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

NCT01774253 | Terminated Phase 2 Results DMC

• 1 other identifier: NMTRCPG007
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 2

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.

Conditions

Pontine Glioma

Keywords

DIPG

Outcome Measures

Primary Outcomes (1)

• Number of Days Participants Experienced Progression Free Survival (PFS)

  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.

  5 years

Secondary Outcomes (4)

• Number of Participants With Adverse Events as a Measure of Safety and Tolerability

  2 years

• Determine the Median Overall Survival (OS) of Participants

  2 years

• Evaluate the Impact of Quality of Life of Children Receiving Vismodegib Using PedsQL Questionnaires

  2 years

• Determine the Response Rates of Participants Based on Activation (or no Activation) of Their Hedgehog Signaling Pathway

  3 years

Study Arms (1)

Vismodegib

EXPERIMENTAL

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

Interventions

Vismodegib DRUG

Also known as: Erivedge

Vismodegib

Eligibility Criteria

• Age: 3 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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:
• Bilirubin ≤ 1.5 times upper limit of normal
• 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:
• mg/dL (for patients ≤ 5 years of age)
• mg/dL (for patients 6 to 10 years of age)

You may not qualify if:

• 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:
• Active, uncontrolled bacterial, viral, or fungal infections requiring systemic therapy
• Diarrhea of any cause ≥ CTCAE grade 2
• Psychiatric illness/social situations that would compromise patient safety or limit compliance with study requirements including maintenance of a compliance/pill diary
• 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.

Sponsors & Collaborators

• Giselle Sholler: lead
• Phoenix Children's Hospital: collaborator

Study Sites (2)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States

Location

Related Links

• Beat Childhood Cancer

MeSH Terms

Interventions

HhAntag691

Results Point of Contact

• Title: Giselle Sholler, MD
• Organization: NMTRC

genevieve.bergendahl@helendevoschildrens.org

6162670335

Study Officials

• Giselle Sholler, MD

  Beat Childhood Cancer at Atrium Health

  STUDY CHAIR

• Albert Cornelius, MD

  Helen DeVos Children's Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Beat Childhood Cancer Chair

Study Record Dates

• First Submitted: January 18, 2013
• First Posted: January 23, 2013
• Study Start: May 1, 2013
• Primary Completion: October 1, 2015
• Study Completion: October 1, 2015
• Last Updated: August 6, 2024
• Results First Posted: October 28, 2016

Record last verified: 2024-08

Locations

US (2)