Osteoid OsteomaPhase 2NCT04658771
MR-HIFU Treatment of Painful Osteoid Osteoma
A Phase 2 study testing MR-HIFU treatment in people with Osteoid Osteoma, run by Children's National Research Institute at 1 site in United States.
Trial summary
To determine treatment safety and efficacy of MR-HIFU ablation of painful Osteoid Osteoma (OO) in children and young adults. Read more about this study
- Who is it for?
- Ages 0 to 30 years
- Conditions the study is for
- Osteoid Osteoma
- What is being tested?
- MR-HIFU treatment
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- 28 Jan 2021to 30 Dec 2025
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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:
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's National Hospital
Washington D.C., District of Columbia, 20010
About this study
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
To determine treatment safety and efficacy of MR-HIFU ablation of painful Osteoid Osteoma (OO) in children and young adults.
Read the detailed 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.
- Conditions
- Osteoid Osteoma
- Being tested
- MR-HIFU treatment
- Medical subject headings
- Osteoma, Osteoid
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MR-HIFU treatmentDevice
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Device: MR-HIFU treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 12 months
Pain relief as assessed by pain medication usage
The patient pain and medication diary will be used
Measured: 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)
Measured: 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)
Measured: 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)
Measured: 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)
Measured: 12 months
Secondary outcomes (3)
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
Measured: 12 months
Number of participants with change in nidus vascularity as assessed via MRI
Number of participants with change in nidus vascularity as assessed via MRI
Measured: 12 months
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
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Dec 2020
- Study start28 Jan 2021
- Primary completion11 Jun 2024
- Study completion30 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Osteoid OsteomaStudies of the same condition, open and under way.
- More trials run by Children's National Research InstituteThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's National Research Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Karun Sharma MD, Children's National Research Institute
- Investigators
- Karun Sharma, MD, PhD, Principal investigator, Children's National Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04658771
- Sponsor's protocol ID
- HIFU OO Phase II
- Status last verified
- Jul 2025
- First submitted
- 13 Nov 2020
- Last updated
- 11 Jul 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04658771
- Acronym / sponsor ID
- HIFU OO Phase II
- Conditions
- Osteoid Osteoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Children's National Research InstituteUniversity, hospital or other
- Study start
- 28 Jan 2021 (actual)
- Primary completion
- 11 Jun 2024 (actual)
- Study completion
- 30 Dec 2025 (estimated)
- First posted
- 8 Dec 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To determine treatment safety and efficacy of MR-HIFU ablation of painful Osteoid Osteoma (OO) in children and young adults.
Detailed 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| All patients | Experimental | Device: MR-HIFU treatmentPatients 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. |
Who may qualify
- Ages
- 0 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (11 listed)
- 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.
Key exclusion criteria (10 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MR-HIFU treatmentMR-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. | Device | All patients |
Primary outcome
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)
3 secondary outcomes
Number of participants with change in nidus size (mm) as assessed via MRI
Time frame: 12 months
Number of participants with change in nidus vascularity as assessed via MRI
Time frame: 12 months
Number of participants with changes in soft tissue and marrow edema as assessed via MRI
Time frame: 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jul 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Karun Sharma, MD, PhD principal investigator
Children's National Research Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing MR-HIFU treatment in people with Osteoid Osteoma, run by Children's National Research Institute at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 11 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jul 2025.