Vascular Function Intervention Trial in Sickle Cell Disease
Status:
Unknown status
Trial end date:
2017-09-01
Target enrollment:
Participant gender:
Summary
Sickle cell disease (SCD) is the most common inherited disorder worldwide affecting 300,000
births annually, most occuring in sub-Saharan Africa (SSA) where poor detection and care
result in high childhood mortality, malnutrition, illness and disability in survivors. SCD is
caused by abnormal haemoglobin, the compound in red blood cells(RBC) that carries oxygen.
Much of the disability in SCD may be caused by vascular damage from the breakdown of damaged
RBC. Research in high-income countries has led to some effective therapies but these are
currently costly and complex. The investigators will test two different formulations of an
affordable, ready-to-use supplementary food (RUSF) specifically tailored for children with
SCD. As well as containing energy, protein, essential fats, vitamins and minerals, the
vascular RUSF (RUSFv) will be fortified with the amino-acids arginine and citrulline and be
delivered with a daily chloroquine dose to create a novel "nutraceutical" intervention.
Arginine is converted to nitric oxide which is essential for vascular health. Arginine levels
are low in SCD because the arginine-degrading enzyme, arginase, is released from RBCs. The
investigators propose that by supplying additional arginine (and citrulline which converts to
arginine) and suppressing arginase activity (an action of chloroquine) the investigators can
improve vascular function. Our study will test this theory, and if provision of RUSF improves
growth in children with SCD.