Safety and Feasibility of MR-Guided High Intensity Focused Ultrasound (MR-HIFU) Ablation of Osteoid Osteoma in Children

This study looks to examine the feasibility and efficacy of using MR-HIFU to ablate Osteoid Osteoma lesions in children and young adults.

Study Overview

• Status: Completed
• Conditions: Osteoid Osteoma
• Intervention / Treatment: Device: MR-HIFU

Detailed Description

Osteoid Osteoma (OO) is a benign, but painful, bone tumor commonly occurring in children and young adults. Common treatment options are surgical excision or, more recently, CT-guided radiofrequency ablation (RFA). RFA is less invasive, but it still requires drilling from the skin through muscle and soft tissue into 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 the need for a scalpel or needle. MR-HIFU has been successfully used to treat painful bone metastases in adult clinical trials and one recent report suggests that it can also be used to treat 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

• Study Type: Interventional
• Enrollment (Actual): 9
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • District of Columbia
    • Washington, District of Columbia, United States, 20012
      • Children's National Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 25 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Diagnosis:

  • All patients with a clinical suspicion of OO based on presence of typical symptoms of localized nocturnal pain that is relieved by NSAIDs and unrelated to trauma or activity.
  • Typical imaging findings on CT and/or MRI. Plain radiographs and bone scans may be obtained by referring physicians and are helpful for confirming the clinical diagnosis but cannot be substituted for a CT or MRI.
  • Non-contrast enhanced or contrast enhanced CT studies are acceptable.
  • Contrast enhanced MRI studies should be performed.
  • Tissue biopsy is not required

• Tumor location:

  • Target lesions can be located in any peripheral bone with acoustic accessibility.
  • Target lesions may be intracortical or juxtacortical in location.
  • Target lesions must be reachable within the normal safety margins of HIFU as specified in the instructions for use.

• Prior therapy:

  ●Patients with prior unsuccessful surgical resection or RFA are eligible for enrollment.

• Laboratory :

  • Hemoglobin > 9 g/dL
  • Platelet count ≥75,000/µL (may receive transfusions)
  • Normal PT, PTT and INR < 1.5 x ULN (including patients on prophylactic anticoagulation)
  • Renal function: Age-adjusted normal serum creatinine (table in protocol) 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 if there is clinical indication for determination.

Exclusion Criteria:

• Clinically significant unrelated systemic illness, such as serious infections, hepatic, renal or other organ dysfunction, which in the judgment of the Principal or Associate Investigator would compromise the patient's ability to tolerate the general anesthetic required for the procedure.
• Implant or prosthesis or scar tissue within the path of the HIFU beam.
• Target <1 cm from nerve plexus, spinal canal, bladder, bowel
• Target <1 cm of the growth plate (physis)
• Lesion in the skull or vertebral body
• Inability to undergo MRI and/or contraindication for MRI
• Inability to tolerate stationary position during HIFU
• Patients currently receiving any investigational agents.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: All Patients; Patients will undergo a one-time procedure of MR-HIFU ablation of OO under sedation or anesthesia. Patients will be monitored for disease status and adverse events for at least 12 months following procedure.	Device: MR-HIFU; 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 the need for a scalpel or needle

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety and feasibility measured by the number of patients who are able to be treated with MR HIFU and do not experience significant treatment related adverse events.	Safety and feasibility will be measured by the number of patients who are able to be treated with MR HIFU and do not experience significant treatment related adverse events.	12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Response	Provide an assessment of MR-HIFU ablation of OO in children by the number of patients who experience a measurable clinical and imaging response.	12 months

Collaborators and Investigators

• Sponsor: Children's National Research Institute
• Investigators: Principal Investigator: Karun Sharma, MD, PhD, Children's National Research Institute

Publications and helpful links

General Publications

• Sharma KV, Yarmolenko PS, Celik H, Eranki A, Partanen A, Smitthimedhin A, Kim A, Oetgen M, Santos D, Patel J, Kim P. Comparison of Noninvasive High-Intensity Focused Ultrasound with Radiofrequency Ablation of Osteoid Osteoma. J Pediatr. 2017 Nov;190:222-228.e1. doi: 10.1016/j.jpeds.2017.06.046. Epub 2017 Aug 17.

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2015
• Primary Completion (Actual): October 3, 2020
• Study Completion (Actual): October 3, 2020

Study Registration Dates

• First Submitted: January 15, 2015
• First Submitted That Met QC Criteria: January 28, 2015
• First Posted (Estimate): January 29, 2015

Study Record Updates

• Last Update Posted (Actual): March 11, 2022
• Last Update Submitted That Met QC Criteria: March 9, 2022
• Last Verified: March 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Bone Tissue; Neoplasms, Connective Tissue; Osteoma; Osteoma, Osteoid
• Other Study ID Numbers: HIFU OO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No