H-PRIME: Hydroxyurea – Pragmatic Reduction in Mortality and Economic burden: A multi-centre Phase III trial to investigate the efficacy of hydroxyurea in children with sickle cell anaemia when administered in a pragmatic fashion.

Imperial College London0 个研究点目标入组 1,800 人开始时间: 2020年2月25日最近更新: 2024年6月3日

适应症

试验速览

2020 Other publications in https://pubmed.ncbi.nlm.nih.gov/32802962/ baseline characteristics (added 21/08/2020)

•1. Aged 1-10 years inclusive
•2. Confirmed Sickle Cell Disease (SCD) (diagnosed either by HPLC or IEF at a qualified laboratory)
•3. Have received conjugate pneumococcal vaccination against Hib and S. pneumoniae (otherwise eligible but unvaccinated children will be vaccinated through the study)
•4. Carer willing/able to provide consent and to bring the child for follow-up visits, as demonstrated by either regular attendance at SCD clinics to date, or attending two visits (one of which may be the screening visit) before randomisation

•1. Already meet criteria for starting hydroxyurea under Uganda National Guidelines 2026 (frequent crises (>5/year), abnormal transcranial Doppler ultrasound velocities, stroke or acute chest syndrome)
•2. Already receiving hydroxyurea
•3. Taking concomitant medications that are contraindicated with any of the trial medications (hydroxyurea, SP, DHA-PQP, penicillin V, cotrimoxazole) (including, but not limited to, nefazodone, verapamil, rifampicin, isoniazid, ethambutol)
•4. Known cancer
•5. A clinical history of previous or existing liver or renal diseases unrelated to sickle cell disease
•6. Known cardiac ventricular dysfunction or failure or a previous history of cardiac arrhythmias
•7. Known HIV (these children should receive cotrimoxazole prophylaxis and many will be receiving antiretrovirals that are contraindicated with one or more trial medications (zidovudine, amprenavir, atazanavir, indinavir, nelfinavir, ritonavir))
•8. Current participation in any other clinical trial of an investigational medicinal product
•9. Presence of acute infection on the day of screening (e.g. symptomatic P. falciparum malaria, pneumonia, septicaemia, meningitis, newly identified tuberculosis) – such children may be enrolled after recovery from an acute infection if they do not meet other exclusion criteria

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