Overview

A Pilot Study of N-acetylcysteine in Thrombotic Thrombocytopenia Purpura

Status:
Completed
Trial end date:
2017-07-01
Target enrollment:
0
Participant gender:
All
Summary
In this study, the investigators want to determine if N-acetylcysteine(NAC), 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 platelet/VWF strings. This will be manifest by a more rapid improvement in the patient's 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.
Phase:
Early Phase 1
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Bloodworks
Bloodworks (Puget Sound Blood Center)
Collaborator:
University of Washington
Treatments:
Acetylcysteine
N-monoacetylcystine
Criteria
Inclusion Criteria:

1. Age >= 18 years of age

2. Diagnosis of suspected TTP (lab evidence of hemolysis, platelet count <120,000,
schistocytes on peripheral smear)

3. Plans for or just initiated therapeutic plasma exchange (TPE), and before 3rd TPE

4. Normal baseline prothrombin time (PT) and activated partial thromboplastin time (aPTT)

5. Anticipated TPE for > 5 days

Exclusion Criteria:

1. Asthma

2. Life expectancy < 1 week

3. Liver function tests abnormal- (ALT, direct bilirubin > three times upper normal
limit)

4. Known underlying bleeding disorder

5. Pregnancy or nursing

6. Known allergy to NAC

7. Phosphodiesterase Type 5 inhibitors, nitroglycerin, or carbamazepine current use