Over 95 Patients Enrolled in Sickle Cell Study
Bahrain Among Top Contributors
Researchers from Bahrain are currently enrolling patients in an important study to decrease the pain of sickle cell disease. Ninety-five patients have been enrolled across the Middle East and Europe and the study will be completing enrolment next year.
Background of this clinical study
The aim of the ongoing study is to explore if taking a new medicine called Sevuparin can reduce the duration and pain of the vaso-occlusive crisis (VOC) in sickle cell patients. Sevuparin was initially designed by prominent scientists at the Karolinska Institute in Sweden and is now being developed by the Swedish company Modus Therapeutics (previously Dilaforette), based in Stockholm. Sevuparin is currently being studied in sickle cell disease centers around the world. After the study protocol was reviewed and accepted by all the responsible national authorities, sickle cell patients with VOC are now treated with Sevuparin in the Netherlands, Turkey, Lebanon, Oman, Bahrain and Jamaica. Three centers are also being added in Saudi Arabia.
Sevuparin is a form of the well know medication Heparin, modified to reduce the blood thinning effect. Heparin is widely used as an anticoagulant (blood thinner) for the treatment of deep vein thrombosis and many other common events. Both Heparin and Low molecular weight heparin (LMWH) have anti-adhesive (anti cell clumping) properties, that, when given in high doses, might also be helpful in the treatment of painful crises in sickle cell patients. During a painful crises the sickle-shaped red blood cells obstruct capillaries and restrict blood flow to an organ resulting in ischaemia, pain, necrosis, and often organ damage. High doses of Heparin and LMWH can reduce or inhibit clumping of the bloods cells and thereby remove the obstruction of the vessels and the pain, but carry a risk of bleeding.
Although Heparin is a very interesting medication to treat VOC, the use of high doses Heparin is prohibited because the anticoagulant activities can result in bleeding. To overcome these problems Swedish scientist have developed Sevuparin. Sevuparin is designed to keep the anti-adhesive (anti cell clumping) properties of heparin while minimizing the anticoagulant activities to prevent bleeding and make the medication safer.
Participation in the Study
You may be able to participate in this important study if you have SCD with episodes of severe pain (known as VOC) and meet the following criteria.
You have been diagnosed with sickle cell disease
You are between 12 and 50 years old
You suffer from painful crises that requires pain relief at hospital
You have used strong opioids for less than 3 days in the last 15 days
The participation in the clinical research study will be after admission to the hospital during a painful crisis. At the hospital patients will receive the normal standard of care treatment including pain medication but will be treated in addition with the novel medication Sevuparin or placebo for 2 to 7 days. During the study patients will be under the care of a doctor.
Early evidence of efficacy and safety
There is evidence that Sevuparin may work to reduce the severity and duration of the painful crisis. Both Heparin and LMWH, when given in high doses, have been shown to reduce pain in sickle cell disease, but these high doses cannot be given to Sicklers due to the risk of bleeding. Sevuparin will have similar anti adhesive effects as heparin on the pain crisis, but being without the anti-coagulant effect, it won’t have the risk of bleeding as heparin. It is important to note that an independent safety committee reviews the data obtained in this study on regular basis to ensure patient safety. After the safety monitoring of the first 75 adult patients in this study, there have been no safety concerns identified.
Participation in clinical research is extremely important as these clinical studies are required to show that the new medication works in patients before it is approved. Your participation in the clinical study will help ensure that new medicines become available for patients suffering from disease.
Taking part in a clinical study is completely voluntary. It is free of charge and can be stopped at any time even without providing any reason and without normal medical care or legal rights being affected. All information that is collected about patients in a clinical study are kept in confidence.
For more information on this important study please call the investigators listed below or talk with your community health physician.