Sarcoma Trials (Osteosarcoma, Rhabdomyosarcoma, Ewing Sarcoma, non-rhabdomyosarcoma)
1. Title: Phase II Study of nab-Paclitaxel in Combination with Gemcitabine for Treatment of Recurrent/Refractory Sarcoma in Teenagers and Young Adults
PI: Javier Oesterheld, MD (Levine Cancer Institute)
Patients who have recurrent or progressive disease following frontline treatment for osteosarcoma or Ewing sarcoma have a dismal prognosis, with less than one-fifth of patients achieving long-term cure. Although many adolescent and young adults with localized bone or soft tissue sarcoma can become long-term survivors, more effective therapies are needed for patients who present with metastatic disease or whose tumors recur after completing therapy.
The FDA has approved the use of gemcitabine and nab-palatal for the treatment of advanced pancreatic cancer and it is thought this combination will be more tolerable and effective than the current regimen of gemcitabine + docetaxel used to treat this population. This trial will look at this combination of nab-paclitaxel and gemcitabine in its ability to prevent the formation or growth of tumors in teenagers and young adults with relapsed or refractory osteosarcoma, Ewing sarcoma, rhabdomyosarcoma and other soft tissue sarcoma. The trial will also look at the length of time during and after treatment that the disease does not get worse, and determine. If nab-paclitaxel combined with gemcitabine is safe and tolerable.
2. Title: A Phase Ib/II Study to Evaluate the Safety, Feasibility and Efficacy of Nivolumab or Nivolumab in Combination with Azacitidine in Patients with Recurrent, Resectable Osteosarcoma
PI: Mihaela Druta, MD (Moffitt Cancer Center) & Patrick Thompson, MD (University of North Carolina)
The purpose of the study is to see if Nivolumab or Nivolumab in combination with Azacitidine given to patients before and after surgery is safe and to see if patients are able to successfully complete the treatment before their surgery without any extended delays in treatment.
This will be the first time both drugs, Nivolumab and Azacitidine are being used in combination to treat Osteosarcoma. Azacitidine is a cytotoxic agent; it prevents cells from reproducing or growing. Nivolumab is a human monoclonal antibody; a type of molecule created in a laboratory that targets cancer cells.
3. Title: Evolutionary inspired therapy for newly diagnosed, metastatic, Fusion Positive Rhabdomyosarcoma
PI: Damon Reed, MD (Moffitt Cancer Center)
Metastatic, fusion positive rhabdomyosarcoma (RMS) have a poor outcome which is worsened with additional risk factors commonly called the Oberlin criteria. Patients that meet all 4 Oberlin criteria have an Event Free Survival (EFS) of less than 20% at 2 years. A more recent publication
demonstrated a 6% 3 year EFS for any alveolar RMS with metastases at diagnosis. Attempts to intensify therapy through combining a series of active regimens has failed to improve survival in this high risk population.
All therapeutic arms on this study are designed to meet the same primary aim of improving the 3 year event free survival from 6% to 35% for these patients. We will investigate 3 arms that translate lessons from ecological theories of extinction and evolutionary principles of integrated pest management to develop resistance management plans and to improve patient outcomes along with a fourth conventional therapy arm.
4. Title: Phase 1 trial of the lsd1 inhibitor sp-2577 in patients with relapsed or refractory Ewing sarcoma
PI: Damon Reed, MD (Moffitt Cancer Center)
This trial is a targeted treatment for individuals diagnosed with refractory or recurrent Ewing sarcoma. Ewing sarcoma is an aggressive, small round blue cell tumor typically presenting as a primary bone tumor in children and young adults.
This trial will be testing LSD1, an enzyme that targets EWS/FLI1 activity; the critical pathophysiologic mechanism of Ewing sarcoma, through disruption of the function of associated proteins represents an intriguing strategy for clinical intervention.
