Clinical Trails

Please be aware that this page may not always be current. Updated information regarding clinical trials can be found searching for "desmoid tumor", “aggressive fibromatosis”, or even “fibromatosis.” at the National Cancer Institute's website.

The following are ongoing or recently closed trials including desmoids:

NEW

Erythromycin Treatment for Readthrough of APC Gene Stop Codon Mutations in Familial Adenomatous Polyposis

This study is currently recruiting participants. 
Last Updated: June 2014
Sponsored by:

Michal Roll

Information provided by: Michal Roll, Tel-Aviv Sourasky Medical Center

Clinical Trials.gov Identifier: NCT02175914


For detailed information click here:

http://www.clinicaltrials.gov/ct2/show/NCT02175914?term=desmoid+tumors&rank=18

Purpose:Colorectal cancer (CRC) is a leading cause for cancer related mortality in the western world with a lifetime risk of 6%. Etiology is complex, while genetic background significantly affects the risk. Around one third of all genetic disorders as well as most cases of Familial Adenomatous Polyposis (FAP) and a large proportion of all sporadic CRC cases occur as a result of premature nonsense mutations (creating a stop codon) in an individual's adenomatous polyposis coli (APC) gene. Nonsense mutations are single-point alterations in the DNA that prematurely halt the protein translation process, producing a shortened, nonfunctional protein. In many of these cases, if the cell can be 'persuaded' to ignore the premature stop codon signal, the resulting protein may be able to ameliorate or stop the disease.

Recently, members of the aminoglycoside family of antibiotics have been found to induce ribosomal read-through of nonsense mutations, leading to expression of a full length, functional protein. Investigators have recently shown that members of the aminoglycoside and macrolide antibiotic families can induce read-through of the nonsense mutations in the APC gene and lead to reduced oncogenic phenotypes in CRC cells and in different mice models.

The aim of this project is to determine the ability of the macrolide antibiotic-erythromycin to induce read-through of the nonsense mutations in the APC gene and to induce expression of a full length, functional APC protein in patients suffering from FAP and to tests its effect on adenoma number and size and on desmoid tumors in these patients. The future goal is to maximize the effect of stop-codon suppressors on APC while minimizing side effects.

In this study investigators will select FAP patients which carry APC nonsense mutations, treat them with erythromycin PO for 4-6 months and examine colonic and duodenal adenomas as well as abdominal desmoid tumors, that will be documented before during and after treatment. In parallel, investigators will test polyp, adenoma and desmoid tissue samples as well as blood samples from these patients for changes in expression levels of the APC protein and related oncogenic markers.

Suppression of nonsense mutations within the APC gene should be of benefit for patients suffering from FAP, attenuated FAP or multiple adenomas and for patients with advanced or diffuse CRC. Furthermore, given the rapid progress being made in the identification of different nonsense mutations in human genes that lead to mostly un-curable disease, the identification of clinically approved compounds that suppress nonsense mutations and that can be administered long-term without significant side effects would open new venues in the treatment of genetic human diseases that arise from pre-mature stop codons in important coding sequences.

Immediate goal: establish the ability of erythromycin to read-through APC nonsense mutation in FAP patients. The read-through effect of erythromycin will be clinically tested by counting and measuring the number and size of both colonic and duodenal adenomas before and over treatment and by measuring the size of known desmoid tumors. Samples of the adenomas and desmoid tumors will be tested by western blot, immunofluorescence and immunohistochemistry for restoration of APC expression and changes in oncogenic markers. These experiments should be conducted within 6 month.


Eligibility:

  • 18 years and older
  • Both genders eligible
  • Will not accept healthy volunteers
Please see site for other eligibility requirements. 

 





 

NEW

Sorafenib Tosylate in Treating Patients With Desmoid Tumors or Aggressive Fibromatosis

This study is currently recruiting participants. 
Last Updated: November 2014
Sponsored by:

National Cancer Institute (NCI)

Information provided by: Mrinal Gounder, MSKCC

Clinical Trials.gov Identifier: NCT02066181


For detailed information click here:

http://clinicaltrials.gov/ct2/show/NCT02066181?term=desmoid+tumors&rank=3
Purpose: This randomized phase III trial compares the effects, good and/or bad, of sorafenib tosylate in treating patients with desmoid tumors or aggressive fibromatosis. Sorafenib tosylate may stop the growth of tumor cells by blocking some of the proteins needed for cell growth.  

Eligibility:

  • 18 years and older
  • Both genders eligible
  • Will not accept healthy volunteers
Please see site for other eligibility requirements. 

 





 

NEW

A Pilot Study Evaluating the Use of mTor Inhibitor Sirolimus in Children and Young Adults With Desmoid-Type Fibromatosis

This study is currently recruiting participants. 
Last Updated: February 2014

Sponsored by:

Maine Medical Center

Information provided by:

Aaron Weiss, Maine Medical Center

Clinical Trials.gov Identifier:

NCT01265030

For detailed information click here:

http://www.clinicaltrials.gov/ct2/show/NCT01265030?term=nct01265030&rank=1

Purpose: Desmoid-type fibromatosis (or desmoid tumor) represents an intermediate grade neoplasm with a striking predilection for locally invasive growth and recurrence following resection. It occurs in children as well as young adults. As a typically localized disease, the historical standard of care for treatment has been surgical resection, with or without ionizing radiation. In some cases where surgical resection or radiation is not feasible, chemotherapy has been used. Two clinical trials conducted in the Pediatric Oncology Group (POG) and the Children's Oncology Group (COG) evaluated the role for either low intensity or non-cytotoxic chemotherapy for children with desmoid tumor that is not amenable to standard therapy. These were largely empirical treatment strategies or based on somewhat anecdotal observations. By better understanding desmoid tumor biology, even more effective therapy targeting a particular protein that is central to the disease can be developed.

Desmoid tumor is well-known to be associated with deregulation of the Adenomatous Polyposis Cell/beta-catenin (APC/β-catenin pathway). This is true of familial cases associated with Gardner's Syndrome and also in sporadic desmoid tumor, nearly all of which display histological or molecular evidence of Adenomatous Polyposis Cell/beta-catenin (APC β-catenin) pathway activation (Alman et al., 1997; Lips et al., 2009). Several new pieces of evidence support the concept that deregulation of the mammalian target of rapamycin (mTOR) cell proliferation/survival pathway may play an important role in tumor biology when the APC/β-catenin pathway is disrupted. Sirolimus, a drug that inhibits mammalian target of rapamycin (mTOR), is currently being evaluated as an anti-cancer agent in a variety of tumor types, but it has not been previously studied in desmoid tumor.

The investigators are conducting this pilot study to begin to explore whether mTOR inhibition may be beneficial for children and young adults with desmoid tumor.  

Eligibility:

  • Age up to 29 years
  • Both genders eligible
  • Will not accept healthy volunteers
Please see site for other eligibility requirements. 

 





 


Trial of Eflornithine Plus Sulindac in Patients with Familial Adenomatous Polyposis (FAP)

This study is currently recruiting participants. 
Last Updated: October 2014
Sponsored by:

Cancer Prevention Pharmaceuticals, Inc.

Information provided by: Cancer Prevention Pharmaceuticals, Inc.

Clinical Trials.gov Identifier: NCT01483144


For detailed information click here:

http://www.clinicaltrials.gov/ct2/show/NCT01483144?term=FAP&rank=6
Purpose: The purpose of this randomized, double-blind, Phase III trial is to determine if the combination of eflornithine plus sulindac is superior to sulindac or eflornithine as single agents in delaying time to the first occurrence of any FAP-related event. This includes: 1) FAP related disease progression indicating the need for excisional intervention involving the colon, rectum, pouch, duodenum and/ or 2) clinically important events which includes progression to more advanced duodenal polyposis, cancer or death.  

Eligibility:

  • Age greater than or equal to 18
  • Both genders eligible
  • Will not accept healthy volunteers
Please see site for other eligibility requirements. 

 





 


Phase II Trial of the Gamma-Secretase Inhibitor PF-03084014 in Adults With Desmoid Tumors/Aggressive Fibromatosis 

This study is active, but not currently recruiting participants.
Last Updated: October 2014
Sponsored by: National Cancer Institue

Information provided by: National Insititutes of Health Clinical Center (CC)
Clinical Trials.gov Identifier: NCT01981551

For detailed information click here:



http://clinicaltrials.gov/ct2/show/NCT01981551?term=kummar&rank=9 
Background:
  • Desmoid tumors (also known as aggressive fibromatosis), are rare, locally invasive, slow-growing soft-tissue tumors. The disease can be either asymptomatic or be associated with severe loss of organ function and significant morbidity.
  • Treatment with the selective small-molecule Gamma-secretase inhibitor PF-03084014 caused significant tumor shrinkage in patients with unresectable desmoid tumors in an early phase clinical trial.
  • The Notch pathway is a key regulator of cell differentiation, proliferation and apoptosis; aberrant signaling via the Notch pathway is associated with carcinogenesis.

Objectives:

  • Primary: Determine the response rate (CR+PR) of PF-03084014 in patients with desmoid tumors/aggressive fibromatosis
  • Secondary: Assess symptom measures at baseline and on study; perform genotyping for germline and somatic mutations in APC and CTNNB1 genes; correlate clinical response to therapy with genotyping data; and assess modulation of the Notch pathway by evaluating notch response genes in tumor biopsies at baseline and after drug administration

Eligibility:

  • Age greater than or equal to18; histologically confirmed desmoid tumor not amenable to curative resection or definitive radiation therapy that has progressed after receiving at least one line of standard treatment; adequate organ function
  • Willingness to provide blood samples and 10 unstained slides or a tumor block for genetic research studies

 

 



 


PAZOPANIB Efficacy and Tolerance in Desmoids Tumors (DESMOPAZ)

This study is currently recruiting participants. 
Last Updated: June 2013
Sponsored by:

Institut Bergonié

Information provided by: Institut Bergonié

Clinical Trials.gov Identifier: NCT01876082


For detailed information click here:

http://clinicaltrials.gov/ct2/show/NCT01876082?term=desmoid&rank=9
Purpose: Desmoids tumors are benign soft tissues tumors characterized by aggressiveness and potential local recurrence. There is a female predominance with a sex ratio of 2/1 and median age at diagnosis is about 30 years.

Only a complete surgical excision is recommended in desmoids tumors. Some forms of desmoid tumors are recurrent and/or symptomatic and are not accessible to a conservative surgical treatment. In these clinical situations, only a medical treatment may achieve tumor control and quality of life maintenance. Place of systemic treatments in the management of desmoids tumors is poorly evaluated. Regarding chemotherapy, methotrexate and vinblastine protocol is actually the best evaluated combination, which allowed observing objective response rate between 40 and 75%. Toxicity was mainly marked by the risk of haematological toxicity.

Pazopanib is an inhibitor of multi-target tyrosine kinase, in oral form, with selective type receptors -1, -2 and -3 of VEGF receptors on the PDGFA and B, and c-Kit. It is currently under clinical development in humans in the treatment of several tumor types.  

Eligibility:

  • 18 years and older
  • Both genders eligible
  • Will not accept healthy volunteers
Please see site for other eligibility requirements. 

 





 


Endoxifen in Adults with Hormone Receptor-Positive Solid Tumors 

This study is currently recruiting participants. 
Last Updated: July 2014
Sponsored by: National Cancer Institute
Information provided by: National Insititutes of Health Clinical Center (CC)
Clinical Trials.gov Identifier: NCT01273168

For detailed information click here:



 http://www.clinicaltrials.gov/ct2/show/NCT01273168?term=desmoid&rank=9

Background:

Some types of cancer cells that have hormone receptors on their surfaces need the hormone estrogen to grow. The drug tamoxifen blocks estrogen from binding to the tumor cells, which helps to slow or stop the growth of cancer. Tamoxifen has been approved for treatment of certain types of estrogen-linked cancers, such as breast and ovarian cancer.

The experimental drug Z-Endoxifen HCl (endoxifen) is related to tamoxifen, and has been shown to work against similar estrogen-linked cancers. In many cancer patients, tamoxifen is turned into endoxifen by enzymes in the liver; however, not all people have the liver enzymes that can turn tamoxifen into endoxifen, which means that the drug cannot work properly. Taking certain other drugs at the same time as tamoxifen can also keep it from turning into endoxifen. Researchers are interested in determining whether endoxifen tablets are effective in slowing or stopping tumor growth in individuals whose hormone-linked tumors have not responded to standard treatment.

Objectives:

To test the safety and effectiveness of daily endoxifen in individuals with hormone receptor-positive solid tumors that have not responded to standard treatment.

Eligibility:

Individuals at least 18 years of age who have been diagnosed with hormone receptor-positive solid tumors (breast or other tumors), desmoid tumors, or gynecologic tumors that have not responded to standard treatment.



  


Trial In Pediatric Patients With Familial Adenomatous Polyposis (FAP) (CHIP)

This study has been terminated and has results.
Last Updated: October 2014
Sponsored by:

Pfizer

Information provided by: Pfizer
Clinical Trials.gov Identifier: NCT00585312

For detailed information click here:

http://clinicaltrials.gov/ct2/show/NCT00585312

To compare the time from randomization to treatment failure over a 5 year period in subjects treated with celecoxib vs placebo.

The trial is currently enrolling subjects (10 to 17 years of age) with FAP and an intact colon into a study assessing the effectiveness and safety of Celebrex versus placebo in the prevention of colorectal polyps. If interested call Carol Burke, M. D. at 216/444-6864 or Hennie Hasson, R.N. at 216/444-6975.


Study to Evaluate Imatinib in Desmoid Tumors

This study is active, but currently not recruiting participants.
Last Updated: March, 2014
Sponsored by:

University of Heidelberg / Novartis

Information provided by: University of Heidelberg
Clinical Trials.gov Identifier: NCT01137916

For detailed information click here:

http://clinicaltrials.gov/show/NCT01137916

A Trial In Patients With Advanced Cancer And Leukemia

This study is ongoing, but not recruiting participants.
Last Updated: November, 2014
Sponsored by:

Pfizer

Information provided by: Pfizer
Clinical Trials.gov Identifier: NCT00878189

For detailed information click here:

http://clinicaltrials.gov/show/NCT00878189

Imatinib in Patients With Desmoid Tumor and Chondrosarcoma (Basket 1)

This study is currently recruiting participants.
Last Updated: September, 2014
Sponsored by:

Italian Sarcoma Group

Information provided by: Italian Sarcoma Group
Clinical Trials.gov Identifier: NCT00928525

For detailed information click here:

http://www.clinicaltrials.gov/ct2/show /NCT00928525


Collecting and Storing Tissue, Blood, and Bone Marrow Samples From Patients With Rhabdomyosarcoma or Other Soft Tissue Sarcoma

This study is currently recruiting participants.
Last Updated: August, 2014
Sponsored by:

Children's Oncology Group National Cancer Institute (NCI)

Information provided by: National Cancer Institute (NCI)
Clinical Trials.gov Identifier: NCT00919269

For detailed information click here:

http://www.clinicaltrials.gov/ ct2/show/NCT00919269


Sulindac and Tamoxifen in Treating Patients with Desmoid Tumors (Phase II)

This study is ongoing, but not currently recruiting participants.
Last Updated: September, 2014
Sponsored by:

Children's Oncology Group
National Cancer Institute (NCI)

Information provided by: National Cancer Institute (NCI)
Clinical Trials.gov Identifier: NCT00068419

For detailed information click here:

http://www.clinicaltrials.gov/ct2/show/ NCT00068419

 

A Pilot Study Evaluating the Use of mTor Inhibitor Sirolimus in Children and Young Adults With Desmoid-Type Firbromatosis

This study is currently recruiting participants.
Last Updated: February 2014
Sponsored by:

University of Chicago

Information provided by: University of Chicago
Clinical Trials.gov Identifier: NCT01265030

For detailed information click here:

http://clinicaltrials.gov/show/NCT01265030

Imatinib Mesylate And Cyclophosphamide In Metronomic Administration: Dose Escalation Study Of Imatinib Mesylate (PALANGI3)

This study has been completed. 
Last Updated: June, 2012
Sponsored by:

Centre Oscar Lambret

Information provided by: Centre Oscar Lambret
Clinical Trials.gov Identifier: NCT01046487

For detailed information click here:

http://clinicaltrials.gov/show/NCT01046487

Radiation Therapy in Treating Patients with Aggressive Fibromatoses (Phase II)

This study has been completed.
Last Updated: August, 2013
Sponsored by:

European Organization for Research and Treatment of Cancer

Information provided by: National Cancer Institute (NCI)
Clinical Trials.gov Identifier: NCT00030680

For detailed information click here:

http://www.clinicaltrials.gov/ct2/show /NCT00030680

Fentanyl Sublingual Spray in Treating Patients With Breakthrough Cancer Pain

This study has been completed.
Last Updated: January, 2014
Sponsored by:

Insys Therapeutics Inc

Information provided by: National Cancer Institute (NCI)
Clinical Trials.gov Identifier: NCT00538850

For detailed information click here:

http://clinicaltrials.gov/show/NCT00538850

MS-275 in Treating Patients With Advanced Solid Tumors or Lymphoma

This study has been completed.
Last Updated: March, 2012
Sponsored by:

National Cancer Institute (NCI)

Information provided by: National Cancer Institute (NCI)
Clinical Trials.gov Identifier: NCT00020579

For detailed information click here:

http://clinicaltrials.gov/show/NCT00020579

Vinblastine and Methotrexate in Treating Children With Desmoid Tumors (Phase II)

This study has been completed.
Last Updated: July, 2014
Sponsored by:

National Cancer Institute (NCI)
Children's Cancer Group
Pediatric Oncology Group

Information provided by: National Cancer Institute (NCI)
Clinical Trials.gov Identifier: NCT00003019

For detailed information click here:

http://www.clinicaltrials.gov/ct2/ show/NCT00003019


Toremifene in Treating Patients With Desmoid Tumors (Phase II)

This study has been completed
Last Updated: July, 2013
Sponsored by:

Lutheran General Hospital

Information provided by: National Cancer Institute (NCI)
Clinical Trials.gov Identifier: NCT00002595

For detailed information click here:

http://www.clinicaltrials.gov/ ct2/show/NCT00002595


Sunitinib in Treating Patients With Metastatic, Locally Advanced, or Locally Recurrent Sarcomas (Phase II)

This study has been completed.
Last Updated: March, 2013
Sponsored by:

Memorial Sloan-Kettering Cancer Center National Cancer Institute (NCI)

Information provided by: National Cancer Institute (NCI)
Clinical Trials.gov Identifier: NCT00474994

For detailed information click here:

http://www.clinicaltrials.gov/ct2/show/ NCT00474994