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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 : February 15, 2021
Sponsor:
Information provided by (Responsible Party):
Karun Sharma MD, Children's National Research Institute

Tracking Information
First Submitted Date  ICMJE November 13, 2020
First Posted Date  ICMJE December 8, 2020
Last Update Posted Date February 15, 2021
Actual Study Start Date  ICMJE January 15, 2021
Estimated Primary Completion Date January 30, 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 11, 2021)
  • 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)
Original Primary Outcome Measures  ICMJE
 (submitted: December 4, 2020)
  • Number of participants with treatment-related adverse events as assessed by CTCAE v.5 [ Time Frame: 12 months ]
    The Common Terminology Criteria for Adverse Events (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 VAS [ Time Frame: 12 months ]
    The Visual Analog Scale (VAS) will be used
  • Pain relief as assessed by using the Pain Interference short form [ Time Frame: 12 months ]
    The PROMIS Pediatric Pain Interference Short Form will be used
  • Quality of life as assessed by using the PedsQL v4.0 [ Time Frame: 12 months ]
    The Pediatric Quality of Life Inventory v4.0 will be used
  • Quality of life as assessed by using the SDS [ Time Frame: 12 months ]
    The Symptom Distress Scale will be used
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 4, 2020)
  • 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
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE MR-HIFU Treatment of Painful Osteoid Osteoma
Official Title  ICMJE Pivotal / Phase II Clinical Trial of Magnetic Resonance-Guided Focused Ultrasound (MR-HIFU) Treatment of Painful Osteoid Osteoma in Children and Young Adults
Brief Summary 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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Osteoid Osteoma
Intervention  ICMJE 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.

Study Arms  ICMJE 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.

Intervention: Device: MR-HIFU treatment
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: December 4, 2020)
30
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE November 30, 2023
Estimated Primary Completion Date January 30, 2023   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 30 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Ann Liew, MS 202-476-6755 aliew@childrensnational.org
Contact: General HIFU trials HIFUtrials@childrensnational.org
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04658771
Other Study ID Numbers  ICMJE HIFU OO Phase II
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: Yes
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Karun Sharma MD, Children's National Research Institute
Study Sponsor  ICMJE Children's National Research Institute
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Karun Sharma, MD, PhD Children's National Research Institute
PRS Account Children's National Research Institute
Verification Date February 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP