MR-HIFU Treatment of Painful Osteoid Osteoma

Osteoid Osteoma Clinical Trial

Official title:

Pivotal / Phase II Clinical Trial of Magnetic Resonance-Guided Focused Ultrasound (MR-HIFU) Treatment of Painful Osteoid Osteoma in Children and Young Adults

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04658771
• Other study ID #: HIFU OO Phase II
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: January 28, 2021
• Est. completion date: June 30, 2025

Study information

• Verified date: January 2024
• Source: Children's National Research Institute
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Description:

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 30
• Est. completion date: June 30, 2025
• Est. primary completion date: December 30, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 0 Years to 30 Years

Eligibility

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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.

Related Conditions & MeSH terms

• Osteoid Osteoma
• Osteoma, Osteoid

Intervention

Device:
• MR-HIFU treatment
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.

Locations

Country	Name	City	State
United States	Children's National Hospital	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Children's National Research Institute

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	Pain relief as assessed by pain medication usage	The patient pain and medication diary will be used	12 months
Primary	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
Primary	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
Primary	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
Primary	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	Number of participants with change in nidus size (mm) as assessed via MRI	Number of participants with change in nidus size (mm) as assessed via MRI	12 months
Secondary	Number of participants with change in nidus vascularity as assessed via MRI	Number of participants with change in nidus vascularity as assessed via MRI	12 months
Secondary	Number of participants with changes in soft tissue and marrow edema as assessed via MRI	Number of participants with changes in soft tissue and marrow edema as assessed via MRI	12 months