NL-OMON52145招募中2 期
A Phase 2, Open-Label, Multicenter Study to Evaluate the Safety and Efficacy of the Oral Pan-RAF Inhibitor DAY101 in Pediatric Patients with RAF-Altered, Recurrent or Progressive Low-Grade Glioma and Advanced Solid Tumors - FIREFLY-1
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 10
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 17（—）
入选标准
- •1. Patients must be age 6 months to 25 years, inclusive, with:
- •a) Arm 1 (Low-Grade Glioma): A relapsed or progressive low-grade glioma with a
- •documented known activating BRAF alteration, as identified through molecular
- •assays as routinely performed at CLIA or other similarly certified laboratories
- •b) Arm 2 (Low-Grade Glioma Extension): A relapsed or progressive low-grade
- •glioma with a documented known or expected to be activating BRAF mutation or
- •RAF fusion, as identified through molecular assays as routinely performed at
- •CLIA-certified or other similarly certified laboratories
- •c) Arm 3 (Advanced Solid Tumor): Locally advanced or metastatic solid tumor with
- •a documented known or expected to be activating RAF fusion, as identified
- •through molecular assays as routinely performed at CLIA-certified or other
- •similarly certified laboratories, that has relapsed or progressed or was
- •nonresponsive to available therapies and for which no standard or available
- •systemic curative therapy exists
- •2. Patients must have histopathologic verification of malignancy at either
- •original diagnosis
- •or relapse.
- •3. Patients must have received at least one line of prior systemic therapy and
- •documented evidence of radiographic progression.
- •4. Patients must have evaluable and/or measurable disease (imaging must be
- •within 28 days of the initiation of treatment) as specified below:
- •a) Arm 1 (Low-Grade Glioma): Must have at least one measurable lesion as defined
- •by RANO criteria (T1-weighted lesion that can be reproducibly measured in at
- •least 2 dimensions of at least 10 mm, visible on 2 or more axial slices that
- •are preferably, at most, 5 mm apart with 0-mm skip)
- •b) Arm 2 (Low-Grade Glioma Extension): Must have evaluable (either
- •unidimensionally measurable lesions, masses with margins not clearly defined,
- •or lesions with maximal perpendicular diameters less than 10 mm) and/or
- •measurable disease as defined by RANO criteria
- •c) Arm 3 (Advanced Solid Tumor): Must have at least one measurable lesion as
- •defined by RECIST v1.1 (>= 10 mm by CT/MRI scan [slice thickness <= 5 mm],
- •>= 20 mm by chest X-ray, or >= 10 mm with calipers by clinical exam, or
- •pathologic lymph nodes with a short axis of >= 15 mm by CT scan/MRI)
- •5. Radiation therapy to the measurable lesion(s) must be completed at least six
- •months prior
- •to administration of DAY101. Patients who have documented radiographic
- •progression less than six months from radiotherapy in one or more measurable
- •lesions are eligible.
- •6. Patients must have Karnofsky (those 16 years and older) or Lansky (those
- •younger than
- •16 years) performance score of at least 50. Patients who are unable to walk
- •because of paralysis, but who are able to sit in a wheelchair, will be
- •considered ambulatory for the purpose of assessing the performance score.
- •7. Patients must have fully recovered from the acute toxic effects of all prior
- •chemotherapy and have undergone the following washout periods, as applicable:
- •a) Myelosuppressive chemotherapy: At least 21 days must have elapsed after the
- •dose of myelosuppressive chemotherapy (42 days if prior nitrosourea).
- •b) Radiation therapy (XRT): At least 14 days must have elapsed after the last
- •fraction of XRT.
- •c) Stem cell transplant or adoptive cell therapy: At least 100 days must have
排除标准
- •Patient*s tumor has an additional previously known or expected to be activating
- •molecular alteration(s) (e.g., histone mutation, IDH1/2 mutations, FGFR
- •mutations or fusions, MYBL alterations, NF-1 somatic or germline mutations).
- •2. Patient has symptoms of clinical progression without radiographically
- •recurrent or
- •radiographically progressive disease.
- •Patient has known or suspected diagnosis of neurofibromatosis type 1 (NF-1)
- •via genetic
- •testing or current diagnostic criteria.
- •4. Patient has history of any major disease, other than the primary malignancy
- •under study,
- •that might interfere with safe protocol participation.
- •5. Patient has a history or current evidence of central serous retinopathy
- •(CSR), retinal vein
- •occlusion (RVO), or ophthalmopathy present at baseline that would be considered
- •a risk factor for CSR or RVO. Ophthalmological findings secondary to
- •long-standing optic pathway glioma (such as visual loss, optic nerve pallor, or
- •strabismus) will NOT be considered significant abnormalities for the purposes
- •of this study.
- •6. Patient has major surgery within 14 days (two weeks) prior to C1D1 (does not
- •central venous access, cyst fenestration or cyst drainage, or
- •ventriculoperitoneal shunt placement or revision).
- •7. Patient has clinically significant active cardiovascular disease, or history
- •of myocardial
- •infarction, or deep vein thrombosis/pulmonary embolism within six months prior
- •to C1D1, ongoing cardiomyopathy, or current prolonged QT interval corrected for
- •heart rate by Fridericia*s formula (QTcF) interval > 470 milliseconds based on
- •triplicate electrocardiogram (ECG) average.
- •8. Patient is currently enrolled in any other investigational treatment study.
- •Participation in
- •a concurrent observational or bio-sampling study is allowed.
- •9. Patient has active systemic bacterial, viral, or fungal infection.
- •10. Patient has nausea and vomiting >= National Cancer Institute (NCI) Common
- •Terminology Criteria for Adverse Events (CTCAE) v5.0 Grade 2, malabsorption
- •requiring supplementation, or significant bowel or stomach resection that would
- •preclude adequate absorption of DAY101.
- •11. Patient is neurologically unstable despite adequate treatment (e.g.,
- •uncontrolled seizures). 12. Patient is currently being treated with a strong
- •CYP2C8 inhibitor or inducer other than
- •those allowed per Section 5.3.2. Medications that are substrates of CYP2C8 are
- •but should be used with caution.
- •13. Patient is pregnant or lactating.
- •14. Patient has a history of any drug reaction with eosinophilia and systemic
- •(DRESS) syndrome or Stevens Johnsons syndrome (SJS), or hypersensitivity to the
- •investigational medicinal product or to any drug with similar chemical
- •structure or to any other excipient present in the pharmaceutical form of the
- •investigational medicinal product.
- •15. There are other unspecified reasons that, in the opinion of the
- •investigator, make the
- •patient unsuitable for enrollment.
研究者
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