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Trial record 54 of 77 for:    DIPG

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01774253
Recruitment Status : Terminated (Lack of enrollment and commercial availability of drug)
First Posted : January 23, 2013
Results First Posted : October 28, 2016
Last Update Posted : February 28, 2017
Sponsor:
Collaborators:
Spectrum Health Hospitals
Phoenix Children's Hospital
Information provided by (Responsible Party):
Giselle Sholler, Spectrum Health Hospitals

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Pontine Glioma
Intervention Drug: Vismodegib
Enrollment 9
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Vismodegib
Hide Arm/Group Description

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

Period Title: Overall Study
Started 9
Completed 0
Not Completed 9
Reason Not Completed
Lack of Efficacy             4
Physician Decision             2
Withdrawal by Subject             2
Death             1
Arm/Group Title Vismodegib
Hide Arm/Group Description

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 Number of Baseline Participants 9
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
<=18 years
9
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Gender  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
Female
4
  44.4%
Male
5
  55.6%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
Hispanic or Latino
1
  11.1%
Not Hispanic or Latino
7
  77.8%
Unknown or Not Reported
1
  11.1%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
8
  88.9%
More than one race
0
   0.0%
Unknown or Not Reported
1
  11.1%
1.Primary Outcome
Title Number of Days Participants Experienced Progression Free Survival (PFS)
Hide Description 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.
Time Frame 5 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Vismodegib
Hide Arm/Group Description:

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 Number of Participants Analyzed 4
Median (Full Range)
Unit of Measure: Days
60
(20 to 143)
2.Secondary Outcome
Title Number of Participants With Adverse Events as a Measure of Safety and Tolerability
Hide Description To determine the safety and tolerability of Vismodegib as a single agent in pediatric and young adult patients with refractory or recurrent pontine glioma
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Vismodegib
Hide Arm/Group Description:

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 Number of Participants Analyzed 9
Measure Type: Number
Unit of Measure: participants
4
3.Secondary Outcome
Title Determine the Median Overall Survival (OS) of Participants
Hide Description Overall Survival (OS) and clinical benefit (ORR + stable disease, SD)
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
Not evaluated due to early closure. Study data does not exist.
Arm/Group Title Vismodegib
Hide Arm/Group Description:

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 Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
4.Secondary Outcome
Title Evaluate the Impact of Quality of Life of Children Receiving Vismodegib Using PedsQL Questionnaires
Hide Description Evaluate the impact of Quality of Life of children receiving Vismodegib using PedsQL questionnaires
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
QOL's not collected due to early closure of study. Data not collected or analyzed threfore no data exists.
Arm/Group Title Vismodegib
Hide Arm/Group Description:

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 Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
5.Secondary Outcome
Title Determine the Response Rates of Participants Based on Activation (or no Activation) of Their Hedgehog Signaling Pathway
Hide Description 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
Time Frame 3 years
Hide Outcome Measure Data
Hide Analysis Population Description
No samples collected for hedgehog pathway. No data exists.
Arm/Group Title Vismodegib
Hide Arm/Group Description:

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 Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame 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.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Vismodegib
Hide Arm/Group Description

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

All-Cause Mortality
Vismodegib
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Vismodegib
Affected / at Risk (%) # Events
Total   1/9 (11.11%)    
General disorders   
Choking event resulting in death * 1 [1]  1/9 (11.11%)  1
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, CTCAE (4.0)
[1]
Subject choked on a hot dog resulting in death. Unrelated to study.
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Vismodegib
Affected / at Risk (%) # Events
Total   4/9 (44.44%)    
Blood and lymphatic system disorders   
Increased GGT * 1  1/9 (11.11%)  1
Gastrointestinal disorders   
Diarrhea * 1  1/9 (11.11%)  1
Gastoesophageal Reflux Disease * 1  1/9 (11.11%)  1
Vomiting * 1  1/9 (11.11%)  1
General disorders   
Alopecia * 1  1/9 (11.11%)  1
Fatigue * 1  1/9 (11.11%)  1
Nervous system disorders   
Ataxia * 1  1/9 (11.11%)  1
Skin and subcutaneous tissue disorders   
Rash * 1  1/9 (11.11%)  1
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, CTCAE (4.0)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Giselle Sholler, MD
Organization: NMTRC
Phone: 6162670335
EMail: genevieve.bergendahl@helendevoschildrens.org
Layout table for additonal information
Responsible Party: Giselle Sholler, Spectrum Health Hospitals
ClinicalTrials.gov Identifier: NCT01774253     History of Changes
Other Study ID Numbers: NMTRCPG007
First Submitted: January 18, 2013
First Posted: January 23, 2013
Results First Submitted: May 10, 2016
Results First Posted: October 28, 2016
Last Update Posted: February 28, 2017