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

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

170 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-11-01

Study Completion Date

2021-10-01

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Thrombotic Microangiopathies Hematologic Diseases

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

PREVENTION

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Experimental Group

N-Acetylcysteine

Group Type EXPERIMENTAL

N-Acetylcysteine

Intervention Type DRUG

50mg/Kg.d, oral

Control Group

Placebo Oral Tablet

Group Type PLACEBO_COMPARATOR

Placebo Oral Tablet

Intervention Type DRUG

50mg/Kg.d, oral

Interventions

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N-Acetylcysteine

50mg/Kg.d, oral

Intervention Type DRUG

Placebo Oral Tablet

50mg/Kg.d, oral

Intervention Type DRUG

Other Intervention Names

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NAC Placebo

Eligibility Criteria

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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.