Acetylcysteine in patients with Sickle Cell Disease: Reducing the incidence of daily life pain in patients with sickle cell disease

Phase 1

Conditions: Sickle Cell
MedDRA version: 15.1Level: LLTClassification code 10040643Term: Sickle cell crisisSystem Organ Class: 100000004850
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Registration Number: EUCTR2012-004892-37-NL

Lead Sponsor: Academic Medical Center

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 140

Inclusion Criteria

-Age 12 years or older
-Sickle cell disease, either homozygous sickle cell disease (HbSS), HbSC sickle cell
disease, HbSß0 or HbSß+ thalassemia.
Genotype needs to be confirmed by high performance liquid chromatography.
-History of at least 1.0 painful crisis per year in the past 3 years.
A crisis will be defined here as a patient defined, painful, sickle cell related episode of at least 24 hours where a subject experienced significant impediments in his/her daily activities, and pain medication had to be taken. A visit to a medical facility is not obligatory in this definition.
-Written informed consent from patient/parent/guardian is given.

Are the trial subjects under 18? yes
Number of subjects for this age range: 30
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 90
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20

Exclusion Criteria

-Chronic blood transfusion or transfusion in the preceding 3 months
-Painful crisis in the last 4 weeks (with respect to the moment of inclusion).
A crisis will be defined as above; a patient defined, painful, sickle cell related episode of at least 24 hours where the subject experienced significant impediments in his/her daily activities, and pain medication had to be taken. A visit to a medical facility is not obligatory in this definition.
-Pregnancy, breast feeding or the desire to get pregnant in the following 7 months
-Known active gastric/duodenal ulcers
-Hydroxycarbamide (HC) treatment with unstable dose in the last 3 months or started on
HC shorter then 6 months prior to study.
-Known poor compliance in earlier trials regarding the completion of pain diaries.
-Insufficient compliance in run-in period.
Participants that do not show up for their follow-up visit without prior notice, that not bring
their diary or with less than 80% of the days in the pain diary filled in, will be excluded from participation in this trial and will not be randomized.