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

Phase 1

Completed

• Conditions: Diffuse Intrinsic Pontine Glioma (DIPG)
• Interventions: Drug: Melphalan hydrochloride

• Registration Number: NCT01688401
• Lead Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

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.

Study Timeline

• Study First Submitted: 2012-09-10
• Study First Submitted QC: 2012-09-14
• Study First Posted: 2012-09-19
• Study Start: 2013-03-08
• Primary Completion: 2017-05
• Study Completion: 2018-11-26
• Status Verified: 2019-03
• Last Update Submitted: 2019-03-26
• Last Update Posted: 2019-03-28

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 3

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
IA melphalan	Melphalan hydrochloride	IA melphalan is administered via the basilar artery.

Primary Outcome Measures

Name	Time	Method
Technical safety as determined by number of participants with toxicity	60 days	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

Name	Time	Method
Long-term Efficacy as assessed by progression free survival	2 years	Number of months until disease progression.

Trial Locations

Locations (1)

The Johns Hopkins Hospital; 🇺🇸 Baltimore, Maryland, United States