MP4CO in adult sickle cell patients

• Research type

  Research Study

• Full title

  A multi-centre, randomised double blind, dose escalation safety study of MP4CO in clinically stable adult sickle cell patients

• IRAS ID

  81809

• Contact name

  Joanna Howard

• Sponsor organisation

  Sangart, Inc.

• Eudract number

  2011-000463-27

• Clinicaltrials.gov Identifier

  01356485

• Research summary

  Sickle cell disease (SCD), also known as sickle cell anaemia is a genetic inherited) disorder which affects the red blood cells in the blood. In SCD the red blood cells are not their normal disc-like shape, instead they become sickle-shaped (like a crescent moon). This can cause issues, such as infections and various other complications. These symptoms come and go and in between the symptoms, patients generally feel well. A severe and sudden symptom due to the ??sickling? of the blood cells is called a sickle cell crisis. Apart from hydroxycarbamide which has been shown to decrease the frequency of painful crisis and reduce hospital admissions there are no other therapies that are licenced. Sangart Inc., the sponsor of this study, is currently developing a potential new treatment for sickle cell crises in patients with SCD, called MP4CO. This study aims to test the safety of different doses of MP4CO. After consenting and being assessed as suitable for the study, patients will be randomly assigned (like tossing a coin) to one of two groups. One group will receive MP4CO, and the other group will receive normal saline (water containing a concentration of salts) solution (control). A Patient will have a 75% chance of being randomised to MP4CO. This study is a double blind study, which means neither the patient, nor their study doctor will know whether the patient receives MP4CO, or the normal saline. Patients will be in the study for approximately 28 days and will undergo a variety of tests and procedures to monitor them. It is expected that no fewer than 32 patients will take part in this research study across different countries.

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  11/LO/1184

• Date of REC Opinion

  8 Nov 2011

• REC opinion

  Further Information Favourable Opinion