Acetylcysteine for Treatment of Sickle Cell Disease

Conditions: sickle cell disease
10018902

Registration Number: NL-OMON30544

Lead Sponsor: Academisch Medisch Centrum

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Pending

Sex: Not specified

Target Recruitment: 10

Inclusion Criteria

1. High performance liquid chromatography confirmed diagnosis of HbSS, HbSC or HbSb genotype
2. Aged 18-65 years
3. Written informed consent

Exclusion Criteria

1. Bloodtransfusion in the preceding four months
2. Pregnancy or the desire to get pregnant in the following 7 months
3. Concommitant use of hydroxyurea, oral anticontraceptives or other estrogen containing preparations, 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 gatsric or duodenal ulcer
6. Concomittant use of anti-hypertensives, sildefanil or nitrates.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Primary outcome measures are the effects of NAC on the laboratory markers<br /><br>described below.<br /><br>* hemoglobin levels, red blood cell counts, reticulocyte counts, leukocyte<br /><br>counts and differentiation, platelet counts, erythrocyte sedimentation rate<br /><br>will be determined with a automated cell counter.<br /><br>* A blood smear will be analyzed microscopically for the number of ISC per<br /><br>field, as well as the number of Heinz bodies<br /><br>* Intra-erythrocytic GSH and GSSG levels<br /><br>* NO availability (ratios of aminoacids involved in arginine metabolism)<br /><br>* SRBC PS exposure will be determined with flow cytometric quantification<br /><br>* Inflammation and endothelial activation (Serum levels of high sensitive CRP,<br /><br>sVCAM-1, ET-1, and IL-8)<br /><br>* Coagulation activation (pro-thrombin fragments (F1.2), D-dimer levels,<br /><br>protein S (free and total) and C activity, vWF-Ag activity)</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Secondary outcome measures are tolerability of study medication at every visit<br /><br>by history taking and by scoring of a NAC for SCD check-list.</p><br>