|Hussain Mulla, PhD|
|+44 (0) 116 2230100|
5 United Kingdom sites
1 Jamaica site
6 to 17 Years
1. Male or female aged from 6 months to 17.99 years of age (i.e. to the day before 18th
2. Diagnosis of sickle cell anemia (HbSS and HbSβº).
3. Parent(s)/legal guardian able and willing to provide written informed consent for the
child to take part in the study.
4. Where applicable, the child should assent to undergo blood sampling for
pharmacokinetic and biochemistry purposes and to allow physiological measurements to
1. Any clinically significant medical condition or abnormality, which, in the opinion of
the investigator, might compromise the safety of the patient or which might interfere
with the study.
2. Hydroxyurea use within 6 months before enrolment.
3. Renal insufficiency (known creatinine more than twice the upper limit for age and
>1.0mg/dL [88.4 micromol/L])).
4. Clinical evidence of hepatic compromise with ALT more than 3 times the upper limit of
5. Other significant organ system dysfunction based on the site investigator’s
6. Severe active infections: fungal, viral or bacterial (as confirmed by culture),
examples include tuberculosis, malaria, active hepatitis, osteomyelitis or any other
illness that would preclude the use of hydroxyurea in normal clinical practice.
7. Active chronic leg ulcers.
8. Known allergy to oral hydroxyurea solution or any of the excipients.
9. Positive pregnancy test for females of child bearing potential (in post-menarcheal
females) before initiation of treatment, unless participant is sexually abstinent.
Note: True abstinence is considered as being in line with the preferred and usual
lifestyle of the subject. Periodic abstinence (such as calendar, ovulation,
symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of
10. Inadequate contraception measures in sexually active females (in post-menarcheal
females) and males of child bearing age.
11. Currently breastfeeding.
12. Participating in another clinical trial of an IMP.
13. Known infection with Human Immunodeficiency Virus (HIV)