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

NCT02076906 | Completed Phase 1 DMC

• 1 other identifier: HIFU Pediatric Solid Tumors
• Study Type: interventional
• Target: 7
• Locations: 1 country
• Sites: 2

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.

Conditions

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

Outcome Measures

Primary Outcomes (1)

• 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.

  28 days following ablative therapy

Secondary Outcomes (3)

• Disease response

  Up to 1 year

• Patient reported outcomes and quality of life measurements

  Up to 1 year

• Immune Markers

  Pre-treatment, 1 day post treatment, 7 days post treatment

Study Arms (1)

MR-HIFU

EXPERIMENTAL

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

Device: Magnetic Resonance High Intensity Focused Ultrasound

Interventions

Magnetic Resonance High Intensity Focused Ultrasound DEVICE

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.

Also known as: MR-HIFU, HIFU, Philips Sonalleve

MR-HIFU

Eligibility Criteria

• Age: Up to 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• AeRang Kim: lead

Study Sites (2)

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

MeSH Terms

Conditions

Rhabdomyosarcoma; Sarcoma, Ewing; Osteosarcoma; Neuroblastoma; Wilms Tumor; Carcinoma, Hepatocellular; Neoplasms, Germ Cell and Embryonal; Desmoid Tumors

Condition Hierarchy (Ancestors)

Myosarcoma; Neoplasms, Muscle Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Sarcoma; Neoplasms, Bone Tissue; Neoplasms, Connective Tissue; Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Neoplasms, Complex and Mixed; Kidney Neoplasms; Urologic Neoplasms; Urogenital Neoplasms; Neoplasms by Site; Neoplastic Syndromes, Hereditary; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Kidney Diseases; Urologic Diseases; Male Urogenital Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Adenocarcinoma; Carcinoma; Liver Neoplasms; Digestive System Neoplasms; Digestive System Diseases; Liver Diseases; Fibroma; Neoplasms, Fibrous Tissue

Study Officials

• AeRang Kim, MD, PhD

  Children's National Research Institute

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: MD, PhD

Study Record Dates

• First Submitted: January 28, 2014
• First Posted: March 4, 2014
• Study Start: April 1, 2014
• Primary Completion: December 27, 2021
• Study Completion: November 24, 2024
• Last Updated: January 1, 2025

Record last verified: 2024-12

Locations

US (2)