Sponsor study identifier
Secondary identifiers
OTHER_GRANT · The Wellcome Trust, UK
interventional
120
1 country
1
Study Start
August 2012
Date type not reportedThis date has not been verified as actual or estimated.
Primary Completion
September 2016
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Study Completion
September 2017
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
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.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
September 2017
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
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Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
February 1, 2012
Actual (reported)August 2012
Date type not reportedThis date has not been verified as actual or estimated.
October 31, 2012
Actual (reported)September 2016
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
September 2017
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
ratio of arginine to ornithine concentration & ratio of arginine to ADMA
We will compare the effects of the RUSFv compared to the simple RUSF on: The ratio of plasma arginine to ornithine \& ratio of arginine to ADMA and adjust for values at baseline Time frame definition: 0 months = baseline 4 months = after 1st four-month intervention period, before washout 1 8 months = after 1st 4 month washout period before 2nd intervention period 12 months = after 2nd four-month intervention period, before washout 2 16 months = after 2nd 4 month washout period (study end)
4 or 12 months
Nitric Oxide dependent endothelial function
Comparison of the effects of the RUSFv to the simple RUSF on vascular function, assessed using flow mediated dilatation (FMDmax), adjusted for baseline values at time 0. Values will also be determined at time point at month 8, in order to check for possible carry-over effect.
months 4 or 12
Linear Growth and Weight Gain
We will compare the effects of RUSF compared to no RUSF on rates of growth by comparison of the 2 intervention periods combined with the two washout periods combined, by conducting measurements at months 0, 4, 8, 12 \& 16.
After 8 months of treatment with RUSFv and RUSF
Haemoglobin concentration
months 0, 4, 8, 12 & 16
Markers of inflammation and vascular activation
months 0, 4 & 12
Markers of haemolysis
months 0, 4 & 12
Frequency of vaso-occlusive painful episodes
Weekly from month 0-16
liver and kidney function clinical chemistry
months 0, 4 & 12
In this intervention period children will receive a vascular ready-to-use supplementary food with added L-Arginine \& L-Citrulline plus daily chloroquine
In this intervention period children will receive a regular ready-to-use supplementary food plus weekly dose of chloroquine
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
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.
Muhimbili University of Heath and Allied Sciences (MUHAS)
Dar es Salaam, Tanzania
Cox SE, Ellins EA, Marealle AI, Newton CR, Soka D, Sasi P, Luca Di Tanna G, Johnson W, Makani J, Prentice AM, Halcox JP, Kirkham FJ. Ready-to-use food supplement, with or without arginine and citrulline, with daily chloroquine in Tanzanian children with sickle-cell disease: a double-blind, random order crossover trial. Lancet Haematol. 2018 Apr;5(4):e147-e160. doi: 10.1016/S2352-3026(18)30020-6. Epub 2018 Mar 13.
Sharon Cox, PhD
London School of Hygiene & Tropical Medicine, UK / Muhimbili Wellcome Programme, Tanzania
First Submitted
February 1, 2012
First Posted
October 31, 2012
Study Start
August 2012
Date type not reportedThis date has not been verified as actual or estimated.
Primary Completion
September 2016
A Phase 2/3 Study of Osivelotor in Adult and Adolescent Participants With SCD
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This estimate has passed. The registry has not confirmed an actual date for this milestone.
Study Completion
September 2017
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Last Updated
July 27, 2016
Record last verified: 2016-07
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Source: ClinicalTrials.gov.
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