drugset / Trial / NCT01800526
A Pilot Study of N-acetylcysteine in Patients With Sickle Cell Disease
Non-randomizedSingle-groupOpen-labelTreatment
Summary
Part 1: A pilot study in patients with homozygous S (HbSS) or hemoglobin S with beta zero thalassemia(HbS-βo thalassemia), with the aim of examining the effect of intravenous NAC treatment on plasma VWF parameters and measures of redox and RBC function. Part 2: A pilot study in patients with sickle cell disease admitted to the hospital in vaso-occlusive crisis to determine the effects of NAC infusions on plasma VWF parameters and measures of redox and RBC function, and on measures of pain and hospital length of stay.
Timeline
- Start
- 2013-03
- Primary completion
- 2020-01-24
- Completion
- 2020-06-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | N-acetylcysteine | Small molecule | 75 mg/kg | Oral |
| Subject | N-acetylcysteine | Small molecule | 150 mg/kg | Oral |
| Subject | N-acetylcysteine | Small molecule | 300 mg/kg | Oral |
| Subject | N-acetylcysteine | Small molecule | 1200 mg | Oral |