AcetylCysteine in the treatment of Sickle Cell Disease

Completed

Conditions: Sickle cell disease
Haematological Disorders
Sickle-cell disorders

Registration Number: ISRCTN28828586

Lead Sponsor: CURAMA Study Group (The Netherlands)

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 10

Inclusion Criteria

1. High performance liquid chromatography confirmed diagnosis of sickle cell anaemia (HbSS), sickle-haemoglobin C disease (HbSC) or sickle cell trait disease (HbSa) genotype
2. Aged 18 to 65 years
3. Written informed consent

Exclusion Criteria

1. Blood transfusion in the preceding four months
2. Pregnancy or the desire to get pregnant in the following seven months
3. Concomitant use of hydroxyurea, vitamin K antagonists or other oral anticoagulants, or contraindications for NAC
4. Impaired renal function of more than 60% (as assessed by the Kockroft-Gauld equation)
5. Known gastric or duodenal ulcer
6. Concomitant use of anti-hypertensives, sildefanil or nitrates

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Primary end-points are the effects of NAC on the laboratory markers (haemoglobin, red blood cell counts, reticulocyte counts, leukocyte counts and differentiation, platelet counts, erythrocyte sedimentation rate, a blood smear will be analysed microscopically for the number of ISC per field, as well as the number of Heinz bodies, intra-erythrocytic reduced glutathione [GSH] and oxidised glutathione [GSSG] levels, NO availability, SRBC phosphatidylserine [PS] exposure, annexin V, creatinine, blood-urea nitrogen [BUN], electrolytes, transaminase levels, albumin levels, lactate dehydrogenase [LDH], indirect bilirubin levels, free haemoglobin levels, high sensitive C-reactive protein [hsCRP], vascular cell adhesion molecule-1 [sVCAM-1], endothelin [ET-1], interleukin-8 [IL-8], pro-thrombin fragments [F1.2], D-dimer levels, protein S [free and total] and C activity, Von Willebrand factor antigen [vWF-Ag] activity).

Secondary Outcome Measures

Name	Time	Method
Tolerability of study medication (in this phase admittedly in a non-controlled fashion) at every visit by history taking and by scoring of a NAC for SCD check-list.