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

Hematologic Diseases Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03252925
• Other study ID #: SOOCHOW-HY-2017
• Status: Completed
• Phase: Phase 3
• Start date: November 1, 2017
• Est. completion date: October 1, 2021

Study information

• Verified date: December 2021
• Source: The First Affiliated Hospital of Soochow University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 170
• Est. completion date: October 1, 2021
• Est. primary completion date: October 1, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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;

Related Conditions & MeSH terms

• Hematologic Diseases
• Thrombotic Microangiopathies
• Vascular Diseases

Intervention

Drug:
• N-Acetylcysteine
50mg/Kg.d, oral
• Placebo Oral Tablet
50mg/Kg.d, oral

Locations

Country	Name	City	State
China	The First affiliated Hospital of SooChow University	Suzhou	Jiangsu

Sponsors (1)

Lead Sponsor	Collaborator
The First Affiliated Hospital of Soochow University

Country where clinical trial is conducted

China, 

References & Publications (4)

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. pii: bcr2016215491. doi: 10.1136/bcr-2016-215491. — View Citation

George JN, López 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. — View 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. — View 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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The Incidence of TA-TMA.	The incidence of TMA after HSCT.	100 days
Secondary	The Level of VWF Multimers	The level of VWF multimers in patients post HSCT.	100 days
Secondary	The Level of Endothelial Micro Particle	The level of endothelial micro particle in patients post HSCT.	100 days
Secondary	The Level of TNF-a	The level of TNF-a in patients post HSCT.	40 days
Secondary	The Level of ROS	The level of ROS in patients post HSCT.	100 days