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A Phase I Study of Fenretinide (NSC #374551) in Children With High Risk Solid Tumors

Phase 1
21 Years
Not Enrolling
Neuroblastoma, Unspecified Childhood Solid Tumor, Protocol Specific

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Trial Information

A Phase I Study of Fenretinide (NSC #374551) in Children With High Risk Solid Tumors


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.

Inclusion Criteria


- 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


- 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


- 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

Type of Study:


Study Design:

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Principal Investigator

Judith G. Villablanca, MD

Investigator Role:

Study Chair

Investigator Affiliation:

Children's Hospital Los Angeles


United States: Food and Drug Administration

Study ID:




Start Date:

March 1998

Completion Date:

Related Keywords:

  • Neuroblastoma
  • Unspecified Childhood Solid Tumor, Protocol Specific
  • recurrent neuroblastoma
  • unspecified childhood solid tumor, protocol specific
  • Neuroblastoma
  • Neoplasms



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