A Phase II Study of Continuous Oral mTOR Inhibitor Everolimus for Recurrent, Radiographic-Progressive Neurofibromatosis Type 1-Associated Pediatric Low-Grade Glioma: A Neurofibromatosis Clinical Trials Consortium Study.
Ullrich NJ, Prabhu SP, Reddy AT, Fisher MJ, Packer R, Goldman S, Robison NJ, Gutmann DH, Viskochil DH, Allen JC, Korf B, Cantor A, Cutter G, Thomas C, Perentesis JP, Mizuno T, Vinks AA, Manley PE, Chi SN, Kieran MW.
Neuro Oncol, 2020 Oct 14;22(10):1527-1535. doi: 10.1093/neuonc/noaa071. PMID:32236425
A randomized placebo-controlled study of lovastatin in children with neurofibromatosis type 1.
Payne JM, Barton B, Ullrich NJ, Cantor A, Hearps SJC, Cutter G, Rosser T, Walsh KS, Gioia GA, Wolters PL, Tonsgard J, Schorry E, Viskochil D, Klesse L, Fisher M, Gutmann DH, Silva AJ, Hunter SJ, Rey-Casserly C, Cantor NL, Byars AW, Stavinoha PL, Ackerson JD, Armstrong CL, Isenberg J, O’Neil SH, Packer RJ, Korf B, Acosta MT, North KN, for the NF Clinical Trials Consortium.
Neurology, 87(24):2575-2584, 2016. PMID:27956565.
Are Some Randomized Clinical Trials Impossible?
Rios JJ, Richards BS, Stevenson DA, Oberlander B, Viskochil D, Gross A, Dombi E, Widemann B, Plotkin SR, May C, Ullrich N, Goldstein R, Rosser T, Jain V, Schorry EK.
J Pediatr Orthop, 2021 Jan;41(1):e90-e93.
Age-dependent changes in sirolimus metabolite formation in patients with neurofibromatosis type 1.
Emoto C1, Fukuda T, Mizuno T, Cox S, Schniedewind B, Christians U, Widemann BC, Fisher MJ, Weiss B, Perentesis J, Vinks AA.
Neuro Oncol. 2015 Apr;17(4):596-603. doi: 10.1093/neuonc/nou235. Epub 2014 Oct 14.
Therapeutic Drug Monitoring, 37(3):395-399, 2015. PMID:25162215
Cabozantinib for neurofibromatosis type 1-related plexiform neurofibromas: a phase 2 trial.
Fisher MJ, Shih C, Rhodes SD, Armstrong AE, Wolters PL, Dombi E, Zhang C, Angus SP, Johnson GL, Packer RJ, Allen JC, Ullrich NJ, Goldman S, Gutmann DH, Plotkin SR, Rosser T, Robertson KA, Widemann BC, Smith AE, Bessler, WK He Y, Park, S-J Mund JA, Jiang L, Bijangi-Vishehsaraei K, Robinson CT, Cutter GR, Korf BR, Neurofibromatosis Clinical Trials Consortium, Blakeley JO, Clapp, DW.
Nature Medicine, 2021 Jan;27(1):165-173. PMID: 33442015.
Cognition, ADHD Symptoms, and Functional Impairment in Children and Adolescents with Neurofibromatosis Type 1.
Payne JM, Haebich KM, MacKenzie R, Walsh KS, Hearps SJC, Coghill D, Barton B, Pride NA, Ullrich NJ, Tonsgard JH, Viskochil D, Schorry EK, Klesse L, Fisher MJ, Gutmann DH, Rosser T, Packer RJ, Korf B, Acosta MT, Bellgrove MA, North KN.
J Atten Disord, In Press. PMID:31838937.
Multicenter prospective phase II and biomarker study of high dose bevacizumab as induction therapy in NF 2 patients with progressive vestibular schwannoma: differential response in pediatric versus adult patients.
Plotkin SR, Duda DG, Muzikansky A, Karajannis MA, Allen J, Blakeley J, Rosser T, Campian JL, Clapp DW, Fisher MJ, Tonsgard J, Ullrich NJ, Thomas C, Cutter G, Korf B, Packer R.
J Clin Oncol, 37(35):3446-3454, 2019. PMID:31626572
NF106: A Neurofibromatosis Clinical Trials Consortium Phase 2 Trial of the MEK Inhibitor Mirdametinib (PD-0325901) in Adolescents and Adults with NF1-Related Plexiform Neurofibromas.
Weiss BD, Wolters PL, Plotkin SR, Widemann BC, Tonsgard JH, Blakeley J, Allen JC, Schorry E, Korf B, Robison NJ, Goldman S, Vinks AA, Emoto C, Fukuda T, Robinson CT, Cutter G, Edwards L, Dombi E, Ratner N, Packer R, Fisher MJ.
J Clin Oncol, In Press
Neurofibromatosis Clinical Trial Consortium.
Packer RJ, Fisher MJ, Cutter G, Cole-Plourde K, Korf BR
Neuro Oncol. 2015 Apr;17(4):596-603. doi: 10.1093/neuonc/nou235. Epub 2014 Oct 14.
J Child Neurol, 33(1):82-91, 2018. PMID:29246097.
Optimizing biologically targeted clinical trials for neurofibromatosis.
Gutmann DH, Blakeley JO, Korf BR, Packer RJ. 2013.
Expert Opin Investig Drugs 22(4):443-462
Population pharmacokinetics of sirolimus in pediatric patients with neurofibromatosis type 1-Erratum.
Scott JR1, Courter JD, Saldaña SN, Widemann BC, Fisher M, Weiss B, Perentesis J, Vinks AA.
Ther Drug Monit. 2015 Jun;37(3):395-9. doi: 10.1097/FTD.0000000000000130.
Ther Drug Monit, 35(3):332-337, 2013. PMID:23666574
Randomized placebo-controlled study of lovastatin in children with neurofibromatosis type 1.
Payne JM1, Barton B1, Ullrich NJ1, Cantor A1, Hearps SJ1, Cutter G1, Rosser T1, Walsh KS1, Gioia GA1, Wolters PL1, Tonsgard J1, Schorry E1, Viskochil D1, Klesse L1, Fisher M1, Gutmann DH1, Silva AJ1, Hunter SJ1, Rey-Casserly C1, Cantor NL1, Byars AW1, Stavinoha PL1, Ackerson JD1, Armstrong CL1, Isenberg J1, O'Neil SH1, Packer RJ1, Korf B1, Acosta MT1, North KN2; NF Clinical Trials Consortium.
Reproducibility of cognitive endpoints in clinical trials: lessons from neurofibromatosis type 1.
Payne JM, Hearps SJC, Walsh KS, Paltin I, Barton B, Ullrich NJ, Haebich KM, Coghill D, Gioia GA, Cantor A, Cutter G, Tonsgard JH, Viskochil D, Rey-Casserly C, Schorry EK, Ackerson JD, Klesse L, Fisher MJ, Gutmann DH, Rosser T, Packer RJ, Korf B, Acosta MT, North KN.
Ann Clin Transl Neurol, 6(12):2555-2565, 2019. PMID:31797581
Sirolimus for progressive neurofibromatosis type 1-associated plexiform neurofibromas: a neurofibromatosis Clinical Trials Consortium phase II study.
Weiss B1, Widemann BC1, Wolters P1, Dombi E1, Vinks A1, Cantor A1, Perentesis J1, Schorry E1, Ullrich N1, Gutmann DH1, Tonsgard J1, Viskochil D1, Korf B1, Packer RJ1, Fisher MJ1.
Ther Drug Monit. 2016 Jan 21. [Epub ahead of print]
Neurology. 2016 Dec 13;87(24):2575-2584. Epub 2016 Nov 9.
Neuro-Oncology, 17(4):596-603, 2015. PMID:25314964
Targeting Sporadic and Neurofibromatosis Type 1 (NF1) Related Refractory Malignant Peripheral Nerve Sheath Tumors (MPNST) in a Phase II Study of Everolimus in Combination with Bevacizumab (SARC016).
Widemann BC, Lu Y, Reinke D, Okuno SH , Meyer CF, Cote GM, Chugh R, Milhem MM, Hirbe AC, Kim A, Turpin B, Pressey JG, Dombi E, Jayaprakash N, Helman LJ, Onwudiwe N, Cichowski K, Perentesis JP.
Sarcoma, 2019 Jul 24; 2019:7656747. eCollection 2019. PMID: 31427883
Targeting Refractory Sarcomas and Malignant Peripheral Nerve Sheath Tumors in a phase I/II study of sirolimus in combination with ganetespib (SARC023).
Kim A, Lu Y, Okuno SH, Reinke DK, Maertens O, Perentesis JP, Basu M, Wolters P, DeRaedt T, Chawla S, Chugh R, Van Tine B, Cichowski K, Widemann BC.
Sarcoma, 2020 Jan 30;2020:5784876. eCollection 2020. PMID: 32089640
Visual outcomes following everolimus targeted therapy for neurofibromatosis type 1-associated optic pathway gliomas in children.
Ullrich NJ, Prabhu SP, Packer RJ, Goldman S, Robison NJ, Allen JC, Viskochil DH, Gutmann DH, Perentesis JP, Korf B, Fisher MJ, Kieran MW on behalf of the NF Clinical Trials Consortium.
Pediatric Blood & Cancer, In Press.
Visual spatial learning outcomes for clinical trials in neurofibromatosis type 1.
Ullrich NJ, Payne JM, Walsh KS, Cutter G, Packer R, North K, Rey-Casserly C; NF Clinical Trials Consortium.
Ann Clin Transl Neurol. 2020 Feb;7(2):245-249. doi: 10.1002/acn3.50976. Epub 2020 Feb 5. PMID: 32022434