NL-OMON49019招募中2 期
The TRAIN study: Trametinib in neurofibromatosis type 1 related symptomatic plexiform neurofibromas - TRAIN study: Trametinib in plexiform neurofibroma
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 30
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
- 18 至 64（—）
入选标准
- •1. Patient with (mosaic) NF1
- •2. Patients with a clinically significant symptomatic plexiform neurofibroma,
- •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.
- •3. Signed, written informed consent
- •4. Age: 18 or higher
- •5. Karnofsky performance level of >=70%
- •6. No standard treatment options = inoperable plexiform neurofibroma. Plexiform
- •neurofibroma 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 plexiform neurofibroma.
- •7. At least one measurable plexiform neurofibroma, defined as a well-demarcated
- •lesion of at least 3 cm measured in one dimension.
- •8. Able to swallow and retain orally administered medication.
- •9. Female Subjects of Childbearing Potential must have negative pregnancy test
- •within 7 days prior study treatment and agrees to use highly effective
- •contraception
- •10. Normal hematological function: Hemoglobin (Hb)>=6 mmol/l, absolute
- •neutrophil count (ANC)>=1.5x109/l, and platelets>=100x109/l
- •11. Normal hepatic function: bilirubin <1.5x the upper limit of normal (UNL),
- •unless gilbert then: bilirubin <3xUNL and AST/ALT <5xUNL
- •12. Normal renal function: creatinine <1.5xUNL
排除标准
- •1. Prior treatment with MEK inhibitor(s)
- •2. Inability to undergo MRI and/or contraindication for MRI examinations
- •3. History of a malignancy within 5 years of inclusion, except squamous cell
- •carcinoma of the skin, cervical premalignant lesions and other curatively
- •treated malignancy
- •4. Prior radiotherapy less than 6 weeks prior to enrollment
- •5. Prior major surgery less than 4 weeks prior to enrollment
- •6. An investigational agent within the past 30 days.
- •7. Enzyme-inducing anticonvulsants, anti-coagulants (including platelet
- •aggregation inhibitors) or other prohibited medication(s) or requirement for
- •prohibited medications
- •8. 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.
- •9. 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
- •10. 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
- •11. 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
- •12. Refractory nausea and vomiting, chronic gastrointestinal diseases (e.g.,
- •inflammatory bowel disease), or significant bowel resection that would preclude
- •adequate absorption.
- •13. Any serious and/or unstable pre-existing medical disorder, psychiatric
- •disorder, or other conditions that could interfere with subject*s safety
- •14. 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
- •15. Pregnant, lactating or actively breastfeeding female subjects
研究者
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