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

NCT01688401 | Completed Phase 1 DMC

• 2 other identifiers: J11164NA_00069122
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

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.

Conditions

Diffuse Intrinsic Pontine Glioma (DIPG)

Keywords

diffuse intrinsic pontine glioma (DIPG); intra-arterial chemotherapy; angiography

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Long-term Efficacy as assessed by progression free survival

  2 years

Other Outcomes (4)

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

  60 days

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

  60 days

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

  60 days

Study Arms (1)

IA melphalan

EXPERIMENTAL

IA melphalan is administered via the basilar artery.

Drug: Melphalan hydrochloride

Interventions

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.

Also known as: Alkeran

IA melphalan

Eligibility Criteria

• Age: 1 Month - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins: lead
• Solving Kids' Cancer: collaborator

Study Sites (1)

The Johns Hopkins Hospital

Baltimore, Maryland, 21287, United States

Location

MeSH Terms

Conditions

Diffuse Intrinsic Pontine Glioma

Interventions

Melphalan

Condition Hierarchy (Ancestors)

Glioma; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Brain Stem Neoplasms; Infratentorial Neoplasms; Brain Neoplasms; Central Nervous System Neoplasms; Nervous System Neoplasms; Neoplasms by Site; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Intervention Hierarchy (Ancestors)

Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Amino Acids, Peptides, and Proteins

Study Officials

• Monica Pearl, M.D.

  Johns Hopkins University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 10, 2012
• First Posted: September 19, 2012
• Study Start: March 8, 2013
• Primary Completion: May 1, 2017
• Study Completion: November 26, 2018
• Last Updated: March 28, 2019

Record last verified: 2019-03

Locations

US (1)