IMR-SCD-OBS - PK and PD of Hydroxyurea in Sickle Cell Disease
Research type
Research Study
Full title
An Observational Study to Characterize the Pharmacokinetics and Pharmacodynamics of Hydroxyurea (Hydroxycarbamide) in Sickle Cell Subjects Currently Receiving a Stable Dose of Hydroxyurea
IRAS ID
222531
Contact name
Jo Howard
Contact email
Sponsor organisation
Imara
Eudract number
2017-000823-28
Duration of Study in the UK
0 years, 4 months, 24 days
Research summary
The purpose of this exploratory, observational study is to examine the pharmacokinetics (PK – how the body processes the drug) and pharmacodynamics (PD – how the drug affects the body) of hydroxyurea (HU) in adult patients with Sickle Cell Disease (SCD) who are currently receiving a stable dose of HU, and to determine how this affects the foetal haemoglobin (HbF) in these patients.
Haemoglobin is the protein that carries oxygen in red blood cells. In patients with sickle cell anaemia the haemoglobin is abnormal causing the red blood cells to “sickle” and the concentration of haemoglobin in blood is lower than normal. Foetal haemoglobin is the haemoglobin expressed in foetuses and at lower levels in children and adult patients with SCD. Induction of foetal haemoglobin production is a promising strategy for the treatment of sickle-cell disease.
The drug being investigated in this study is hydroxyurea (HU) which causes changes in the blood, which reduce the frequency of sickle cell crises and the need for transfusion in some patients with sickle cell disease.
The study comprises of a ≤28 day screening period and a 6 week observational period. Participants will continue receiving their prescribed dose of HU according to standard of care. Participants will attend the hospital every other week during the 6 week observational period, for blood sample collection. At screening, a blood sample will be taken sent for genetic analyses. At the subsequent visits the blood samples will be taken to measure the concentration of HU in the blood, the haemoglobin level and type, as well as a naturally occurring substance (a “peptide”) that may be affected by treatment of SCD.
This study will be conducted at a single study site in the UK in approximately 12 eligible participants.
REC name
London - Riverside Research Ethics Committee
REC reference
17/LO/0474
Date of REC Opinion
24 May 2017
REC opinion
Further Information Favourable Opinion