MR-HIFU Treatment of Painful Osteoid Osteoma

NCT04658771 | Active Not Recruiting Phase 2 DMC FDA Device

• 1 other identifier: HIFU OO Phase II
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

To determine treatment safety and efficacy of MR-HIFU ablation of painful Osteoid Osteoma (OO) in children and young adults.

Conditions

Osteoid Osteoma

Outcome Measures

Primary Outcomes (6)

• Number of participants with treatment-related adverse events as assessed by the Common Terminology Criteria for Adverse Events (CTCAE) v.5

  CTCAE v.5 will be used

  12 months

• Pain relief as assessed by pain medication usage

  The patient pain and medication diary will be used

  12 months

• Pain relief as assessed using the Visual Analog Scale (VAS)

  The VAS is a continuous numerical pain scale ranging from 0 (no pain) to 10 (worst possible pain)

  12 months

• Pain relief as assessed by using the PROMIS Pain Interference short form

  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)

  12 months

• Quality of life as assessed by using the Pediatric Quality of Life Inventory (PedsQL) v4.0

  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)

  12 months

• Quality of life as assessed by using the Symptom Distress Scale (SDS)

  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)

  12 months

Secondary Outcomes (3)

• Number of participants with change in nidus size (mm) as assessed via MRI

  12 months

• 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

Study Arms (1)

All patients

EXPERIMENTAL

Patients will undergo MR-HIFU ablation procedure of OO under general anesthesia. Patients will be monitored for disease status and adverse events for 12 months following procedure. Patients with incomplete or partial response at 28 days after MR-HIFU will be permitted a second MR-HIFU procedure. If there is still incomplete pain relief without medication use by 28 days following the second MR-HIFU treatment, patients will be offered standard of care treatment with RFA and followed for one year.

Device: MR-HIFU treatment

Interventions

MR-HIFU treatment DEVICE

MR-HIFU is an innovative technology that allows for non-invasive thermal ablation of tissues and tumors by integrating an external high intensity focused ultrasound (HIFU) transducer with a conventional MRI scanner. The focused ultrasound transducer is placed outside of the body and generates and focuses acoustic energy to heat tumors or other pre-defined focal regions of tissues inside the body. Because the acoustic energy is precisely focused over tiny areas of tissue, potential thermal injury to surrounding tissues is minimal. Cell death inside the selected treatment area occurs via coagulative necrosis while avoiding injury to intervening and adjacent tissues.

All patients

Eligibility Criteria

• Age: 0 Years - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Children's National Research Institute: lead

Study Sites (1)

Children's National Hospital

Washington D.C., District of Columbia, 20010, United States

Location

MeSH Terms

Conditions

Osteoma, Osteoid

Condition Hierarchy (Ancestors)

Osteoma; Neoplasms, Bone Tissue; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms

Study Officials

• Karun Sharma, MD, PhD

  Children's National Research Institute

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director of Interventional Radiology

Study Record Dates

• First Submitted: November 13, 2020
• First Posted: December 8, 2020
• Study Start: January 28, 2021
• Primary Completion: June 11, 2024
• Study Completion: December 30, 2025
• Last Updated: July 11, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)