Trial Outcomes & Findings for Topotecan by Intracerebral Clysis for Recurrent Primary Malignant Brain Tumors (NCT NCT00308165)
NCT ID: NCT00308165
Last Updated: 2025-06-27
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
16 participants
Primary outcome timeframe
During treatment, up to 5 Days
Results posted on
2025-06-27
Participant Flow
Participant milestones
| Measure |
Topotecan 0.02 mg/mL
Cohort 1: Patients received 0.02 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.04 mg/mL
Cohort 2: Patients received 0.04 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.0667 mg/mL
Cohort 3: Patients received 0.0667 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.1 mg/mL
Cohort 4: Patients received 0.1 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.133 mg/mL
Cohort 5: Patients received 0.133 mg/mL infusion of topotecan through convection-enhanced delivery.
|
|---|---|---|---|---|---|
|
Dose Level 1: 0.02 mg/mL
STARTED
|
3
|
0
|
0
|
0
|
0
|
|
Dose Level 1: 0.02 mg/mL
COMPLETED
|
3
|
0
|
0
|
0
|
0
|
|
Dose Level 1: 0.02 mg/mL
NOT COMPLETED
|
0
|
0
|
0
|
0
|
0
|
|
Dose Level 2: 0.04 mg/mL
STARTED
|
0
|
3
|
0
|
0
|
0
|
|
Dose Level 2: 0.04 mg/mL
COMPLETED
|
0
|
3
|
0
|
0
|
0
|
|
Dose Level 2: 0.04 mg/mL
NOT COMPLETED
|
0
|
0
|
0
|
0
|
0
|
|
Dose Level 3: 0.0667 mg/mL
STARTED
|
0
|
0
|
3
|
0
|
0
|
|
Dose Level 3: 0.0667 mg/mL
COMPLETED
|
0
|
0
|
3
|
0
|
0
|
|
Dose Level 3: 0.0667 mg/mL
NOT COMPLETED
|
0
|
0
|
0
|
0
|
0
|
|
Dose Level 4: 0.1 mg/mL
STARTED
|
0
|
0
|
0
|
3
|
0
|
|
Dose Level 4: 0.1 mg/mL
COMPLETED
|
0
|
0
|
0
|
3
|
0
|
|
Dose Level 4: 0.1 mg/mL
NOT COMPLETED
|
0
|
0
|
0
|
0
|
0
|
|
Dose Level 5: 0.133 mg/mL
STARTED
|
0
|
0
|
0
|
0
|
4
|
|
Dose Level 5: 0.133 mg/mL
COMPLETED
|
0
|
0
|
0
|
0
|
4
|
|
Dose Level 5: 0.133 mg/mL
NOT COMPLETED
|
0
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Topotecan by Intracerebral Clysis for Recurrent Primary Malignant Brain Tumors
Baseline characteristics by cohort
| Measure |
Topotecan 0.02 mg/mL
n=3 Participants
Cohort 1: Patients received 0.02 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.04 mg/mL
n=3 Participants
Cohort 2: Patients received 0.04 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.0667 mg/mL
n=3 Participants
Cohort 3: Patients received 0.0667 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.1 mg/mL
n=3 Participants
Cohort 4: Patients received 0.1 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.133 mg/mL
n=4 Participants
Cohort 5: Patients received 0.133 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Total
n=16 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
3 Participants
n=21 Participants
|
13 Participants
n=8 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
3 Participants
n=8 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
5 Participants
n=8 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
11 Participants
n=8 Participants
|
PRIMARY outcome
Timeframe: During treatment, up to 5 DaysOutcome measures
| Measure |
Topotecan 0.02 mg/mL
n=3 Participants
Cohort 1: Patients received 0.02 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.04 mg/mL
n=3 Participants
Cohort 2: Patients received 0.04 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.0667 mg/mL
n=3 Participants
Cohort 3: Patients received 0.0667 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.1 mg/mL
n=3 Participants
Cohort 4: Patients received 0.1 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.133 mg/mL
n=4 Participants
Cohort 5: Patients received 0.133 mg/mL infusion of topotecan through convection-enhanced delivery.
|
|---|---|---|---|---|---|
|
Number of Participants With Dose Limiting Toxicities
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
2 Participants
|
PRIMARY outcome
Timeframe: During treatment, up to 5 DaysIf 2 participants within a cohort develop dose-limiting toxicity (DLT), the prior dose level is considered the maximum tolerated dose (MTD). Dose-Limiting Toxicities (DLT) will be defined as any new (or increased from baseline) treatment-related (drug and/or device) neurological deficits as exhibited on neurological examination with severity of grade 3 or higher.
Outcome measures
| Measure |
Topotecan 0.02 mg/mL
n=16 Participants
Cohort 1: Patients received 0.02 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.04 mg/mL
Cohort 2: Patients received 0.04 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.0667 mg/mL
Cohort 3: Patients received 0.0667 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.1 mg/mL
Cohort 4: Patients received 0.1 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.133 mg/mL
Cohort 5: Patients received 0.133 mg/mL infusion of topotecan through convection-enhanced delivery.
|
|---|---|---|---|---|---|
|
Maximum Tolerated Dose (MTD)
|
0.1 mg/ml
|
—
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Treatment to progression, Up to 8 yearsPopulation: 1 participant in Cohort 1 died before progression due to disease complications unrelated to treatment. 2 participants in Cohort 5 had not reached progression. These participants were excluded from the analysis.
Time to tumor progression/recurrence in weeks.
Outcome measures
| Measure |
Topotecan 0.02 mg/mL
n=2 Participants
Cohort 1: Patients received 0.02 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.04 mg/mL
n=3 Participants
Cohort 2: Patients received 0.04 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.0667 mg/mL
n=3 Participants
Cohort 3: Patients received 0.0667 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.1 mg/mL
n=3 Participants
Cohort 4: Patients received 0.1 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.133 mg/mL
n=2 Participants
Cohort 5: Patients received 0.133 mg/mL infusion of topotecan through convection-enhanced delivery.
|
|---|---|---|---|---|---|
|
Time to Tumor Progression/Recurrence
|
70.5 Weeks
Standard Deviation 74.2
|
6.7 Weeks
Standard Deviation 2.3
|
31.7 Weeks
Standard Deviation 6.4
|
36.7 Weeks
Standard Deviation 32.5
|
15.0 Weeks
Standard Deviation 11.3
|
SECONDARY outcome
Timeframe: Treatment to Time of Death, Up to 8 YearsPopulation: Death had not occurred in 1 participant in Cohort 1, 1 participant in Cohort 4, and 2 participants in Cohort 5. These participants were excluded from the analysis.
Time to death measured in weeks
Outcome measures
| Measure |
Topotecan 0.02 mg/mL
n=2 Participants
Cohort 1: Patients received 0.02 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.04 mg/mL
n=3 Participants
Cohort 2: Patients received 0.04 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.0667 mg/mL
n=3 Participants
Cohort 3: Patients received 0.0667 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.1 mg/mL
n=2 Participants
Cohort 4: Patients received 0.1 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.133 mg/mL
n=2 Participants
Cohort 5: Patients received 0.133 mg/mL infusion of topotecan through convection-enhanced delivery.
|
|---|---|---|---|---|---|
|
Time to Death
|
80 weeks
Standard Deviation 101.8
|
46 weeks
Standard Deviation 33.5
|
54 weeks
Standard Deviation 19.2
|
35.7 weeks
Standard Deviation 47.8
|
27.3 weeks
Standard Deviation 28.6
|
Adverse Events
Topotecan 0.02 mg/mL
Serious events: 0 serious events
Other events: 0 other events
Deaths: 2 deaths
Topotecan 0.04 mg/mL
Serious events: 0 serious events
Other events: 0 other events
Deaths: 3 deaths
Topotecan 0.0667 mg/mL
Serious events: 0 serious events
Other events: 0 other events
Deaths: 3 deaths
Topotecan 0.1 mg/mL
Serious events: 0 serious events
Other events: 0 other events
Deaths: 2 deaths
Topotecan 0.133 mg/mL
Serious events: 2 serious events
Other events: 0 other events
Deaths: 2 deaths
Serious adverse events
| Measure |
Topotecan 0.02 mg/mL
n=3 participants at risk
Cohort 1: Patients received 0.02 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.04 mg/mL
n=3 participants at risk
Cohort 2: Patients received 0.04 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.0667 mg/mL
n=3 participants at risk
Cohort 3: Patients received 0.0667 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.1 mg/mL
n=3 participants at risk
Cohort 4: Patients received 0.1 mg/mL infusion of topotecan through convection-enhanced delivery.
|
Topotecan 0.133 mg/mL
n=4 participants at risk
Cohort 5: Patients received 0.133 mg/mL infusion of topotecan through convection-enhanced delivery.
|
|---|---|---|---|---|---|
|
Nervous system disorders
Parietal syndrome
|
0.00%
0/3
|
0.00%
0/3
|
0.00%
0/3
|
0.00%
0/3
|
25.0%
1/4
|
|
Nervous system disorders
Upper extremity weakness
|
0.00%
0/3
|
0.00%
0/3
|
0.00%
0/3
|
0.00%
0/3
|
25.0%
1/4
|
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place