N-Acetylcysteine in Patients With Sickle Cell Disease

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.

Conditions

• Sickle Cell Disease

Interventions

DRUG

N-Acetylcysteine

DRUG

Placebo

Sponsors & Collaborators

• Erasmus Medical Center

  collaborator OTHER

• Haga Hospital

  collaborator OTHER

• University Medical Center Groningen

  collaborator OTHER

• ZonMw: The Netherlands Organisation for Health Research and Development

  collaborator OTHER

• Fonds NutsOhra

  collaborator OTHER

• Stichting Janivo

  collaborator UNKNOWN

• CHU Brugmann, Brussels

  collaborator OTHER

• Erasme University Hospital

  collaborator OTHER

• Centre Hospitalier Régional de la Citadelle

  collaborator OTHER

• University Hospital St Luc, Brussels

  collaborator OTHER

• Centre Hospitalier Universitaire Saint Pierre

  collaborator OTHER

• Queen Fabiola Children's University Hospital

  collaborator OTHER

• Guy's and St Thomas' NHS Foundation Trust

  collaborator OTHER

• Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

  lead OTHER

Principal Investigators

• Bart Biemond, MD, PhD · Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

• Karin Fijnvandraat, MD, PhD · Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

Study Design

Allocation

RANDOMIZED

Purpose

PREVENTION

Masking

QUADRUPLE

Model

PARALLEL

Eligibility

Min Age

12 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2013-04-30

Primary Completion

2016-06-30

Completion

2016-06-30

Countries

• Belgium
• Netherlands
• United Kingdom

Study Locations