Clinical trial NCT01688401: 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
Overall status: COMPLETED
Phase: PHASE1
Conditions: Diffuse Intrinsic Pontine Glioma (DIPG)
Keywords: diffuse intrinsic pontine glioma (DIPG)
intra-arterial chemotherapy
angiography
Lead sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Sponsor class: OTHER
Collaborators: name: Solving Kids' Cancer
First posted: 2012-09-19
Enrollment: 3
Sex: ALL
Minimum age (years): 1
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

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.

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.

Primary outcomes:
measure: Technical safety as determined by number of participants with toxicity
timeFrame: 60 days
Secondary outcomes:
measure: Long-term Efficacy as assessed by progression free survival
timeFrame: 2 years
Other outcomes:
measure: Immediate Efficacy as assessed by number of participants with decrease in required steroid dose
timeFrame: 60 days
measure: Immediate Efficacy as assessed by number of participants with decrease in tumor size on MRI
timeFrame: 60 days
measure: Immediate Efficacy as assessed by number of participants with decrease in the degree of enhancement on MRI
timeFrame: 60 days
measure: Immediate Efficacy as assessed by number of participants with improved neurological examination
timeFrame: 60 days
Locations:
city: Baltimore
country: United States
facility: The Johns Hopkins Hospital
state: Maryland
Study officials:
affiliation: Johns Hopkins University
name: Monica Pearl, M.D.
role: PRINCIPAL_INVESTIGATOR
References: