Trial Outcomes & Findings for Erivedge (Vismodegib) in the Treatment of Pediatric Patients With Refractory Pontine Glioma (NCT NCT01774253)

Last Updated: 2024-08-06

Results Overview

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 the appearance of new lesions.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

9 participants

Primary outcome timeframe

5 years

Results posted on

2024-08-06

Participant Flow

Participant milestones

Participant milestones
Measure	Vismodegib Vismodegib will be dosed at 150mg-300mg orally (max dose: 300mg) once a day on days 1 to 28 of a 28-day cycle. In the absence of unacceptable toxicity or disease progression, treatment may continue for as long as tolerated. Vismodegib
Overall Study STARTED	9
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	9

Reasons for withdrawal

Reasons for withdrawal
Measure	Vismodegib Vismodegib will be dosed at 150mg-300mg orally (max dose: 300mg) once a day on days 1 to 28 of a 28-day cycle. In the absence of unacceptable toxicity or disease progression, treatment may continue for as long as tolerated. Vismodegib
Overall Study Lack of Efficacy	4
Overall Study Physician Decision	2
Overall Study Withdrawal by Subject	2
Overall Study Death	1

Baseline Characteristics

Erivedge (Vismodegib) in the Treatment of Pediatric Patients With Refractory Pontine Glioma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Vismodegib n=9 Participants Vismodegib will be dosed at 150mg-300mg orally (max dose: 300mg) once a day on days 1 to 28 of a 28-day cycle. In the absence of unacceptable toxicity or disease progression, treatment may continue for as long as tolerated. Vismodegib
Age, Categorical <=18 years	9 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	5 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	1 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	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: 5 years

Outcome measures

Outcome measures
Measure	Vismodegib n=4 Participants Vismodegib will be dosed at 150mg-300mg orally (max dose: 300mg) once a day on days 1 to 28 of a 28-day cycle. In the absence of unacceptable toxicity or disease progression, treatment may continue for as long as tolerated. Vismodegib
Number of Days Participants Experienced Progression Free Survival (PFS)	60 Days Interval 20.0 to 143.0

SECONDARY outcome

Timeframe: 2 years

To determine the safety and tolerability of Vismodegib as a single agent in pediatric and young adult patients with refractory or recurrent pontine glioma

Outcome measures

Outcome measures
Measure	Vismodegib n=9 Participants Vismodegib will be dosed at 150mg-300mg orally (max dose: 300mg) once a day on days 1 to 28 of a 28-day cycle. In the absence of unacceptable toxicity or disease progression, treatment may continue for as long as tolerated. Vismodegib
Number of Participants With Adverse Events as a Measure of Safety and Tolerability	4 participants

SECONDARY outcome

Timeframe: 2 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: 2 years

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

Evaluate the impact of Quality of Life of children receiving Vismodegib using PedsQL questionnaires

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 3 years

Population: No samples collected for hedgehog pathway. No data exists.

To determine the objective response rates (partial and complete response) for patients without and with evidence of activation of Hedgehog signaling pathway in their tumors

Outcome measures

Outcome data not reported

Adverse Events

Vismodegib

Serious events: 1 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Vismodegib n=9 participants at risk Vismodegib will be dosed at 150mg-300mg orally (max dose: 300mg) once a day on days 1 to 28 of a 28-day cycle. In the absence of unacceptable toxicity or disease progression, treatment may continue for as long as tolerated. Vismodegib
General disorders Choking event resulting in death	11.1% 1/9 • Number of events 1 • New adverse events collected from first dose of study drug to 30 days past last dose of study drug. Related adverse events followed until resolution.

Other adverse events

Other adverse events
Measure	Vismodegib n=9 participants at risk Vismodegib will be dosed at 150mg-300mg orally (max dose: 300mg) once a day on days 1 to 28 of a 28-day cycle. In the absence of unacceptable toxicity or disease progression, treatment may continue for as long as tolerated. Vismodegib
General disorders Alopecia	11.1% 1/9 • Number of events 1 • New adverse events collected from first dose of study drug to 30 days past last dose of study drug. Related adverse events followed until resolution.
Nervous system disorders Ataxia	11.1% 1/9 • Number of events 1 • New adverse events collected from first dose of study drug to 30 days past last dose of study drug. Related adverse events followed until resolution.
Gastrointestinal disorders Diarrhea	11.1% 1/9 • Number of events 1 • New adverse events collected from first dose of study drug to 30 days past last dose of study drug. Related adverse events followed until resolution.
General disorders Fatigue	11.1% 1/9 • Number of events 1 • New adverse events collected from first dose of study drug to 30 days past last dose of study drug. Related adverse events followed until resolution.
Gastrointestinal disorders Gastoesophageal Reflux Disease	11.1% 1/9 • Number of events 1 • New adverse events collected from first dose of study drug to 30 days past last dose of study drug. Related adverse events followed until resolution.
Blood and lymphatic system disorders Increased GGT	11.1% 1/9 • Number of events 1 • New adverse events collected from first dose of study drug to 30 days past last dose of study drug. Related adverse events followed until resolution.
Skin and subcutaneous tissue disorders Rash	11.1% 1/9 • Number of events 1 • New adverse events collected from first dose of study drug to 30 days past last dose of study drug. Related adverse events followed until resolution.
Gastrointestinal disorders Vomiting	11.1% 1/9 • Number of events 1 • New adverse events collected from first dose of study drug to 30 days past last dose of study drug. Related adverse events followed until resolution.

Additional Information

Giselle Sholler, MD

NMTRC

Phone: 6162670335

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place