Learn about our trials
Please contact Hans Shuhaiber at hshuhaiber@ufl.edu and/or (312) 286-1059 for information about these Neurofibromatosis clinical trials.
| Trial Number | Trial Name | Trial Description |
|---|---|---|
| NCT04924608 Neurofibromatosis Type 1 | Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas (KOMET) | Phase III, Multicenter, International Study with a Parallel, Randomized, Double-blind, Placebo-controlled, 2 Arm Design to Assess the Efficacy and Safety of Selumetinib in Adult Participants with NF Who Have Symptomatic, Inoperable Plexiform (KOMET). UF Health is only site in Florida. Sponsored through AstraZeneca. |
| NCT03962543 Neurofibromatosis Type 1 | MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas | Phase 2b Trial of the MEK 1/2 Inhibitor (MEKi) PD-0325901 in Adult and Pediatric Patients with Neurofibromatosis Type 1 (NF1)-Associated Inoperable Plexiform Neurofibromas (PNs) That Are Causing Significant Morbidity. Sponsored with SpringWorks therapeutics. |
| NCT05005845 Neurofibromatosis Type 1 | https://clinicaltrials.gov/ct2/show/NCT05005845 | This is a randomized, double-blind, vehicle-controlled, parallel group dose response study evaluating the safety and effectiveness of 2 concentrations of NFX-179 Gel in subjects with cutaneous neurofibromas. At Visit 1, the investigator will identify 10 Target cNFs that fulfill the enrollment criteria. The Target cNFs must be located on the subject’s face, anterior trunk, or upper extremities. Two Target cNFs must be on the face and 8 must be on the anterior trunk or upper extremities. The study medication will be applied topically QD to the Target cNFs for 182days (26 weeks). During the duration of the study subjects will be evaluated for safety and efficacy. |
| NCT04954001 Neurofibromatosis Type 1 | Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis Type 1 | FCN-159 is a highly active MEK1/2 inhibitor that was designed, synthesized and screened on the basis of the structure of trametinib. FCN-159 is an orally available and highly potent selective inhibitor of MEK1/2, which is expected to be a targeted therapy for the treatment of advanced solid tumors and neurofibromatosis type 1. |
| NCT05130866 Neurofibromatosis Type 1 | https://clinicaltrials.gov/ct2/show/NCT05130866 | This is a parallel-group, two-staged, Phase 2/3, randomized, multi-center study to investigate the efficacy and safety of REC-2282 in patients with progressive NF2 mutated meningiomas. |
| NCT05085561 Neurofibromatosis Type 1 | The Symptomatic Cerebral Cavernous Malformation Trial of REC-994 | This is a multi-center, randomized, double-blind, placebo-controlled study to investigate the safety, efficacy and pharmacokinetics of REC-994 (200 mg and 400 mg) compared to placebo in subjects with symptomatic cerebral cavernous malformation (CCM). |
To learn more see https://www.ctsi.ufl.edu/.