|- candidate number||14242|
|- NTR Number||NTR3806|
|- ISRCTN||ISRCTN wordt niet meer aangevraagd.|
|- Date ISRCTN created|
|- date ISRCTN requested|
|- Date Registered NTR||22-jan-2013|
|- Secondary IDs||2012-004892-37 / NL 41205.018.12; EudraCT / CCMO|
|- Public Title||N-Acetylcysteine in Patients With Sickle Cell Disease (NAC).|
|- Scientific Title||N-Acetylcysteine in Patients With Sickle Cell Disease: Reducing the Incidence of Daily Life Pain.|
|- ACRONYM||NAC trial|
|- hypothesis||Our primary hypothesis is that the drug N-Acetylcysteine, a scavenger of free oxygen radicals and a precursor of glutathione, can reduce the frequency of daily life pain in patients with Sickle Cell Disease by reducing oxidative stress.
|- Healt Condition(s) or Problem(s) studied||Sickle cell disease, Pain, Painful crisis, Sickle Cell anemia|
|- Inclusion criteria||1. Age 12 years or older;|
2. Sickle cell disease, either homozygous sickle cell disease (HbSS), compound heterozygous sickle cell disease (HbSC), HbSβ0 or HbSβ+ thalassemia;
3. History of at least 1.0 painful crisis per year in the past 3 years (visit to medical facility is not required).
|- Exclusion criteria||1. Chronic blood transfusion or transfusion in the preceding 3 months;|
2. Painful crisis in the last 4 weeks (with respect to the moment of inclusion);
3. Pregnancy, breast feeding or the desire to get pregnant in the following 7 months;
4. Known active gastric/duodenal ulcers;
5. Hydroxycarbamide (HC) treatment with change in dose in the last 3 months or started on HC shorter then 6 months prior to study;
6. Known poor compliance in earlier trials regarding the completion of pain diaries;
7. Insufficient compliance in run-in period;
8. Known hypersensitivity to acetylcysteine or one of the other components of the study medication.
|- mec approval received||yes|
|- multicenter trial||yes|
|- Type||2 or more arms, randomized|
|- planned startdate ||1-mrt-2013|
|- planned closingdate||1-okt-2014|
|- Target number of participants||140|
|- Interventions||Experimental: |
N-Acetylcysteine (N-Acetylcysteine 600mg 1 oral tablet twice daily during 6 months).
(Placebo 1 oral tablet twice daily during 6 months).
|- Primary outcome||The frequency of SCD related pain in daily life in patients with Sickle Cell Disease evaluated over a period of 6 months.|
|- Secondary outcome||1. The severity of SCD related pain in daily life;|
2. The incidence and severity of painful crises;
3. The frequency and length of hospital admissions;
4. The health-related Quality of Life;
5. The SCD-related societal costs;
6. The tolerability of NAC;
7. Frequency of use of pain medication at home;
8. Frequency of SCD complications (e.g. acute chest syndrome);
9. The changes in hematological markers of oxidative stress, hemolysis, hypercoagulability, inflammation, erythrocyte adhesion and endothelial dysfunction.
|- Timepoints||Evaluated over a 6 month intervention period with montly follow-up visits.|
|- Trial web site||N/A|
|- status||open: patient inclusion|
|- CONTACT FOR PUBLIC QUERIES||MD. J. Sins|
|- CONTACT for SCIENTIFIC QUERIES||Dr. B.J. Biemond|
|- Sponsor/Initiator ||Academic Medical Center (AMC), Amsterdam|
(Source(s) of Monetary or Material Support)
|Academic Medical Center (AMC), Amsterdam, ZON-MW, The Netherlands Organization for Health Research and Development|
|- Brief summary||The primary aim of this study is to evaluate the effect of the drug N-Acetylcysteine on the frequency of pain in daily life in patients with Sickle Cell Disease (SCD).
Pain is an invalidating hallmark of this disease and has a considerable impact on the Quality of Life of patients and the medical health care system. Oxidative stress is hypothesized to play a central role in its pathophysiology. In pilot studies the administration of N-Acetylcysteine (NAC) resulted in a reduction of oxidative stress. Moreover, administration of NAC seemed to decrease hospitalization for painful crises in a small pilot study in patients with SCD.
This study will be performed as a multicenter, randomized, controlled trial where patients will be treated with either NAC or placebo for a period of 6 months. We expect that NAC can reduce the frequency of pain in patients with SCD, thereby improving their quality of life and participation in society.
|- Main changes (audit trail)|
|- RECORD||22-jan-2013 - 26-apr-2013|