Contact information
Type
Scientific
Primary contact
Dr B.J. Biemond
ORCID ID
Contact details
Academic Medical Centre (AMC)
Department of Clinical Chemistry
P.O. Box 22660
Amsterdam
1100 DD
Netherlands
+31 (0)20 566 7391
b.j.biemond@amc.uva.nl
Additional identifiers
EudraCT number
ClinicalTrials.gov number
Protocol/serial number
NTR1013
Study information
Scientific title
Acronym
NAC in SCD
Study hypothesis
We hypothesise that treatment of sickle cell patients with N-acetylcysteine (NAC) results in reduced red cell phosphatidylserine (PS) exposure, reduced endothelial activation, increased nitric oxide (NO) availability, reduced coagulation activation and reduced inflammation detectable with specific laboratory testing, as well as a reduction of irreversibly sickled cells (ISC's) and Heinz Body formation.
Ethics approval
Ethics approval received from the local medical ethics committee
Study design
Randomised, active controlled, parallel group trial
Primary study design
Interventional
Secondary study design
Randomised controlled trial
Trial setting
Not specified
Trial type
Treatment
Patient information sheet
Condition
Sickle cell disease
Intervention
N-acetylcysteine 1200 mg or 2400 mg a day.
Intervention type
Drug
Phase
Not Specified
Drug names
N-acetylcysteine
Primary outcome measures
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
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.
Overall trial start date
01/10/2007
Overall trial end date
31/12/2008
Reason abandoned
Eligibility
Participant 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
Participant type
Patient
Age group
Adult
Gender
Not Specified
Target number of participants
10
Participant 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
Recruitment start date
01/10/2007
Recruitment end date
31/12/2008
Locations
Countries of recruitment
Netherlands
Trial participating centre
Academic Medical Centre (AMC)
Amsterdam
1100 DD
Netherlands
Funders
Funder type
Research organisation
Funder name
CURAMA Study Group (The Netherlands)
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
Results and Publications
Publication and dissemination plan
Not provided at time of registration
Intention to publish date
Participant level data
Not provided at time of registration
Results - basic reporting
Publication summary