Posted January 10, 2023

Neurofibromatosis Clinical Trials Consortium
Dr. Bruce Korf, University of Alabama, Birmingham

For fiscal year 2021 (FY21), the Congressionally Directed Medical Research Programs (CDMRP) is funding research through an Other Transaction Agreement (OTA), a first for the Neurofibromatosis Research Program (NFRP). This type of research agreement provides additional flexibility above and beyond that of a traditional grant or contract. Some benefits and features include increased opportunities for information exchange, cost sharing, and negotiation of intellectual property and data rights. This new OTA is funding the Neurofibromatosis Clinical Trials Consortium (NFCTC), which was established by the Department of Defense’s NFRP in 2006 to develop and perform phase I and II clinical trials for the management and treatment of Neurofibromatosis (NF) complications in children and adults. The consortium includes a Steering Committee led by Dr. Michael Fisher at the Children’s Hospital of Philadelphia and an Operations Center at the University of Alabama at Birmingham, under the direction of Dr. Bruce Korf. The NFCTC is further rounded out with a Data Coordinating Center at the Children’s Hospital of Philadelphia and 25 clinical trial sites. These clinical sites have expanded since the initial award, from 9 to 15 primary sites as well as an additional 10 affiliate sites. 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. The NFRP’s commitment to identify and support testing of promising therapeutic therapies for NF patients is evident by the consistent support of the NFCTC since 2005, with additional NFCTC awards executed by the NFRP in FY06, FY11, and FY16. The NFCTC has convened committees to identify promising new treatments more rapidly for multiple complications of NF-type 1 (NF1), NF-type 2 (NF2), and Schwannomatosis; these committees use this information to develop new clinical trials. Since the NFCTC was established initially with an initial 9 primary sites, 15 clinical trials have been launched. More than 480 participants have been enrolled, and the NFCTC has worked together with pharmaceutical companies and patient support organizations to leverage more than $5 million in additional funding to support and expand its number of clinical trials.

Under the new OTA, the NFCTC team will continue to accelerate progress towards reliable treatments for all forms of NF. Specifically, the Consortium will open multiple new clinical trials, some of which are already in preparation, for multiple complications of NF1, NF2, and Schwannomatosis, including attention deficit disorder, malignant peripheral nerve sheath tumors, brain tumors, plexiform neurofibromas, cutaneous neurofibromas, bone disorders, hearing nerve tumors, and tumors of the membranes that cover the brain and spinal cord, and pain due to tumors or tumor treatment. In addition, the NFCTC is developing a registry of participants, a collection of imaging studies (MRI, PET, etc.), and a biorepository of tissue and blood samples to enable long-term outcome tracking. Furthermore, the NFCTC is working on a collaboration with an NF-focused clinical trials group in Europe (EUropean-Patient-cEntric clinicAl tRial pLatforms [EU-PEARL]) to compare trial results across the U.S. and Europe.

The NFCTC is committed to serving the NF community by conducting clinical trials as rapidly and reliably as possible for all forms of NF while always respecting the safety and rights of study participants. To date, there have been significant successes in the development of new treatments such as Selumentinib, the first treatment for plexiform neurofibromas, but considerable work remains to be done to address the full range of complications of NF1, NF2, and Schwannomatosis. The NFCTC looks forward to continuing to advance its efforts on behalf of the NF patient community and to strengthening its partnerships with that community to ensure new therapeutic developments can be moved into clinical practice as rapidly and responsibly as possible.

Links:
Public and Technical Abstracts: Neurofibromatosis Research Program Clinical Trial Consortium Award FY21
Public and Technical Abstracts: Neurofibromatosis Clinical Consortium Award FY16
Public and Technical Abstracts: Neurofibromatosis Clinical Consortium Award FY11

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