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

NCT03252925 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: SOOCHOW-HY-2017
• Study Type: interventional
• Target: 170
• Locations: 1 country
• Sites: 1

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.

Conditions

Thrombotic Microangiopathies; Hematologic Diseases

Keywords

N-acetylcysteine; Complement; Thrombotic Microangiopathies

Outcome Measures

Primary Outcomes (1)

• The Incidence of TA-TMA.

  The incidence of TMA after HSCT.

  100 days

Secondary Outcomes (4)

• The Level of VWF Multimers

  100 days

• The Level of Endothelial Micro Particle

  100 days

• The Level of TNF-α

  40 days

• The Level of ROS

  100 days

Study Arms (2)

Experimental Group

EXPERIMENTAL

N-Acetylcysteine

Drug: N-Acetylcysteine

Control Group

PLACEBO COMPARATOR

Placebo Oral Tablet

Drug: Placebo Oral Tablet

Interventions

N-Acetylcysteine DRUG

50mg/Kg.d, oral

Also known as: NAC

Experimental Group

Placebo Oral Tablet DRUG

50mg/Kg.d, oral

Also known as: Placebo

Control Group

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• Be sensitive to NAC;
• Bronchial asthma；
• Peptic ulcer；
• Severe organ damage；
• Pregnancy and breastfeeding women；

Sponsors & Collaborators

• The First Affiliated Hospital of Soochow University: lead

Study Sites (1)

The First affiliated Hospital of SooChow University

Suzhou, Jiangsu, 215006, China

Location

Related Publications (4)

• 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.

  PMID: 24819072 BACKGROUND

• 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.

  PMID: 28011677 BACKGROUND

• 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.

  PMID: 27284100 BACKGROUND

• 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.

  PMID: 26245827 BACKGROUND

MeSH Terms

Conditions

Thrombotic Microangiopathies; Hematologic Diseases

Interventions

Acetylcysteine

Condition Hierarchy (Ancestors)

Thrombocytopenia; Blood Platelet Disorders; Hemic and Lymphatic Diseases; Cytopenia

Intervention Hierarchy (Ancestors)

Cysteine; Amino Acids, Sulfur; Sulfur Compounds; Organic Chemicals; Amino Acids; Amino Acids, Peptides, and Proteins

Limitations and Caveats

Several outcome indicators are entirely inconsistent with our study's actual course of execution. First, the occurrence of TA-TMA was not limited to the first 100 days after hematopoietic stem cell transplantation. Secondly, the detection of VWF multimers, endothelial microparticles, and ROS was not performed according to the protocol.

Results Point of Contact

• Title: Yue Han
• Organization: The First Affiliated Hospital of Soochow University

hanyue@suda.edu.cn

13901551669

Study Officials

• Depei Wu, MD. PhD.

  The First Affiliated Hospital of Soochow University

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 22, 2017
• First Posted: August 17, 2017
• Study Start: November 1, 2017
• Primary Completion: October 1, 2021
• Study Completion: October 1, 2021
• Last Updated: February 14, 2022
• Results First Posted: February 14, 2022

Record last verified: 2021-12

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)