USC’s Clinical Trials Centre is working with the Sunshine Coast Haematology and Oncology Centre at Buderim to conduct a trial of a new treatment for people with Primary Cold Agglutinin Disease (CAgD).

This disease is a rare form of autoimmune haemolytic anaemia, in which the body mistakenly attacks and destroys its own red blood cells. There is currently no cure for the condition.

People with severe Primary CAgD can require numerous blood transfusions to manage their anaemia which, on its own, presents inherent risks.

Patients can also experience fatigue, dizziness, headaches, cold hands and feet, pale skin, dark urine, jaundice, chest pain, pain in the back or legs, vomiting or diarrhea or even heart problems.

USC Clinical Trials Centre Director Lucas Litewka said the trial would research whether a new investigational product could increase haemoglobin levels in order to reduce or eliminate the need for blood transfusions.

“Patients aged 18 years or over who have been medically diagnosed with Primary CAgD can participate in the trial,” he said. “Participants will be asked to attend 16 clinic visits over an initial six-month period, then fortnightly visits for up to two years.”

Principal investigator Dr Sorab Shavaksha said the rare condition could be difficult to manage because there was no single established treatment.

“Often for these rare diseases we rely on treatment that is primarily intended for other conditions that are similar, rather than on something that has been developed specifically for the disease itself,” he said.

“This new investigational product offers an exciting new opportunity to see whether we can manage this condition more effectively with targeted therapy.”

Those who meet the criteria and are enrolled in the study will be reimbursed for their time. Patients interested in participating can go to www.usc.edu.au/trials or call (07) 5456 3797.

— Terry Walsh