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Trial Outcomes & Findings for Safety and Efficacy Study of TPI-287 in Neuroblastoma and Medulloblastoma (NCT NCT01483820)

NCT ID: NCT01483820

Last Updated: 2024-08-06

Results Overview

Phase I portion of trial- To determine the safety and tolerability of TPI 287 as a single agent in pediatric and young adult patients with refractory or recurrent neuroblastoma or medulloblastoma. Adverse events collected from time of first dose to 30 days past last dose and until all related events resolved, average of one year.

Recruitment status

TERMINATED

Study phase

PHASE1/PHASE2

Target enrollment

8 participants

Primary outcome timeframe

length of study +30 days

Results posted on

2024-08-06

Participant Flow

Participant milestones

Participant milestones
Measure
TPI 287
Subjects will receive six cycles of intravenous (IV) TPI 287 at a dose of 125 mg/m2 on Days 1, 8 and 15 of a 21-day cycle.
Overall Study
STARTED
8
Overall Study
COMPLETED
0
Overall Study
NOT COMPLETED
8

Reasons for withdrawal

Reasons for withdrawal
Measure
TPI 287
Subjects will receive six cycles of intravenous (IV) TPI 287 at a dose of 125 mg/m2 on Days 1, 8 and 15 of a 21-day cycle.
Overall Study
Lack of Efficacy
6
Overall Study
Adverse Event
1
Overall Study
Death
1

Baseline Characteristics

Safety and Efficacy Study of TPI-287 in Neuroblastoma and Medulloblastoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
TPI 287
n=8 Participants
Subjects will receive six cycles of intravenous (IV) TPI 287 at a dose of 125 mg/m2 on Days 1, 8 and 15 of a 21-day cycle.
Age, Categorical
<=18 years
8 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
0 Participants
n=5 Participants
Age, Categorical
>=65 years
0 Participants
n=5 Participants
Sex: Female, Male
Female
4 Participants
n=5 Participants
Sex: Female, Male
Male
4 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
1 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
7 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
0 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=5 Participants
Race (NIH/OMB)
White
8 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants

PRIMARY outcome

Timeframe: length of study +30 days

Phase I portion of trial- To determine the safety and tolerability of TPI 287 as a single agent in pediatric and young adult patients with refractory or recurrent neuroblastoma or medulloblastoma. Adverse events collected from time of first dose to 30 days past last dose and until all related events resolved, average of one year.

Outcome measures

Outcome measures
Measure
TPI 287
n=8 Participants
Subjects will receive six cycles of intravenous (IV) TPI 287 at a dose of 125 mg/m2 on Days 1, 8 and 15 of a 21-day cycle.
Number of Participants With Adverse Events as a Measure of Safety and Tolerability
6 participants

SECONDARY outcome

Timeframe: 6 months

Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) 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

Outcome measures
Measure
TPI 287
n=6 Participants
Subjects will receive six cycles of intravenous (IV) TPI 287 at a dose of 125 mg/m2 on Days 1, 8 and 15 of a 21-day cycle.
Number of Participants With Overall Response Assessed Using RECIST Criteria
0 participants

SECONDARY outcome

Timeframe: 3 years

Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 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
TPI 287
n=6 Participants
Subjects will receive six cycles of intravenous (IV) TPI 287 at a dose of 125 mg/m2 on Days 1, 8 and 15 of a 21-day cycle.
Number of Days Participants Experienced Progression Free Survival (PFS)
46 Days

SECONDARY outcome

Timeframe: 3 years

Population: Not evaluated due to early closure. Study data does not exist.

Overall Survival (OS) and clinical benefit (ORR + stable disease, SD)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 3 years

Population: QOL's not collected due to early closure of study. Data not collected or analyzed threfore no data exists.

To evaluate the impact of QOL of children receiving TPI287 using PedsQL questionnaires

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Population: PK's not run due to early closure of study. Data not collected or analyzed threfore no data exists.

To evaluate the pharmacokinetics (PK) of TPI 287 in the Phase I population of this trial.

Outcome measures

Outcome data not reported

Adverse Events

TPI 287

Serious events: 2 serious events
Other events: 6 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
TPI 287
n=8 participants at risk
Subjects will receive six cycles of intravenous (IV) TPI 287 at a dose of 125 mg/m2 on Days 1, 8 and 15 of a 21-day cycle.
Nervous system disorders
Seizure
12.5%
1/8 • Number of events 1 • From date of first dose of TPI 287 until 30 days past last dose. Related AE's collected until resolution.
General disorders
Progression of Disease resulting in Death
25.0%
2/8 • Number of events 2 • From date of first dose of TPI 287 until 30 days past last dose. Related AE's collected until resolution.

Other adverse events

Other adverse events
Measure
TPI 287
n=8 participants at risk
Subjects will receive six cycles of intravenous (IV) TPI 287 at a dose of 125 mg/m2 on Days 1, 8 and 15 of a 21-day cycle.
Hepatobiliary disorders
alanine aminotransferase (ALT) increase
25.0%
2/8 • Number of events 2 • From date of first dose of TPI 287 until 30 days past last dose. Related AE's collected until resolution.
Blood and lymphatic system disorders
Anemia
12.5%
1/8 • Number of events 1 • From date of first dose of TPI 287 until 30 days past last dose. Related AE's collected until resolution.
Hepatobiliary disorders
Aspartate aminotransferase (AST) increase
12.5%
1/8 • Number of events 1 • From date of first dose of TPI 287 until 30 days past last dose. Related AE's collected until resolution.
Gastrointestinal disorders
Diarrhea
12.5%
1/8 • Number of events 2 • From date of first dose of TPI 287 until 30 days past last dose. Related AE's collected until resolution.
General disorders
fatigue
12.5%
1/8 • Number of events 1 • From date of first dose of TPI 287 until 30 days past last dose. Related AE's collected until resolution.
Endocrine disorders
Hypoglycemia
12.5%
1/8 • Number of events 2 • From date of first dose of TPI 287 until 30 days past last dose. Related AE's collected until resolution.
Blood and lymphatic system disorders
Hypocalcemia
12.5%
1/8 • Number of events 1 • From date of first dose of TPI 287 until 30 days past last dose. Related AE's collected until resolution.
Blood and lymphatic system disorders
Hypokalemia
12.5%
1/8 • Number of events 1 • From date of first dose of TPI 287 until 30 days past last dose. Related AE's collected until resolution.
Blood and lymphatic system disorders
Hypophosphatemia
12.5%
1/8 • Number of events 1 • From date of first dose of TPI 287 until 30 days past last dose. Related AE's collected until resolution.
General disorders
Infusion related reaction
25.0%
2/8 • Number of events 2 • From date of first dose of TPI 287 until 30 days past last dose. Related AE's collected until resolution.
Blood and lymphatic system disorders
Lymphocytopenia
12.5%
1/8 • Number of events 5 • From date of first dose of TPI 287 until 30 days past last dose. Related AE's collected until resolution.
Blood and lymphatic system disorders
Neutropenia
25.0%
2/8 • Number of events 2 • From date of first dose of TPI 287 until 30 days past last dose. Related AE's collected until resolution.
Blood and lymphatic system disorders
Leukopenia
25.0%
2/8 • Number of events 3 • From date of first dose of TPI 287 until 30 days past last dose. Related AE's collected until resolution.
Nervous system disorders
Nervous system disorders
12.5%
1/8 • Number of events 1 • From date of first dose of TPI 287 until 30 days past last dose. Related AE's collected until resolution.

Additional Information

Giselle Sholler, MD

NMTRC

Phone: 6162670335

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place

Restriction type: LTE60