Mirdametinib Monotherapy in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF).
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorJohns Hopkins University
About this trial
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.
Eligibility criteria
Qualifiers
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
Disqualifiers
alanine transaminase (ALT) value of > 2.0 x upper limit of normal (ULN);
total bilirubin value of > 1.5 x ULN (isolated bilirubin > 1.5 x ULN is acceptable if bilirubin is fractionated and direct bilirubin < 35%);
Participant has a history of malignancy associated hypercalcemia;
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;
Trial design
Treatments tested in this trial
- Mirdametinib
Treatment groups
24 Participants
are divided into 2 treatment groupsSponsors and collaborators
Johns Hopkins University
Lead sponsor
SpringWorks Therapeutics, Inc.
Collaborator
Source ClinicalTrials.gov