Neoplasms

All Conditions

Digestive System Diseases

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Diseases and Abnormalities at or Before Birth

Skin and Connective Tissue Diseases

Symptoms and General Pathology

Rare Diseases

Osteosarcoma

Neuroblastoma

Sarcoma

Liver Neoplasms

Wilms Tumor

Sarcoma, Ewing

Rhabdomyosarcoma

Neoplasms, Germ Cell and Embryonal

Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type

Neuroectodermal Tumors, Primitive, Peripheral

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Neuroepithelial

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Neoplasms, Bone Tissue

Neoplasms, Connective Tissue

Myosarcoma

Kidney Neoplasms

Urologic Neoplasms

Urogenital Neoplasms

Syndrome

Neoplastic Syndromes, Hereditary

Urogenital Diseases

Female Urogenital Diseases

Pregnancy Complications

Female Urogenital Diseases and Pregnancy Complications

Kidney Diseases

Urologic Diseases

Male Urogenital Diseases

Genetic Diseases, Inborn

Gastrointestinal Neoplasms

Digestive System Neoplasms

Gastrointestinal Diseases

Liver Diseases

Soft Tissue Sarcoma

Germ Cells Tumors

Wilms' Tumor

Ewing Sarcoma

Neuroepithelioma

Antineoplastic Agents

All Drugs and Chemicals

Anti-Infective Agents

Doxorubicin

Liposomal doxorubicin

Anti-Bacterial Agents

Antibiotics, Antitubercular

Enzyme Inhibitors

LTLD will be administered intravenously in combination with MR-HIFU ablation on day 1 of every 21 day cycle. There will be two potential dose escalation of LTLD with highest dose not to exceed the adult recommended MTD. Patients may receive up to a total of 6 cycles.

LTLD at dose determined from Part A will be administered intravenously on day 1 of every 21 day cycle. MR-HIFU induced MHT will follow immediately post LTLD infusion for one hour to target area with target temperatures of 40-45°C. Patients may receive up to a total of 6 cycles

Magnetic resonance (MR)-high intensity focused ultrasound (HIFU) provides precise controlled delivery of heat by focusing ultrasound energy inside a lesion using an external applicator that is completely non-invasive and non-ionizing.

Magnetic resonance high intensity focused ultrasound

A heat-activated formulation of liposomal doxorubicin with unique property of heat-activated release of doxorubicin, an active agent in most pediatric solid tumors.

Lyso-thermosensitive liposomal doxorubicin

AeRang Kim, MD, PhD

This study is looking to determine the maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) of lyso-thermosensitive liposomal doxorubicin (LTLD) administered in combination with MR-HIFU in children with relapsed/refractory 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, and germ cell tumors.

This is phase 1 trial of LTLD with MR-HIFU induced heating in children and young adults with relapsed/refractory solid tumors.

Part A of the trial will be a traditional dose escalation study to determine the pediatric MTD/RP2D of LTLD combined with MR-HIFU ablation which allows for release of doxorubicin in the ablation zone and peri-ablation margins.

Part B of the trial will combine LTLD at the MTD/RP2D with MR-HIFU induced mild hyperthermia (MHT) in an expanded cohort.

A Phase I Study of Lyso-thermosensitive Liposomal Doxorubicin and MR-HIFU for Pediatric Refractory Solid Tumors

A Phase I Study of Lyso-thermosensitive Liposomal Doxorubicin (LTLD, ThermoDox®) and Magnetic Resonance-Guided High Intensity Focused Ultrasound (MR-HIFU) for Relapsed or Refractory Solid Tumors in Children, Adolescents, and Young Adults

To determine the maximum tolerated dose (MTD) or recommended phase 2 dose (RP2D) of LTLD administered in combination with MR-HIFU ablation in children with relapsed/refractory solid tumors by examining blood samples collected from participants

To define and describe the toxicities of LTLD administered in combination with MR-HIFU as measured by reportable adverse events of participants as per the CTCAE v 4.0

Analyze blood samples of participants to characterize the pharmacokinetic properties of LTLD administered in combination with MR-HIFU

To determine the feasibility of LTLD administered at the recommended dose in combination with MR-HIFU induced mild hyperthermia (MHT) as determined by patient outcomes and adverse events

To preliminarily determine the antitumor activity of LTLD with MR-HIFU within the confines of a phase 1 study by examining the response of target lesions using RECIST criteria v1.1

To determine changes in symptoms and quality of life in children treated with LTLD and MR-HIFU by administering the Symptom Distress Scale and Peds QL v4.0 to participants

To determine the changes in immune markers in children treated with LTLD and MR-HIFU by examining blood samples from participants

AeRang Kim

MD, PhD

Behaviors and Mental Disorders

Fibromatosis, Aggressive

Aggression

Fibroma

Neoplasms, Fibrous Tissue

Desmoid Tumor

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

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.

Magnetic Resonance High Intensity Focused Ultrasound

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.

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.

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

Safety and Feasibility Study of Using MR-guided High Intensity Focused Ultrasound (HIFU) for the Ablation of Relapsed or Refractory Pediatric Solid Tumors

Toxicity will be evaluated using CTCAEv4. Adverse events will be summarized descriptively with tabulations of adverse event type, severity, and relationship to study treatment.

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.

We will use a validated symptom distress scale and quality of life metrics pre- and post-therapy at scheduled intervals.

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

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
A Phase I Study of Lyso-thermosensitive Liposomal Doxorubicin and MR-HIFU for Pediatric Refractory Solid Tumors	NCT02536183	Pediatric Cance... Solid Tumors Rhabdomyosarcom... Ewing Sarcoma Soft Tissue Sar... Osteosarcoma Neuroblastoma Wilms Tumor Hepatic Tumor Germ Cell Tumor...	Magnetic resona... Lyso-thermosens...	- 30 Years	Children's National Research Institute
MR-guided High Intensity Focused Ultrasound (HIFU) on Pediatric Solid Tumors	NCT02076906	Relapsed Pediat... Refractory Pedi... Rhabdomyosarcom... Ewing Sarcoma Osteosarcoma Neuroblastoma Wilms Tumor Hepatic Tumor Germ Cell Tumor Desmoid Tumor	Magnetic Resona...	- 30 Years	Children's National Research Institute
A Phase I Study of Lyso-thermosensitive Liposomal Doxorubicin and MR-HIFU for Pediatric Refractory Solid Tumors	NCT02536183	Pediatric Cance... Solid Tumors Rhabdomyosarcom... Ewing Sarcoma Soft Tissue Sar... Osteosarcoma Neuroblastoma Wilms Tumor Hepatic Tumor Germ Cell Tumor...	Magnetic resona... Lyso-thermosens...	- 30 Years	Children's National Research Institute
MR-guided High Intensity Focused Ultrasound (HIFU) on Pediatric Solid Tumors	NCT02076906	Relapsed Pediat... Refractory Pedi... Rhabdomyosarcom... Ewing Sarcoma Osteosarcoma Neuroblastoma Wilms Tumor Hepatic Tumor Germ Cell Tumor Desmoid Tumor	Magnetic Resona...	- 30 Years	Children's National Research Institute

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