Clinical study of patient-proposed healthcare services with dabrafenib and trametinib for BRAF V600 mutant pediatric glioma

JRCT ID: jRCTs071210071

Registered date:16/09/2021

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: BRAF V600 mutant progressive glioma
• Date of first enrollment: 16/09/2021
• Target sample size: 4
• Study type: Interventional
• Intervention(s): mokecular target therapy

Outcome(s)

• Primary Outcome: Best overall response up to 16 weeks after treatment initiation
• Secondary Outcome: progression free survival (PFS), adverse evemt

Key inclusion & exclusion criteria

• Age minimum: >= 12month old
• Age maximum: < 15age old
• Gender: Both

Include criteria

1. 12 months to 14 years old. Minimum body weight of 7 kg.
2. Diagnosis of BRAF V600 mutant glioma on cancer multi-gene test covered by the national health insurance.
3. No standard therapy, or the standard therapy was terminated due to ineffectiveness or toxicity.
4. Any lesion detected by MRI with contrast within 28 days before registration (Patient without measurable lesion is permitted).
5. Performance Score is 50 or more by Lansky scale
6. No anti-cancer therapy including chemotherapy, molecular target therapy, immunotherapy, endocrine therapy or radiation therapy within 28days before starting the study drugs
7. If receiving glucocorticoid, the dosage is stable or tapered for at least 7 days before starting the study drugs
8. Appropriate bone marrow function without G-CSF
9. Appropriate kidney, liver, and cardiac functions
10. Written informed consent provided by proxies. Informed assent can be provided by a patients with the age of 7 years or more.

Exclude criteria

1. Having malignancies other than BRAFV600 mutant glioma
2. Having a history of malignancies with RAS gene activation or BRAF fusions
3. Having history of takin RAF inhibitors, MEK inhibitors, or ERK inhibitors
4. Having allergies to dabrafenib, trametinib, or any components of these drugs
5. Treated by auto/allogenic stem cell transplant within 3 months before starting the study drugs
6. Having a current or past history of severe cardiac disease
7. Positive test in any of HIV antibody testing, HBs antigen testing, or HCV antibody testing
8. Decided to be excluded by study investigators

Related Information

• Primary Sponsor: Koga Yuhki