Development of a Ready-to-use Nutraceutical Food for Patients With Sickle Cell Disease (SCD): Testing of Vascular Support Components

Locations

1 Tanzania site

Age

8 to 11 Years

Genotypes

HbSS

Phase

Phase 2/Phase 3

Study type

Interventional

Gender

All

Interventions

Dietary Supplement

Drug

Compensation

Unknown

About the study

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.

participation requirements

– Aged 8-11 years old at enrolment and resident within urban Dar-es-Salaam

– Enrolled in Muhimbili Sickle Cohort and attending routine Muhimbili National Hospital
sickle clinics

– Homozygous for hemoglobin S (HbSS) phenotype confirmed by electrophoresis and high
performance liquid chromatography (HPLC)

participation restrictions

– >95th percentile for body mass index (BMI) for age using British 1990 growth standards

– Receiving hydroxyurea therapy or significant other long-term drug therapy

– Diagnosis with clinically significant non-SCD related disease including:

– Stage III or above HIV – or receiving ART therapy regardless of AIDS stage

– Tuberculosis infection

– Blood transfusion within previous 30 days

– Previously diagnosed clinical pulmonary hypertension or cardiac dysfunction or
clinical signs of pulmonary hypertension (loud pulmonary second heart sound) or heart
failure (displaced apex beat, high jugular venous pressure, enlarged liver, peripheral
oedema)

– Low visual acuity at baseline (<6/9 using a modified (for Tanzania) Snellen chart or previously diagnosed chronic eye disorder likely to suggest retinopathy or macular degeneration - Significant hepatic/renal dysfunction assessed by clinical chemistry panel at baseline - Epilepsy, psoriasis or currently taking any drugs listed as interacting with chloroquine

Locations

  • Dar es Salaam, Tanzania, Muhimbili University of Heath and Allied Sciences (MUHAS)