A Safety and Efficacy Study of NAC in Patients With TA-TMA

January 19, 2022 updated by: The First Affiliated Hospital of Soochow University

A Safety and Efficacy Study of N-acetylcysteine in Patients With Transplant-Associated Thrombotic Microangiopathy

HSCT associated thrombotic microangiopathy(TA-TMA) is a heterogeneous, fatal disorder seen within 100 days post-transplant and presents with thrombocytopenia, hemolysis, acute renal failure, mental status changes and involvement of other organs. N-Acetylcysteine (NAC) is a small, simple molecule that began as a generic drug almost 40 years ago. It has since been approved by the FDA for many indications. The investigators conducted an prospective clinical trial to evaluate the safety and efficiency of NAC in patients with TA-TMA.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

Hematopoietic stem cell transplantation (HSCT) has been commonly used as a potentially curative option in the treatment of various hematological malignancies. However, it may end up with serious complications in various systems, including the hemostatic system. HSCT associated thrombotic microangiopathy(TA-TMA) is a heterogeneous, fatal disorder seen within 100 days post-transplant and presents with thrombocytopenia, hemolysis, acute renal failure, mental status changes and involvement of other organs. Since the pathophysiology has not been clarified, there are no established treatments for TA-TMA several agents seem to have successful results. N-Acetylcysteine (NAC) is a small, simple molecule that began as a generic drug almost 40 years ago. It has since been approved by the FDA for many indications. Studies showed NAC reduce plasma VWF multimers and VWF multimeric size without an effect on the bleeding time in vitro and in vivo, thus proposed as a possible supplementary treatment for TTP. The investigators conducted an prospective clinical trial to evaluate the safety and efficiency of NAC in patients with TA-TMA.

Study Type

Interventional

Enrollment (Actual)

170

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
    • Jiangsu
      • Suzhou, Jiangsu, China, 215006
        • The First Affiliated Hospital of Soochow University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Patients be scheduled to undergo HSCT;
  2. Not received decitabine 6 month ago;
  3. Without severe organ damage;
  4. ECOG 0-2;
  5. Informed consent were obtained.

Exclusion Criteria:

  1. Be sensitive to NAC;
  2. Bronchial asthma；
  3. Peptic ulcer；
  4. Severe organ damage；
  5. Pregnancy and breastfeeding women；

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Experimental Group
N-Acetylcysteine
Drug: N-Acetylcysteine
50mg/Kg.d, oral
Other Names:
  • NAC
Placebo Comparator: Control Group
Placebo Oral Tablet
Drug: Placebo Oral Tablet
50mg/Kg.d, oral
Other Names:
  • Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The Incidence of TA-TMA.
Time Frame: 100 days
The incidence of TMA after HSCT.
100 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The Level of VWF Multimers
Time Frame: 100 days
The level of VWF multimers in patients post HSCT.
100 days
The Level of Endothelial Micro Particle
Time Frame: 100 days
The level of endothelial micro particle in patients post HSCT.
100 days
The Level of TNF-α
Time Frame: 40 days
The level of TNF-α in patients post HSCT.
40 days
The Level of ROS
Time Frame: 100 days
The level of ROS in patients post HSCT.
100 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

The First Affiliated Hospital of Soochow University

Investigators

  • Study Chair: Depei Wu, MD. PhD., The First Affiliated Hospital of Soochow University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 1, 2017

Primary Completion (Actual)

October 1, 2021

Study Completion (Actual)

October 1, 2021

Study Registration Dates

First Submitted

May 22, 2017

First Submitted That Met QC Criteria

August 16, 2017

First Posted (Actual)

August 17, 2017

Study Record Updates

Last Update Posted (Actual)

February 14, 2022

Last Update Submitted That Met QC Criteria

January 19, 2022

Last Verified

December 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SOOCHOW-HY-2017

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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