Intra-arterial Chemotherapy for the Treatment of Progressive Diffuse Intrinsic Pontine Gliomas (DIPG).

Study Overview
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Brief Summary
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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
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Delivering the chemotherapeutic agent directly to the tumor via the arterial system avoids the complications and adverse events associated with toxicity from systemic chemotherapy.

Official Title
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Intra-arterial Chemotherapy for the Treatment of Progressive Diffuse Intrinsic Pontine Gliomas (DIPG).

Conditions
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Diffuse Intrinsic Pontine Glioma (DIPG)

Intervention / Treatment
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* Drug: Melphalan hydrochloride


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 1 Month
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Other
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: IA melphalan<br>IA melphalan is administered via the basilar artery. | Drug: Melphalan hydrochloride<br>* 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.<br>* Other names: Alkeran;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 60 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Long-term Efficacy as assessed by progression free survival | Number of months until disease progression. | 2 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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diffuse intrinsic pontine glioma (DIPG), intra-arterial chemotherapy, angiography