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Completed

NCT Number: NCT01688401

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

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.

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Key information

Conditions

Diffuse Intrinsic Pontine Glioma (DIPG) Brain Diseases Brain Neoplasms Brain Stem Neoplasms Central Nervous System Diseases Central Nervous System Neoplasms Diffuse Intrinsic Pontine Glioma Glioma Infratentorial Neoplasms Neoplasms Neoplasms by Histologic Type Neoplasms by Site Neoplasms, Germ Cell and Embryonal Neoplasms, Glandular and Epithelial Neoplasms, Nerve Tissue Neoplasms, Neuroepithelial Nervous System Diseases Nervous System Neoplasms Neuroectodermal Tumors

Age range

1 month–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

The Johns Hopkins Hospital

Baltimore, Maryland, 21287, United States

About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Melphalan Hydrochloride

Drug

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.

Other names: Alkeran

Primary outcomes

  1. Technical safety as determined by number of participants with toxicity

    Time frame: 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 outcomes

  1. Long-term Efficacy as assessed by progression free survival

    Time frame: 2 years

    Number of months until disease progression.

Other outcomes

  1. Immediate Efficacy as assessed by number of participants with decrease in required steroid dose

    Time frame: 60 days

  2. Immediate Efficacy as assessed by number of participants with decrease in tumor size on MRI

    Time frame: 60 days

  3. Immediate Efficacy as assessed by number of participants with decrease in the degree of enhancement on MRI

    Time frame: 60 days

  4. Immediate Efficacy as assessed by number of participants with improved neurological examination

    Time frame: 60 days

Sponsors and collaborators

Lead sponsor

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Other

Collaborators

  • Solving Kids' Cancer

Registry information

Important dates

Study start
2013
Primary completion
2017
Study completion
2018
First posted
Sep 19, 2012
Registry last updated
Mar 28, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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