Study of Busulfan for Refractory Central Nervous System (CNS) Tumors

NCT00836628 | Withdrawn Phase 1 DMC

• 1 other identifier: CNS 1100
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

This protocol is aimed at establishing a maximum tolerated dose (MTD) for submyeloablative doses of Busulfex ® with the hope that a tolerable, submyeloablative dose can be established to test efficacy as alternative therapy for refractory pediatric brain tumors.

Conditions

Refractory Brain Tumors

Study Arms (1)

experimental

EXPERIMENTAL

Drug: Busulfan

Interventions

Busulfan DRUG

Also known as: Busulfex ®

experimental

Eligibility Criteria

• Age: 3 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age: Age \>2 year and ≤ 21 years Histologic Diagnosis Any histological proven (confirmed by institutional pathology report; pathology slides from outside referring outside institutions are not required.) recurrent or progressive CNS tumor. (optic pathway and brainstem gliomas allowed without histologic verification, but must have diagnostic imaging).
• Life Expectancy Patients must have a life expectancy of ≥ 2 months. Prior Therapy There is no limit to the number of prior therapies a patient has received
• Must be ≥ 3 weeks from myelosuppressive chemotherapy (6 weeks from nitrosoureas) and have demonstrated recovery (ANC ≥ 1000/uL) from their last course of chemotherapy
• ≥ 6 months following allogeneic stem cell transplantation
• ≥ 3 months following autologous stem cell transplantation
• ≥ 3 months from craniospinal radiation
• ≥ 4 weeks from focal radiation
• ≥ 7 days from any past biologic/immunotherapy
• ≥ 1 week from any hematopoietic growth factors Concomitant Medication
• Patients taking Itraconazole or Phenytoin will be excluded. Patients must be off of these medications for at least 3 days prior to entering this trial. If the patient is taking phenytoin for seizures at the time of study enrollment, it must be stopped at least 3 days prior to starting therapy and Clonazepam will be substituted during the Busulfex ® infusions and for 24 hours following the infusion.
• Patients on growth stimulating factors, such as GCSF, will be allowed to continue these medications only as indicated in the study.
• Patients may be taking steroids while participating in this trial, but should be on a stable dose for \>1 week prior to enrollment.
• Medications interacting with the CYP3A4 substrate should also be avoided while the patient is on study.
• Patients should also be on Pneumocystis prophylaxis while participating in this study. Pentamidine will be required, with a recommended dose of 4 mg/kg given intravenously every month. Pentamidine should continue throughout the duration of the trial.
• Organ Function Requirements Adequate Bone Marrow Function Defined As

You may not qualify if:

• Pregnancy/Contraception: patients who are pregnant or breast-feeding will not be eligible.
• Patients of childbearing potential must practice an effective method of birth control while participating on the study.
• Females \> 13 years of age or those who have achieved menarche must have a negative pregnancy test prior to study entry.

Sponsors & Collaborators

• Ann & Robert H Lurie Children's Hospital of Chicago: lead

MeSH Terms

Conditions

Brain Neoplasms

Interventions

Busulfan

Condition Hierarchy (Ancestors)

Central Nervous System Neoplasms; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Intervention Hierarchy (Ancestors)

Butylene Glycols; Glycols; Alcohols; Organic Chemicals; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds

Study Officials

• Stewart Goldman, MD

  Ann & Robert H Lurie Children's Hospital of Chicago

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Chair of Hem/Onc/Stem Cell

Study Record Dates

• First Submitted: February 3, 2009
• First Posted: February 4, 2009
• Study Start: July 1, 2008
• Primary Completion: October 1, 2010
• Study Completion: October 1, 2010
• Last Updated: August 5, 2020

Record last verified: 2020-08