Clinical Trials

ClinicalTrials.gov Identifier: NCT06188741

Description: Plexiform neurofibromas (PN) are known to cause significant morbidity in children with NF1. The recent FDA approval for selumetinib in children 2 years and older with inoperable symptomatic PN was based on the finding that selumetinib shrinks the majority of PN in children with NF1 and results in clinically meaningful benefit such as improvement in pain or range of motion. However, many morbidities, such as blindness or nerve damage, cannot be fully reversed with PN shrinkage. Therefore, there remains a critical need in this patient population to determine if young participants with PN in high-risk locations may benefit from early medical intervention prior to the development of clinical problems. This study will determine whether participants with asymptomatic PN in high-risk locations can potentially benefit from early treatment with selumetinib.

Key Eligibility Criteria: 

• Age: > 1 and ≤ 8 years of age at the time of study enrollment
• Diagnosis of NF1 based on the 2021 revised consensus criteria
• No known PN prior to enrollment

ClinicalTrials.gov Identifier: NCT06541847

Description: The trial will be an open label, single arm, phase 2 study in 20 participants. The study will assess the tolerability and efficacy of HLX-1502 in participants with NF1 16 years of age or older with progressive and/or symptomatic PN.

Key Eligibility Criteria: 

• Age: ≥16 years of age at the time of study enrollment.
• Diagnosis of NF1 based on the 2021 revised consensus criteria
• PN(s) that are progressive OR are causing significant morbidity
• Note: Although prior MEKi therapy is not a requirement, patients should be counseled on the availability of FDA-approved MEKi therapies prior to enrollment

ClinicalTrials.gov Identifier: NCT06502171

Description: Based on the clinical activity of both selumetinib and cabozantinib as monotherapies in clinical trials, the demonstrated activity of these agents in reduced doses in preclinical studies, and the non-overlapping toxicity profiles, the study will assess the tolerability and efficacy of selumetinib and cabozantinib in combination in participants with NF1 ≥16 years old with progressive and/or symptomatic PN in a phase 1/1b/2 clinical trial.

Key Eligibility Criteria: 

• Age: ≥16 years of age at the time of study enrollment.
• Diagnosis of NF1 based on the 2021 revised consensus criteria
• PN(s) that are progressive OR are causing significant morbidity
• Prior treatment with cabozantinib or selumetinib is permitted for participants on Phase 1 of this study only (not Phase 1b/2)

ClinicalTrials.gov Identifier: NCT03871257

Description: This phase III trial studies if selumetinib works just as well as the standard treatment with carboplatin/vincristine (CV) for subjects with NF1-associated low grade glioma (LGG), and to see if selumetinib is better than CV in improving vision in subjects with LGG of the optic pathway (vision nerves). Selumetinib is a drug that works by blocking some enzymes that low-grade glioma tumor cells need for their growth. This results in killing tumor cells. Drugs used as chemotherapy, such as carboplatin and vincristine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. It is not yet known whether selumetinib works better in treating patients with NF1-associated low-grade glioma compared to standard therapy with carboplatin and vincristine.

Key Eligibility Criteria:

• Age: ≥2 years and ≤ 21 years at the time of study enrollment.
• Diagnosis of NF1 based on clinical criteria and/or germline genetic testing
• Newly diagnosed or have previously diagnosed NF-1 associated LGG that has not been treated with any modality other than surgery

ClinicalTrials.gov Identifier: NCT06712875

Description: This is a pilot study evaluating the toxicity and early efficacy of dabrafenib and/or trametinib combined with nivolumab for the treatment of BRAF-altered or NF altered gliomas. 

Key Eligibility Criteria:

• Cohort A: Patients ≥1 and ≤26 years of age with a recurrent or progressive low grade glioma harboring a KIAA1549-BRAF fusion, or a newly diagnosed high grade glioma harboring a KIAA1549-BRAF fusion
• Cohort B: Patients ≥1 and ≤26 years of age with NF-1 associated or altered low-grade glioma that is recurrent or progressive, high-grade glioma that is newly diagnosed or recurrent, or transforming glioma that is newly diagnosed or recurrent

ClinicalTrials.gov Identifier: NCT03611868

Description: Phase 2 study assessing the overall response rate of APG-115 in combination with pembrolizumab in the treatment of patients with advanced solid tumors. Children's is enrolling for Cohort F for patients with metastatic, refractory, or unresectable malignant peripheral nerve sheath tumors.

Key Eligibility Criteria

• Malignant peripheral nerve sheath tumor
• Ages ≥ 12 years

ClinicalTrials.gov Identifier: NCT03611868

Description: This is a phase 0/1/2, multi-site study to evaluate the MEK inhibitor Selumetinib with the MDM2 Inhibitor APG-115 in patients with Neurofibromatosis Type 1 and pre-malignant and malignant peripheral nerve sheath tumors

Key Eligibility Criteria

• Malignant peripheral nerve sheath tumor
• Ages ≥ 12 years