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Fenretinide in Treating Children With Solid Tumors

Primary Purpose

Neuroblastoma, Unspecified Childhood Solid Tumor, Protocol Specific

Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
fenretinide
Sponsored by
National Cancer Institute (NCI)
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Neuroblastoma focused on measuring recurrent neuroblastoma, unspecified childhood solid tumor, protocol specific

Eligibility Criteria

undefined - 21 Years (Child, Adult)All SexesDoes not accept healthy volunteers

DISEASE CHARACTERISTICS: Histologically confirmed malignant solid tumor that is refractory to conventional therapy or recurrent neuroblastoma treated with myeloablative therapy and autologous stem cell transplant in second complete or partial response Bone marrow metastases with granulocytopenia, anemia, and/or thrombocytopenia are eligible PATIENT CHARACTERISTICS: Age: Under 21 at diagnosis Performance status: CCG 0-2 Life expectancy: At least 2 months Absolute neutrophil count at least 750/mm3 Platelet count at least 50,000/mm3 Hemoglobin at least 7.0 g/dL Bilirubin no greater than 1.5 mg/dL SGOT and SGPT less than 2.5 times normal Creatinine no greater than 1.5 g/dL OR creatinine clearance at least 50 mL/min OR radioisotope GFR at least 50 mL/min Seizure disorders controlled with anticonvulsants allowed No CNS toxicity greater than grade 2 Not pregnant Fertile patients must use effective contraception PRIOR CONCURRENT THERAPY: At least 1 month since prior autologous stem cell transplantation No prior allogeneic transplantation At least 2 weeks since prior chemotherapy (4 weeks for nitrosourea) and recovered No other concurrent chemotherapy No concurrent immunomodulating agents (including steroids) Concurrent corticosteroid therapy for increased intracranial pressure allowed Concurrent dexamethasone for CNS tumor allowed At least 2 weeks since prior radiotherapy Concurrent radiotherapy to localized lesions allowed At least 2 weeks since prior retinoids Prior isotretinoin or 9-cis-retinoic acid allowed

Sites / Locations

  • Cancer Center and Beckman Research Institute, City of Hope
  • Long Beach Memorial Medical Center
  • Children's Hospital Los Angeles
  • Jonsson Comprehensive Cancer Center, UCLA
  • Children's Hospital of Orange County
  • UCSF Cancer Center and Cancer Research Institute
  • Children's National Medical Center
  • Indiana University Cancer Center
  • University of Michigan Comprehensive Cancer Center
  • University of Minnesota Cancer Center
  • Mayo Clinic Cancer Center
  • Children's Mercy Hospital
  • Cancer Institute of New Jersey
  • NYU School of Medicine's Kaplan Comprehensive Cancer Center
  • Memorial Sloan-Kettering Cancer Center
  • Herbert Irving Comprehensive Cancer Center
  • Children's Hospital Medical Center - Cincinnati
  • Children's Hospital of Columbus
  • Children's Hospital of Philadelphia
  • Children's Hospital of Pittsburgh
  • Vanderbilt-Ingram Cancer Center
  • University of Texas - MD Anderson Cancer Center
  • Primary Children's Medical Center
  • Children's Hospital and Regional Medical Center - Seattle
  • University of Wisconsin Comprehensive Cancer Center
  • Princess Margaret Hospital for Children

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Arm I

Arm Description

Patients receive oral fenretinide 3 times a day on days 1-7. Treatment repeats every 3 weeks for up to 8 courses. Patients may receive an additional 22 courses of therapy in the presence of stable or responding residual tumor. Patients with recurrent neuroblastoma, after prior myeloablative therapy with no measurable disease, will stop treatment after 8 courses. Cohorts of 3-6 patients receive escalating doses of fenretinide until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 6 patients experience dose limiting toxicity.

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
November 1, 1999
Last Updated
February 6, 2013
Sponsor
National Cancer Institute (NCI)
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1. Study Identification

Unique Protocol Identification Number
NCT00003191
Brief Title
Fenretinide in Treating Children With Solid Tumors
Official Title
A Phase I Study of Fenretinide (NSC #374551) in Children With High Risk Solid Tumors
Study Type
Interventional

2. Study Status

Record Verification Date
August 2006
Overall Recruitment Status
Completed
Study Start Date
March 1998 (undefined)
Primary Completion Date
March 2005 (Actual)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
National Cancer Institute (NCI)

4. Oversight

5. Study Description

Brief Summary
Phase I trial to study the effectiveness of fenretinide in treating children who have solid tumors that have not responded to standard therapy. Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die.
Detailed Description
OBJECTIVES: I. Determine the maximum tolerated dose of fenretinide (HPR) in children with high risk solid tumors. II. Determine the toxicities of HPR in these patients. III. Determine the pharmacokinetics of HPR in these patients. IV. Determine the CSF level of HPR in patients whom cerebrospinal fluid is obtained for routine purposes while on this study. V. Determine the effect of HPR on plasma retinol levels in these patients. VI. Determine the activity of HPR in these patients. VII. Determine the antitumor activity of HPR on minimal residual bone marrow disease in neuroblastoma. OUTLINE: This is a dose escalation study. Patients receive oral fenretinide 3 times a day on days 1-7. Treatment repeats every 3 weeks for up to 8 courses. Patients may receive an additional 22 courses of therapy in the presence of stable or responding residual tumor. Patients with recurrent neuroblastoma, after prior myeloablative therapy with no measurable disease, will stop treatment after 8 courses. Cohorts of 3-6 patients receive escalating doses of fenretinide until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 6 patients experience dose limiting toxicity. Patients are followed until death.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neuroblastoma, Unspecified Childhood Solid Tumor, Protocol Specific
Keywords
recurrent neuroblastoma, unspecified childhood solid tumor, protocol specific

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
18 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Arm I
Arm Type
Experimental
Arm Description
Patients receive oral fenretinide 3 times a day on days 1-7. Treatment repeats every 3 weeks for up to 8 courses. Patients may receive an additional 22 courses of therapy in the presence of stable or responding residual tumor. Patients with recurrent neuroblastoma, after prior myeloablative therapy with no measurable disease, will stop treatment after 8 courses. Cohorts of 3-6 patients receive escalating doses of fenretinide until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 6 patients experience dose limiting toxicity.
Intervention Type
Drug
Intervention Name(s)
fenretinide

10. Eligibility

Sex
All
Maximum Age & Unit of Time
21 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
DISEASE CHARACTERISTICS: Histologically confirmed malignant solid tumor that is refractory to conventional therapy or recurrent neuroblastoma treated with myeloablative therapy and autologous stem cell transplant in second complete or partial response Bone marrow metastases with granulocytopenia, anemia, and/or thrombocytopenia are eligible PATIENT CHARACTERISTICS: Age: Under 21 at diagnosis Performance status: CCG 0-2 Life expectancy: At least 2 months Absolute neutrophil count at least 750/mm3 Platelet count at least 50,000/mm3 Hemoglobin at least 7.0 g/dL Bilirubin no greater than 1.5 mg/dL SGOT and SGPT less than 2.5 times normal Creatinine no greater than 1.5 g/dL OR creatinine clearance at least 50 mL/min OR radioisotope GFR at least 50 mL/min Seizure disorders controlled with anticonvulsants allowed No CNS toxicity greater than grade 2 Not pregnant Fertile patients must use effective contraception PRIOR CONCURRENT THERAPY: At least 1 month since prior autologous stem cell transplantation No prior allogeneic transplantation At least 2 weeks since prior chemotherapy (4 weeks for nitrosourea) and recovered No other concurrent chemotherapy No concurrent immunomodulating agents (including steroids) Concurrent corticosteroid therapy for increased intracranial pressure allowed Concurrent dexamethasone for CNS tumor allowed At least 2 weeks since prior radiotherapy Concurrent radiotherapy to localized lesions allowed At least 2 weeks since prior retinoids Prior isotretinoin or 9-cis-retinoic acid allowed
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Judith G. Villablanca, MD
Organizational Affiliation
Children's Hospital Los Angeles
Official's Role
Study Chair
Facility Information:
Facility Name
Cancer Center and Beckman Research Institute, City of Hope
City
Duarte
State/Province
California
ZIP/Postal Code
91010-3000
Country
United States
Facility Name
Long Beach Memorial Medical Center
City
Long Beach
State/Province
California
ZIP/Postal Code
90806
Country
United States
Facility Name
Children's Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027-0700
Country
United States
Facility Name
Jonsson Comprehensive Cancer Center, UCLA
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095-1781
Country
United States
Facility Name
Children's Hospital of Orange County
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
UCSF Cancer Center and Cancer Research Institute
City
San Francisco
State/Province
California
ZIP/Postal Code
94143-0128
Country
United States
Facility Name
Children's National Medical Center
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010-2970
Country
United States
Facility Name
Indiana University Cancer Center
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202-5289
Country
United States
Facility Name
University of Michigan Comprehensive Cancer Center
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109-0752
Country
United States
Facility Name
University of Minnesota Cancer Center
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Facility Name
Mayo Clinic Cancer Center
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Children's Mercy Hospital
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Facility Name
Cancer Institute of New Jersey
City
New Brunswick
State/Province
New Jersey
ZIP/Postal Code
08901
Country
United States
Facility Name
NYU School of Medicine's Kaplan Comprehensive Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States
Facility Name
Memorial Sloan-Kettering Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Facility Name
Herbert Irving Comprehensive Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Children's Hospital Medical Center - Cincinnati
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229-3039
Country
United States
Facility Name
Children's Hospital of Columbus
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205-2696
Country
United States
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Children's Hospital of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
Vanderbilt-Ingram Cancer Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232-6838
Country
United States
Facility Name
University of Texas - MD Anderson Cancer Center
City
Houston
State/Province
Texas
ZIP/Postal Code
77030-4009
Country
United States
Facility Name
Primary Children's Medical Center
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84113
Country
United States
Facility Name
Children's Hospital and Regional Medical Center - Seattle
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Facility Name
University of Wisconsin Comprehensive Cancer Center
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53792-6164
Country
United States
Facility Name
Princess Margaret Hospital for Children
City
Perth
State/Province
Western Australia
ZIP/Postal Code
6001
Country
Australia

12. IPD Sharing Statement

Citations:
PubMed Identifier
16849757
Citation
Children's Oncology Group (CCG 09709); Villablanca JG, Krailo MD, Ames MM, Reid JM, Reaman GH, Reynolds CP. Phase I trial of oral fenretinide in children with high-risk solid tumors: a report from the Children's Oncology Group (CCG 09709). J Clin Oncol. 2006 Jul 20;24(21):3423-30. doi: 10.1200/JCO.2005.03.9271. Erratum In: J Clin Oncol. 2006 Sep 1;24(25):4223. Reynolds, Patrick C [corrected to Reynolds, C Patrick].
Results Reference
result
Citation
Villablanca JG, Ames MW, Reid JM, et al.: Phase I trial of oral [N-(-4-hydroxyphenyl)retinamide] (4-HPR) in children with resistant/recurrent solid tumors: a children's cancer group study (CCG 09709). [Abstract] Proceedings of the American Society of Clinical Oncology 21: A-1588, 2002.
Results Reference
result

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Fenretinide in Treating Children With Solid Tumors

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