Actively Recruiting
Phase 1/2 Study of Mirdametinib + Vinblastine for Newly Diagnosed/Previously Untreated PLGG + Activation of MAPK
Led by St. Justine's Hospital · Updated on 2026-02-19
50
Participants Needed
5
Research Sites
376 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This is a 3-part open-label study (feasibility phase, treatment phase and follow-up phase) of orally administered mirdametinib in combination with intravenous vinblastine chemotherapy in patients with PLGG with activation of MAPK pathway. Feasibility Phase: The maximum tolerated/recommended phase 2 dose (MTD/RP2D) of the mirdametinib plus vinblastine combination will be assessed using a modified Rolling-6 design. Treatment Phase: Patients will receive mirdametinib twice daily (continuously) at a fixed dose (2mg/m2 po BID up to 4 mg BID) for a total of 13 cycles (28 days cycle). Weekly intravenous vinblastine at MTD will be given for a total of 17 cycles. If adverse events occur, two dose reductions are allowed. Follow-up Phase: Following the end of treatment, patients will be scheduled for a follow-up visit every 6 months for 36 months to evaluate PFS, TTP and OS.
CONDITIONS
Official Title
Phase 1/2 Study of Mirdametinib + Vinblastine for Newly Diagnosed/Previously Untreated PLGG + Activation of MAPK
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed written informed consent prior to study participation.
- Willing and able to comply with scheduled visits, treatments, laboratory tests, and MRI disease assessments.
- Aged 2 to 25 years at the start of mirdametinib treatment.
- Body surface area of at least 0.40 m2.
- Diagnosis of pediatric low-grade glioma (PLGG) with NF1 gene mutation, KIAA1549-BRAF fusion, or MAPK pathway alteration except BRAF V600E mutation.
- Tumor tissue available (paraffin-embedded block or fresh frozen if possible); NF1 patients without tissue can enroll if no surgery or biopsy done.
- Baseline MRI completed.
- Life expectancy greater than 6 months.
- Lansky or Karnofsky performance score of 50 or higher.
- Normal organ and bone marrow function as specified in the study protocol.
- Use of highly effective contraception for fertile male and female patients.
- Able to take and keep oral medication without using a nasogastric or gastrostomy tube.
You will not qualify if you...
- Receiving other investigational agents.
- QTcB interval of 480 msec or more or resting left ventricular ejection fraction of 49% or less.
- Any other malignancy that is currently clinically significant or requires active treatment.
- Tumor with BRAF V600E mutation.
- Prior systemic or radiation therapy.
- Severe or uncontrollable medical conditions.
- Blood pressure above the 95th percentile for age, height, and gender.
- High risk for serious retinopathy or retinal vein occlusion.
- Known HIV, hepatitis B, or hepatitis C infection.
- Major surgery within 2 weeks prior to enrollment.
- History of allergic reactions to compounds chemically similar to mirdametinib.
- Pregnant or breastfeeding.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 5 locations
1
Stollery Children's Hospital
Edmonton, Alberta, Canada, T6G 2E1
Actively Recruiting
2
Childrens and Womens Health Centre of British Columbia - British Columbia Childrens Hospital
Vancouver, British Columbia, Canada, V6H 3V4
Actively Recruiting
3
Children's Hospital, London Health Sciences Centre
London, Ontario, Canada, N6A5W9
Actively Recruiting
4
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1E8
Actively Recruiting
5
CHU Sainte-Justine
Montreal, Quebec, Canada, H3T 1C5
Actively Recruiting
Research Team
S
Sébastien Perreault, M.D, FRCPC
CONTACT
M
MIRV Study Team
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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