Trial Outcomes & Findings for Depocyt® With Sorafenib in Neoplastic Meningitis (NCT NCT00964743)

Results Overview

Safety and tolerability of sorafenib with DepoCyt. Toxicities were to be reported using tables and descriptive statistics by type and grade. All patients were to be followed up until death.

Recruitment status

TERMINATED

Study phase

NA

Target enrollment

2 participants

Primary outcome timeframe

6 Months

Results posted on

2013-09-09

Participant Flow

Participant milestones

Participant milestones
Measure	Intrathecal DepoCyt and Oral Sorafenib This is a single arm pilot study. Investigators planned to enroll approximately 10 patients to receive concurrent intrathecal DepoCyt and oral Sorafenib. DepoCyt: through a reservoir every 2 weeks for 5 doses, then every 4 weeks for an additional 5 doses (a total of 10 DepoCyt treatments). Oral Sorafenib: at 400 mg twice a day throughout the treatment course until disease progression or death.
Overall Study STARTED	2
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Intrathecal DepoCyt and Oral Sorafenib This is a single arm pilot study. Investigators planned to enroll approximately 10 patients to receive concurrent intrathecal DepoCyt and oral Sorafenib. DepoCyt: through a reservoir every 2 weeks for 5 doses, then every 4 weeks for an additional 5 doses (a total of 10 DepoCyt treatments). Oral Sorafenib: at 400 mg twice a day throughout the treatment course until disease progression or death.
Overall Study Neither patient completed 8 weeks of tre	2

Baseline Characteristics

Depocyt® With Sorafenib in Neoplastic Meningitis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Intrathecal DepoCyt and Oral Sorafenib n=2 Participants This is a single arm pilot study. Investigators planned to enroll approximately 10 patients to receive concurrent intrathecal DepoCyt and oral Sorafenib. DepoCyt: through a reservoir every 2 weeks for 5 doses, then every 4 weeks for an additional 5 doses (a total of 10 DepoCyt treatments). Oral Sorafenib: at 400 mg twice a day throughout the treatment course until disease progression or death.
Age, Categorical <=18 years	0 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=93 Participants
Age, Categorical >=65 years	2 Participants n=93 Participants
Sex: Female, Male Female	1 Participants n=93 Participants
Sex: Female, Male Male	1 Participants n=93 Participants
Region of Enrollment United States	2 participants n=93 Participants

PRIMARY outcome

Timeframe: 6 Months

Population: All participants

Safety and tolerability of sorafenib with DepoCyt. Toxicities were to be reported using tables and descriptive statistics by type and grade. All patients were to be followed up until death.

Outcome measures

Outcome measures
Measure	Intrathecal DepoCyt and Oral Sorafenib n=2 Participants This is a single arm pilot study. Investigators planned to enroll approximately 10 patients to receive concurrent intrathecal DepoCyt and oral Sorafenib. DepoCyt: through a reservoir every 2 weeks for 5 doses, then every 4 weeks for an additional 5 doses (a total of 10 DepoCyt treatments). Oral Sorafenib: at 400 mg twice a day throughout the treatment course until disease progression or death.
Number of Participants With Adverse Events (AEs)	2 participants

SECONDARY outcome

Timeframe: 6 Months

Population: The study closed early due to low accrual of 2 of 10 expected patients. Neither patient completed 8 weeks of treatment as outlined in the protocol. Both patients expired before reaching the 6 month Progression Free Survival endpoint.

Kaplan-Meier analysis of PFS was to be performed and the PFS at 6 months in the study patients were be empirically described. All patients were to be followed up until death.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 6 Months

Population: The study closed early due to low accrual of 2 of 10 expected patients. Neither patient completed 8 weeks of treatment as outlined in the protocol. Both patients expired before reaching the 6 month Progression Free Survival endpoint.

Several secondary endpoints were to be analyzed in a descriptive fashion. All patients were to be followed up until death.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 6 Months

Population: The study closed early due to low accrual of 2 of 10 expected patients. Neither patient completed 8 weeks of treatment as outlined in the protocol. Both patients expired before reaching the 6 month Progression Free Survival endpoint.

CSF sorafenib level was to be measured over time, and the means and standard errors of the sorafenib level were to be plotted at specific sampling time points. CSF sorafenib levels may also have been correlated with patients' PFS, OS, or cytology using descriptive statistical methods (e.g., KM analysis stratified by high vs. low CSF sorafenib levels). The log transformation of lab values were to be employed on the continuous variables whenever necessary.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 6 Months

Population: The study closed early due to low accrual of 2 of 10 expected patients. Neither patient completed 8 weeks of treatment as outlined in the protocol. Both patients expired before reaching the 6 month Progression Free Survival endpoint.

CSF and serum VEGF levels were to be measured over time, and the means and standard errors of the respective VEGF levels were to be plotted at specific sampling time points. The respective VEGF levels may also have been correlated with patients' PFS, OS, or cytology using descriptive statistical methods similarly as mentioned above. The log transformation of lab values were to be employed on the continuous variables whenever necessary.

Outcome measures

Outcome data not reported

Adverse Events

Intrathecal DepoCyt and Oral Sorafenib

Serious events: 2 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Intrathecal DepoCyt and Oral Sorafenib n=2 participants at risk This is a single arm pilot study. Investigators planned to enroll approximately 10 patients to receive concurrent intrathecal DepoCyt and oral Sorafenib. DepoCyt: through a reservoir every 2 weeks for 5 doses, then every 4 weeks for an additional 5 doses (a total of 10 DepoCyt treatments). Oral Sorafenib: at 400 mg twice a day throughout the treatment course until disease progression or death.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Death not associated with CTCAE term - Death not otherwise specified (NOS)	50.0% 1/2 • Number of events 1 • 8 months First On Study (9/10/09) to last Off Study (5/18/10)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Death not associated with CTCAE term - Disease progression not otherwise specified (NOS)	50.0% 1/2 • Number of events 1 • 8 months First On Study (9/10/09) to last Off Study (5/18/10)

Other adverse events

Other adverse events
Measure	Intrathecal DepoCyt and Oral Sorafenib n=2 participants at risk This is a single arm pilot study. Investigators planned to enroll approximately 10 patients to receive concurrent intrathecal DepoCyt and oral Sorafenib. DepoCyt: through a reservoir every 2 weeks for 5 doses, then every 4 weeks for an additional 5 doses (a total of 10 DepoCyt treatments). Oral Sorafenib: at 400 mg twice a day throughout the treatment course until disease progression or death.
Blood and lymphatic system disorders Hemoglobin	50.0% 1/2 • Number of events 1 • 8 months First On Study (9/10/09) to last Off Study (5/18/10)
Cardiac disorders Hypotension	50.0% 1/2 • Number of events 1 • 8 months First On Study (9/10/09) to last Off Study (5/18/10)
General disorders Fever (in the absence of neutropenia, where neutropenia is defined as ANC <1.0 x 10^9/L	50.0% 1/2 • Number of events 1 • 8 months First On Study (9/10/09) to last Off Study (5/18/10)
Skin and subcutaneous tissue disorders Ulceration	50.0% 1/2 • Number of events 1 • 8 months First On Study (9/10/09) to last Off Study (5/18/10)
Gastrointestinal disorders Anorexia	50.0% 1/2 • Number of events 1 • 8 months First On Study (9/10/09) to last Off Study (5/18/10)
Gastrointestinal disorders Dehydration	50.0% 1/2 • Number of events 1 • 8 months First On Study (9/10/09) to last Off Study (5/18/10)
Infections and infestations Infection with unknown ANC-Mucosa	50.0% 1/2 • Number of events 1 • 8 months First On Study (9/10/09) to last Off Study (5/18/10)
Blood and lymphatic system disorders Edema: limb	50.0% 1/2 • Number of events 1 • 8 months First On Study (9/10/09) to last Off Study (5/18/10)
Nervous system disorders Somnolence/depressed level of consciousness	50.0% 1/2 • Number of events 1 • 8 months First On Study (9/10/09) to last Off Study (5/18/10)
Nervous system disorders Cognitive disturbance	50.0% 1/2 • Number of events 1 • 8 months First On Study (9/10/09) to last Off Study (5/18/10)

Additional Information

Edward Pan, M.D.

H. Lee Moffitt Cancer Center and Research Institute

Phone: 813-745-3871

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place