CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 58 enrolled
Drug / intervention
Sirolimusdrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT00634270
NCT00634270Phase 2Completed
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A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas

University of Alabama at Birmingham·interventional·Posted Mar 13, 2008·Updated Nov 17, 2017

In Brief

A Phase 2 clinical trial evaluating Sirolimus for Neurofibromatosis Type 1. Completed, enrolled 58 participants across 9 sites.

Detailed Summary

Treatment Overview This phase II study will evaluate the activity of sirolimus in children and adults with NF1 and inoperable plexiform neurofibromas that have the potential to cause significant morbidity. The following disease strata will be studied: Stratum 1: Progressive plexiform neurofibroma(s) that have the potential to cause significant morbidity. The endpoint will be time to tumor progression based on volumetric tumor measurements. Stratum 2: Plexiform neurofibromas without documented radiographic progression at trial entry. The endpoint will be radiographic response. As of May 2009, Stratum 2 was closed to enrollment. Stratum 1 is active.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsNeurofibromatosis Type 1
CountriesUnited States
CollaboratorsBoston Children's Hospital, Children's Hospital of Philadelphia, Children's National Research Institute, Children's Hospital Medical Center, Cincinnati, National Cancer Institute (NCI), University of Chicago, University of Utah, Washington University School of Medicine

Timeline

Phase 2CompletedFinished
20082009201020112012201320142015201620172018201920202021202220232024202520262027
First PostedMar 13, 2008
Enrollment StartApr 1, 2008
Primary CompletionNov 1, 2014
Study CompletionDec 1, 2015
TodayJul 2, 2026
Enrollment to primary: 6.6 yearsPosted 18.3 years ago

Interventions

Sirolimusdrug

This phase II study will evaluate children and adults with neurofibromatosis type-1 (NF1) and plexiform neurofibromas treated with sirolimus. It is divided in two strata. The first stratum will evaluate time to progression (TTP) in children and adults with NF1 and progressive plexiform neurofibromas with the potential to cause significant morbidity treated with sirolimus. The second stratum will evaluate objective radiographic response to sirolimus in children and adults with NF1 and inoperable plexiform neurofibromas with the potential to cause significant morbidity that do not have documented progression of the PN at time of trial entry.