Mirdametinib Monotherapy in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF). (NCT06159166) | Clinical Trial Compass
RecruitingPhase 1/2
Mirdametinib Monotherapy in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF).
United States24 participantsStarted 2024-02-12
Plain-language summary
This is a Phase 1/2a, open-label, non-randomized, multi-dose study of mirdametinib monotherapy in adults with NF1 and cNF.
In both Phases of the study, participation in the study will comprise three periods: screening, treatment and post-study safety follow-up to be performed at the NF1 and cNF specialty center: Johns Hopkins University.
Who can participate
Age range18 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Meet the diagnostic criteria for NF12
✓. ≥ 18 years of age
✓. Have a minimum of 24 measurable cNF (2 target areas of ≥6 measurable cNF)
✓. Participants must have cNF that meet eligibility criteria located within the two study designated target areas or outside of the target areas amenable to biopsy. If biopsy is taken within the target area there must be a minimum of 6 cNF remaining for long term surveillance after biopsy. Participants must be willing to undergo pre-, and on-treatment tumor biopsies providing fresh tumor tissue; there should be no contraindication for serial biopsy
✓. Karnofsky performance level of ≥ 80%.
✓. Adequate organ and bone marrow function as defined by the following Screening laboratory values:
✕. Participant has an active parathyroid disorder, hyperphosphatemia at Screening (serum phosphorus \> 1 x ULN), or serum calcium (mg/dL) x serum phosphorus (mg/dL) product \> 70 at Screening;
✕. Any clinically significant active or known history of liver disease, or known hepatic or biliary abnormalities (with the exception of Gilbert's syndrome or asymptomatic gallstones);
✕. Hepatitis serology will be tested at Screening. Participants who are hepatitis B surface antigen (HBsAg) positive or hepatitis C virus (HCV) antibody positive at Screening must not be enrolled until further definite testing with hepatitis B virus (HBV) deoxyribonucleic acid (DNA) titers is \< 500 IU/mL or HCV ribonucleic acid (RNA) polymerase chain reaction test is negative;
✕. Lymphoma, leukemia, or any malignancy (including malignant glioma or MPNST) within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years;