Ignite Creation Date:
2024-05-06 @ 1:25 AM
Last Modification Date:
2024-10-26 @ 11:04 AM
Study NCT ID:
NCT01808521
Status:
COMPLETED
Last Update Posted:
2017-09-20
First Post:
2013-02-22
Brief Title:
A Pilot Study of N-acetylcysteine in Thrombotic Thrombocytopenia Purpura
Sponsor:
Bloodworks
Organization:
Bloodworks
Study Overview
Official Title:
A Pilot Study of N-acetylcysteine in Suspected Thrombotic Thrombocytopenia Purpura
Status:
COMPLETED
Status Verified Date:
2017-09
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:
NACinTTP
Brief Summary:
In this study the investigators want to determine if N-acetylcysteineNAC given intravenously will decrease complications in patients with Thrombotic Thrombocytopenia Purpura TTP who are receiving treatment with therapeutic plasma exchange TPE The investigators want to determine through anti-oxidant activity if NAC will have additional efficacy in TTP by improving cleavage of the patients VWF by ADAMTS13 and preventing propagation of plateletVWF strings This will be manifest by a more rapid improvement in the patients platelet count decrease in number of days requiring TPE and decrease in microvascular thrombotic complications The investigators will additionally 1 Assess safety of NAC by evaluating subjects for adverse events and significant adverse events 2 Determine effects on TTP by measuring clinical and research laboratory values 3 Determine drug effects by measuring clinical and research laboratory values
Detailed Description:
Thrombotic thrombocytopenic purpura TTP is a rare hemostatic disorder with life threatening consequences secondary to microvascular thrombosis While the use of therapeutic plasma exchange TPE has greatly improved survival end organ damage resistance to therapy and relapses occur in many patients Ultra-large von Willebrand factor multimers ULVWF are pathogenic in TTP The investigators have found that N-acetylcysteine NAC cleaves ULVWF in vitro and in vivo in the ADAMTS13 deficient mice that are at increased risk of TTP NAC is well tolerated in humans at intravenous doses used for treatment of acetaminophen overdose This dosage correlates with that producing an effect in the murine studies noted above and thus is an attractive treatment for patients with TTP By cleaving VWF and preventing propagation of plateletVWF strings the investigators hypothesize that NAC treatment will decrease complications in patients with TTP receiving treatment with TPE This will be manifest by a more rapid improvement in platelet count decrease in number of days requiring plasma exchange and decrease in microvascular thrombotic complications To prepare for a larger trial the investigators propose a pilot study in 3 patients with suspected TTP at the University of Washington UW Medical Center The study will be approved by the UW IRB prior to study initiation Patients who consent to the study will receive daily NAC infusions beginning after the first TPE in doses used for acetaminophen overdose Blood samples will be collected for laboratory assays to determine optimal timing for sample collection in the larger multicenter trial and to pilot the data collection forms The investigators will also evaluate safety and patient tolerability
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