N-Acetylcysteine for Treatment of Sickle Cell Disease - N-acetylcystein for Sickle Cell Disease

Status

Not yet recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2006-005889-40-NL

Enrollment

Registered

2006-11-02

Start date

2007-08-14

Completion date

Unknown

Last updated

2012-03-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Patients homozygous for sickle cell disease MedDRA version: 8.1 Level: LLT Classification code 10040644 Term: Sickle cell disease

Interventions

Trade Name: fluimicil Pharmaceutical Form: Tablet INN or Proposed INN: ACETYLCYSTEINE CAS Number: 616911 Current Sponsor code: ACETYLCYSTEINE Other descriptive name: ACETYLCYSTEINE Concentration unit:

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Ten patients with SCD (HbSS HbSC or HbSb) will be included. Inclusion criteria 1. High performance liquid chromatography confirmed diagnosis of HbSS HbSC or HbSb genotype 2. Aged 18-65 years 3. Written informed consent Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: 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, 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.

Design outcomes

Primary

Measure	Time frame
Main Objective: To determine whether NAC therapy results in decreased red cell phosphatidylserine exposure, endothelial activation, inflammation, and reduction clotting activation in the steady state. We hypothesize that treatment of sickle cell patients with NAC results in reduced red cell phosphatidylserine exposure, reduced endothelial activation, increased NO availability, reduced coagulation activation and reduced inflammation detectable with specific laboratory testing, as well as a reduction of irreversibly sickled cells and Heinz Body formation ;Secondary Objective: Secondary outcome measures are tolerability of study medication at every visit by history taking and by scoring of a NAC for SCD check-list (see appendix B). ;Primary end point(s): Primary outcome measures are the effects of NAC on the laboratory markers described below. • hemoglobin levels, red blood cell counts, reticulocyte counts, leukocyte counts and differentiation, platelet counts, erythrocyte sedimentation rate will be determined with a automated cell counter. • A blood smear will be analyzed microscopically for the number of ISC per field, as well as the number of Heinz bodies • Intra-erythrocytic GSH and GSSG levels • NO availability (ratios of aminoacids involved in arginine metabolism) • SRBC PS exposure will be determined with flow cytometric quantification • Inflammation and endothelial activation (Serum levels of high sensitive CRP, sVCAM-1, ET-1, and IL-8) • Coagulation activation (pro-thrombin fragments (F1.2), D-dimer levels, protein S (free and total) and C activity, vWF-Ag activity)	—

Measure

Time frame

Main Objective: To determine whether NAC therapy results in decreased red cell phosphatidylserine exposure, endothelial activation, inflammation, and reduction clotting activation in the steady state. We hypothesize that treatment of sickle cell patients with NAC results in reduced red cell phosphatidylserine exposure, reduced endothelial activation, increased NO availability, reduced coagulation activation and reduced inflammation detectable with specific laboratory testing, as well as a reduction of irreversibly sickled cells and Heinz Body formation ;Secondary Objective: Secondary outcome measures are tolerability of study medication at every visit by history taking and by scoring of a NAC for SCD check-list (see appendix B). ;Primary end point(s): Primary outcome measures are the effects of NAC on the laboratory markers described below. • hemoglobin levels, red blood cell counts, reticulocyte counts, leukocyte counts and differentiation, platelet counts, erythrocyte sedimentation rate will be determined with a automated cell counter. • A blood smear will be analyzed microscopically for the number of ISC per field, as well as the number of Heinz bodies • Intra-erythrocytic GSH and GSSG levels • NO availability (ratios of aminoacids involved in arginine metabolism) • SRBC PS exposure will be determined with flow cytometric quantification • Inflammation and endothelial activation (Serum levels of high sensitive CRP, sVCAM-1, ET-1, and IL-8) • Coagulation activation (pro-thrombin fragments (F1.2), D-dimer levels, protein S (free and total) and C activity, vWF-Ag activity)

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Countries

Netherlands

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026