A Phase I Study of Lyso-thermosensitive Liposomal Doxorubicin and MR-HIFU for Pediatric Refractory Solid Tumors
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.
- Pediatric Cancer
- Solid Tumors
- Ewing Sarcoma
- Soft Tissue Sarcomas
- Wilms Tumor
- Hepatic Tumor
- Germ Cell Tumors
- Eligible Ages
- Under 30 Years
- Eligible Genders
- Accepts Healthy Volunteers
- Part A: ≤21 years of age
- Part B: ≤ 30 years of age.
- Histologically confirmed malignant solid tumor.
- Patient must have at least one tumor located in areas accessible to HIFU, which will be defined as the target lesion(s).
- Radiographically evaluable or measurable solid tumor target lesion(s).
- Malignant tumor that is relapsed or refractory and with no other potentially curative treatment options available.
- Must have completed an appropriate washout period from prior therapy.
- Karnofsky or Lansky performance level ≥ 50%.
- Must have appropriate hematologic, hepatic and renal function.
- Adequate cardiac function with ejection fraction > 50%
- Clinically significant unrelated systemic illness.
- Patients who are pregnant or breast-feeding.
- Implant or prosthesis or scar tissue within the path of the HIFU beam.
- Target lesion <1 cm from nerve plexus, spinal canal, and bowel.
- Lesion in the skull.
- Inability to undergo MRI and/or contraindication for MRI.
- Inability to tolerate stationary position during HIFU.
- Previous history of hypersensitivity to doxorubicin or its liposomal formulations.
- Phase 1
- Study Type
- Intervention Model
- Single Group Assignment
- Primary Purpose
- None (Open Label)
|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||
Children's National and nearby locations
- NCT ID
- AeRang Kim
Study ContactAnn Liew, MS, CCRP
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.