MR-HIFU Treatment of Painful Osteoid Osteoma
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|ClinicalTrials.gov Identifier: NCT04658771|
Recruitment Status : Recruiting
First Posted : December 8, 2020
Last Update Posted : December 3, 2021
|Condition or disease||Intervention/treatment||Phase|
|Osteoid Osteoma||Device: MR-HIFU treatment||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||30 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Actual Study Start Date :||January 28, 2021|
|Estimated Primary Completion Date :||June 30, 2022|
|Estimated Study Completion Date :||June 30, 2023|
Experimental: All patients
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
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.
- Number of participants with treatment-related adverse events as assessed by the Common Terminology Criteria for Adverse Events (CTCAE) v.5 [ Time Frame: 12 months ]CTCAE v.5 will be used
- Pain relief as assessed by pain medication usage [ Time Frame: 12 months ]The patient pain and medication diary will be used
- Pain relief as assessed using the Visual Analog Scale (VAS) [ Time Frame: 12 months ]The VAS is a continuous numerical pain scale ranging from 0 (no pain) to 10 (worst possible pain)
- Pain relief as assessed by using the PROMIS Pain Interference short form [ Time Frame: 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)
- Quality of life as assessed by using the Pediatric Quality of Life Inventory (PedsQL) v4.0 [ Time Frame: 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)
- Quality of life as assessed by using the Symptom Distress Scale (SDS) [ Time Frame: 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)
- Number of participants with change in nidus size (mm) as assessed via MRI [ Time Frame: 12 months ]Number of participants with change in nidus size (mm) as assessed via MRI
- Number of participants with change in nidus vascularity as assessed via MRI [ Time Frame: 12 months ]Number of participants with change in nidus vascularity as assessed via MRI
- Number of participants with changes in soft tissue and marrow edema as assessed via MRI [ Time Frame: 12 months ]Number of participants with changes in soft tissue and marrow edema as assessed via MRI
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04658771
|Contact: Ann Liew, MSemail@example.com|
|Contact: General HIFU trials||HIFUtrials@childrensnational.org|
|United States, District of Columbia|
|Children's National Hospital||Recruiting|
|Washington, District of Columbia, United States, 20010|
|Contact: Karun Sharma, MD, PhD 202-476-3791 firstname.lastname@example.org|
|Contact: Ann Liew, MS 202-476-6755 email@example.com|
|Principal Investigator: Karun Sharma, MD, PhD|
|Principal Investigator:||Karun Sharma, MD, PhD||Children's National Research Institute|