NCT01808521

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
3

participants targeted

Target at below P25 for early_phase_1

Timeline
Completed

Started May 2013

Longer than P75 for early_phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 22, 2013

Completed
17 days until next milestone

First Posted

Study publicly available on registry

March 11, 2013

Completed
2 months until next milestone

Study Start

First participant enrolled

May 1, 2013

Completed
4.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2017

Completed
Last Updated

September 20, 2017

Status Verified

September 1, 2017

Enrollment Period

4.2 years

First QC Date

February 22, 2013

Last Update Submit

September 18, 2017

Conditions

Purpura, Thrombotic ThrombocytopenicTTP

Keywords

Purpura, Thrombotic ThrombocytopenicTTP

Outcome Measures

Primary Outcomes (1)

  • Changes in platelet count

    The platelet count will be measured before, daily during 4 days of NAC infusion, the subsequent 3 days and on the day of hospital discharge which is estimated to be at 1-2 weeks post infusion. Changes in platelet count over time will be reported.

    Daily for 7 days and at hospital discharge, expected to be at 1-2 weeks post infusion.

Secondary Outcomes (4)

  • Laboratory measures of VWF activity

    Daily for 7 days and at hospital dischargewhich is estimated to be at 1-2 weeks post-infusion

  • Laboratory measures of ADAMTS13 activity

    Daily for 7 days and at hospital discharge which is estimated to be at 1-2 weeks post-infusion

  • Laboratory measures of red blood cell (RBC) hemolysis and oxidation

    Daily for 7 days and at hospital discharge which is estimated to be at 1-2 weeks post-infusion

  • Safety of NAC infusion

    Over the study period

Study Arms (1)

N-acetylcysteine

EXPERIMENTAL

IV administration of N-acetylcysteine (Acetadote) at 150mg/Kg loading bolus over 60 minutes followed by 150mg/Kg over 17 hours if loading dose was well tolerated.

Drug: N-Acetylcysteine

Interventions

N-AcetylcysteineDRUG

IV administration of N-Acetylcysteine at 150mg/kg over 60 min first, then if well tolerated, 150mb/kg over 17 hours

Also known as: Acetadote
N-acetylcysteine

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Age \>= 18 years of age
  • Diagnosis of suspected TTP (lab evidence of hemolysis, platelet count \<120,000, schistocytes on peripheral smear)
  • Plans for or just initiated therapeutic plasma exchange (TPE), and before 3rd TPE
  • Normal baseline prothrombin time (PT) and activated partial thromboplastin time (aPTT)
  • Anticipated TPE for \> 5 days

You may not qualify if:

  • Asthma
  • Life expectancy \< 1 week
  • Liver function tests abnormal- (ALT, direct bilirubin \> three times upper normal limit)
  • Known underlying bleeding disorder
  • Pregnancy or nursing
  • Known allergy to NAC
  • Phosphodiesterase Type 5 inhibitors, nitroglycerin, or carbamazepine current use

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Puget Sound Blood Center

Seattle, Washington, 98104, United States

Location

MeSH Terms

Conditions

Purpura, Thrombotic Thrombocytopenic

Interventions

Acetylcysteine

Condition Hierarchy (Ancestors)

Purpura, ThrombocytopenicPurpuraBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesThrombotic MicroangiopathiesThrombocytopeniaBlood Platelet DisordersCytopeniaThrombophiliaHemorrhagePathologic ProcessesPathological Conditions, Signs and SymptomsSkin ManifestationsSigns and Symptoms

Intervention Hierarchy (Ancestors)

CysteineAmino Acids, SulfurSulfur CompoundsOrganic ChemicalsAmino AcidsAmino Acids, Peptides, and Proteins

Study Officials

  • Barbara A Konkle, MD

    Bloodworks

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
early phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 22, 2013

First Posted

March 11, 2013

Study Start

May 1, 2013

Primary Completion

July 1, 2017

Study Completion

July 1, 2017

Last Updated

September 20, 2017

Record last verified: 2017-09

Locations

US(1)