Vascular Function Intervention Trial in sickle cell disease

Not Applicable

Completed

• Conditions: Sickle Cell Disease; Haematological Disorders; Other sickle-cell disorders

• Registration Number: ISRCTN74331412
• Lead Sponsor: ondon School of Hygiene and Tropical Medicine (UK)

Brief Summary

2018 results in https://pubmed.ncbi.nlm.nih.gov/29548623/ (added 17/12/2020)

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2012-02-02
• Study Completion: 2014-04-09
• Last Update Posted: 5 January 2021

Recruitment & Eligibility

• Status: Completed
• Sex: All
• Target Recruitment: 119

Inclusion Criteria

1. Aged 8-11 years old at enrolment and resident within urban Dar-es-Salaam
2. Enrolled in MSC and attending routine MNH sickle clinics
3. Haemoglobin phenotype SS (HbSS) confirmed by electrophoresis and High-performance liquid chromatography (HPLC)

Exclusion Criteria

1. >95th percentile for body mass index (BMI) for age using British 1990 growth standards
2. Receiving hydroxyurea (HU) therapy or significant other long-term drug therapy
3. Diagnosis with clinically significant non-SCD related disease including:
3.1. Stage III or above human immunodeficiency virus (HIV) ? or receiving antiretroviral therapy (ART) therapy regardless of Acquired immune deficiency syndrome (AIDS) stage
3.2. Tuberculosis infection
3.3. Blood transfusion within previous 30 days
4. 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)
5. 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
6. Significant hepatic/renal dysfunction assessed by clinical chemistry panel at baseline
7. Epilepsy, psoriasis or currently taking any drugs listed as interacting with chloroquine

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<br> 1. Compare the effects of the RUSFv compared to the simple RUSF on:<br> 1.1. Plasma arginine concentrations, the ratio of plasma arginine to ornithine & ratio of arginine to ADMA measured at baseline, 4 and 12 months<br> 1.2. NO-dependent endothelial function (vascular function) assessed using flow mediated dilatation (FMDmax) measured at baseline, 4, 8 and 12 months<br> 2. Compare the effects of RUSF compared to no RUSF by comparison of the two intervention periods combined with the two washout periods combined, on: linear growth and weight gain measured at baseline, 4, 8, 12 and 16 months<br>

Secondary Outcome Measures

Name	Time	Method
<br> 1. Haemoglobin concentration measured at baseline, 4, 8, 12 and 16 months<br> 2. Markers of inflammation and vascular activation measured at baseline, 4, 8 and 12 months<br> 3. Markers of haemolysis measured at baseline, 4, 8 and 12 months<br> 4. Frequency of vasoocclusive crisis (VOC) painful episodes: study personnel will administer detailed questionnaires at weekly home visits to assess the frequency of all sickle and non-sickle associated morbidity and health seeking behaviour, with a focus on painful episodes. Participatory research will be used to determine the likely application and optimal formatting of pain diaries to be completed by patients and families in addition to the standard questionnaire.<br>