Ignite Creation Date:
2024-05-05 @ 11:09 PM
Last Modification Date:
2024-10-26 @ 10:29 AM
Study NCT ID:
NCT01265030
Status:
COMPLETED
Last Update Posted:
2023-06-26
First Post:
2010-12-06
Brief Title:
A Pilot Study Evaluating the Use of mTor Inhibitor Sirolimus in Children and Young Adults With Desmoid-Type Fibromatosis
Sponsor:
MaineHealth
Organization:
MaineHealth
Study Overview
Official Title:
A Pilot Study Evaluating the Use of mTor Inhibitor Sirolimus in Children and Young Adults With Desmoid-Type Fibromatosis
Status:
COMPLETED
Status Verified Date:
2023-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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 Childrens 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 Cellbeta-catenin APCβ-catenin pathway This is true of familial cases associated with Gardners Syndrome and also in sporadic desmoid tumor nearly all of which display histological or molecular evidence of Adenomatous Polyposis Cellbeta-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 proliferationsurvival pathway may play an important role in tumor biology when the APCβ-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
Desmoid tumor is well-known to be associated with deregulation of the Adenomatous Polyposis Cellbeta-catenin APCβ-catenin pathway This is true of familial cases associated with Gardners Syndrome and also in sporadic desmoid tumor nearly all of which display histological or molecular evidence of Adenomatous Polyposis Cellbeta-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 proliferationsurvival pathway may play an important role in tumor biology when the APCβ-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
Detailed Description:
We propose a translational research project that will directly test the hypothesis that mTOR is active in desmoid tumor in children and young adults Activity will be assessed by clinical and histological studies following a course of pre-operative chemotherapy using sirolimus Clinical response will be measured using validated pain assessment scales because desmoid tumor size is unlikely to change during the course of pre-operative chemotherapy in this study Histological response will be based on quantifying the phosphorylation of following mTOR targets thr389p-p70S6K p-4E-BP1 and ser473p-AKT
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
True
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 44574 | OTHER_GRANT | Desmoid Tumor Research Foundation | None |