Effect of Hydroxyurea as Treatment for Primary Desmoid Tumors

This study has been withdrawn prior to enrollment.
(Study was administratively withdrawn by the IRB; no subjects were enrolled)
Sponsor:
Information provided by:
Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier:
NCT00978146
First received: September 15, 2009
Last updated: June 23, 2011
Last verified: June 2011
  Purpose

The purpose of this study is to examine the response rate of desmoid tumors to hydroxyurea. The investigators hypothesize that hydroxyurea will be a safe, non-toxic alternative to aggressive surgery or chemotherapy for this difficult to treat tumor.


Condition Intervention Phase
Desmoid Tumors
Fibromatosis
Drug: Hydroxyurea
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Effect of Hydroxyurea as Treatment for Primary Desmoid Tumors in Adults and Children

Resource links provided by NLM:


Further study details as provided by Children's Hospital of Philadelphia:

Primary Outcome Measures:
  • The primary outcome of this study is change in tumor size. [ Time Frame: 1 year ] [ Designated as safety issue: No ]

Estimated Enrollment: 10
Study Start Date: October 2009
Estimated Study Completion Date: January 2015
Estimated Primary Completion Date: January 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Desmoid tumor
Patients with desmoid tumors
Drug: Hydroxyurea
Patients will take oral hydroxyurea with a starting dose of 20 mg/kg daily. Patients will remain on hydroxyurea as long as there is a response or stability in tumor size and associated toxicities are manageable. Maximum treatment duration will be one year.

Detailed Description:

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.

  Eligibility

Ages Eligible for Study:   up to 21 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients </= 21 years of age will be eligible
  • 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 study
  • Patients receiving additional treatment for tumor other than pain control
  • Patients having taken an investigational drug within the past 30 days
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00978146

Sponsors and Collaborators
Children's Hospital of Philadelphia
Investigators
Study Chair: Richard B Womer, M.D. Children's Hospital of Philadelphia
  More Information

No publications provided

Responsible Party: Naomi J. Balamuth, M.D., Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier: NCT00978146     History of Changes
Other Study ID Numbers: CHP-914
Study First Received: September 15, 2009
Last Updated: June 23, 2011
Health Authority: United States: Institutional Review Board

Additional relevant MeSH terms:
Fibromatosis, Aggressive
Fibroma
Neoplasms, Fibrous Tissue
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Neoplasms
Hydroxyurea
Antineoplastic Agents
Therapeutic Uses
Pharmacologic Actions
Antisickling Agents
Hematologic Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors

ClinicalTrials.gov processed this record on September 16, 2014