COBALT: Coversin in patients with PNH
Research type
Research Study
Full title
COBALT: Coversin Global Study: An Open-Label, Safety and Efficacy Trial in PNH Patients
IRAS ID
208269
Contact name
Anita Hill
Contact email
Sponsor organisation
Akari Therapeutics Plc
Eudract number
2016-002067-33
Duration of Study in the UK
0 years, 7 months, 0 days
Research summary
Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, life-threatening disease of the blood, characterised by the destruction of red blood cells by the immune system. Breakdown of the red blood cells causes dark urine and anaemic symptoms such as fatigue. The biggest risk of PNH is blood clots. PNH is a genetic disease and occurs due to a mutation in a gene that results in loss of protective proteins on the cells allowing them to be destroyed by part of the immune system. The incidence of PNH is 1-2 in one million.
Most therapies for PNH aim to ease symptoms. Iron supplements and folic acid are usually given to patients to offset the high turnover of red blood cells. Blood transfusions may be required, or warfarin to reduce the risk of blood clots however both have risks and are not always effective. Bone marrow transplantation is the only cure for PNH, however this is not an available option for many patients. The only current therapy for PNH is Eculizumab which inhibits the 'complement system', a part of the body's immune system, however this drug must be given by a healthcare professional every 2 weeks. There is an unmet need for the use of a more convenient treatment with fewer risks.
Akari Therapeutics PLC have developed a new drug, Coversin, that has been designed to inhibit the complement system.
This study is being done to assess whether Coversin is safe and effective when given to patients with PNH.
This is a multicentre study which will take place across Europe. It is anticipated that up to 10 patients ≥18 years old with newly diagnosed PNH will be enrolled worldwide.
Patients will receive 4 doses of 30 mg Coversin every 12 hours over 2 days by injection under the skin, followed by daily maintenance doses of Coversin for 88 days.
REC name
London - Riverside Research Ethics Committee
REC reference
16/LO/1161
Date of REC Opinion
18 Jul 2016
REC opinion
Favourable Opinion