Trial Outcomes & Findings for Trial of RAD001 and Erlotinib With Recurrent Head and Neck Squamous Cell Carcinoma (NCT NCT00942734)

Last Updated: 2025-05-01

Results Overview

Tumor assessments performed by Computed Tomography (CT) scan or Magnetic Resonance Imaging (MRI) after 4 weeks, 12 weeks, then every 8 weeks thereafter. Participants who received at least one dose of RAD001+erlotinib and who die before 12 weeks, counted as having progressive disease. Response Evaluation Criteria in Solid Tumors (RECIST) defined as Complete Response (CR): Disappearance of all target lesions; Partial Response (PR): \>30% decrease in sum of longest diameter (LD) of target lesions, taking as reference baseline sum LD. Progression-free survival defined as stable disease or better. Progressive Disease (PD): \>20% increase in sum of LD of target lesions, taking as reference smallest sum LD recorded since treatment started or appearance of one or more new lesions; Stable Disease (SD): Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference smallest sum LD since treatment started.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

49 participants

Primary outcome timeframe

12 weeks

Results posted on

2025-05-01

Participant Flow

Recruitment Period: July 17, 2009 to February 08, 2013. Recruitment was done in various medical clinics in the United States.

Of the forty-nine participants registered, six participants were ineligible and not treated.

Participant milestones

Participant milestones
Measure	RAD001 + Erlotinib RAD001 5 mg orally and Erlotinib 150 mg orally every day of each 28 day study cycle.
Overall Study STARTED	43
Overall Study COMPLETED	35
Overall Study NOT COMPLETED	8

Reasons for withdrawal

Reasons for withdrawal
Measure	RAD001 + Erlotinib RAD001 5 mg orally and Erlotinib 150 mg orally every day of each 28 day study cycle.
Overall Study Adverse Event	2
Overall Study Protocol Violation	3
Overall Study Disease Progression	2
Overall Study Physician Decision	1

Baseline Characteristics

Trial of RAD001 and Erlotinib With Recurrent Head and Neck Squamous Cell Carcinoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	RAD001 + Erlotinib n=43 Participants RAD001 5 mg orally and Erlotinib 150 mg orally every day of each 28 day study cycle.
Age, Continuous	61 years n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	35 Participants n=5 Participants
Region of Enrollment United States	43 participants n=5 Participants

PRIMARY outcome

Timeframe: 12 weeks

Population: Eight participants were not evaluable.

Outcome measures

Outcome measures
Measure	RAD001 + Erlotinib n=35 Participants RAD001 5 mg orally and Erlotinib 150 mg orally every day of each 28 day study cycle.
12-Week Progression-Free Survival (PFS)	48.57 Percentage of Participants

Adverse Events

RAD001 + Erlotinib

Serious events: 1 serious events

Other events: 43 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	RAD001 + Erlotinib n=43 participants at risk RAD001 5 mg orally and Erlotinib 150 mg orally every day of each 28 day study cycle.
Metabolism and nutrition disorders hypercalcaemia	2.3% 1/43 • Adverse events collected through 12 weeks of treatment (± 3 days).

Other adverse events

Other adverse events
Measure	RAD001 + Erlotinib n=43 participants at risk RAD001 5 mg orally and Erlotinib 150 mg orally every day of each 28 day study cycle.
Skin and subcutaneous tissue disorders ACNE	62.8% 27/43 • Number of events 27 • Adverse events collected through 12 weeks of treatment (± 3 days).
Gastrointestinal disorders ANOREXIA	7.0% 3/43 • Number of events 3 • Adverse events collected through 12 weeks of treatment (± 3 days).
Metabolism and nutrition disorders CHOLESTEROL	4.7% 2/43 • Number of events 2 • Adverse events collected through 12 weeks of treatment (± 3 days).
Skin and subcutaneous tissue disorders DERMATOLOGY/SKIN	11.6% 5/43 • Number of events 5 • Adverse events collected through 12 weeks of treatment (± 3 days).
Gastrointestinal disorders DIARRHEA	41.9% 18/43 • Number of events 18 • Adverse events collected through 12 weeks of treatment (± 3 days).
General disorders DIZZINESS	4.7% 2/43 • Number of events 2 • Adverse events collected through 12 weeks of treatment (± 3 days).
Gastrointestinal disorders DYSPHAGIA	4.7% 2/43 • Number of events 2 • Adverse events collected through 12 weeks of treatment (± 3 days).
Respiratory, thoracic and mediastinal disorders DYSPNEA	4.7% 2/43 • Number of events 2 • Adverse events collected through 12 weeks of treatment (± 3 days).
General disorders EDEMA: HEAD AND NECK	20.9% 9/43 • Number of events 9 • Adverse events collected through 12 weeks of treatment (± 3 days).
General disorders EDEMA: LIMB	4.7% 2/43 • Number of events 2 • Adverse events collected through 12 weeks of treatment (± 3 days).
General disorders FATIGUE	41.9% 18/43 • Number of events 18 • Adverse events collected through 12 weeks of treatment (± 3 days).
Investigations FEVER WITHOUT NEUTROPINIA	4.7% 2/43 • Number of events 2 • Adverse events collected through 12 weeks of treatment (± 3 days).
Skin and subcutaneous tissue disorders HAND-FOOT SYNDROME	4.7% 2/43 • Number of events 2 • Adverse events collected through 12 weeks of treatment (± 3 days).
Blood and lymphatic system disorders HEMOGLOBIN	11.6% 5/43 • Number of events 5 • Adverse events collected through 12 weeks of treatment (± 3 days).
Metabolism and nutrition disorders HYPERGLYCEMIA	4.7% 2/43 • Number of events 2 • Adverse events collected through 12 weeks of treatment (± 3 days).
Metabolism and nutrition disorders HYPERTRIGLYCERIDEMIA	7.0% 3/43 • Number of events 3 • Adverse events collected through 12 weeks of treatment (± 3 days).
Metabolism and nutrition disorders HYPOKALEMIA	4.7% 2/43 • Number of events 2 • Adverse events collected through 12 weeks of treatment (± 3 days).
Blood and lymphatic system disorders LEUKOCYTES	4.7% 2/43 • Number of events 2 • Adverse events collected through 12 weeks of treatment (± 3 days).
Gastrointestinal disorders MUCOSITIS	41.9% 18/43 • Number of events 18 • Adverse events collected through 12 weeks of treatment (± 3 days).
Skin and subcutaneous tissue disorders NAIL CHANGES	9.3% 4/43 • Number of events 4 • Adverse events collected through 12 weeks of treatment (± 3 days).
Gastrointestinal disorders NAUSEA	20.9% 9/43 • Number of events 9 • Adverse events collected through 12 weeks of treatment (± 3 days).
Gastrointestinal disorders PAIN (ORAL CAVITY)	4.7% 2/43 • Number of events 2 • Adverse events collected through 12 weeks of treatment (± 3 days).
Blood and lymphatic system disorders PLATELETS LEVEL	11.6% 5/43 • Number of events 5 • Adverse events collected through 12 weeks of treatment (± 3 days).
Skin and subcutaneous tissue disorders PRURITUS	9.3% 4/43 • Number of events 4 • Adverse events collected through 12 weeks of treatment (± 3 days).
Skin and subcutaneous tissue disorders RASH/DESQUAMATION	11.6% 5/43 • Number of events 5 • Adverse events collected through 12 weeks of treatment (± 3 days).
Gastrointestinal disorders VOMITING	11.6% 5/43 • Number of events 5 • Adverse events collected through 12 weeks of treatment (± 3 days).
General disorders WEIGHT LOSS	25.6% 11/43 • Number of events 11 • Adverse events collected through 12 weeks of treatment (± 3 days).

Additional Information

Vali Papadimitrakopoulou, MD

University of Texas (UT) MD Anderson Cancer Center

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place