About the SAPPHIRE study

Logo SAPPHIRE AP 12009

Anaplastic astrocytoma is a type of aggressive tumor that affects the central nervous system. It is a primary brain tumor, which means an abnormal cancerous growth of body tissue originating in the brain. Tumors are defined as refractory, if they have been resistant to previous radio- or chemotherapy or other therapeutic treatment. They are defined as recurrent, if they have been initially successfully treated, but have returned. The treatment of recurrent or refractory anaplastic astrocytoma remains a challenge, and medical solutions are needed to improve the quality of life and to extend survival times in patients with this severe illness.

The ongoing SAPPHIRE Phase III study is designed for patients diagnosed with refractory or recurrent anaplastic astrocytoma only. The acronym SAPPHIRE stands for: Efficacy and Safety of AP 12009 in Adult Patients with Recurrent or Refractory Anaplastic Astrocytoma (WHO grade III) as Compared to Standard Treatment with Temozolomide or BCNU: A Randomized, Actively Controlled, Open-label Clinical Phase III Study. The study will assess the efficacy, safety, and effect on quality of life of trabedersen (AP 12009) in these patients, compared with standard chemotherapies. Patients will be randomly assigned to one of two treatment groups:

  1. Trabedersen (AP 12009)
  2. Standard chemotherapy – oral temozolomide [TMZ] (or intravenous BCNU, if the patient has received TMZ previously or TMZ therapy is not appropriate)

Trabedersen is administered via a method defined as convection-enhanced delivery (CED), using a system, which delivers the drug directly into the brain tumor (Figure 1). This means that the drug is targeted to the exact site of the tumor, preventing the need for it to travel from the rest of the body through the brain’s protective barrier, known as the “blood–brain barrier”. Patients participating in the SAPPHIRE study, who receive trabedersen therapy, will undergo minimal surgery to have the drug delivery system fitted.

Figure 1. Components of the trabedersen drug delivery system and surgical procedure

  • External parts (1, 2): The portable infusion pump (1)contains trabedersen in a reservoir. The pump is connected to an infusion line (2), which is connected to the port chamber (3).
  • Implanted parts (3–5): The port chamber (3) is implanted in the frontal part of the chest wall and connected to the port catheter and the intratumoral catheter (4). The catheters are directed under the skin and end in the brain tumor (5).
Components of the trabedersen drug delivery system and surgical procedure

The drug delivery system includes a portable pump, which you can wear attached to a belt around your hip, allowing you to move around freely. This means you will only have to return to the hospital once a week, and you can pursue your daily activities. The maximum length of the treatment with trabedersen is 21 weeks.

If you or someone you know has been diagnosed with recurrent or refractory anaplastic astrocytoma and is possibly interested in participating in the SAPPHIRE study, please click here.