2024-516593-30-00招募中2 期
Trametinib in neurofibromatosis type 1 related symptomatic plexiform neurofibromas
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)1 个研究点 分布在 1 个国家目标入组 30 人开始时间: 2024年9月6日最近更新:
适应症
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Tumor volume on volumetric MRI analysis
研究概览
简要总结
To determine whether trametinib can induce shrinkage in plexiform neurofibromas. Response to treatment is defined as a tumor volume decrease from baseline of at least 20%, monitored by using volumetric MRI analysis.
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 years 至 64 years（18-64 Years）
- 接受健康志愿者
- 否
入选标准
- •Patient with (mosaic) NF1
- •Normal hematological function: Hemoglobin (Hb)≥6 mmol/l, absolute neutrophil count (ANC)≥1.5x109/l, and platelets≥100x109/l
- •Normal hepatic function: bilirubin <1.5x the upper limit of normal (UNL), unless gilbert then: bilirubin <3xUNL and AST/ALT <5xUNL
- •Normal renal function: creatinine <1.5xUNL
- •Patients with a clinically significant symptomatic plexiform neurofibroma (PNF), such as (but not limited to) head and neck lesions that could compromise the airway or great vessels, brachial or lumbar plexus lesions that could cause nerve compression and loss of function, lesions that could result in major deformity (e.g., orbital lesions) or are significantly disfiguring, lesions of the extremity that cause limb hypertrophy or loss of function, and painful lesions. This will be determined by the treating physician.
- •Signed, written informed consent
- •Age: 18 or higher
- •Karnofsky performance level of ≥70%
- •No standard treatment options = inoperable PNF PNF that cannot be surgically completely removed without risk for substantial morbidity due to invasiveness, high vascularity or encasement of, or close proximity to, vital structures of the PNF.
- •At least one measurable PNF, defined as a well-demarcated lesion of at least 3 cm measured in one dimension.
- •Able to swallow and retain orally administered medication.
- •Female Subjects of Childbearing Potential must have negative pregnancy test within 7 days prior study treatment and agrees to use highly effective contraception
排除标准
- •Prior treatment with MEK inhibitor(s)
- •Risk factors for gastrointestinal perforation, including history of diverticulitis, metastases to the gastrointestinal tract and concomitant use of medications with a recognized risk of gastrointestinal perforation
- •Any evidence of severe or uncontrolled systemic disease, active infection, active bleeding diatheses, or renal transplant, including any patient known to have hepatitis B, hepatitis C, or human immunodeficiency virus (HIV) will be excluded.
- •Refractory nausea and vomiting, chronic gastrointestinal diseases (e.g., inflammatory bowel disease), or significant bowel resection that would preclude adequate absorption.
- •Any serious and/or unstable pre-existing medical disorder, psychiatric disorder, or other conditions that could interfere with subject’s safety
- •Known severe hypersensitivity to trametinib or any excipient of trametinib or history of allergic reactions attributed to compounds of similar chemical or biologic composition to trametinib
- •Pregnant, lactating or actively breastfeeding female subjects
- •Inability to undergo MRI and/or contraindication for MRI examinations
- •History of a malignancy within 5 years of inclusion, except squamous cell carcinoma of the skin, cervical premalignant lesions and other curatively treated malignancy
- •Prior radiotherapy less than 6 weeks prior to enrollment
- •Prior major surgery less than 4 weeks prior to enrollment
- •An investigational agent within the past 30 days
- •Enzyme-inducing anticonvulsants, anti-coagulants (including platelet aggregation inhibitors) or other prohibited medication(s) or requirement for prohibited medications
- •Left ventricular dysfunction, New York Heart Association Class II, III, or IV heart failure, acute coronary syndrome within the past 6 months, clinically significant uncontrolled arrhythmias, and uncontrolled hypertension.
- •A history of retinal vein occlusion (RVO) or predisposing factors for RVO, including uncontrolled glaucoma or ocular hypertension, uncontrolled hypertension, uncontrolled diabetes mellitus, or a history of hyperviscosity or hypercoagulability syndromes
结局指标
主要结局
Tumor volume on volumetric MRI analysis
Tumor volume on volumetric MRI analysis
次要结局
- Numeric pain rating scale (NRS-11)
- Pain interference (PROMIS)
- QLQ-SF36
- Medical photography
- Adverse events according to CTCAEv5.0
- Time to first significant progression defined as >20% volumetric growth of the index lesion(s)
- Incidence of surgical interventions
研究者
Christine Noordhoek
Scientific
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
研究点 (1)
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