Trial Outcomes & Findings for Letrozole and RAD001 With Advanced or Recurrent Endometrial Cancer (NCT NCT01068249)
NCT ID: NCT01068249
Last Updated: 2025-11-18
Results Overview
Objective response or stable disease rate monitored as patients accrue and are evaluated following the example of Thall and Simon. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) 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
ACTIVE_NOT_RECRUITING
Study phase
PHASE2
Target enrollment
42 participants
Primary outcome timeframe
at 8 weeks of treatment, then every 12 weeks, up to 2 years
Results posted on
2025-11-18
Participant Flow
All recruitments were done in a medical clinic setting.
42 participants signed the consent, 3 participants were screen failures
Participant milestones
| Measure |
RAD001 + Letrozole
RAD001 10 mg oral tablet daily + Letrozole 2.5 oral tablet daily for 28 days
|
|---|---|
|
Overall Study
STARTED
|
39
|
|
Overall Study
COMPLETED
|
35
|
|
Overall Study
NOT COMPLETED
|
4
|
Reasons for withdrawal
| Measure |
RAD001 + Letrozole
RAD001 10 mg oral tablet daily + Letrozole 2.5 oral tablet daily for 28 days
|
|---|---|
|
Overall Study
Death
|
1
|
|
Overall Study
Withdrawal by Subject
|
3
|
Baseline Characteristics
Letrozole and RAD001 With Advanced or Recurrent Endometrial Cancer
Baseline characteristics by cohort
| Measure |
RAD001 + Letrozole
n=42 Participants
RAD001 10 mg oral tablet daily + Letrozole 2.5 oral tablet daily for 28 days
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=202 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
27 Participants
n=202 Participants
|
|
Age, Categorical
>=65 years
|
15 Participants
n=202 Participants
|
|
Age, Continuous
|
59.9 years
n=202 Participants
|
|
Sex: Female, Male
Female
|
42 Participants
n=202 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=202 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
4 Participants
n=202 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
37 Participants
n=202 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=202 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=202 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=202 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=202 Participants
|
|
Race (NIH/OMB)
Black or African American
|
4 Participants
n=202 Participants
|
|
Race (NIH/OMB)
White
|
36 Participants
n=202 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=202 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=202 Participants
|
|
Region of Enrollment
United States
|
42 participants
n=202 Participants
|
PRIMARY outcome
Timeframe: at 8 weeks of treatment, then every 12 weeks, up to 2 yearsObjective response or stable disease rate monitored as patients accrue and are evaluated following the example of Thall and Simon. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) 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
Outcome measures
| Measure |
RAD001 + Letrozole
n=35 Participants
RAD001 10 mg oral tablet daily + Letrozole 2.5 oral tablet daily for 28 days
|
|---|---|
|
Number of Participants With Objective Response Rate
Complete Response (CR)
|
9 Participants
|
|
Number of Participants With Objective Response Rate
Partial Response (PR)
|
2 Participants
|
SECONDARY outcome
Timeframe: from study entry (1st treatment) to date of tumor progression, date of death, or, for patients alive without tumor progression, date of last follow-up, assessed up to 2 yearsPFS is the time from first dose of study treatment until the first date of either objective disease progression or death due to any cause. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1), as a 30% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions. Median PFS was estimated using the Kaplan-Meier method.
Outcome measures
| Measure |
RAD001 + Letrozole
n=35 Participants
RAD001 10 mg oral tablet daily + Letrozole 2.5 oral tablet daily for 28 days
|
|---|---|
|
Median Progression Free Survival (PFS)
|
3 months
Interval 1.9 to 15.7
|
SECONDARY outcome
Timeframe: from (1st treatment) to death or, for living patients, date of last contact, up to 24.4 monthsOS is defined as the interval from first dose of study treatment to the date of death, irrespective of the cause of death; subjects still alive will be censored at the date of the last contact
Outcome measures
| Measure |
RAD001 + Letrozole
n=35 Participants
RAD001 10 mg oral tablet daily + Letrozole 2.5 oral tablet daily for 28 days
|
|---|---|
|
Median Overall Survival (OS)
|
14 months
Interval 9.5 to 24.4
|
SECONDARY outcome
Timeframe: from study entry (1st treatment) to date of tumor progression, date of death, or, for patients alive without tumor progression, date of last follow-up, up to 2 yearsOutcome measures
| Measure |
RAD001 + Letrozole
n=35 Participants
RAD001 10 mg oral tablet daily + Letrozole 2.5 oral tablet daily for 28 days
|
|---|---|
|
Number of Participants With Disease Progression
|
31 Participants
|
SECONDARY outcome
Timeframe: Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatmentToxicity was assessed using the National Cancer Institute Common Terminology Criteria for Adverse Events (version 3.0).
Outcome measures
| Measure |
RAD001 + Letrozole
n=35 Participants
RAD001 10 mg oral tablet daily + Letrozole 2.5 oral tablet daily for 28 days
|
|---|---|
|
Number of Participants With Adverse Events (All Grades)
|
28 Participants
|
Adverse Events
RAD001 + Letrozole
Serious events: 0 serious events
Other events: 28 other events
Deaths: 1 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
RAD001 + Letrozole
n=35 participants at risk
RAD001 10 mg oral tablet daily + Letrozole 2.5 oral tablet daily for 28 days
|
|---|---|
|
Nervous system disorders
Neuropathy- sensory
|
34.3%
12/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Skin and subcutaneous tissue disorders
Rash: acneiform
|
40.0%
14/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
22.9%
8/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
General disorders
Fatigue
|
80.0%
28/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Metabolism and nutrition disorders
Hypertriglyceridemia
|
80.0%
28/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Metabolism and nutrition disorders
Hypercholesterolemia
|
77.1%
27/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Gastrointestinal disorders
Mucositis
|
71.4%
25/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Blood and lymphatic system disorders
Anemia
|
65.7%
23/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
60.0%
21/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Gastrointestinal disorders
Nausea
|
57.1%
20/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Gastrointestinal disorders
Vomiting
|
28.6%
10/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Gastrointestinal disorders
Anorexia
|
45.7%
16/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
31.4%
11/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Investigations
Elevated AST
|
5.7%
2/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Investigations
Elevated ALT
|
40.0%
14/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Investigations
Hypomagnesemia
|
37.1%
13/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Investigations
Hypokalemia
|
31.4%
11/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Gastrointestinal disorders
Diarrhea
|
34.3%
12/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
37.1%
13/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Blood and lymphatic system disorders
Edema: limbs
|
31.4%
11/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Nervous system disorders
Headache
|
42.9%
15/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Blood and lymphatic system disorders
Leukopenia
|
37.1%
13/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
25.7%
9/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Gastrointestinal disorders
Taste alteration
|
25.7%
9/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Metabolism and nutrition disorders
Weight loss
|
22.9%
8/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Gastrointestinal disorders
Abdominal pain
|
14.3%
5/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
General disorders
Chills
|
14.3%
5/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Nervous system disorders
Dizziness
|
14.3%
5/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Skin and subcutaneous tissue disorders
Dry Skin
|
17.1%
6/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
General disorders
Fever
|
14.3%
5/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Respiratory, thoracic and mediastinal disorders
Hemorrhage - nasal
|
14.3%
5/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
General disorders
Insomnia
|
14.3%
5/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
14.3%
5/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Skin and subcutaneous tissue disorders
Nail Disorder
|
17.1%
6/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Blood and lymphatic system disorders
Neutrophils decreased
|
14.3%
5/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Musculoskeletal and connective tissue disorders
Pain - extremities
|
20.0%
7/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Musculoskeletal and connective tissue disorders
Pain - other
|
14.3%
5/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
General disorders
Sweating
|
20.0%
7/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Infections and infestations
Urinary Tract infection
|
14.3%
5/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Gastrointestinal disorders
Dry mouth
|
14.3%
5/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Investigations
Alkaline phosphatase increased
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Psychiatric disorders
Anxiety
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Ear and labyrinth disorders
Auditory/ear (other)
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Hepatobiliary disorders
Bladder pain
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Blood and lymphatic system disorders
Blood/bone marrow (other)
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Injury, poisoning and procedural complications
Bruising
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Cardiac disorders
Cardiac arrhythmia (other)
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Psychiatric disorders
Confusion
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Metabolism and nutrition disorders
Dehydration
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Skin and subcutaneous tissue disorders
Dermatology/skin (other)
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Gastrointestinal disorders
Dysphagia
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Endocrine disorders
Endocrine (other)
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Gastrointestinal disorders
Enteritis
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Gastrointestinal disorders
GI (other)
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Infections and infestations
Infection
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Infections and infestations
Infection (other)
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Musculoskeletal and connective tissue disorders
Joint pain
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Respiratory, thoracic and mediastinal disorders
Paranasal sinus infection
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngolaryngeal pain
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary hemorrhage
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Metabolism and nutrition disorders
Serum glucose decreased
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Metabolism and nutrition disorders
Serum potassium increased
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Cardiac disorders
Sinus tachycardia
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Infections and infestations
Sinusitis
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Injury, poisoning and procedural complications
Stomal ulcer
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
|
Eye disorders
Watering eyes
|
2.9%
1/35 • Adverse events were reported from first dose of study treatment until end of study treatment at 28 days plus 30 days post treatment, assessed up to 24.4 months
|
Additional Information
Dr. Pamela Soliman,Professor, Gyn Onc & Reproductive Med
UT MD Anderson Cancer Center
Phone: (713) 745-2352
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place