Patients with haemophilia A could soon have access to new possible treatments for this serious disorder. This is thanks to a ground-breaking phase 1 clinical trial that investigated the efficacy of a new application of an existing drug in patients.
The trial was conducted by experts from the University of the Free State (UFS) and the South African clinical research organisation Farmovs, as well as the Adult Bleeding Disorder Clinic at the Universitas Academic Hospital in Bloemfontein. It ran from January until July last year and formed part of a global clinical research study.
Prof. Marius Coetzee, an affiliated associate professor in the Department of Haematology and Cell Biology at the UFS, was the principal investigator of the study. He and his co-researchers, Dr Jaco Joubert and Dr Leriska Haupt, work with patients with haemophilia A on a regular basis.
According to Coetzee, haemophilia A is an inherited bleeding disorder that occurs in about 1 in 10 000 people, mostly males.
“It is caused by defects in clotting factor VIII. People with haemophilia bleed longer than others. They often bleed into their joints and muscles, but can bleed anywhere and bleeds can often be life-threatening,” said Coetzee.
“The results were encouraging and will probably lead to further phase 2 and 3 studies on the drug. For patients suffering from this disease, the results mean new options for treatment.”
Farmovs has conducted more than 3 000 International Council on Harmonisation Good Clinical Practice (ICH-GCP) compliant phase 1 to 3 clinical trials for clients globally. Farmovs has a wealth of bioequivalence and phase 1 experience, clinical trial expertise and patient recruitment success through network-investigator sites managed by the Site Management Organisation (SMO) division.
According to Reinard Mc Pherson, chief medical officer at Farmovs, reports such as these highlight the incredible impact that collaborative research efforts can have on local patient care in the short term and on global patient care in the longer term.
“South Africa is well-known for its clinical research legacy over many years. With 47 years of clinical research experience, Farmovs continues to contribute to this legacy. Our collaborative model combines external clinical expertise in challenging patient populations with Farmovs’ expertise in planning and executing clinical trial protocols to create success stories such as the one being shared today,” said Mc Pherson.
The Adult Bleeding Disorder Clinic at Universitas Academic Hospital has prior experience in clinical research collaboration with Farmovs.
The clinic was started in the 1970s and was recognised as a haemophilia treatment centre by the World Federation of Hemophilia in 2000. It serves the entire Free State and Northern Cape.
It has one of the largest cohorts of patients with hereditary haemorrhagic telangiectasia (HHT) in Africa, and also has expertise in Von Willebrand disease.
