Trial intelligence / Profile preview

Sirolimus in NF1 Related Plexiform Neurofibromas

NCT00634270Phase 2

Key facts

Status: Completed
Start: Apr 2008
Primary completion: Nov 2014
Countries: 1
Sponsor: University of Alabama at Birmingham
Modality: Small Molecules

Drugs: sirolimus
Conditions: neurofibromatosis type 1 (NF1)

Overview

Phase 2, open-label, single-arm trial of sirolimus in neurofibromatosis type 1 (NF1), sponsored by University of Alabama at Birmingham. Primary endpoints: Time to Disease Progression Based on Volumetric MRI at 24 months, Objective Radiographic Responses Based on Volumetric MRI Measurements at 48 weeks and 2 more. Started Apr 2008; status: completed.

Design

1 arm

Study design: Non-randomizedOpen-label

Sirolimus
Sirolimus oral solution administered orally BID on a continuous dosing schedule.

Who it enrols

Patients >= 3 years old with NF1 and inoperable plexiform neurofibromas (PN) that have the potential to cause significant morbidity. Stratum 1 requires progressive PN, while Stratum 2 does not require documented progression. Patients must have adequate organ function and no prior mTOR inhibitor treatment.

Endpoints

Primary

Time to Disease Progression Based on Volumetric MRI24 months
Objective Radiographic Responses Based on Volumetric MRI Measurements48 weeks
Toxicity48 weeks
Pharmacokinetic Profile of Sirolimus (Clearance)Day 1

Secondary

Quality of Life During Treatment48 weeks
Reduction in Pain48 weeks
p70s6 Kinase Activity in Peripheral Blood Mononuclear Cells48 weeks

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