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

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 fifteen clinical sites, ten collaborating affiliate sites, and an Operations Center at the University of Alabama at Birmingham under the direction of Dr. Bruce Korf. 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) RAD001 in Combination with Bevacizumab for Patients with Sporadic and NF1-Related Refractory MPNST (Collaboration with the Sarcoma Alliance for Research through Collaboration (SARC))

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
(4) INFUSE Bone Graft for treatment of NF1-Related Tibial Pseudarthrosis
(5) Phase I/II Trial of Ganetespib in Combination with Sirolimus for Patients with Refractory MPNST (Collaboration with SARC)
(6) Phase I/II Study of MEK162 for Children with Low-Grade Gliomas and Other Ras/Raf/MAP Pathway Activated Tumors (Collaboration with non-consortium sites for the NF1-specific cohort)
(7) Phase II Trial of Selumetinib in Combination with Sirolimus for Patients with Refractory MPNST (Collaboration with SARC)

Approved NFCTC Clinical Trials (FY16 NFRP Award)

(1) A Phase II Study of Binimetinib in Children and Adults with NF1 associated Plexiform Neurofibromas
(2) Open-label, Phase 2 Clinical Trial of Crizotinib for Children and Adults with Neurofibromatosis Type 2 and Progressive Vestibular Schwannomas
(3) Phase II Trial of Poly-ICLC for Progressive, Previously Treated Low-Grade Gliomas in Children and Young Adults with Neurofibromatosis Type 1
(4) A Phase 1/2 Trial of the MEK inhibitor selumetinib and bromodomain inhibitor AZD5153 with durvalumab (MEDI4736), a PD-L1 antibody for sarcomas including malignant peripheral nerve sheath tumors

Consortium Sites
(As listed on

Operations Center
University of Alabama at Birmingham

Boston/Harvard Center for NF and Allied Disorders
Children's Hospital at Westmead, University of Sydney
Children's Hospital of Los Angeles
Children's National Medical Center
Children’s Hospital of Philadelphia/University of Pennsylvania
Cincinnati Children's Hospital Medical Center
Indiana University
Mayo Clinic
National Cancer Institute
New York University Medical Center
University of Alabama at Birmingham
University of Chicago
University of Texas Southwestern
University of Utah
Washington University

Affiliate Sites
Ann & Robert H. Lurie Children's Hospital of Chicago
Children's Healthcare of Atlanta/ Emory University
Johns Hopkins Hospital
Massachusetts General Hospital
Dana Farber Cancer Institute
Memorial Sloan Kettering Cancer Center
Royal Children’s Hospital/ Murdoch Children’s Research Institute
Texas Scottish Rite Hospital
University of California, Los Angeles
University of Minnesota

General Publications

Packer RJ, Fisher MJ, Cutter G, et al. 2018. Neurofibromatosis Clinical Trial Consortium. J Child Neurol 33(1): 82-91. doi: 10.1177/0883073817739196.

Gutmann DH, Blakeley JO, Korf BR, et al. 2013. Optimizing biologically targeted clinical trials for neurofibromatosis. Expert Opin Investig Drugs 22(4):443-462.

Last updated Thursday, May 26, 2022