Back to resultsIntra-arterial Chemotherapy for the Treatment of Progressive Diffuse Intrinsic Pontine Gliomas (DIPG).
Primary Purpose
Diffuse Intrinsic Pontine Glioma (DIPG)
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Melphalan hydrochloride
Sponsored by
About this trial
This is an interventional other trial for Diffuse Intrinsic Pontine Glioma (DIPG) focused on measuring diffuse intrinsic pontine glioma (DIPG), intra-arterial chemotherapy, angiography
Eligibility Criteria
Inclusion Criteria:
- Pediatric patients of all ages with progressive DIPG.
- Consensus following presentation of the case at the multidisciplinary Pediatric Neuro-Oncology conference, which includes participation of neuro-oncology, neurosurgery, radiation oncology, interventional neuroradiology and neurology.
Exclusion Criteria:
Documented hypercoagulable disorders or vasculopathies
- INR value more than a Grade 1 toxicity by CTCAE v 4.0 criteria (>1 - 1.5 x ULN; >1 - 1.5 times above baseline if on anticoagulation).
- APTT value more than a Grade 1 toxicity by CTCAE v 4.0 criteria (>ULN - 1.5 x ULN).
- Platelets less than 50 x 103/mm3
- Absolute neutrophil count less than 500/ mm3
- Pregnancy
- Documented severe allergic reaction to IV iodinated contrast, specifically bronchospasm and anaphylaxis.
Sites / Locations
- The Johns Hopkins Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
IA melphalan
Arm Description
IA melphalan is administered via the basilar artery.
Outcomes
Primary Outcome Measures
Technical safety as determined by number of participants with toxicity
Number of participants with grades 3-5 intracranial hemorrhage, grades 3-5 stroke, as defined by the Nervous system disorder CTCAE, and requirement of blood transfusion.
Secondary Outcome Measures
Long-term Efficacy as assessed by progression free survival
Number of months until disease progression.
Full Information
NCT ID
NCT01688401
First Posted
September 10, 2012
Last Updated
March 26, 2019
Sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Collaborators
Solving Kids' Cancer
1. Study Identification
Unique Protocol Identification Number
NCT01688401
Brief Title
Intra-arterial Chemotherapy for the Treatment of Progressive Diffuse Intrinsic Pontine Gliomas (DIPG).
Official Title
Intra-arterial Chemotherapy for the Treatment of Progressive Diffuse Intrinsic Pontine Gliomas (DIPG).
Study Type
Interventional
2. Study Status
Record Verification Date
March 2019
Overall Recruitment Status
Completed
Study Start Date
March 8, 2013 (Actual)
Primary Completion Date
May 2017 (Actual)
Study Completion Date
November 26, 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Collaborators
Solving Kids' Cancer
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The goal of this pilot study is to determine if intra-arterial (IA) chemotherapy is safe in the treatment of progressive diffuse intrinsic pontine gliomas (DIPG). IA administration of the chemotherapeutic agent enhances the regional distribution of the drug, thereby increasing the local delivered dose while minimizing systemic toxicity. It also provides a treatment option for these patients at the time of tumor recurrence.
Detailed Description
Delivering the chemotherapeutic agent directly to the tumor via the arterial system avoids the complications and adverse events associated with toxicity from systemic chemotherapy.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Diffuse Intrinsic Pontine Glioma (DIPG)
Keywords
diffuse intrinsic pontine glioma (DIPG), intra-arterial chemotherapy, angiography
7. Study Design
Primary Purpose
Other
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
3 (Actual)
8. Arms, Groups, and Interventions
Arm Title
IA melphalan
Arm Type
Experimental
Arm Description
IA melphalan is administered via the basilar artery.
Intervention Type
Drug
Intervention Name(s)
Melphalan hydrochloride
Other Intervention Name(s)
Alkeran
Intervention Description
Drug administered intra-arterially (injection in the artery).
Standard dose: Cycle 1: 1 mg, intra-arterial (IA) delivery. Cycle 2: 2 mg, intra-arterial (IA) delivery.
Duration of treatment: Eight weeks total - two cycles of IA chemotherapy, separated by four weeks.
Primary Outcome Measure Information:
Title
Technical safety as determined by number of participants with toxicity
Description
Number of participants with grades 3-5 intracranial hemorrhage, grades 3-5 stroke, as defined by the Nervous system disorder CTCAE, and requirement of blood transfusion.
Time Frame
60 days
Secondary Outcome Measure Information:
Title
Long-term Efficacy as assessed by progression free survival
Description
Number of months until disease progression.
Time Frame
2 years
Other Pre-specified Outcome Measures:
Title
Immediate Efficacy as assessed by number of participants with decrease in required steroid dose
Time Frame
60 days
Title
Immediate Efficacy as assessed by number of participants with decrease in tumor size on MRI
Time Frame
60 days
Title
Immediate Efficacy as assessed by number of participants with decrease in the degree of enhancement on MRI
Time Frame
60 days
Title
Immediate Efficacy as assessed by number of participants with improved neurological examination
Time Frame
60 days
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Month
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Pediatric patients of all ages with progressive DIPG.
Consensus following presentation of the case at the multidisciplinary Pediatric Neuro-Oncology conference, which includes participation of neuro-oncology, neurosurgery, radiation oncology, interventional neuroradiology and neurology.
Exclusion Criteria:
Documented hypercoagulable disorders or vasculopathies
INR value more than a Grade 1 toxicity by CTCAE v 4.0 criteria (>1 - 1.5 x ULN; >1 - 1.5 times above baseline if on anticoagulation).
APTT value more than a Grade 1 toxicity by CTCAE v 4.0 criteria (>ULN - 1.5 x ULN).
Platelets less than 50 x 103/mm3
Absolute neutrophil count less than 500/ mm3
Pregnancy
Documented severe allergic reaction to IV iodinated contrast, specifically bronchospasm and anaphylaxis.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Monica Pearl, M.D.
Organizational Affiliation
Johns Hopkins University
Official's Role
Principal Investigator
Facility Information:
Facility Name
The Johns Hopkins Hospital
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Intra-arterial Chemotherapy for the Treatment of Progressive Diffuse Intrinsic Pontine Gliomas (DIPG).
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