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

The Neurofibromatosis Clinical Trials Consortium (NFCTC, http://www.uab.edu/nfconsortium) 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.

Fiscal Year 2004 (FY04): The NFRP and National Institutes of Health sponsored a workshop in 2004 to discuss and prioritize perceived barriers to the clinical translation of basic NF research and to determine strategies to overcome those barriers. Members of the NFRP FY04-FY05 Integration Panel and other subject matter experts identified the following barriers in NF clinical research: (1) fund/resource availability, (2) patient recruitment and long-term tracking, (3) lack of standardized consent forms, (4) communication among sites in multi-institutional studies, (5) data collection and analysis, (6) tissue banking and distribution, and (7) institutional and Army regulatory approval processes. They recommended the formation of an NF consortium that included an operations center to provide administrative, statistical, data management, regulatory, and protocol writing support to individual clinical sites.

FY05: The NFRP offered funding for two separate award mechanisms to support the development of a NF consortium - the NF Consortium Development Operations Center Award and NF Consortium Development Site Awards. These mechanisms were designed such that the recipient of the FY05 NF Consortium Development Operations Center Award would collaborate with the recipients of the FY05 NF Consortium Development Site Awards to submit an application, clinical protocols, and associated clinical documents to the NF Consortium Award mechanism to be offered the following year. The Development Award was funded for $3M in total costs to the Operations Center and $30K to each Clinical Site.

The University of Alabama at Birmingham received The NF Consortium Development Operations Center Award. In addition, nine NF Consortium Development Site Awards were made to institutions across the United States, including an UAB site.

FY06: The NFRP offered the NF Consortium Award to support a major goal/product-driven consortium of exceptional investigators to conceive, develop, and conduct collaborative pilot and Phase I and II clinical evaluations of promising therapeutic agents or approaches for the management or treatment of NF1. This award mechanism offered up to $6 million (M) in direct costs ($9M total cost); a maximum of $2M could be requested for the Operations Center and up to $4M for the Consortium's clinical studies.

The NFCTC received the FY06 NF Consortium Award to (1) develop innovative biologically based therapies for the more common and/or devastating complications of NF1; (2) develop a framework to expeditiously translate advances in the understanding of the molecular pathogenesis of specific clinical abnormalities seen in patients with NF1 to the effective treatment of these disorders, and (3) implement sequential hypothesis-driven translational trials for multiple NF1 clinical manifestations, building on previous studies, so as to more rapidly improve the outcome for children and adults with NF1.

During its first NFRP funding period, the Consortium focused its activities and directed initial scientific efforts toward four disease-specific areas as outlined in the NF Consortium Award application: (1) plexiform neurofibromas, (2) neurocognitive function, (3) optic gliomas, and (4) malignant peripheral nerve sheath tumors (MPNST).

FY11: The NFRP offered the Clinical Consortium Award to support a consortium of exceptional institutions and investigators to conceive, design, develop, and conduct collaborative Phase I and II clinical evaluations of promising therapeutic agents for the management or treatment of NF1, NF2, and schwannomatosis. This award mechanism offered up to $9M (total cost) to fund the one Operations Center and at least five clinical sites.

The NFCTC received this $9M award with the primary goal to develop, perform, and expeditiously complete biologically based clinical trials for children and adults with significant complications of NF. Expanding on the work performed in the first five years of funding, the Consortium planned to expand its scope of activities to include NF2 and schwannomatosis, with trials aimed at both children and adults.

FY16: The NFRP again offered the Clinical Consortium Award, intended to support a major goal/product-driven Consortium of exceptional institutions and investigators that will accelerate the clinical translation of basic NF research and ultimately decrease the impact of the diseases. This award mechanism offered up to $9M (total cost) to fund the one Operations Center and at least five clinical sites.

The NFCTC will conduct a minimum of four clinical trials during this period of performance. Historically, the Consortium has leveraged funding from others sources, such as institutions, foundations, and pharmaceutical companies to perform more clinical trials than can be supported solely by NFRP funding, and the Consortium is planning to continue with this model moving forward.