Mirdametinib Monotherapy in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF).

Part of paid clinical trials in Baltimore, Maryland.

Sponsor
Johns Hopkins University
Study ID
NCT06159166
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Cutaneous Neurofibroma
  • Monotherapy
  • NF1

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Mirdametinib — DRUG
    This study is designed to assess Mirdametinib safety and efficacy in two phases: phase 1 tests the safety of up to four dose regimens of Mirdametinib, administered continuously or intermittently (3 weeks on/1 week off) to identify up to two recommended phase 2 doses. Phase 2a of the study will test the safety and efficacy of the recommended phase 2 dose(s) in adults with NF1 and cNF. The following dosing strategy will be assessed in participants ≥ 18 years old with NF1 and a minimum of 12 measurable cNF who desire systemic treatment of their cNF due to disfigurement, pain or itching. Each treatment cycle in this study is 28 days.treatment cycle in this study is 28 days.

Study Details

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.

Key Dates

Start date
Feb 12, 2024
Status verified
Apr 2026
Primary completion
Nov 15, 2027
Completion
Nov 15, 2028

Study Design

Enrollment
24 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Phase 1
    For the Phase 1 portion, treatment will be administered continuously (Dose regimens 1, 2, 4) or intermittently (Dose regimen 3; 3 weeks on/1 week off) in 28-day cycles). All participants will receive study drug until: 1. cessation of study treatment due to death, intolerance, or withdrawal of consent from the study; 2. completion of 24 cycles of treatment (unless the investigator's benefit-risk assessment supports continued treatment); 3. participants enroll in the phase 2a portion of the study; 4. disease progression; or 5. Investigator's decision. Treatment period ends with the administration of the last dose. The study ends 30 days after the last dose
  • Experimental: Phase 2
    For the Phase 2 portion, treatment will be administered based on recommended RP2D from Phase 1. All participants will receive study drug until: 1. cessation of study treatment due to death, intolerance, or withdrawal of consent from the study; 2. completion of 24 cycles of treatment (unless the investigator's benefit-risk assessment supports continued treatment; 3. disease progression; or 4. Investigator's decision. Treatment period ends with the administration of the last dose. The study ends 30 days after the last dose.

Primary Outcome Measure

Assess the safety of continuous and three week on-one week off dosing of mirdametinib in adults with NF1 and cutaneous neurofibromas (cNFs). [ Time Frame: Up to 28 days ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Johns Hopkins HospitalBaltimoreMaryland21287
Joshua Roberts
[email protected]
410-955-6827

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By research site
Johns Hopkins Hospital· Baltimore, MD

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