N-Acetylcysteine in Patients With Sickle Cell Disease

• Status: Completed
• Trial end date: 2016-06-01
• Target enrollment: 0
• Participant gender: All

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
• Accepts Healthy Volunteers?: No

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
• Treatments: Acetylcysteine; N-monoacetylcystine

Criteria

Inclusion Criteria:

• Age 12 years or older

• Sickle cell disease, either homozygous sickle cell disease (HbSS), compound heterozygous sickle cell disease (HbSC), HbSβ0 or HbSβ+ thalassemia

• History of at least 1.0 painful crisis per year in the past 3 years (visit to medical facility is not required)

Exclusion Criteria:

• Chronic blood transfusion or transfusion in the preceding 3 months

• Painful crisis in the last 4 weeks (with respect to the moment of inclusion)

• Pregnancy, breast feeding or the desire to get pregnant in the following 7 months

• Known active gastric/duodenal ulcers

• Hydroxycarbamide (HC) treatment with unstable dose in the last 3 months or started on HC shorter then 6 months prior to study

• Known poor compliance in earlier trials regarding the completion of pain diaries

• Insufficient compliance in run-in period

• Known hypersensitivity to acetylcysteine or one of the other components of the study medication

• Use of pain medication for sickle-cell related pains on more than 15 days per month in the past 6 months ('chronic pain').