Overview | Consortium History | Management and Oversight | Research Site Details | Approved Trial Details

The Neurofibromatosis Clinical Trials Consortium (NFCTC, was established by the Department of Defense Neurofibromatosis Research Program (NFRP) to develop and perform clinical trials for the treatment of neurofibromatosis (NF) complications in children and adults. The Consortium is composed of thirteen clinical sites, five collaborating sites, and an Operations Center at the University of Alabama at Birmingham under the direction of Dr. Bruce Korf1. The purpose of the Operations Center is to provide administrative, data management, and statistical support to the NFCTC. Each of the clinical and collaborating sites has expertise in the treatment and management of NF and an established patient population available for clinical trials.

Approved NFCTC Clinical Trials (FY06 NFRP Award)

(1) STOPN: Sirolimus for the Treatment of NF1-Related Plexiform Neurofibromas
(2) STARS: Lovastatin for the Treatment of Learning Disabilities in Children with NF1
(3) RAD001: RAD001 for Children with NF1 and Chemotherapy-Refractory Radiographic Progressive Low-Grade Gliomas
(4) MPNST: RAD001 in Combination with Bevacizumab for Patients with Sporadic and NF1-Related Refractory MPNST

Approved NFCTC Clinical Trials (FY11 NFRP Award)

(1) Bevacizumab for NF2-Related Progressive Vestibular Schwannomas
(2) Cabozantinib (XL184) for NF1-Related Plexiform Neurofibromas
(3) PD-0325901 for NF1-Related Plexiform Neurofibromas

Consortium Sites
(As listed on

Operations Center
(1) University of Alabama at Birmingham

Original Sites
(2) Boston/Harvard Center for NF and Allied Disorders
(3) Children's National Medical Center
(4) Cincinnati Children's Hospital Medical Center
(5) National Cancer Institute
(6) University of Chicago
(7) Children's Hospital of Pennsylvania
(8) University of Utah
(9) Washington University

New Sites
(10) Children's Hospital at Westmead, University of Sydney
(11) Indiana University
(12) New York University Medical Center
(13) Children's Hospital of Los Angeles

Affiliate Sites
(A1) Ann & Robert H. Lurie Children's Hospital of Chicago
(A2) John Hopkins Hospital
(A3) Massachusetts General Hospital
(A4) Dana Farber Cancer Institute
(A5) University of California Los Angeles

1Originally, Dr. Jeannette Lee