No registrations found.
ID
Source
Brief title
Health condition
Sickle Cell Disease
Sickle Cell Anemia
Painful Crisis
Pain
Sikkelcel ziekte
Sikkelcel anemie
Pijnlijke crise
Pijn
Sponsors and support
- Academic Medical Center, Amsterdam
Intervention
Outcome measures
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.
Background 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.
Study objective
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.
Study design
Evaluated over a 6 month intervention period with montly follow-up visits.
Intervention
Experimental:
N-Acetylcysteine (N-Acetylcysteine 600mg 1 oral tablet twice daily during 6 months).
Placebo Comparator:
(Placebo 1 oral tablet twice daily during 6 months).
Academic Medical Center, Amsterdam
J. Sins
Amsterdam
The Netherlands
+31 (0)20 5661693
j.w.sins@amc.nl
Academic Medical Center, Amsterdam
J. Sins
Amsterdam
The Netherlands
+31 (0)20 5661693
j.w.sins@amc.nl
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.
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| NTR-new | NL3660 |
| NTR-old | NTR3806 |
| Other | EudraCT / CCMO : 2012-004892-37 / NL 41205.018.12; |
| ISRCTN | ISRCTN wordt niet meer aangevraagd. |