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MEK Inhibitor

Mirdametinib for Neurofibromatosis (Mirda Trial)

Phase 1 & 2

Recruiting

Led By Carlos Romo, MD

Research Sponsored by Johns Hopkins University

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Have a minimum of 24 measurable cNF with 2 target areas of at least 6 measurable cNF each

Be at least 18 years of age

Must not have

History of retinal pathology or risk factors for retinal vein occlusion

Malabsorption syndrome or preexisting gastrointestinal conditions that may impair absorption of study treatment

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 28 days

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing the drug mirdametinib in adults with NF1 and cNF. Participants will go through three phases: screening, treatment, and a follow-up period for safety.

Who is the study for?

Adults over 18 with Neurofibromatosis type 1 (NF1) and at least 12 measurable cutaneous neurofibromas (cNF). They must have a certain level of overall health, including specific blood cell counts and performance status. Participants need to be able to undergo tumor biopsies.

What is being tested?

The trial is testing Mirdametinib as a single-agent therapy for adults with NF1 and cNF. It's an open-label study, meaning both doctors and patients know what treatment is being given, conducted in multiple doses across three phases: screening, treatment, and follow-up.

What are the potential side effects?

While the side effects are not explicitly listed here, common ones associated with similar medications may include skin reactions at the site of neurofibroma treatment, gastrointestinal issues like nausea or diarrhea, fatigue, liver enzyme changes, rash or other skin changes.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have at least 24 measurable skin growths, with 2 areas having at least 6 each.

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I am 18 years old or older.

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My eligible neurofibromas are in the study's specified areas or can be biopsied if outside those areas.

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I can carry out normal activities with minimal symptoms.

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I agree to follow the specified contraception guidelines.

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My organ and bone marrow functions are within normal ranges.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have a history of eye problems related to the retina.

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I have a condition that affects how my body absorbs medication.

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I have been treated with MEK inhibitors before.

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I have had high calcium levels due to cancer.

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I do not have any significant liver diseases or abnormalities.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 28 days

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 28 days

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 28 days for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Assess the safety of continuous and three week on-one week off dosing of mirdametinib in adults with NF1 and cutaneous neurofibromas (cNFs).

Define the recommended phase 2 dose (RP2D) of mirdametinib in adults with NF1 and cNF.

• Evaluate the preliminary anti-tumor activity of mirdametinib monotherapy in adults with NF1 and cNFs.

Side effects data

From 2023 Phase 1 & 2 trial • 6 Patients • NCT05054374

33%

CPK increased

33%

Lymphocyte count decreased

17%

Alkaline phosphatase increased

17%

Aspartate aminotransferase increased

100%

80%

60%

40%

20%

Study treatment Arm

Arm 1, Part 1 - Mirdametinib in Combination With Fulvestrant

Arm 1, Part 2 - Mirdametinib in Combination With Fulvestrant

Arm 2, Part 1 - Mirdametinib as Single Agent

Arm 2, Part 2 - Mirdametinib as Single Agent

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Phase 2Experimental Treatment1 Intervention

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.

Group II: Phase 1Experimental Treatment1 Intervention

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Mirdametinib

2021

Completed Phase 2

~10

0 / 11Eligibility criteria met