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MR-guided High Intensity Focused Ultrasound (HIFU) on Pediatric Solid Tumors

Primary Purpose

Relapsed Pediatric Solid Tumors, Refractory Pediatric Solid Tumors, Rhabdomyosarcoma

Status
Active
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Magnetic Resonance High Intensity Focused Ultrasound
Sponsored by
AeRang Kim
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Relapsed Pediatric Solid Tumors focused on measuring 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

Eligibility Criteria

undefined - 30 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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:

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:

    1. Peripheral absolute neutrophil count (ANC) of ≥750/µL
    2. 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)

Exclusion Criteria:

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

Inability to tolerate stationary position during HIFU

Patients currently receiving other anticancer agents.

Patients currently receiving other investigational agents.

Sites / Locations

  • Children's National Medical Center
  • Cincinnati Children's Hospital Medical Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

MR-HIFU

Arm Description

Magnetic Resonance High Intensity Focused Ultrasound (MR-HIFU) ablative therapy for children with recurrent or relapsed solid tumors.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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.
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.
Immune Markers
Changes in immune markers from baseline and post therapy will be evaluated.

Full Information

First Posted
January 28, 2014
Last Updated
August 9, 2023
Sponsor
AeRang Kim
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1. Study Identification

Unique Protocol Identification Number
NCT02076906
Brief Title
MR-guided High Intensity Focused Ultrasound (HIFU) on Pediatric Solid Tumors
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
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
April 2014 (Actual)
Primary Completion Date
December 27, 2021 (Actual)
Study Completion Date
November 24, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
AeRang Kim

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Relapsed Pediatric Solid Tumors, Refractory Pediatric Solid Tumors, Rhabdomyosarcoma, Ewing Sarcoma, Osteosarcoma, Neuroblastoma, Wilms Tumor, Hepatic Tumor, Germ Cell Tumor, Desmoid Tumor
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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
14 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
MR-HIFU
Arm Type
Experimental
Arm Description
Magnetic Resonance High Intensity Focused Ultrasound (MR-HIFU) ablative therapy for children with recurrent or relapsed solid tumors.
Intervention Type
Device
Intervention Name(s)
Magnetic Resonance High Intensity Focused Ultrasound
Other Intervention Name(s)
MR-HIFU, HIFU, Philips Sonalleve
Intervention Description
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.
Primary Outcome Measure Information:
Title
Toxicity
Description
Toxicity will be evaluated using CTCAEv4. Adverse events will be summarized descriptively with tabulations of adverse event type, severity, and relationship to study treatment.
Time Frame
28 days following ablative therapy
Secondary Outcome Measure Information:
Title
Disease response
Description
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.
Time Frame
Up to 1 year
Title
Patient reported outcomes and quality of life measurements
Description
We will use a validated symptom distress scale and quality of life metrics pre- and post-therapy at scheduled intervals.
Time Frame
Up to 1 year
Title
Immune Markers
Description
Changes in immune markers from baseline and post therapy will be evaluated.
Time Frame
Pre-treatment, 1 day post treatment, 7 days post treatment

10. Eligibility

Sex
All
Maximum Age & Unit of Time
30 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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: 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) Exclusion Criteria: 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 Inability to tolerate stationary position during HIFU Patients currently receiving other anticancer agents. Patients currently receiving other investigational agents.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
AeRang Kim, MD, PhD
Organizational Affiliation
Children's National Research Institute
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's National Medical Center
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States

12. IPD Sharing Statement

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MR-guided High Intensity Focused Ultrasound (HIFU) on Pediatric Solid Tumors

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