Treating Metastatic Breast or Pancreatic Cancer in People with an Inherited BRCA or PALB2 Mutation Using Chemotherapy and Patients’ 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 are no restrictions 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 Involves

Before receiving treatment, participants will undergo a procedure called apheresis to collect a type of cell called a stem cell from your blood. The stem cells are like seeds from which bone marrow will grow. 

Participants will undergo 2 cycles of treatment. For each cycle:

• participants will be hospitalized for their treatments.
• participants will receive an IV (in the vein) injection of two chemotherapy drugs, melphalan and BCNU and vitamin B12b and vitamin C and low dose ethanol (alcohol).
• 2 days after these treatments, participants will receive an injection of the treated stem cells.
• participants will have daily blood tests to monitor the recovery of their blood counts.

Cycle 2 will begin 6 weeks after the first study treatment.

After finishing the second study treatment, participants will be followed for 1 year to watch for side effects. Participants will have follow up visits at 1, 3, 6, 9, and 12 months after the second study treatment.

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 genetic condition known as a G6PD deficiency.