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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 : April 22, 2022
Phoenix Children's Hospital
Information provided by (Responsible Party):
Wake Forest University Health Sciences

Brief Summary:

The purpose of this research study is to evaluate an investigational drug (Vismodegib) for Pontine Glioma that is growing or has come back (reoccurred). This study will look at the tumors response to the study drug, Vismodegib, and will also look at the safety and tolerability of Vismodegib.

Vismodegib has been tested in multiple adult clinical trials and one pediatric trial. Laboratory testing in pontine gliomas suggests that this drug may be effective in treating this disease.

Condition or disease Intervention/treatment Phase
Pontine Glioma Drug: Vismodegib Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 9 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II, Open-label, Multi-center Study of Erivedge (Vismodegib) in the Treatment of Pediatric Patients With Refractory Pontine Glioma.
Study Start Date : May 2013
Actual Primary Completion Date : October 2015
Actual Study Completion Date : October 2015

Resource links provided by the National Library of Medicine

Drug Information available for: Vismodegib

Arm Intervention/treatment
Experimental: 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.
Drug: Vismodegib
Other Name: Erivedge

Primary Outcome Measures :
  1. Number of Days Participants Experienced Progression Free Survival (PFS) [ Time Frame: 5 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 the appearance of new lesions.

Secondary Outcome Measures :
  1. Number of Participants With Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 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

  2. Determine the Median Overall Survival (OS) of Participants [ Time Frame: 2 years ]
    Overall Survival (OS) and clinical benefit (ORR + stable disease, SD)

  3. Evaluate the Impact of Quality of Life of Children Receiving Vismodegib Using PedsQL Questionnaires [ Time Frame: 2 years ]
    Evaluate the impact of Quality of Life of children receiving Vismodegib using PedsQL questionnaires

  4. Determine the Response Rates of Participants Based on Activation (or no Activation) of Their Hedgehog Signaling Pathway [ Time Frame: 3 years ]
    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

Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Subjects must have radiographically proven diffuse intrinsic pontine glioma and confirmation of residual disease after initial therapy or at the time of recurrence/progression as confirmed by MRI of the brain
  • Subjects must be age ≥3 years and ≤ 18 years
  • Diffuse intrinsic pontine glioma with measurable disease after receiving radiotherapy either concurrent with or followed by ≤ 2 prior courses of chemotherapy
  • Measurable disease as defined by:

Measurable tumor >10mm by MRI

  • Karnofsky performance status (PS) 60-100% (for patients > 16 years of age) OR Lansky PS 60-100% (for patients ≤ 16 years of age)
  • Body surface area > 0.67 m2 and ≤ 2.21 m2
  • Life expectancy of at least 2 months
  • A negative urine pregnancy test is required for female participants of child bearing potential (≥13 years of age or after onset of menses)
  • Acceptable liver function as defined by:

    1. Bilirubin ≤ 1.5 times upper limit of normal
    2. AST (SGOT), ALT (SGPT) and Alkaline phosphatase ≤ 2.5 times upper limit of normal
  • Creatinine clearance or radioisotope GFR ≥ 70 mL/min OR serum creatinine based on age as follows:

    • 0.8 mg/dL (for patients ≤ 5 years of age)
    • 1.0 mg/dL (for patients 6 to 10 years of age)
    • 1.2 mg/dL (for patients 11 to 15 years of age)
    • 1.5 mg/dL (for patients > 15 years of age)
  • Acceptable hematologic status as defined by:

    1. Granulocyte ≥ 1500 cells/mm3
    2. Platelet count ≥ 100,000 (plt/mm3)
    3. Serum albumin ≥ 2.5 g/dL
  • Urinalysis:

    a. No clinically significant abnormalities

  • Acceptable coagulation status as defined by:

    1. PT/INR less than 1.5
    2. PTT within normal limits
  • Subjects must be able to swallow and retain oral medication
  • Female post-pubertal study subjects need to agree to use one of the more effective birth control methods during treatment and for 7 (seven) months after treatment is stopped. These methods include total abstinence (no sex), oral contraceptives ("the pill"), an intrauterine device (IUD), levonorgestrol implants (Norplant), or medroxyprogesterone acetate injections (Depo-provera shots).
  • Male post-pubertal study subjects need to agree to use condoms with spermicide, even after a vasectomy, during sexual intercourse with female partners while being treated with Erivedge capsule and for 2 months after the last dose to avoid exposing an embryo or fetus to Vismodegib.
  • Voluntarily signed and dated a written IRB-approved informed consent by parent or legal guardian of subject

Exclusion Criteria:

  • Concurrent anti-cancer therapy (chemotherapy, radiation therapy, surgery, immunotherapy, hormonal therapy, biologic therapy) other than the ones specified in the protocol. Patients must have discontinued the above cancer therapies for generally about 3 weeks (8 weeks for radiotherapy) prior to the first dose of study medication, as well as recovered from toxicity (to ≤ than grade 2 except for alopecia) induced by previous treatments.
  • Currently receiving another investigational medicinal product.
  • Uncontrolled concurrent illness including, but not limited to:

    1. Active, uncontrolled bacterial, viral, or fungal infections requiring systemic therapy
    2. Diarrhea of any cause ≥ CTCAE grade 2
    3. Psychiatric illness/social situations that would compromise patient safety or limit compliance with study requirements including maintenance of a compliance/pill diary
    4. Any kind of malabsorption syndrome significantly affecting gastrointestinal function
  • Pregnant or nursing female patients. NOTE: If a female patient becomes pregnant or suspects that she is pregnant while participating in this study, she should stop taking study drug and immediately inform her treating physician immediately.
  • Prior therapy with a Hedgehog inhibitor
  • Unwillingness or inability to comply with procedures required in this protocol
  • Serious nonmalignant disease (e.g., hydronephrosis, liver failure, or other conditions) that could compromise protocol objectives in the opinion of the investigator and/or the sponsor.
  • History of Congestive Heart Failure (CHF) or ventricular arrhythmia requiring medication.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01774253

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United States, Arizona
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
United States, Michigan
Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States, 49503
Sponsors and Collaborators
Wake Forest University Health Sciences
Phoenix Children's Hospital
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Study Chair: Giselle Sholler, MD Beat Childhood Cancer at Atrium Health
Principal Investigator: Albert Cornelius, MD Helen DeVos Children's Hospital
Additional Information:
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Responsible Party: Wake Forest University Health Sciences
ClinicalTrials.gov Identifier: NCT01774253    
Other Study ID Numbers: NMTRCPG007
First Posted: January 23, 2013    Key Record Dates
Results First Posted: October 28, 2016
Last Update Posted: April 22, 2022
Last Verified: November 2020
Keywords provided by Wake Forest University Health Sciences:
Additional relevant MeSH terms:
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Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue