A Prospective Open Label, Pharmacokinetic Study of an Oral Hydroxyurea Solution in Children With Sickle Cell Anemia.

Study point of contact

Hussain Mulla, PhD
+44 (0) 116 2230100
[email protected]

Locations

5 United Kingdom sites

1 Jamaica site

Age

6 to 17 Years

Genotypes

HbSS

Phase

Phase 2

Study type

Interventional

Gender

All

Interventions

Drug

Compensation

Unknown

About the study

An open label, safety and pharmacokinetic study of oral hydroxyurea solution administered to
children from 6 months to 17.99 years (i.e. to the day before 18th birthday), with a 12 15
month treatment period for each participant. The study treatment duration will be for 6
months at the maximum tolerated dose [MTD], which is usually reached by 6 months after
initiation of treatment. For patients in whom time to MTD is longer than 6 months or not
achieved at all, the maximum duration of study treatment will be 15 months.

participation requirements

1. Male or female aged from 6 months to 17.99 years of age (i.e. to the day before 18th
birthday).

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
be made.

participation restrictions

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
normal.

5. Other significant organ system dysfunction based on the site investigator’s
discretion.

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
contraception.

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)

Locations

  • Kingston, Jamaica, Dr Angela E Rankine- Mullings [Recruiting]
  • Birmingham, United Kingdom, Birmingham Women's and Children's NHS Foundation Trust [Recruiting]
  • Liverpool, United Kingdom, Alder Hey Children's NHS Foundation Trust [Recruiting]
  • London, United Kingdom, Evelina London Children's Hospital [Recruiting]
  • London, United Kingdom, King's College Hospital NHS Foundation Trust [Recruiting]
  • London, United Kingdom, The Royal London Children's Hospital, Barts Health NHS Trust [Recruiting]