Vascular Function Intervention Trial in Sickle Cell Disease

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.

Conditions

• Sickle Cell Disease

Interventions

DIETARY_SUPPLEMENT

Vascular ready-to-use supplementary food

Daily vascular ready-to-use supplementary food containing protein, energy and fortified with 1 x recommended daily allowance(RDA) of vitamins and minerals except for folic acid = 1mg and with no iron fortificant included and fortified with arginine and citrulline amino acids

DIETARY_SUPPLEMENT

Regular Ready-to-use supplementary food

Daily ready-to-use supplementary food containing protein, energy and fortified with 1 x RDA vitamins and minerals except for folic acid = 1mg and with no iron fortificant included.

DRUG

Chloroquine

Sponsors & Collaborators

• Wellcome Trust

  collaborator OTHER

• London School of Hygiene and Tropical Medicine

  lead OTHER

Principal Investigators

• Sharon Cox, PhD · London School of Hygiene & Tropical Medicine, UK / Muhimbili Wellcome Programme, Tanzania

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Model

CROSSOVER

Eligibility

Min Age

8 Years

Max Age

11 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2012-08-31

Primary Completion

2016-09-30

Completion

2017-09-30

Countries

• Tanzania

Study Locations