Pharmacy and Poisons Board: Applications

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Protocol No:

ECCT/14/04/01

Date of Protocol:

11-03-2014

Study Title:	REALIZING EFFECTIVENESS ACROSS CONTINENTS WITH HYDROXYUREA (REACH): A PHASE I/II PILOT STUDY OF HYDROXYUREA FOR CHILDREN WITH SICKLE CELL ANAEMIA.
	The inherited disease sickle cell anaemia is common in Kenya where more than 4000 children are born with the condition each year. On the basis of positive data from clinical trials, an increasing number of patients with sickle cell anaemia in the North are now being treated with hydroxyurea. No trials of hydroxyurea have been conducted in Africa where the safety and effectiveness of the drug are therefore unknown. We aim to conduct a multi-center phase I/II clinical trial to investigate the safety of hydroxyurea as a supportive treatment in children with sickle cell disease in Kenya, Angola, Uganda and the DRC. In total, 600 children aged 12 months - 10 years (150 in each site) will be enrolled into the study. All will be placed on hydroxyurea and monitored closely to investigate both safety and efficacy of the drug ~~over a 48-month period~~ until they reach the age of 18-years. If successful, we hope that this study will lead to a larger trial that will shape national management policies for all children born with sickle cell anaemia in Africa.
Study Objectives:	a) General objectives To conduct an early phase clinical trial to establish whether or not hydroxyurea could potentially provide both a safe and effective option for the management of children living with SCA in the African context. If no major safety concerns are revealed in this early phase trial it is anticipated that this trial will be followed with a larger, definitive, late-phase trial investigating safety and efficacy in a broad range of hospital settings in Africa. b) Specific objectives i) To monitor the safety of hydroxyurea therapy, specifically documenting significant haematological toxicities (cytopaenias) and infections. ii) To evaluate the benefits of hydroxyurea therapy, using both laboratory (i.e. fetal haemoglobin and haemoglobin concentrations and white blood cell counts) and clinical parameters (such as pain, hospitalization and growth parameters). iii) To investigate the feasibility of hydroxyurea dose-escalation on laboratory and clinical parameters. iv) To explore the pharmacokinetic and genetic basis for any observed inter-patient variability in the clinical or laboratory response to hydroxyurea vi) To evaluate the economic cost of providing hydroxyurea therapy in the REACH study sites.
Laymans Summary:	The inherited disease sickle cell anaemia is common in Kenya where more than 4000 children are born with the condition each year. On the basis of positive data from clinical trials, an increasing number of patients with sickle cell anaemia in the North are now being treated with hydroxyurea. No trials of hydroxyurea have been conducted in Africa where the safety and effectiveness of the drug are therefore unknown. We aim to conduct a multi-center phase I/II clinical trial to investigate the safety of hydroxyurea as a supportive treatment in children with sickle cell disease in Kenya, Angola and the DRC. In total, 450 children aged 12 months - 10 years (150 in each site) will be enrolled into the study. All will be placed on hydroxyurea and monitored closely to investigate both safety and efficacy of the drug over a 48 month period. If successful, we hope that this study will lead to a larger trial that will shape national management policies for all children born with sickle cell anaemia in Africa.
Abstract of Study:	The inherited disease sickle cell anaemia is common in Kenya where more than 4000 children are born with the condition each year. On the basis of positive data from clinical trials, an increasing number of patients with sickle cell anaemia in the North are now being treated with hydroxyurea. No trials of hydroxyurea have been conducted in Africa where the safety and effectiveness of the drug are therefore unknown. We aim to conduct a multi-center phase I/II clinical trial to investigate the safety of hydroxyurea as a supportive treatment in children with sickle cell disease in Kenya, Angola and the DRC. In total, 450 children aged 12 months - 10 years (150 in each site) will be enrolled into the study. All will be placed on hydroxyurea and monitored closely to investigate both safety and efficacy of the drug over a 48 month period. If successful, we hope that this study will lead to a larger trial that will shape national management policies for all children born with sickle cell anaemia in Africa.