Ignite Creation Date:
2024-05-06 @ 1:26 AM
Last Modification Date:
2024-10-26 @ 11:03 AM
Study NCT ID:
NCT01800526
Status:
COMPLETED
Last Update Posted:
2020-07-09
First Post:
2013-01-21
Brief Title:
A Pilot Study of N-acetylcysteine in Patients With Sickle Cell Disease
Sponsor:
Bloodworks
Organization:
Bloodworks
Study Overview
Official Title:
A Pilot Study of N-acetylcysteine in Patients With Sickle Cell Disease
Status:
COMPLETED
Status Verified Date:
2020-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
NACinSCD
Brief Summary:
Part 1 A pilot study in patients with homozygous S HbSS or hemoglobin S with beta zero thalassemiaHbS-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
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
Detailed Description:
Two primary processes dominate the complications associated with sickle cell disease SCD vasoocclusion and hemolysis The plasma and vessel wall adhesive protein von Willebrand factor VWF is thought to be involved in both of these processes so strategies aimed at reducing its secretion or reactivity which could decrease complications in patients with SCD are being tested
Based on prior studies N-acetylcysteine NAC treatment may decrease VWF activity in patients with SCD and may be a useful adjunctive treatment in this disorder
Part 1 enrolls stable outpatients with homozygous S HbSS or hemoglobin S with beta zero thalassemia HbS-βo thalassemia with the aim of examining the effect of NAC treatment on VWF parameters measures of oxidation and RBC fragments Patients receive IV NAC first at 150 mgkg over 8 hours and if tolerated at a later date at 300 mgkg over 8 hours in the University of Washington Clinical Research Center Blood is collected for laboratory assessment Subjects are later offered enrollment in an oral phase
Part 2 patients with a history of vaso-occlusive crisis VOC are approached in the outpatient setting to discuss the study When admitted for VOC subjects receive NAC as an IV infusion75 mgkg every 6 hours for up to 5 days Blood for laboratory assays are collected each morning and pain assessment is performed prior to and following each NAC infusion
Based on prior studies N-acetylcysteine NAC treatment may decrease VWF activity in patients with SCD and may be a useful adjunctive treatment in this disorder
Part 1 enrolls stable outpatients with homozygous S HbSS or hemoglobin S with beta zero thalassemia HbS-βo thalassemia with the aim of examining the effect of NAC treatment on VWF parameters measures of oxidation and RBC fragments Patients receive IV NAC first at 150 mgkg over 8 hours and if tolerated at a later date at 300 mgkg over 8 hours in the University of Washington Clinical Research Center Blood is collected for laboratory assessment Subjects are later offered enrollment in an oral phase
Part 2 patients with a history of vaso-occlusive crisis VOC are approached in the outpatient setting to discuss the study When admitted for VOC subjects receive NAC as an IV infusion75 mgkg every 6 hours for up to 5 days Blood for laboratory assays are collected each morning and pain assessment is performed prior to and following each NAC infusion
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None