Clinical Trials

Study SCD-105 – Phase 1b Sickle cell study (Europe and Jamaica)

Details
This multi-center, randomized, double blind, dose escalation safety study of MP4CO in clinically stable adult sickle cell patients will be conducted at major sickle cell centers in France, Jamaica and the UK. The primary objective is to evaluate the safety of MP4CO infusion in clinically stable adult sickle cell patients who are not currently experiencing a painful vaso-occlusive crisis (VOC).

The initial release of CO from MP4CO is predicted to have a therapeutic effect including immediate stabilization of Hb S to prevent further polymerization and reverse existing sickling, vasodilation of capillaries, and anti-inflammatory properties. The subsequent circulation of the MP4 molecule as an oxygen therapeutic agent (after converting to MP4OX following oxygenation in the lungs) will help to 1) preferentially oxygenate ischemic cells, 2) reverse partially sickled red cells, and 3) improve oxygenation of local tissues, thereby potentially ameliorating the painful VOC caused by red blood cell sickling.

A minimum of 32 male and female patients, aged ≥ 18 years old will be enrolled in this study. Patients eligible for inclusion must be diagnosed with sickle cell disease based on Hb SS, or S/β0 Thalassemia genotype, and must be clinically stable and not experiencing an acute episode of pain. Patients meeting inclusion criteria will be assigned to receive one of 8 doses of investigational product, either MP4CO (treated group) or normal saline (control group) administered as intravenous infusions, Safety variables will be evaluated at different time points from randomization through Day 28 follow-up visit. Specific procedures including physical examination, vital signs, and laboratory assessments will be conducted at defined time-points throughout the study. [Protocol details can be found at http://clinicaltrials.gov/ct2/show/NCT01356485]