Desmoid TumorPhase 2NCT00068419
Sulindac and Tamoxifen in Treating Patients With Desmoid Tumor
A Phase 2 study testing tamoxifen citrate, sulindac and laboratory biomarker analysis in people with Desmoid Tumor, run by Children's Oncology Group at 1 site in United States.
Trial summary
This phase II trial is studying how well giving sulindac together with tamoxifen works in treating patients with desmoid tumor. Sulindac may stop the growth of cancer cells by blocking the enzymes necessary for cancer cell growth. Hormone therapy using tamoxifen may fight cancer by blocking the use of estrogen. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Desmoid Tumor
- What is being tested?
- tamoxifen citrate, sulindac and laboratory biomarker analysis
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2004to 26 Apr 2010
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Histologically confirmed desmoid tumor, meeting 1 of the following criteria:
- Newly diagnosed disease
- Not previously treated
- Not amenable to complete surgical resection and/or radiotherapy
- If surgical resection was attempted, there must be gross residual disease measurable by MRI
- Radiographically documented recurrent or progressive disease
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- No prior chemotherapy or radiotherapy for the present recurrence
- Tumors that progressed on prior chemotherapy are allowed provided patients have not received chemotherapy for this recurrence
- Measurable disease by gadolinium-enhanced MRI
- No other fibroblastic lesions or fibromatoses
- Lipofibromatosis or desmoplastic fibroma of the bone allowed
- Performance status - Karnofsky Score 50-100% (patients over age 16)
- Performance status - Lansky Score 50-100% (patients age 16 and under)
- At least 8 weeks
- Absolute neutrophil count at least 1,000/mm^3
- Platelet count at least 100,000/mm^3 (transfusion independent)
- Hemoglobin at least 10.0 g/dL (transfusion allowed)
- No hemophilia
- No von Willebrand disease
- No other clinically significant bleeding diathesis
- Bilirubin no greater than 1.5 times upper limit of normal (ULN)
- Alanine aminotransferase (ALT) less than 2.5 times ULN
- Creatinine adjusted according to age as follows:
- No greater than 0.4 mg/dL (≤ 5 months)
- No greater than 0.5 mg/dL (6 months -11 months)
- No greater than 0.6 mg/dL (1 year-23 months)
- No greater than 0.8 mg/dL (2 years-5 years)
- No greater than 1.0 mg/dL (6 years-9 years)
- No greater than 1.2 mg/dL (10 years-12 years)
- No greater than 1.4 mg/dL (13 years and over [female])
- No greater than 1.5 mg/dL (13 years to 15 years [male])
- No greater than 1.7 mg/dL (16 years and over [male])
- Creatinine clearance or radioisotope glomerular filtration rate at least 70 mL/min
- No prior deep venous thrombosis
- Electrocardiogram (EKG) normal
- Chest x-ray normal
- No prior significant gastrointestinal hemorrhage
- No prior peptic ulcer disease
- Not pregnant or nursing
- Fertile patients must use effective nonhormonal contraception
- No evidence of active graft-versus-host disease
- No allergy to aspirin
- Recovered from prior immunotherapy
- At least 7 days since prior anticancer biologic agents
- At least 6 months since prior allogeneic stem cell transplantation
- More than 1 week since prior growth factors
- No concurrent immunomodulating agents
- No prior nonsteroidal anti-inflammatory drugs (NSAIDs) for desmoid tumor
- More than 2 weeks since prior myelosuppressive chemotherapy (4 weeks for nitrosoureas) and recovered
- No concurrent anticancer chemotherapy
- No prior estrogen antagonists for desmoid tumor
- No concurrent hormonal contraceptives
- No concurrent steroids except for non tumor indications (e.g., asthma or severe allergic reactions)
- No concurrent NSAIDs for desmoid tumor
- Occasional NSAIDs for musculoskeletal or other pain are allowed
- Recovered from prior radiotherapy
- No concurrent adjuvant radiotherapy
- No concurrent participation in another COG therapeutic study
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Oncology Group
Monrovia, California, 91016
About this study
Official title: A Phase II Study of Sulindac and Tamoxifen in Patients With Desmoid Tumors That Are Recurrent or Not Amenable to Standard Therapy
This phase II trial is studying how well giving sulindac together with tamoxifen works in treating patients with desmoid tumor. Sulindac may stop the growth of cancer cells by blocking the enzymes necessary for cancer cell growth. Hormone therapy using tamoxifen may fight cancer by blocking the use of estrogen. Combining sulindac with tamoxifen may kill more cancer cells.
Read the detailed description
PRIMARY OBJECTIVES:
- To estimate the safety and efficacy of sulindac and tamoxifen in patients with recurrent desmoid tumor (DT) and primary DT that is not readily amenable to surgery or radiation therapy.
SECONDARY OBJECTIVES:
- Determine the tumor response rate in patients treated with this regimen.
II. Correlate changes in Magnetic Resonance Imaging (MRI) signal features of the tumor with clinical outcome in patients treated with this regimen.
III. Correlate pathological studies of cyclooxygenase-2 (COX-2) and estrogen/progesterone receptor expression in the tumor with clinical outcome in patients treated with this regimen.
IV. Collect information about clinical factors that make a tumor unresectable at diagnosis and resectable during the four courses of study treatment.
- Determine whether short-term endocrine toxicity is associated with treatment with this regimen in these patients.
OUTLINE: This is a multicenter study.
Patients receive oral sulindac and oral tamoxifen twice daily for up to 12 months (four 3-month courses) in the absence of disease progression or unacceptable toxicity. Patients who achieve a complete response (CR) receive 1 additional month of treatment beyond documentation of CR.
After completion of study treatment, patients are followed for 5 years.
- Conditions
- Desmoid Tumor
- Being tested
- tamoxifen citrate, sulindac, laboratory biomarker analysis
- Medical subject headings
- Desmoid Tumors
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
tamoxifen citrateDrug
Also called Nolvadex, TAM, tamoxifen, TMX
Given orally
sulindacDrug
Also called Aflodac, Algocetil, Clinoril, SULIN
Given orally
laboratory biomarker analysisOther
Correlative studies
| Study group | Role | What they receive |
|---|---|---|
| Treatment (enzyme inhibitor therapy, anti-estrogen therapy) | Experimental | Patients receive oral sulindac and oral tamoxifen citrate twice daily for up to 12 months (four 3-month courses) in the absence of disease progression or unacceptable toxicity. Patients who achieve a complete response (CR) receive 1 additional month of treatment beyond documentation of CR. Receives: Drug: tamoxifen citrate; Drug: sulindac; Other: laboratory biomarker analysis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Patients Failure Free at 2 Years Following Study Entry
Kaplan Meier estimate of failure free survival at 2 years, where failure free survival is defined as the time to relapse, progression, second malignancy, and death whichever occurs first.
Measured: Up to 2 years
Percentage of Patients Experiencing a Grade 3 or Higher Adverse Event During Therapy.
The percentage of patients experiencing a grade 3 or higher adverse event as assessed by the National Cancer Institute Common Toxicity Terminology for Adverse Events v3.0
Measured: Up to 12 months
Secondary outcomes (4)
Percentage of Patients With Tumor Response From Imaging
Percentage of patients with a tumor response where tumor response is assessed according to Response Evaluation Criteria in Solid Tumors (RECIST)
Measured: Baseline up to 5 years
Mean Change in Response Measured by MRI
The mean change in response measured by MRI. Response is assessed by the lesion size which is derived from the sum of the longest of the three orthogonal diameters (from MRI) of each target lesion.
Measured: From baseline to up to 5 years
Percentage of Patients Failure Free at 2 Years by Pathological Response
The failure free survival is compared by the log-rank test between patient subgroups defined by pathological response of cyclooxygenase-2 (COX-2) and estrogen/progesterone receptor expression
Measured: From enrollment to up to 2 years
Percentage of Patients Experiencing Short-term Endocrine Toxicity
The percentage of patients experiencing short-term endocrine toxicity between treatment groups is compared using the chi-square test
Measured: At study entry
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Sep 2003 (estimated)
- Study startFeb 2004
- Primary completion26 Apr 2010
- Study completion26 Apr 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Desmoid TumorStudies of the same condition, open and under way.
- More trials run by Children's Oncology GroupThe 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
- 70 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Oncology Group
- Sponsor type
- Research network
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Stephen Skapek, MD, Principal investigator, Children's Oncology Group
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00068419
- Sponsor's protocol ID
- ARST0321
- Other IDs
- NCI-2009-00424, CDR0000322260, COG-ARST0321, U10CA098543
- Status last verified
- Feb 2019
- First submitted
- 10 Sep 2003
- Last updated
- 19 Feb 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 19 Feb 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Sulindac and Tamoxifen in Patients With Desmoid Tumors That Are Recurrent or Not Amenable to Standard Therapy
- ClinicalTrials.gov ID
- NCT00068419
- Acronym / sponsor ID
- ARST0321
- Conditions
- Desmoid Tumor
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 70 participants (actual)
- Lead sponsor
- Children's Oncology GroupResearch network
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Feb 2004
- Primary completion
- 26 Apr 2010 (actual)
- Study completion
- 26 Apr 2010 (actual)
- First posted
- 11 Sep 2003 (estimated)
- Results
- Posted 4 Dec 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase II trial is studying how well giving sulindac together with tamoxifen works in treating patients with desmoid tumor. Sulindac may stop the growth of cancer cells by blocking the enzymes necessary for cancer cell growth. Hormone therapy using tamoxifen may fight cancer by blocking the use of estrogen. Combining sulindac with tamoxifen may kill more cancer cells.
Detailed description
PRIMARY OBJECTIVES:
- To estimate the safety and efficacy of sulindac and tamoxifen in patients with recurrent desmoid tumor (DT) and primary DT that is not readily amenable to surgery or radiation therapy.
SECONDARY OBJECTIVES:
- Determine the tumor response rate in patients treated with this regimen.
II. Correlate changes in Magnetic Resonance Imaging (MRI) signal features of the tumor with clinical outcome in patients treated with this regimen.
III. Correlate pathological studies of cyclooxygenase-2 (COX-2) and estrogen/progesterone receptor expression in the tumor with clinical outcome in patients treated with this regimen.
IV. Collect information about clinical factors that make a tumor unresectable at diagnosis and resectable during the four courses of study treatment.
- Determine whether short-term endocrine toxicity is associated with treatment with this regimen in these patients.
OUTLINE: This is a multicenter study.
Patients receive oral sulindac and oral tamoxifen twice daily for up to 12 months (four 3-month courses) in the absence of disease progression or unacceptable toxicity. Patients who achieve a complete response (CR) receive 1 additional month of treatment beyond documentation of CR.
After completion of study treatment, patients are followed for 5 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (enzyme inhibitor therapy, anti-estrogen therapy) | Experimental | Drug: tamoxifen citrate, Drug: sulindac, Other: laboratory biomarker analysisPatients receive oral sulindac and oral tamoxifen citrate twice daily for up to 12 months (four 3-month courses) in the absence of disease progression or unacceptable toxicity. Patients who achieve a complete response (CR) receive 1 additional month of treatment beyond documentation of CR. |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (58 listed)
- Histologically confirmed desmoid tumor, meeting 1 of the following criteria:
- Measurable disease by gadolinium-enhanced MRI
- No other fibroblastic lesions or fibromatoses
- Performance status - Karnofsky Score 50-100% (patients over age 16)
- Performance status - Lansky Score 50-100% (patients age 16 and under)
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| tamoxifen citrateAlso: Nolvadex, TAM, tamoxifen, TMXGiven orally | Drug | Treatment (enzyme inhibitor therapy, anti-estrogen therapy) |
| sulindacAlso: Aflodac, Algocetil, Clinoril, SULINGiven orally | Drug | Treatment (enzyme inhibitor therapy, anti-estrogen therapy) |
| laboratory biomarker analysisCorrelative studies | Other | Treatment (enzyme inhibitor therapy, anti-estrogen therapy) |
Primary outcome
Percentage of Patients Failure Free at 2 Years Following Study Entry
Time frame: Up to 2 years
Kaplan Meier estimate of failure free survival at 2 years, where failure free survival is defined as the time to relapse, progression, second malignancy, and death whichever occurs first.
Percentage of Patients Experiencing a Grade 3 or Higher Adverse Event During Therapy.
Time frame: Up to 12 months
The percentage of patients experiencing a grade 3 or higher adverse event as assessed by the National Cancer Institute Common Toxicity Terminology for Adverse Events v3.0
4 secondary outcomes
Percentage of Patients With Tumor Response From Imaging
Time frame: Baseline up to 5 years
Mean Change in Response Measured by MRI
Time frame: From baseline to up to 5 years
Percentage of Patients Failure Free at 2 Years by Pathological Response
Time frame: From enrollment to up to 2 years
Percentage of Patients Experiencing Short-term Endocrine Toxicity
Time frame: At study entry
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Feb 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Stephen Skapek, MD principal investigator
Children's Oncology Group
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing tamoxifen citrate, sulindac and laboratory biomarker analysis in people with Desmoid Tumor, run by Children's Oncology Group at 1 site in United States.
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 58 inclusion and 0 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: finished, as of the sponsor's last update on 19 Feb 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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?
Yes. Results are posted in the registry (first posted 4 Dec 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Percentage of Patients Failure Free at 2 Years Following Study Entry (time frame: Up to 2 years). The study lists 2 primary outcomes in all. 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 19 Feb 2020.