A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas

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.

Conditions

• Neurofibromatosis Type 1

Interventions

DRUG

Sirolimus

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.

Sponsors & Collaborators

• Boston Children's Hospital

  collaborator OTHER

• Children's Hospital of Philadelphia

  collaborator OTHER

• Children's National Research Institute

  collaborator OTHER

• Children's Hospital Medical Center, Cincinnati

  collaborator OTHER

• National Cancer Institute (NCI)

  collaborator NIH

• University of Chicago

  collaborator OTHER

• University of Utah

  collaborator OTHER

• Washington University School of Medicine

  collaborator OTHER

• University of Alabama at Birmingham

  lead OTHER

Principal Investigators

• Bruce Korf, MD · The University of Alabama at Birmingham

• Brian Weiss, MD · Children's Hospital Medical Center, Cincinnati

• Roger Packer, MD · Children's National Medical Center - Chairman of the NF Consortium

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

3 Years

Max Age

75 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2008-04-30

Primary Completion

2014-11-30

Completion

2015-12-31

Countries

• United States

Study Locations