain	30.0% 9/30 All the adverse events are reported here regardless of attribution.
Skin and subcutaneous tissue disorders Dermatology/Skin - Other: blister	3.3% 1/30 All the adverse events are reported here regardless of attribution.
General disorders Pain: Pain NOS	3.3% 1/30 All the adverse events are reported here regardless of attribution.
Gastrointestinal disorders pain: stomach	3.3% 1/30 All the adverse events are reported here regardless of attribution.

Additional Information

Stephanie Blank, MD

Perlmutter Cancer Center at NYU Langone

Phone: 212-731-5705

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place