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New treatment option for NF1
Official title Mirdametinib Monotherapy in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF).
ClinicalTrials.gov ID: NCT06159166
What this study is testing
What is Mirdametinib?
Mirdametinib is an investigational medicine, being studied as a potential treatment for nf1.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Meet the diagnostic criteria for NF12
- ≥ 18 years of age
- Have a minimum of 24 measurable cNF (2 target areas of ≥6 measurable cNF) a. Measurable is defined as: i. non-pedunculated (no stalk) ii. surrounded...
- Participants must have cNF that meet eligibility criteria located within the two study designated target areas or outside of the target areas...
- Karnofsky performance level of ≥ 80%.
You likely can't join if
- Participant has a altered screening values:
- 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...
- Any clinically significant active or known history of liver disease, or known hepatic or biliary abnormalities (with the exception of Gilbert's...
See the full eligibility criteria
Who can join
- Meet the diagnostic criteria for NF12
- ≥ 18 years of age
- Have a minimum of 24 measurable cNF (2 target areas of ≥6 measurable cNF) a. Measurable is defined as: i. non-pedunculated (no stalk) ii. surrounded by uninvolved skin and not adjacent to another cNF lesion iii...
- 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...
- Karnofsky performance level of ≥ 80%.
- Adequate organ and bone marrow function as defined by the following Screening laboratory values:
- Absolute neutrophil count ≥ 1500 cells/µL;
- Platelets ≥ 100 x 103/µL;
- Hemoglobin ≥ 9.5 g/dL;
- Serum albumin ≥ 2.8 g/dL;
- Calculated creatinine clearance at Screening ≥ 60 mL/min (by Cockcroft-Gault formula) OR a normal serum creatinine.
- Participant is willing and able to comply with all aspects of the protocol
- Ability to understand and the willingness to sign written informed consent document(s).
- Women of childbearing potential (WOCBP) must not be pregnant or breastfeeding during any portion of the study and must use an adequate method to avoid pregnancy during the study period and for 6 months after treatment...
- In order for a woman to be determined not of childbearing potential, she must have ≥ 12 months of non-therapy-induced amenorrhea or be surgically or medically sterile.
- WOCBP must have a negative serum pregnancy test result at Screening and a negative urine pregnancy test result at the Baseline visit prior to the first dose of study treatment.
- The Investigator is responsible for review of medical history, menstrual history, and recent sexual activity to decrease the risk for inclusion of a woman with an early undetected pregnancy.
- Male participants are eligible to participate if they agree to the following during the treatment period and for at least 90 days after the last dose of study treatment:
- Refrain from donating sperm PLUS either:
- Be abstinent from heterosexual intercourse as their preferred and usual lifestyle (abstinent on a long term and persistent basis) and agree to remain abstinent until 90 days after the last study drug treatment; OR
- Must agree to use a male condom when having sexual intercourse with a WOCBP and their female partner must utilize one the approved methods of birth control below: Approved Methods of birth control for this study are:
- Total abstinence
- Male or female sterilization (vasectomy in males or surgical removal of ovaries or uterus in females)
- Unsterilized male study participants must use a male condom and their female partner must use one of the methods below:
- Unsterilized female study participants must use one of the following highly effective methods listed below: Acceptable birth control methods which are considered highly effective if they result in a failure rate of less...
- Combined (estrogen and progestogen containing) hormonal contraceptive that stops the release of eggs from the ovary (oral, intravaginal, or transdermal)
- Progestogen-only hormonal contraception that stops the release of eggs from the ovary (oral, injectable, implantable)
- Intrauterine device (IUD)
- Intrauterine hormone-releasing system (IUS)
- Bilateral tubal occlusion or bilateral tubal ligation
What rules you out
- Participant has a altered screening values:
- 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;
- 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...
- 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...
- Breast cancer within the past 3 years;
- Active optic glioma or other low-grade glioma requiring treatment with chemotherapy or radiation therapy. a. Participants not requiring treatment are eligible. Ophthalmological findings secondary to long-standing optic...
- Abnormal QT interval corrected by Fridericia's formula (\> 450 msec for male participants, \> 470 msec for female participants, or \> 480 msec for participants with known bundle branch block), calculated from triplicate...
- Participant has experienced any of the following within 6 months (24 weeks) of signing informed consent/assent: clinically significant cardiac disease, myocardial infarction, severe/unstable angina, coronary/peripheral...
- A recorded LVEF \< 55% at screening or within 3 years of signing informed consent/assent, OR has a history of congestive heart failure;
- Participants with a history of, or evidence of, retinal pathology on ophthalmologic examination that is considered a risk factor for central serous retinopathy, retinal vein occlusion (RVO), or neovascular macular...
- Intraocular pressure \> 21 mmHg;
- Serum cholesterol \> 300 mg/dL;
- Serum triglycerides \> 300 mg/dL;
- Hyperglycemia (fasting blood glucose \> 125 mg/dL or random blood glucose \> 200 mg/dL);
- Hypertension (BP ≥ 140/90 mm Hg)
- History of glaucoma;
- Known history of a positive human immunodeficiency virus (HIV) antibody test;
- Known malabsorption syndrome or preexisting gastrointestinal conditions that may impair absorption of mirdametinib (e.g., gastric bypass, lap band, or other gastric procedures). Delivery of mirdametinib via nasogastric...
- Previously treated with MEK inhibitor including mirdametinib (PD-0325901) and had to stop treatment due to adverse event.
- Currently receiving therapy with a MEK inhibitor including mirdametinib (PD-0325901) or treated with a MEK inhibitor in the 12 months prior to prior to first dose of study treatment.
- Received radiation therapy within the 6 months prior to prior to first dose of study treatment. Participants who have received radiation to the orbit at any time are excluded.
- Pregnant or breastfeeding women may not take study drug.
- Current enrollment or past participation in any other clinical study (excluding observational studies) within 28 days of first dose of study treatment.
- Known sensitivity to the study treatment, or components thereof, or drug or other allergy that, could compromise safety of the subject
- Participant is receiving systemic (oral, inhaled, of IV/SC) or ocular glucocorticoid therapy (with the exception of participants with endocrine deficiencies who are allowed to receive physiologic or stress doses of...
- Participants are excluded if they have severe and/or uncontrolled medical disease or social situation, which could compromise participation in the study (e.g. uncontrolled diabetes, uncontrolled hypertension, severe...
- Participants is receiving systemic treatment of a pan-cytochrom 450 (CYP) inducers such as rifampin or ritonavir within 14-days prior to first dose of study treatment Drug Development and Drug Interactions
The study team makes the final eligibility decision.
Where it's taking place
- Baltimore, Maryland, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Baltimore, Maryland, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.