A Pilot Study of Thermodox and MR-HIFU for Treatment of Relapsed Solid Tumors
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|ClinicalTrials.gov Identifier: NCT04791228|
Recruitment Status : Not yet recruiting
First Posted : March 10, 2021
Last Update Posted : August 3, 2021
|Condition or disease||Intervention/treatment||Phase|
|Solid Tumors Soft Tissue Sarcoma Ewing Sarcoma Malignant Epithelial Neoplasm Rhabdomyosarcoma Wilms Tumor Hepatic Tumor Germ Cell Tumor Bone Metastases||Device: Magnetic Resonance-Guided High Intensity Focused Ultrasound Drug: Lyso-thermosensitive Liposomal Doxorubicin||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||14 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Pilot Study of Lyso-thermosensitive Liposomal Doxorubicin (LTLD, ThermoDox®) and Magnetic Resonance-Guided High Intensity Focused Ultrasound (MR-HIFU) for Treatment of Relapsed or Refractory Solid Tumors|
|Estimated Study Start Date :||October 1, 2021|
|Estimated Primary Completion Date :||June 1, 2024|
|Estimated Study Completion Date :||June 1, 2024|
Experimental: All Patients
LTLD 50 mg/m2 will be administered intravenously over 30 minutes on day 1 of every 21-day cycle. MR-HIFU hyperthermia will follow infusion (+/- 30 minutes) for one hour to a target area with a target temperature of 40-45°C followed by ablation therapy (>55°C). The HIFU hyperthermia regimen will have a duration of at least 60 minutes and will then be followed by ablation therapy. Patients may receive up to a total of 6 cycles. Subsequent treatment cycles may treat alternative target lesions. Disease status will be evaluated using standard imaging techniques (CT/MR) post each cycle.
Device: Magnetic Resonance-Guided High Intensity Focused Ultrasound
Magnetic resonance (MR)-guided high intensity focused ultrasound (HIFU) provides precise and controlled delivery of heat by focusing ultrasound energy inside a lesion using an external applicator without the need for a scalpel or needle.
Additional therapeutic advantages of this modality include its range of bioeffects, including both high temperature tumor ablation via coagulative necrosis, and effects of lower temperature, mild hyperthermia that can help to enhance local drug delivery to tumors. Both tumor ablation and hyperthermia may be employed to potentiate the effects of chemotherapy.
Other Name: MR-HIFU
Drug: Lyso-thermosensitive Liposomal Doxorubicin
LTLD combines doxorubicin with lyso-thermosensitive liposomes that can selectively deliver drug to tumors and when exposed to temperatures greater than 40°C, rapidly and locally releases doxorubicin in high concentrations from systemically administered LTLD.
If combined with hyperthermia, doxorubicin will be released in the heated tumor margins and in any areas within the tumor that were not completely ablated and increase the likelihood of complete tumor necrosis. LTLD will extend tumor cell death to the hyperthermic regions in the peri-ablation zones and minimize the possibility of incomplete ablation or tumor recurrence.
- Primary objective 1: Response of treated target lesion(s) assessed by CT or MRI [ Time Frame: At the end of every 21-day cycle ]The Response Evaluation Criteria in Solid Tumors (RECIST v1.1) will be used
- Primary objective 2: The number of participants with treatment-related adverse events as assessed by the Common Terminology Criteria of Adverse Events (CTCAE) v.5 [ Time Frame: Up to thirty days after last dose of protocol therapy ]CTCAE v.5 will be used
- Exploratory objective 1: Response of non-target lesion(s) assessed by CT or MRI [ Time Frame: At the end of every 21-day cycle ]Lesions that are evaluable or measurable but not in the treated location(s) will be collected
- Exploratory objective 2: Participant reported target tumor pain intensity assessed using the Numerical Rating Scale-11 (NRS-11) [ Time Frame: At the end of every 21-day cycle ]The NRS-11 consists of a 11-point numeric scale to assess pain intensity from 0 (no pain) to 10 (worst pain)
- Exploratory objective 3: Participant reported impact of pain on daily activities assessed using the PROMIS Pain Interference Scale (PROMIS-PI) [ Time Frame: At the end of every 21-day cycle ]The PROMIS-PI consists of 8 questions on pain. Each question will be rated from 0 (never), 1 (almost never), 2 (sometimes), 3 (often), and 4 (almost always)
- Exploratory objective 4: Blood samples taken to see adaptive immune response and immune suppression [ Time Frame: At 1 day and 1 week after MR-HIFU treatment on first 21-day cycle ]To determine changes in pharmacodynamics of immune markers
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04791228
|Contact: General HIFU trials||202-476-6755||HIFUtrials@childrensnational.org|
|Principal Investigator:||AeRang Kim, MD, PhD||Children's National Research Institute|