MP4CO for the Acute Treatment of Vaso-occlusive Crises V1
Research type
Research Study
Full title
A Phase 2 Multi-center, Randomized, Double-blind, Comparator-Controlled Dose Finding Study to Evaluate MP4CO for the Acute Treatment of Vaso-occlusive Crises in Subjects with Sickle Cell Disease
IRAS ID
135556
Contact name
Swee Lay Thein
Contact email
Sponsor organisation
Sangart, Inc.
Eudract number
2013-001600-11
ISRCTN Number
n/a
Research summary
This study will enroll about 200 patients with sickle cell disease who have prior history of at least one vaso-occlusive crisis (VOC) requiring hospitalisation within the last 24 months. The study will take place globally at multiple study centres. Patients enrolled in the study will either receive the investigational drug, MP4CO, or a control of standard fluid treatment (normal saline - salt water) solution.
This is a dose escalation study. After the treatment of the first group of patients, the data will be reviewed and if it is deemed safe to do so, the next group of patients to be treated will receive an increased dose. A total of 5 treatment groups receiving increasing doses of investigational product may be included in the study.
On presentation of a VOC and admittance to hospital, eligible patients enrolled in the study will receive either the investigational product, MP4CO, or the control through 2 separate infusions each given over a 2 hour period, with the first given at hour 0 and the second given at hour 24. After the patient has received the initial treatment, the patient will followed for 12 weeks to allow further information on the effects and safety of the study drug to be collected. The total duration of the study is expected to be 18 months. If the patient after 60 days of their first VOC, experiences a second VOC, they will be eligible to receive a second study treatment of either MP4CO or normal saline. Due to the crossover design, the patient will not receive the same product used to treat the first VOC.
REC name
London - Riverside Research Ethics Committee
REC reference
13/LO/1257
Date of REC Opinion
25 Sep 2013
REC opinion
Further Information Favourable Opinion