N-Acetylcysteine in Patients With Sickle Cell Disease
Status:
Completed
Trial end date:
2016-06-01
Target enrollment:
Participant gender:
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. The investigators expect
that NAC can reduce the frequency of pain in patients with SCD, thereby improving their
quality of life and participation in society.
Phase:
Phase 3
Details
Lead Sponsor:
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Collaborators:
Centre Hospitalier Régional de la Citadelle Centre Hospitalier Universitaire Saint Pierre CHU Brugmann, Brussels Erasme University Hospital Erasmus Medical Center Fonds NutsOhra Guy's and St Thomas' NHS Foundation Trust Haga Hospital Queen Fabiola Children's University Hospital Stichting Janivo University Hospital St Luc, Brussels University Medical Center Groningen ZonMw: The Netherlands Organisation for Health Research and Development