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

Primary Purpose

Thrombotic Microangiopathies, Hematologic Diseases

Status
Completed
Phase
Phase 3
Locations
China
Study Type
Interventional
Intervention
N-Acetylcysteine
Placebo Oral Tablet
Sponsored by
The First Affiliated Hospital of Soochow University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional prevention trial for Thrombotic Microangiopathies focused on measuring N-acetylcysteine, Complement, Thrombotic Microangiopathies

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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；

Sites / Locations

  • The First affiliated Hospital of SooChow University

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Experimental Group

Control Group

Arm Description

N-Acetylcysteine

Placebo Oral Tablet

Outcomes

Primary Outcome Measures

The Incidence of TA-TMA.
The incidence of TMA after HSCT.

Secondary Outcome Measures

The Level of VWF Multimers
The level of VWF multimers in patients post HSCT.
The Level of Endothelial Micro Particle
The level of endothelial micro particle in patients post HSCT.
The Level of TNF-α
The level of TNF-α in patients post HSCT.
The Level of ROS
The level of ROS in patients post HSCT.

Full Information

First Posted
May 22, 2017
Last Updated
January 19, 2022
Sponsor
The First Affiliated Hospital of Soochow University
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1. Study Identification

Unique Protocol Identification Number
NCT03252925
Brief Title
A Safety and Efficacy Study of NAC in Patients With TA-TMA
Official Title
A Safety and Efficacy Study of N-acetylcysteine in Patients With Transplant-Associated Thrombotic Microangiopathy
Study Type
Interventional

2. Study Status

Record Verification Date
December 2021
Overall Recruitment Status
Completed
Study Start Date
November 1, 2017 (Actual)
Primary Completion Date
October 1, 2021 (Actual)
Study Completion Date
October 1, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
The First Affiliated Hospital of Soochow University

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Thrombotic Microangiopathies, Hematologic Diseases
Keywords
N-acetylcysteine, Complement, Thrombotic Microangiopathies

7. Study Design

Primary Purpose
Prevention
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
170 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Experimental Group
Arm Type
Experimental
Arm Description
N-Acetylcysteine
Arm Title
Control Group
Arm Type
Placebo Comparator
Arm Description
Placebo Oral Tablet
Intervention Type
Drug
Intervention Name(s)
N-Acetylcysteine
Other Intervention Name(s)
NAC
Intervention Description
50mg/Kg.d, oral
Intervention Type
Drug
Intervention Name(s)
Placebo Oral Tablet
Other Intervention Name(s)
Placebo
Intervention Description
50mg/Kg.d, oral
Primary Outcome Measure Information:
Title
The Incidence of TA-TMA.
Description
The incidence of TMA after HSCT.
Time Frame
100 days
Secondary Outcome Measure Information:
Title
The Level of VWF Multimers
Description
The level of VWF multimers in patients post HSCT.
Time Frame
100 days
Title
The Level of Endothelial Micro Particle
Description
The level of endothelial micro particle in patients post HSCT.
Time Frame
100 days
Title
The Level of TNF-α
Description
The level of TNF-α in patients post HSCT.
Time Frame
40 days
Title
The Level of ROS
Description
The level of ROS in patients post HSCT.
Time Frame
100 days

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patients be scheduled to undergo HSCT; Not received decitabine 6 month ago; Without severe organ damage; ECOG 0-2; Informed consent were obtained. Exclusion Criteria: Be sensitive to NAC; Bronchial asthma； Peptic ulcer； Severe organ damage； Pregnancy and breastfeeding women；
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Depei Wu, MD. PhD.
Organizational Affiliation
The First Affiliated Hospital of Soochow University
Official's Role
Study Chair
Facility Information:
Facility Name
The First affiliated Hospital of SooChow University
City
Suzhou
State/Province
Jiangsu
ZIP/Postal Code
215006
Country
China

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
24819072
Citation
George JN, Lopez JA, Konkle BA. N-Acetylcysteine: an old drug, a new insight, a potentially effective treatment for thrombotic thrombocytopenic purpura. Transfusion. 2014 May;54(5):1205-7. doi: 10.1111/trf.12561. No abstract available.
Results Reference
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PubMed Identifier
28011677
Citation
Tersteeg C, Roodt J, Van Rensburg WJ, Dekimpe C, Vandeputte N, Pareyn I, Vandenbulcke A, Plaimauer B, Lamprecht S, Deckmyn H, Lopez JA, De Meyer SF, Vanhoorelbeke K. N-acetylcysteine in preclinical mouse and baboon models of thrombotic thrombocytopenic purpura. Blood. 2017 Feb 23;129(8):1030-1038. doi: 10.1182/blood-2016-09-738856. Epub 2016 Dec 23.
Results Reference
background
PubMed Identifier
27284100
Citation
Acedillo RR, Govind M, Kashgary A, Clark WF. Treatment of severe, refractory and rapidly evolving thrombotic thrombocytopenic purpura. BMJ Case Rep. 2016 Jun 9;2016:bcr2016215491. doi: 10.1136/bcr-2016-215491.
Results Reference
background
PubMed Identifier
26245827
Citation
Rottenstreich A, Hochberg-Klein S, Rund D, Kalish Y. The role of N-acetylcysteine in the treatment of thrombotic thrombocytopenic purpura. J Thromb Thrombolysis. 2016 May;41(4):678-83. doi: 10.1007/s11239-015-1259-6.
Results Reference
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A Safety and Efficacy Study of NAC in Patients With TA-TMA

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