NCT00028522

Brief Summary

This phase I trial is studying the side effects and best dose of R(+)XK469 in treating patients with advanced neuroblastoma. Drugs used in chemotherapy work in different ways to stop cancer cells from dividing so they stop growing or die.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
85

participants targeted

Target at P75+ for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 1, 2001

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

January 4, 2002

Completed
1.1 years until next milestone

First Posted

Study publicly available on registry

January 27, 2003

Completed
6.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2009

Completed
Last Updated

December 16, 2013

Status Verified

December 1, 2013

Enrollment Period

7.8 years

First QC Date

January 4, 2002

Last Update Submit

December 13, 2013

Conditions

Disseminated NeuroblastomaLocalized Unresectable NeuroblastomaRecurrent NeuroblastomaRegional Neuroblastoma

Outcome Measures

Primary Outcomes (3)

  • Maximum tolerated dose of XK469 in pediatric patients with advanced neuroblastoma

    Defined as the highest dose studied for which the incidence of dose-limiting toxicity (DLT) was less than 33%.

    Day 29 of course 1

  • DLT

    Defined as the occurrence of any of the following: 1) grade 3 or higher nonhematologic toxicity except fatigue, alopecia, nausea, vomiting, 2) grade 4 thrombocytopenia or anemia, 3) any fever accompanied by granulocyte count \< 1000/mm\^3 (grade 3 or 4 neutropenia), 4) failure to recover absolute neutrophil count 1500/μL

    Day 29 of course 1

  • Recommended phase II dose

    Generally defined as the MTD. For both schedules A and B and the pediatric dosing schedule, once the recommended phase II dose has been tentatively defined, a total of 12 evaluable patients will be studied to ensure the feasibility of this dose for phase II trials. If interindividual pharmacokinetic variability is high, additional patients will be enrolled (maximum of 20 at the phase II dose) to permit adequate pharmacological characterization of XK469 and the relationship of interindividual pharmacokinetic variability to toxicity.

    Day 29 of course 1

Secondary Outcomes (4)

  • Metabolism of XK469 in pediatric patients

    Days 1-3 of course 1

  • Pharmacokinetics of XK469 in pediatric patients

    Days 1-3 of course 1

  • Pharmacodynamics of XK469 in pediatric patients

    Continuously over the course of study treatment

  • Antineoplastic activity of XK469 for neuroblastoma

    Every 2 courses

Study Arms (1)

Treatment (chemotherapy)

EXPERIMENTAL

SCHEDULE A: Patients receive R(+)XK469 IV over 30 minutes on days 1, 3, and 5. Courses repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Cohorts of 3-6 patients receive escalating doses of R(+)XK469 until the recommended phase II dose or MTD is determined. The MTD is defined as the dose preceding that at which 2 of 6 patients experience dose-limiting toxicity. Once the MTD is determined, additional patients are accrued and treated at the recommended phase II dose (for a maximum of 20 patients treated at that dose). SCHEDULE B: Once the recommended phase II dose is determined on schedule A, additional patients are accrued and receive escalating doses of R(+)XK469 IV over 30-60 minutes on day 1, beginning at a reduced dose. Courses repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Dose escalation continues as in Schedule A.

Drug: R(+)XK469

Interventions

R(+)XK469DRUG

Given IV

Also known as: XK469
Treatment (chemotherapy)

Eligibility Criteria

Age5 Years - 20 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Histologically confirmed high-risk neuroblastoma that has relapsed or is refractory to standard therapy
  • No active brain metastases
  • Previously treated brain metastases allowed if there is no requirement for corticosteroids or anticonvulsants
  • Performance status - Karnofsky performance status 70-100% or Lansky score ≥ 70 for your pediatric patients
  • More than 3 months
  • WBC at least 3,000/mm\^3
  • Absolute neutrophil count at least 1,500/mm\^3
  • Platelet count at least 100,000/mm\^3
  • Bilirubin normal (unless due to documented Gilbert's syndrome)
  • Creatinine less than 1.5 times upper limit of normal
  • No symptomatic congestive heart failure
  • No unstable angina pectoris
  • No cardiac arrhythmia
  • Not pregnant or nursing
  • Negative pregnancy test
  • +17 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of Chicago Comprehensive Cancer Center

Chicago, Illinois, 60637-1470, United States

Location

MeSH Terms

Conditions

Neuroblastoma

Interventions

XK 469

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, PeripheralNeuroectodermal Tumors, PrimitiveNeoplasms, NeuroepithelialNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms by Histologic TypeNeoplasmsNeoplasms, Glandular and EpithelialNeoplasms, Nerve Tissue

Study Officials

  • Susan Cohn

    University of Chicago Comprehensive Cancer Center

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
NIH
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 4, 2002

First Posted

January 27, 2003

Study Start

December 1, 2001

Primary Completion

September 1, 2009

Last Updated

December 16, 2013

Record last verified: 2013-12

Locations

US(1)