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NCT01265030 · ClinicalTrials.gov registry record · Phase 1

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

A Phase 1 study of Desmoid Tumor, sponsored by MaineHealth.

Completed
Registry status
Phase 1
Development phase
9
Enrollment target
8
Study locations

NCT01265030 is a Phase 1 study of Desmoid Tumor that has completed, run by MaineHealth. The registered enrollment target is 9 participants, below the 87-participant average among 4 other Desmoid Tumor trials with a reported enrollment target (90% lower). The trial reports 8 study locations across 7 states.

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The verdict

NCT01265030, a Phase 1 study of Desmoid Tumor, has completed, sponsored by MaineHealth.

COMPLETED
Registry status
Phase 1
Development phase
9 participants
Enrollment target
8
Study locations

Study 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 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 tu

Conditions Studied

Interventions

  • DRUG Sirolimus

Study Locations (8)

California

  • UCLA Medical Center - Los Angeles
  • Rady Children's Hospital - San Diego

Florida

  • University of Florida College of Medicine - Gainesville

Maine

  • Maine Medical Center - Portland

Minnesota

  • University of Minnesota - Minneapolis

Missouri

  • Children's Mercy Hospital - Kansas City

Washington

  • Seattle Children's Hospital - Seattle

Wisconsin

  • Medical College of Wisconsin - Milwaukee

Trial Details

FieldValue
Enrollment Target 9 participants
Start Date 2014-02
Est. Completion 2021-12-22
Phase Phase 1

Sponsor

MaineHealth

26 total trials

What the Registry Record Tells You About NCT01265030

The ClinicalTrials.gov registry entry for NCT01265030 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 9 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 87-participant average among 4 other Desmoid Tumor trials with a reported enrollment target (90% lower). The listed sponsor is MaineHealth, which has 26 total studies on file at ClinicalTrials.gov.

The record links to 1 condition, with Desmoid Tumor appearing as the primary indexed condition, and to 1 intervention - of which Sirolimus is the first listed.

NCT01265030 reports 8 study locations spanning 7 distinct geographic areas - top geographies include California, Florida, Maine.

Frequently Asked Questions

What is clinical trial NCT01265030 about?

NCT01265030 is a clinical study titled "A Pilot Study Evaluating the Use of mTor Inhibitor Sirolimus in Children and Young Adults With Desmoid-Type Fibromatosis". 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 tre...

What is the current status of trial NCT01265030?

This trial is currently completed. It is a Phase 1 study. The enrollment target is 9 participants. The study started on 2014-02. Estimated completion is 2021-12-22.

What conditions does trial NCT01265030 study?

This clinical trial studies the following conditions: Desmoid Tumor.

What interventions are being tested in trial NCT01265030?

The interventions under investigation include: Sirolimus (DRUG).

Who is sponsoring clinical trial NCT01265030?

This trial is sponsored by MaineHealth, which has 26 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT01265030 being conducted?

This trial has 8 study locations across California, Florida, Maine, Minnesota, Missouri. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.