Relapsed Pediatric Solid TumorsPhase 1NCT02076906
MR-guided High Intensity Focused Ultrasound (HIFU) on Pediatric Solid Tumors
A Phase 1 study testing Magnetic Resonance High Intensity Focused Ultrasound in people with Relapsed Pediatric Solid Tumors and Refractory Pediatric Solid Tumors, run by AeRang Kim at 2 sites in United States.
Trial summary
The purpose of this study is to determine if Magnetic Resonance guided High Intensity Focused Ultrasound ablative therapy is safe and feasible for children, adolescents, and young adults with refractory or relapsed solid tumors. Read more about this study
- Who is it for?
- Ages Up to 30 years
- Conditions the study is for
- Relapsed Pediatric Solid Tumors, Refractory Pediatric Solid Tumors, Rhabdomyosarcoma
- What is being tested?
- Magnetic Resonance High Intensity Focused Ultrasound
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 2 sitesUnited States
- Study dates
- Apr 2014to 24 Nov 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 Up to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- AGE: ≤ 30 years of age.
- DIAGNOSIS:
- Histologically confirmed malignant solid tumors, which may include but are not limited to rhabdomyosarcoma and other soft tissue sarcomas, Ewing's sarcoma family of tumors, osteosarcoma, neuroblastoma, Wilms' tumor, hepatic tumors, germ cell tumors, and desmoid tumors.
- TUMOR LOCATION: Target lesion(s) must be located in bone or soft tissue in close proximity to bone. Target lesions must be reachable within the normal safety margins of HIFU as specified in the instructions for use.
- TARGET LESION(S): Radiographically evaluable or measurable solid tumor target lesion(s).
- THERAPEUTIC OPTIONS:
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- Malignant Tumor: The patient's cancer must have relapsed after or failed to respond to frontline curative therapy and there must not be other potentially curative treatment options available. Curative therapy may include surgery, radiation therapy, chemotherapy, or any combination of these modalities.
- PRIOR THERAPY:
- Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering on this study.
- No limitation on the number of prior chemotherapy regimens that the patient may have received prior to study entry.
- Myelosuppressive chemotherapy: The last dose of all myelosuppressive anticancer drugs must be at least 3 weeks prior to study entry.
- Immunotherapy: The last dose of immunotherapy (monoclonal antibody or vaccine) must be at least 4 weeks prior to study entry.
- Biologic (anti-cancer agent): The last dose of all biologic agents for the treatment of the patient's cancer (such as retinoids or tyrosine kinase inhibitors) must be at least 7 days prior to study entry.
- Radiation therapy: The last dose of radiation to more than 25 % of marrow containing bones (pelvis, spine, skull) must be at least 4 weeks prior to study entry. The last dose of all other local palliative (limited port) radiation must be at least 2 weeks prior to study entry.
- Stem Cell Transplantation. At least 2 months post-autologous stem cell transplant or at least 3 months post-allogeneic transplant and recovered from toxicities without evidence of graft versus host disease and on stable doses of immunosuppressive medications if required.
- Growth Factors. The last dose of colony stimulating factors, such as filgrastim, sargramostim, and erythropoietin, must be at least 1 week prior to study entry, the last dose of long-acting colony stimulating factors, such as pegfilgrastim, must be at least 2 weeks prior to study entry.
- CONCURRENT THERAPIES:
- No other anti-cancer therapy (chemotherapy, biological therapy, radiation therapy) is permitted during HIFU treatment and post treatment follow up for tolerability (see section 3.3).
- PERFORMANCE STATUS:
- Patients > 16 years old must have a Karnofsky performance level ≥ 50%, and children ≤ 16 years old must have a Lansky performance level ≥ 50% (See Appendix I).
- Patients who are unable to walk because of paralysis or motor weakness, but who are up in a wheelchair will be considered ambulatory for the purpose of calculating the performance score.
- HEMATOLOGIC FUNCTION:
- Peripheral absolute neutrophil count (ANC) of ≥750/µL
- Platelet count ≥75,000/µL (may receive transfusions)
- RENAL FUNCTION: Age-adjusted normal serum creatinine OR a creatinine clearance ≥60 mL/min/1.73 m2.
- ADEQUATE PULMONARY FUNCTION: Defined as no dyspnea at rest, and a pulse oximetry >94% on room air if there is clinical indication for determination.
- Normal PT, PTT and INR < 1.5 x ULN (including patients on prophylactic anticoagulation)
Who cannot join
- Clinically significant unrelated systemic illness, such as serious infections, hepatic, renal or other organ dysfunction, which in the judgment of the Principal or Associate Investigator would compromise the patient's ability to tolerate the general anesthetic required for the procedure.
- Implant or prosthesis or scar tissue within the path of the HIFU beam.
- Target <1 cm from nerve plexus, spinal canal, bladder, bowel
- Target in contact with hollow viscera
- Lesion in the skull
- Inability to undergo MRI and/or contraindication for MRI
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- Inability to tolerate stationary position during HIFU
- Patients currently receiving other anticancer agents.
- Patients currently receiving other investigational agents.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
Official title: Safety and Feasibility Study of Using MR-guided High Intensity Focused Ultrasound (HIFU) for the Ablation of Relapsed or Refractory Pediatric Solid Tumors
The purpose of this study is to determine if Magnetic Resonance guided High Intensity Focused Ultrasound ablative therapy is safe and feasible for children, adolescents, and young adults with refractory or relapsed solid tumors.
Read the detailed description
Magnetic Resonance (MR)-guided high intensity focused ultrasound (HIFU) is an innovative technique that allows for non-invasive thermal ablation of tissue. Advantages over conventional local tumor control such as surgery, radiation, or radiofrequency are that MR-HIFU is completely non-invasive, non-ionizing, and enables ablation of large tumor volumes with avoidance of adjacent tissue injury. This study will evaluate the safety and feasibility of MR-HIFU ablative therapy in children, adolescents, and young adults with refractory or relapsed solid tumors that are located in bone or soft tissue in close proximity to bone. Patients ≤ 30 years of age with refractory or relapsed solid tumors with measurable target lesions that are located in bone or soft tissue in close proximity to bone are eligible. Tolerability will be defined during the 14 days following MR-HIFU ablation. Patients will continue to be followed for tumor response and secondary outcomes for up to one year post ablation treatment.
- Conditions
- Relapsed Pediatric Solid Tumors, Refractory Pediatric Solid Tumors, Rhabdomyosarcoma, Ewing Sarcoma, Osteosarcoma, Neuroblastoma
- Being tested
- Magnetic Resonance High Intensity Focused Ultrasound
- Keywords
- relapsed pediatric solid tumors, refractory pediatric solid tumors, high intensity focused ultrasound, rhabdomyosarcoma, Ewing's sarcoma, osteosarcoma, neuroblastoma, Wilms' tumor, hepatic tumors, germ cell tumors, desmoid tumor
- Medical subject headings
- Rhabdomyosarcoma, Sarcoma, Ewing, Osteosarcoma, Neuroblastoma, Wilms Tumor, Carcinoma, Hepatocellular, Neoplasms, Germ Cell and Embryonal, Desmoid Tumors
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Magnetic Resonance High Intensity Focused UltrasoundDevice
Also called MR-HIFU, HIFU, Philips Sonalleve
MR-High intensity focused ultrasound (HIFU) is an ultrasound based technology which allows for non-invasive thermal ablation of tumors under the guidance of MR thermography.
| Study group | Role | What they receive |
|---|---|---|
| MR-HIFU | Experimental | Magnetic Resonance High Intensity Focused Ultrasound (MR-HIFU) ablative therapy for children with recurrent or relapsed solid tumors. Receives: Device: Magnetic Resonance High Intensity Focused Ultrasound |
What is being measured
The primary outcome is the main question the study is designed to answer.
Toxicity
Toxicity will be evaluated using CTCAEv4. Adverse events will be summarized descriptively with tabulations of adverse event type, severity, and relationship to study treatment.
Measured: 28 days following ablative therapy
Secondary outcomes (3)
Disease response
We will preliminarily define tumor response from MR-HIFU ablative therapy assessed according to a modified Response Evaluation Criteria in Solid Tumors (RECIST). We will also evaluate functional tumor response using FDG-PET post MR-HIFU ablative treatment for patients with PET avid target lesions.
Measured: Up to 1 year
Patient reported outcomes and quality of life measurements
We will use a validated symptom distress scale and quality of life metrics pre- and post-therapy at scheduled intervals.
Measured: Up to 1 year
Immune Markers
Changes in immune markers from baseline and post therapy will be evaluated.
Measured: Pre-treatment, 1 day post treatment, 7 days post treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Mar 2014 (estimated)
- Study startApr 2014
- Primary completion27 Dec 2021
- Study completion24 Nov 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Relapsed Pediatric Solid TumorsStudies of the same condition, open and under way.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- AeRang Kim
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, AeRang Kim, Children's National Research Institute
- Investigators
- AeRang Kim, MD, PhD, Principal investigator, Children's National Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02076906
- Sponsor's protocol ID
- HIFU Pediatric Solid Tumors
- Status last verified
- Dec 2024
- First submitted
- 28 Jan 2014
- Last updated
- 1 Jan 2025
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 1 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Feasibility Study of Using MR-guided High Intensity Focused Ultrasound (HIFU) for the Ablation of Relapsed or Refractory Pediatric Solid Tumors
- ClinicalTrials.gov ID
- NCT02076906
- Acronym / sponsor ID
- HIFU Pediatric Solid Tumors
- Conditions
- Relapsed Pediatric Solid Tumors, Refractory Pediatric Solid Tumors, Rhabdomyosarcoma, Ewing Sarcoma, Osteosarcoma, Neuroblastoma
- Keywords
- relapsed pediatric solid tumors, refractory pediatric solid tumors, high intensity focused ultrasound, rhabdomyosarcoma, Ewing's sarcoma, osteosarcoma, neuroblastoma, Wilms' tumor
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- AeRang KimUniversity, hospital or other
- Study start
- Apr 2014 (actual)
- Primary completion
- 27 Dec 2021 (actual)
- Study completion
- 24 Nov 2024 (actual)
- First posted
- 4 Mar 2014 (estimated)
- 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.
The purpose of this study is to determine if Magnetic Resonance guided High Intensity Focused Ultrasound ablative therapy is safe and feasible for children, adolescents, and young adults with refractory or relapsed solid tumors.
Detailed description
Magnetic Resonance (MR)-guided high intensity focused ultrasound (HIFU) is an innovative technique that allows for non-invasive thermal ablation of tissue. Advantages over conventional local tumor control such as surgery, radiation, or radiofrequency are that MR-HIFU is completely non-invasive, non-ionizing, and enables ablation of large tumor volumes with avoidance of adjacent tissue injury. This study will evaluate the safety and feasibility of MR-HIFU ablative therapy in children, adolescents, and young adults with refractory or relapsed solid tumors that are located in bone or soft tissue in close proximity to bone. Patients ≤ 30 years of age with refractory or relapsed solid tumors with measurable target lesions that are located in bone or soft tissue in close proximity to bone are eligible. Tolerability will be defined during the 14 days following MR-HIFU ablation. Patients will continue to be followed for tumor response and secondary outcomes for up to one year post ablation treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MR-HIFU | Experimental | Device: Magnetic Resonance High Intensity Focused UltrasoundMagnetic Resonance High Intensity Focused Ultrasound (MR-HIFU) ablative therapy for children with recurrent or relapsed solid tumors. |
Who may qualify
- Ages
- Up to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (27 listed)
- AGE: ≤ 30 years of age.
- Histologically confirmed malignant solid tumors, which may include but are not limited to rhabdomyosarcoma and other soft tissue sarcomas, Ewing's sarcoma family of tumors, osteosarcoma, neuroblastoma, Wilms' tumor, hepatic tumors, germ cell tumors, and desmoid tumors.
- TUMOR LOCATION: Target lesion(s) must be located in bone or soft tissue in close proximity to bone. Target lesions must be reachable within the normal safety margins of HIFU as specified in the instructions for use.
- TARGET LESION(S): Radiographically evaluable or measurable solid tumor target lesion(s).
- Malignant Tumor: The patient's cancer must have relapsed after or failed to respond to frontline curative therapy and there must not be other potentially curative treatment options available. Curative therapy may include surgery, radiation therapy, chemotherapy, or any combination of these modalities.
Key exclusion criteria (9 listed)
- Clinically significant unrelated systemic illness, such as serious infections, hepatic, renal or other organ dysfunction, which in the judgment of the Principal or Associate Investigator would compromise the patient's ability to tolerate the general anesthetic required for the procedure.
- Implant or prosthesis or scar tissue within the path of the HIFU beam.
- Target <1 cm from nerve plexus, spinal canal, bladder, bowel
- Target in contact with hollow viscera
- Lesion in the skull
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 |
|---|---|---|
| Magnetic Resonance High Intensity Focused UltrasoundAlso: MR-HIFU, HIFU, Philips SonalleveMR-High intensity focused ultrasound (HIFU) is an ultrasound based technology which allows for non-invasive thermal ablation of tumors under the guidance of MR thermography. | Device | MR-HIFU |
Primary outcome
Toxicity
Time frame: 28 days following ablative therapy
Toxicity will be evaluated using CTCAEv4. Adverse events will be summarized descriptively with tabulations of adverse event type, severity, and relationship to study treatment.
3 secondary outcomes
Disease response
Time frame: Up to 1 year
Patient reported outcomes and quality of life measurements
Time frame: Up to 1 year
Immune Markers
Time frame: Pre-treatment, 1 day post treatment, 7 days post treatment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jan 2025.
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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.
AeRang Kim, MD, PhD principal investigator
Children's National Research Institute
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Magnetic Resonance High Intensity Focused Ultrasound in people with Relapsed Pediatric Solid Tumors and Refractory Pediatric Solid Tumors, run by AeRang Kim at 2 sites 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 27 inclusion and 9 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: finished, as of the sponsor's last update on 1 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Toxicity (time frame: 28 days following ablative therapy). 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 1 Jan 2025.