Pivotal / Phase II Clinical Trial of Magnetic Resonance-Guided Focused Ultrasound (MR-HIFU) Treatment of Painful Osteoid Osteoma in Children and Young Adults
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 30
- 试验地点
- 2
- 主要终点
- Pain relief as assessed using the Visual Analog Scale (VAS)
研究概览
简要总结
To determine treatment safety and efficacy of MR-HIFU ablation of painful Osteoid Osteoma (OO) in children and young adults.
详细描述
Osteoid Osteoma (OO) is a benign, but painful, bone tumor commonly occurring in children and young adults. Definitive treatment options are CT-guided radiofrequency ablation (RFA) and less commonly, open surgical resection. RFA is less invasive than surgery, but it still requires drilling from the skin through muscle and bone. It also exposes the patient and operator to ionizing radiation.
Magnetic Resonance guided High Intensity Focused Ultrasound (MR-HIFU) provides precise and controlled delivery of focused ultrasound energy inside a lesion using an external applicator, without any incisions or drilling. MR-HIFU has been successfully used to treat painful bone metastases in adult clinical trials. Several recent reports, including two publications from our group, show that MR-HIFU is a feasible and safe treatment for OO.
MR-HIFU ablation of OO may provide a better alternative to surgical resection or RFA as it is completely non-invasive and does not require ionizing radiation. These two qualities of MR-HIFU are especially beneficial in growing children and young adults. Furthermore, MR-HIFU OO ablation is quick, with expected total procedure time of less than two hours. Such short treatments offer additional safety benefits from reduced anesthesia / sedation requirement compared to surgery and RFA.
The investigator's group has performed the first pilot clinical trial in the U.S. to evaluate MR-HIFU treatment for painful osteoid osteoma (PI: K.V. Sharma, NCT02349971). This trial shows that MR-HIFU ablation is feasible, well tolerated, and can be safely performed in a pediatric cohort. Based on these results, the investigators propose a pivotal clinical trial designed to evaluate treatment efficacy as the next logical step required for clinical translation. The investigators hypothesize that noninvasive and radiation-free MR-HIFU ablation for osteoid osteoma will be a clinically effective treatment, with success rate comparable to that currently reported for RFA.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 0 Years 至 30 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≤30 years.
- •Diagnosis of OO: All patients diagnosed with painful OO based on characteristic clinical history of pain relieved with NSAID medications AND confirmatory imaging findings characteristic of OO on CT and/or MRI scans.
- •Location of OO: eligible lesions must be located in a bone with acoustic accessibility and within the normal safety margins of MR-HIFU, as specified in the instructions for use.
- •Prior therapy: Patients with prior unsuccessful treatments including CT-RFA and/or surgical resection are eligible for enrollment.
- •Patients < 18 years old must be accompanied by a legal guardian at the time of recruitment.
- •Laboratory:
- •Complete Blood Count (CBC) values within acceptable range for safe administration of anesthesia. Hemoglobin > 9 g/dL.
- •PT, PTT and INR < 1.5 x ULN (including patients on prophylactic anticoagulation)
- •Basic metabolic panel (BMP) and/or Complete metabolic panel (CMP) values within acceptable range for safe administration of anesthesia.
- •Adequate renal function: Age-adjusted normal serum creatinine (see table below) OR a creatinine clearance ≥60 mL/min/1.73 m2 for safe contrast administration
- •Adequate pulmonary function: Defined as no dyspnea at rest, and a pulse oximetry >94% on room air.
排除标准
- •Diagnosis of bone lesion other than OO in the judgement of the Principal Investigator.
- •Clinically significant unrelated systemic illness, such as serious infections, hepatic, renal or other organ dysfunction, which would compromise the patient's ability to tolerate the general anesthetic required for the procedure in the judgment of the Principal Investigator.
- •Implant, prosthesis or scar tissue that would interfere with a safe completion of MR-HIFU. ablation in the judgement of the Principal Investigator.
- •Treatment area nidus <1 cm from a major nerve, spinal canal, bladder, or bowel.
- •Target <1 cm of growth plate (physis).
- •Lesion in the skull.
- •Lesion in the vertebral body.
- •Inability to undergo MRI and/or contraindication for MRI.
- •Inability to tolerate stationary position during MR-HIFU.
- •Patients currently receiving any investigational agents.
结局指标
主要结局
Pain relief as assessed using the Visual Analog Scale (VAS)
时间窗: 12 months
The VAS is a continuous numerical pain scale ranging from 0 (no pain) to 10 (worst possible pain)
Number of participants with treatment-related adverse events as assessed by the Common Terminology Criteria for Adverse Events (CTCAE) v.5
时间窗: 12 months
CTCAE v.5 will be used
Quality of life as assessed by using the Pediatric Quality of Life Inventory (PedsQL) v4.0
时间窗: 12 months
The PedsQL is a 23-item, 4 scale, and 2 domain instrument measuring physical, social, emotional, and cognitive well-being. Each question will be rated from 0 (never) to 4 (almost always)
Pain relief as assessed by using the PROMIS Pain Interference short form
时间窗: 12 months
The PROMIS Pain Interference Short Form consists of 8 questions on pain. Each question will be rated from 0 (never), 1 (almost never), 2 (sometimes), 3 (often), and 4 (almost always)
Pain relief as assessed by pain medication usage
时间窗: 12 months
The patient pain and medication diary will be used
Quality of life as assessed by using the Symptom Distress Scale (SDS)
时间窗: 12 months
SDS is a 10-item Likert format instrument that measures symptom intensity and distress. Each question will be rated from 1 (no problems in the area) to 5 (worst problems in this area)
次要结局
- Number of participants with change in nidus vascularity as assessed via MRI(12 months)
- Number of participants with changes in soft tissue and marrow edema as assessed via MRI(12 months)
- Number of participants with change in nidus size (mm) as assessed via MRI(12 months)
研究者
Karun Sharma MD
Director of Interventional Radiology
Children's National Research Institute