Desmoid TumorsPhase 2NCT00978146
Effect of Hydroxyurea as Treatment for Primary Desmoid Tumors
A Phase 2 study testing Hydroxyurea in people with Desmoid Tumors and Fibromatosis, run by Children's Hospital of Philadelphia.
Trial summary
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. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Desmoid Tumors, Fibromatosis
- What is being tested?
- Hydroxyurea
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Oct 2009to Jan 2015
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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
About this study
Official title: Effect of Hydroxyurea as Treatment for Primary Desmoid Tumors in Adults and Children (CHP-914)
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.
Read the 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.
- Conditions
- Desmoid Tumors, Fibromatosis
- Being tested
- Hydroxyurea
- Medical subject headings
- Desmoid Tumors, Fibroma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HydroxyureaDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Desmoid tumor | Experimental | Patients with desmoid tumors Receives: Drug: Hydroxyurea |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary outcome of this study is change in tumor size.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2009 (estimated)
- Study startOct 2009
- Primary completionJan 2015 (estimated)
- Study completionJan 2015 (estimated)
Why it stopped: Study was administratively withdrawn by the IRB; no subjects were enrolled
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Desmoid TumorsStudies of the same condition, open and under way.
- More trials run by Children's Hospital of PhiladelphiaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Philadelphia
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Richard B Womer, M.D., Study chair, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00978146
- Sponsor's protocol ID
- 09-007317
- Status last verified
- Apr 2017
- First submitted
- 15 Sep 2009
- Last updated
- 20 Apr 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 20 Apr 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Hydroxyurea as Treatment for Primary Desmoid Tumors in Adults and Children (CHP-914)
- ClinicalTrials.gov ID
- NCT00978146
- Acronym / sponsor ID
- 09-007317
- Conditions
- Desmoid Tumors, Fibromatosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study was administratively withdrawn by the IRB; no subjects were enrolled
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Children's Hospital of PhiladelphiaUniversity, hospital or other
- Study start
- Oct 2009
- Primary completion
- Jan 2015 (estimated)
- Study completion
- Jan 2015 (estimated)
- First posted
- 16 Sep 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Desmoid tumor | Experimental | Drug: HydroxyureaPatients with desmoid tumors |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HydroxyureaPatients 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. | Drug | Desmoid tumor |
Primary outcome
The primary outcome of this study is change in tumor size.
Time frame: 1 year
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Apr 2017.
Related trials for Desmoid Tumors
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FOG-001 in Locally Advanced or Metastatic Solid Tumors
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A Study of a New Drug, Nirogacestat, for Treating Desmoid Tumors That Cannot be Removed by Surgery
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Richard B Womer, M.D. study chair
Children's Hospital of Philadelphia
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Hydroxyurea in people with Desmoid Tumors and Fibromatosis, run by Children's Hospital of Philadelphia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 20 Apr 2017. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The primary outcome of this study is change in tumor size. (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Apr 2017.