Pontine GliomaPhase 2NCT01774253
Erivedge (Vismodegib) in the Treatment of Pediatric Patients With Refractory Pontine Glioma
A Phase 2 study testing Vismodegib in people with Pontine Glioma, run by Giselle Sholler at 2 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 to 18 years
- Conditions the study is for
- Pontine Glioma
- What is being tested?
- Vismodegib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 2 sitesUnited States
- Study dates
- May 2013to Oct 2015
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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)
Show all 30Show fewer
- 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:
- 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:
- Granulocyte ≥ 1500 cells/mm3
- Platelet count ≥ 100,000 (plt/mm3)
- Serum albumin ≥ 2.5 g/dL
- Urinalysis:
- No clinically significant abnormalities
- Acceptable coagulation status as defined by:
- PT/INR less than 1.5
- 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
Who cannot join
- 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
Show all 12Show fewer
- 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.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503
Phoenix Children's Hospital
Phoenix, Arizona, 85016
About this study
Official title: A Phase II, Open-label, Multi-center Study of Erivedge (Vismodegib) in the Treatment of Pediatric Patients With Refractory Pontine Glioma.
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
- Being tested
- Vismodegib
- Keywords
- DIPG
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VismodegibDrug
Also called Erivedge
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Vismodegib |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 5 years
Secondary outcomes (4)
Number of Participants With Adverse Events as a Measure of Safety and Tolerability
To determine the safety and tolerability of Vismodegib as a single agent in pediatric and young adult patients with refractory or recurrent pontine glioma
Measured: 2 years
Determine the Median Overall Survival (OS) of Participants
Overall Survival (OS) and clinical benefit (ORR + stable disease, SD)
Measured: 2 years
Evaluate the Impact of Quality of Life of Children Receiving Vismodegib Using PedsQL Questionnaires
Evaluate the impact of Quality of Life of children receiving Vismodegib using PedsQL questionnaires
Measured: 2 years
Determine the Response Rates of Participants Based on Activation (or no Activation) of Their Hedgehog Signaling Pathway
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
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jan 2013 (estimated)
- Study startMay 2013
- Primary completionOct 2015
- Study completionOct 2015
Why it stopped: Lack of enrollment and commercial availability of drug
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Pontine GliomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Giselle Sholler
- Sponsor type
- University, hospital or other
- Collaborators
- Phoenix Children's Hospital
- Responsible party
- Sponsor-investigator, Giselle Sholler, Milton S. Hershey Medical Center
- Investigators
- Giselle Sholler, MD, Study chair, Beat Childhood Cancer at Atrium HealthAlbert Cornelius, MD, Principal investigator, Helen DeVos Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01774253
- Sponsor's protocol ID
- NMTRCPG007
- Status last verified
- Aug 2024
- First submitted
- 18 Jan 2013
- Last updated
- 6 Aug 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 6 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Open-label, Multi-center Study of Erivedge (Vismodegib) in the Treatment of Pediatric Patients With Refractory Pontine Glioma.
- ClinicalTrials.gov ID
- NCT01774253
- Acronym / sponsor ID
- NMTRCPG007
- Conditions
- Pontine Glioma
- Keywords
- DIPG
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Lack of enrollment and commercial availability of drug
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Giselle ShollerUniversity, hospital or other
- Collaborators
- Phoenix Children's Hospital
- Study start
- May 2013
- Primary completion
- Oct 2015 (actual)
- Study completion
- Oct 2015 (actual)
- First posted
- 23 Jan 2013 (estimated)
- Results
- Posted 28 Oct 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Vismodegib | Experimental | Drug: VismodegibVismodegib 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. |
Who may qualify
- Ages
- 3 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (30 listed)
- 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
Key exclusion criteria (12 listed)
- 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:
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VismodegibAlso: Erivedge | Drug | Vismodegib |
Primary outcome
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.
4 secondary outcomes
Number of Participants With Adverse Events as a Measure of Safety and Tolerability
Time frame: 2 years
Determine the Median Overall Survival (OS) of Participants
Time frame: 2 years
Evaluate the Impact of Quality of Life of Children Receiving Vismodegib Using PedsQL Questionnaires
Time frame: 2 years
Determine the Response Rates of Participants Based on Activation (or no Activation) of Their Hedgehog Signaling Pathway
Time frame: 3 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Aug 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Beat Childhood CancerLink provided by the sponsor.
Related trials for Pontine Glioma
RecruitingPhase 1
PEACH TRIAL- Precision Medicine and Adoptive Cellular Therapy
Neuroblastoma, Diffuse Intrinsic Pontine Glioma
Active, not recruitingPhase 1/2
A Study of the Drug Selinexor With Radiation Therapy in Patients With Newly-Diagnosed Diffuse Intrinsic Pontine (DIPG) Glioma and High-Grade Glioma (HGG)
Malignant Glioma
Active, not recruitingPhase 2
Pediatric Trial of Indoximod With Chemotherapy and Radiation for Relapsed Brain Tumors or Newly Diagnosed DIPG
Glioblastoma, Medulloblastoma
Not yet recruitingPhase 1
IL-8 Receptor-modified CD70 CAR T Cell Therapy in CD70+ Newly Diagnosed and Recurrent Pediatric High-grade Glioma (pHGG) and Newly Diagnosed Diffuse Intrinsic Pontine Glioma (ndDIPG)
High-grade Glioma, Diffuse Intrinsic Pontine Glioma
RecruitingPhase 2
A Study of VRT106 in Combination With Radiotherapy in Adult Patients With Diffuse Midline Glioma / Diffuse Intrinsic Pontine Glioma
Diffuse Midline Glioma, H3 K27M-Mutant, Diffuse Intrinsic Pontine Glioma
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Giselle Sholler, MD study chair
Beat Childhood Cancer at Atrium Health
Albert Cornelius, MD principal investigator
Helen DeVos Children's Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Vismodegib in people with Pontine Glioma, run by Giselle Sholler at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 30 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 6 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 28 Oct 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Days Participants Experienced Progression Free Survival (PFS) (time frame: 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Aug 2024.