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Effect of Hydroxyurea as Treatment for Primary Desmoid Tumors in Adults and Children

Phase 2
21 Years
Not Enrolling
Desmoid Tumors, Fibromatosis

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Trial Information

Effect of Hydroxyurea as Treatment for Primary Desmoid Tumors in Adults and Children

The treatment of desmoid tumors is complicated by benign histology and potentially
aggressive and recurrent behavior. Invasive or intensive treatments with surgery or
radiation therapy can lead to good tumor control, but at the expense of significant side
effects along with a high risk of local recurrence after surgical excision. Low dose
regimens of standard chemotherapy drugs have been favored as a conservative first-line
treatment; however, refractory and recurrent tumors are not uncommon. The purpose of this
study is to investigate the efficacy and safety of using hydroxyurea, an anti-neoplastic
agent with relatively few side-effects, as a novel treatment for primary, unresectable
desmoid tumors, desmoid tumors that have had an incomplete primary resection, or desmoid
tumors that have recurred after other therapy.

Patients presenting to our oncology center with a new diagnosis of desmoid tumor, or a
recurrent desmoid tumor, will be eligible for this study. After informed consent, physical
examination, and baseline imaging studies (CT/MRI), patients will receive oral hydroxyurea
at a starting dose of 20 mg/kg daily. Laboratory tests will be performed at set intervals
to maintain the appropriate dose and monitor for cytopenias. Clinical evaluations, physical
examinations, and/or imaging studies will be conducted every three months to assess for
changes in tumor size. Patients will remain on hydroxyurea as long as there is a response
or stability in tumor size and associated toxicities are manageable. Data will be collected
regarding the response to the drug, the duration of the response, and the occurrence of
adverse events. The goal of the analysis will be to determine the efficacy of hydroxyurea
as a treatment modality for desmoid tumors.

Inclusion Criteria:

- Patients
- Histologically-confirmed diagnosis of desmoid tumor

- Measurable disease

- Stable hematologic, renal and hepatic parameters

- Negative pregnancy test for women of childbearing potential

Exclusion Criteria:

- Presence of a second neoplastic process

- Pregnant or breastfeeding women, fetuses, and prisoners will not be included in this

- Patients receiving additional treatment for tumor other than pain control

- Patients having taken an investigational drug within the past 30 days

Type of Study:


Study Design:

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

The primary outcome of this study is change in tumor size.

Outcome Time Frame:

1 year

Safety Issue:


Principal Investigator

Richard B Womer, M.D.

Investigator Role:

Study Chair

Investigator Affiliation:

Children's Hospital of Philadelphia


United States: Institutional Review Board

Study ID:




Start Date:

October 2009

Completion Date:

January 2015

Related Keywords:

  • Desmoid Tumors
  • Fibromatosis
  • Fibroma
  • Fibromatosis, Aggressive