SHARON: A Clinical Trial for Metastatic Cancer With an Inherited BRCA or PALB2 Mutation Using Chemotherapy and Patients’ Own Stem Cells

About the Study

The purpose of this study is to see whether the combination of melphalan, BCNU, vitamin B12b, and vitamin C, followed by autologous (self) bone marrow stem cell infusion, is safe and effective for treating patients with advanced pancreatic cancer or Stage IV, HER2-negative breast cancer who have a BRCA1, BRCA2 or PALB2 inherited mutation. All of these treatments are given intravenously (by vein).

Melphalan and BCNU are both chemotherapy drugs that work by targeting the processes that cancer cells use to grow and spread. Vitamin B12b and vitamin C work together to block cancer cell energy production and prevent cancer cells from repairing the damage caused by the chemotherapy drugs. Autologous bone marrow stem cell infusion involves using healthy blood-forming cells from a patient's own body to replace diseased or damaged bone marrow. The stem cell infusions will help decrease the side effects of the study treatment. This study is open to people who have already received a PARP inhibitor, as well as those who have not. There is no restriction on how many prior lines of treatment a patient has received before enrolling.

You can learn more on the study website at: www.SharonTrial.com. 

What the Study Entails

• All participants will receive the combination of melphalan, BCNU, vitamin B12b, and vitamin C, followed by autologous bone marrow stem cell infusion.
• If you decide to take part in this study, you will undergo a procedure called apheresis to collect and preserve blood stem cells for the autologous bone marrow stem cell infusion. The stem cells collected from your blood are like seeds from which bone marrow will grow. 
• About 1 week after the apheresis, you will be admitted to the hospital for your first study treatment. You will get melphalan, BCNU, vitamin B12b, and vitamin C, and then you will have an autologous bone marrow stem cell infusion 2 days later. You may be able to leave the hospital before you have your stem cell infusion, depending on how you respond to the study treatment.
• About 6 weeks after your first study treatment, you will stay in the hospital again to get a second treatment. You will also have a second stem cell infusion.
• After each stem cell infusion, you will have daily blood tests to monitor the recovery of your blood counts.

After you finish your second study treatment, the study doctor will continue to follow your condition for 1 year and watch you for side effects. The study doctor or a member of the study team will call you monthly to check if you are having any side effects, and you will come to the clinic 1, 3, 6, 9, and 12 months after your second study treatment.

Sites

New York
New York City, NY: Memorial Sloan Kettering Cancer Center 
Lead researcher: Dr. Kenneth Yu

People 18 years or older may be eligible if they:

• have been diagnosed with stage 4 pancreatic cancer or stage 4, HER2-negative breast cancer.
• have an inherited BRCA1, BRCA2 or PALB2 mutation.
• are able to walk and do routine activities for more than half of their normal waking hours.

The study is not open to people who:

• have metastatic disease to the brain, spinal cord, eye, or heart.
• have a current biliary tract infection.
• have a history of only one biliary tract infection and fewer than 30 days have passed since discontinuation of antibiotic treatment.
• have a history of more than one biliary tract infection and after discussion between the site study team and sponsor medical monitor and careful evaluation for suitability the patient is deemed to be unsuitable for the trial due to risk of recurring biliary tract infections.
• have a G6PD deficiency.