A Phase 2 Study of the MEK Inhibitor Trametinib in Children With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
Study on the Effectiveness of an Investigational Medication for Juvenile Myelomonocytic Leukemia
Brief description of study.
Detailed description of study
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: juvenile myelomonocytic leukemia
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Age: 21 years or below
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Gender: All
Inclusion Criteria
Patients must have had histologic verification of juvenile myelomonocytic leukemia (JMML) at original diagnosis and currently have relapsed or refractory disease
Patients with refractory or relapsed JMML must have had at least one cycle of intensive frontline therapy or a deoxyribonucleic acid (DNA) demethylating agent with persistence of disease, defined by clinical symptoms or the presence of a clonal abnormality
Patients must have a Lansky or Karnofsky performance status score of >= 50, corresponding to Eastern Cooperative Oncology Group (ECOG) categories 0, 1 or 2
Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to study enrollment
Patients must not be known to be refractory to red blood cell or platelet transfusions
Exclusion Criteria
Patients who are pregnant or breast-feeding
Patients requiring corticosteroids who have not been on a stable or decreasing dose of corticosteroid for the 7 days prior to enrollment
Patients who are currently receiving another investigational drug
Patients who are currently receiving other anti-cancer agents
Patients who are receiving cyclosporine, tacrolimus or other agents to prevent either graft-versus-host disease post bone marrow transplant or organ rejection post-transplant
Any medications for treatment of left ventricular systolic dysfunction
Patients who are unable to swallow capsules or liquid
Patients who have an uncontrolled infection
Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study
Patients with a history of hepatic sinusoid obstructive syndrome (veno-occlusive disease) within the prior 3 months
Patients with a history of current evidence/risk of retinal vein occlusion (RVO) or central serous retinopathy (CSR)
Patients with a history of RVO or CSR, or predisposing factors to RVO or CSR (e.g., uncontrolled glaucoma or ocular hypertension, uncontrolled systemic disease(s) such as hypertension, diabetes mellitus, or a history of hyperviscosity or hypercoagulability syndromes)
This study investigates how well an investigational medication works for patients with juvenile myelomonocytic leukemia (JMML) that has returned or did not respond to treatment. JMML is a rare type of blood cancer that affects young children. The purpose of this study is to determine how well the investigational medication can reduce the cancer in children with this condition.
Participants will undergo certain procedures to evaluate their response to the investigational medication. This includes regular medical check-ups and tests to monitor the effectiveness of the treatment and any side effects that may occur.
- Who can participate: Children with juvenile myelomonocytic leukemia that has come back or does not respond to treatment may participate. They must have fully recovered from prior treatments, not be refractory to blood or platelet transfusions, have undergone at least one cycle of prior therapy, and have a performance status score of 50 or higher. Participants should not be pregnant or breastfeeding and must not have certain medical conditions or be on specific medications.
- Study details: Participants will take trametinib, an investigational medication, to see if it can reduce their cancer. They will be monitored for any changes in their condition and for any side effects.
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