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

Phase 3

Completed

• Conditions: Thrombotic Microangiopathies; Hematologic Diseases
• Interventions: Drug: Placebo Oral Tablet; Drug: N-Acetylcysteine

• Registration Number: NCT03252925
• Lead Sponsor: The First Affiliated Hospital of Soochow University

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.

Study Timeline

• Study First Submitted: 2017-05-22
• Study First Submitted QC: 2017-08-16
• Study First Posted: 2017-08-17
• Study Start: 2017-11-01
• Primary Completion: 2021-10-01
• Study Completion: 2021-10-01
• Status Verified: 2021-12
• Results First Submitted: 2022-01-03
• Results First Submitted QC: 2022-01-19
• Last Update Submitted: 2022-01-19
• Results First Posted: 2022-02-14
• Last Update Posted: 2022-02-14

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 170

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Control Group	Placebo Oral Tablet	Placebo Oral Tablet
Experimental Group	N-Acetylcysteine	N-Acetylcysteine

Primary Outcome Measures

Name	Time	Method
The Incidence of TA-TMA.	100 days	The incidence of TMA after HSCT.

Secondary Outcome Measures

Name	Time	Method
The Level of Endothelial Micro Particle	100 days	The level of endothelial micro particle in patients post HSCT.
The Level of TNF-α	40 days	The level of TNF-α in patients post HSCT.
The Level of ROS	100 days	The level of ROS in patients post HSCT.
The Level of VWF Multimers	100 days	The level of VWF multimers in patients post HSCT.

Trial Locations

Locations (1)

The First affiliated Hospital of SooChow University; 🇨🇳 Suzhou, Jiangsu, China