Trial Outcomes & Findings for Compassionate Use of Mifepristone in Brain/Nervous System and Other Cancers (NCT NCT00832871)

Results Overview

The time from the date of response (not the beginning of treatment unless there is stable disease) to disease progression. Response and progression are evaluated according to Response Evaluation Criteria in Solid Tumors (RECIST) (version 1.0). Target lesions are assessed by computerized tomography (CT) or magnetic resonance imaging (MRI): Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Stable Disease (SD), neither sufficient decrease in the sum of the longest diameter of target lesions to qualify for PR nor sufficient increase in the sum of the longest diameter of target lesions to qualify for Progressive Disease; Progressive Disease (PD), 20% 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

Recruitment status

TERMINATED

Study phase

NA

Target enrollment

4 participants

Primary outcome timeframe

5 years

Results posted on

2018-06-29

Participant Flow

Participant milestones

Participant milestones
Measure	Mifepristone 200 mg RU-486 (Mifepristone) daily Mifepristone: Mifepristone 200 mg will be administered orally
Overall Study STARTED	4
Overall Study COMPLETED	4
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Compassionate Use of Mifepristone in Brain/Nervous System and Other Cancers

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Mifepristone n=4 Participants 200 mg RU-486 (Mifepristone) daily Mifepristone: Mifepristone 200 mg will be administered orally
Age, Continuous	45 years n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Region of Enrollment United States	4 participants n=5 Participants

PRIMARY outcome

Timeframe: 5 years

Population: Only one patient achieved stable disease. This patient's duration of response could therefore be reported. The other three patients progressed on treatment.

The time from the date of response (not the beginning of treatment unless there is stable disease) to disease progression. Response and progression are evaluated according to Response Evaluation Criteria in Solid Tumors (RECIST) (version 1.0). Target lesions are assessed by computerized tomography (CT) or magnetic resonance imaging (MRI): Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Stable Disease (SD), neither sufficient decrease in the sum of the longest diameter of target lesions to qualify for PR nor sufficient increase in the sum of the longest diameter of target lesions to qualify for Progressive Disease; Progressive Disease (PD), 20% 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

Outcome measures

Outcome measures
Measure	Mifepristone n=1 Participants 200 mg RU-486 (Mifepristone) daily Mifepristone: Mifepristone 200 mg will be administered orally
Duration of Response	44 days Interval 44.0 to 44.0

SECONDARY outcome

Timeframe: Up to 8 weeks after the end of study treatment or until any adverse events are resolved (whichever is longest)

Population: All patients received at least one dose of study medication and are included in this analysis.

Toxicity will be evaluated per National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE), version 3.0. Frequency and severity of adverse events will be tabulated using counts the following events of interest, which are related to possible adrenal insufficiency: nausea, vomiting, lethargy, dizziness, fatigue, anorexia, and skin rash. Any grade of these events that are self-reported by patients as well as events identified by physician assessment (e.g. physical exam) will be included.

Outcome measures

Outcome measures
Measure	Mifepristone n=4 Participants 200 mg RU-486 (Mifepristone) daily Mifepristone: Mifepristone 200 mg will be administered orally
Toxicity Associated With Adrenal Insufficiency Nausea	0 percentage of participants
Toxicity Associated With Adrenal Insufficiency Vomiting	0 percentage of participants
Toxicity Associated With Adrenal Insufficiency Lethargy	0 percentage of participants
Toxicity Associated With Adrenal Insufficiency Dizziness	0 percentage of participants
Toxicity Associated With Adrenal Insufficiency Fatigue	0 percentage of participants
Toxicity Associated With Adrenal Insufficiency Anorexia	0 percentage of participants
Toxicity Associated With Adrenal Insufficiency Skin Rash	0 percentage of participants

OTHER_PRE_SPECIFIED outcome

Timeframe: 5 years

The time from patient entry into the protocol to death by any cause.

Outcome measures

Outcome measures
Measure	Mifepristone n=4 Participants 200 mg RU-486 (Mifepristone) daily Mifepristone: Mifepristone 200 mg will be administered orally
Overall Survival	24.2 Months Standard Deviation 28.5

Adverse Events

Mifepristone

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Mifepristone n=4 participants at risk 200 mg RU-486 (Mifepristone) daily Mifepristone: Mifepristone 200 mg will be administered orally
Blood and lymphatic system disorders Edema: limb	25.0% 1/4 • Number of events 1
Blood and lymphatic system disorders Edema: trunk	25.0% 1/4 • Number of events 1
Nervous system disorders Headache	25.0% 1/4 • Number of events 1
Eye disorders Watery eye	25.0% 1/4 • Number of events 1
Eye disorders Diplopia	25.0% 1/4 • Number of events 1

Additional Information

Dr. Fa-chyi Lee

University of New Mexico Comprehensive Cancer Center

Phone: 505-925-0405

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place