N-AcetylCysteine in the treatment of Sickle Cell Disease
| ISRCTN | ISRCTN28828586 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN28828586 |
| Secondary identifying numbers | NTR1013 |
- Submission date
- 23/08/2007
- Registration date
- 23/08/2007
- Last edited
- 08/09/2008
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Haematological Disorders
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Record updated in last year
Plain English summary of protocol
Not provided at time of registration
Contact information
Dr B.J. Biemond
Scientific
Scientific
Academic Medical Centre (AMC)
Department of Clinical Chemistry
P.O. Box 22660
Amsterdam
1100 DD
Netherlands
| Phone | +31 (0)20 566 7391 |
|---|---|
| b.j.biemond@amc.uva.nl |
Study information
| Study design | Randomised, active controlled, parallel group trial |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Randomised controlled trial |
| Study setting(s) | Not specified |
| Study type | Treatment |
| Scientific title | |
| Study acronym | NAC in SCD |
| Study objectives | 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(s) | Ethics approval received from the local medical ethics committee |
| Health condition(s) or problem(s) studied | Sickle cell disease |
| Intervention | N-acetylcysteine 1200 mg or 2400 mg a day. |
| Intervention type | Drug |
| Pharmaceutical study type(s) | |
| Phase | Not Specified |
| Drug / device / biological / vaccine name(s) | N-acetylcysteine |
| Primary outcome measure | 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 study start date | 01/10/2007 |
| Completion date | 31/12/2008 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Lower age limit | 18 Years |
| Sex | Not Specified |
| Target number of participants | 10 |
| Key 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 |
| Key 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 |
| Date of first enrolment | 01/10/2007 |
| Date of final enrolment | 31/12/2008 |
Locations
Countries of recruitment
- Netherlands
Study participating centre
Academic Medical Centre (AMC)
Amsterdam
1100 DD
Netherlands
1100 DD
Netherlands
Sponsor information
CURAMA Study Group (The Netherlands)
Research organisation
Research organisation
c/o Dr B.J. Biemond
Academic Medical Centre (AMC)
Department of Clinical Chemistry
P.O. Box 22660
Amsterdam
1100 DD
Netherlands
Funders
Funder type
Research organisation
CURAMA Study Group (The Netherlands)
No information available
Results and Publications
| Intention to publish date | |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | Not provided at time of registration |
| IPD sharing plan |
