AZA Combined With NAC for PIT After HSCT

November 8, 2021 updated by: The First Affiliated Hospital of Soochow University

Azacitidine (AZA) Combined With N-Acetyl-L-cysteine (NAC) for Prolonged Isolated Thrombocytopenia (PIT) After Hematopoietic Stem Cell Transplantation (HSCT)

Prolonged isolated thrombocytopenia (PIT) that is refractory to conventional treatments has remained a critical complication after allogeneic hematopoietic cell transplantation since decades years ago. Recombinant human thrombopoietin (rhTPO) is the main therapy in clinical practice, but remains low efficiency for PIT. Demethylating drugs have shown thier potential in high-risk myelodysplastic syndromes (MDS) and acte myeloid leukemia (AML). In addition, decitabine has demonstrated its efficacy of over 70% for response rate in treatment for PIT in early clinical trials with elusive mechanism. Preliminary experiments revealed that PIT was associated with abnormality of oxidation microenvironment, and N-Acetyl-L-cysteine (NAC) was the most commonly used antioxidant. Therefore, the investigators have been wondering whether Azacitidine in combination with NAC could improve PIT post HSCT and explore the possible mechanism of it.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Detailed Description

Prolonged isolated thrombocytopenia (PIT) that is refractory to conventional treatments has remained a critical complication after allogeneic hematopoietic cell transplantation since decades years ago. Recombinant human thrombopoietin (rhTPO) is the main therapy in clinical practice, but remains low efficiency for PIT. Demethylating drugs have shown thier potential in high-risk myelodysplastic syndromes (MDS) and acte myeloid leukemia (AML). In addition, decitabine has demonstrated its efficacy of over 70% for response rate in treatment for PIT in early clinical trials with elusive mechanism. Preliminary experiments revealed that PIT was associated with abnormality of oxidation microenvironment, and N-Acetyl-L-cysteine (NAC) was the most commonly used antioxidant. Furthermore, AZA had shown its potential in immune regulation. Therefore, the investigators have been wondering whether Azacitidine in combination with NAC could improve PIT post HSCT and explore the possible mechanism of it.

Study Type

Interventional

Enrollment (Anticipated)

100

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

  • Name: Depei Wu, PhD,MD

Study Locations

  • China
    • Jiangsu
      • Suzhou, Jiangsu, China, 215000
        • The First Affiliated Hospital of Soochow University
        • Contact:
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 65 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Platelet count ≤ 30 × 10^9/L persistently at day 60 post-HSCT or later;
  • Neutrophil and hemoglobin were well recovered;
  • Full donor chimerism was achieved;

Exclusion Criteria:

  • Patients with malignancy relapse;
  • Active infections;
  • Grade Ⅲ-Ⅳ acute graft-versus-host disease or severe chronic graft-versus-host disease according to National Institute of Health criteria;
  • Severe organ damage;
  • Thrombosis requiring treatment;

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
No Intervention: Control group
receive supportive care
Experimental: AZA+NAC group
AZA 50mg Subcutaneous daily d1-d5 + NAC 600mg oral bid d1-28， 28 days for one cycle
Drug: Azacitidine
28 days for one cycle, evaluation post 3 cycles of treatment. Continue if response exists, otherwise, quit the trial and seek for other therapies.
Other Names:
  • AZA
Drug: N Acetyl L Cysteine
28 days for one cycle, evaluation post 3 cycles of treatment. Continue if response exists, otherwise, quit the trial and seek for other therapies.
Other Names:
  • NAC

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
platelet reconstruction
Time Frame: From date of randomization until the date of platelet reconstruction, assessed up to 100 days
platelet count above 50*10^9/L independent of transfusion
From date of randomization until the date of platelet reconstruction, assessed up to 100 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
overall survival
Time Frame: From date of randomization until the date of death from any cause, assessed up to 1 year
the time from the date of day 1 post HSCT to the date of death due to any cause
From date of randomization until the date of death from any cause, assessed up to 1 year
overall response rate
Time Frame: From date of randomization until the date of platelet count between 30*10^9/L and 50*10^9/L, assessed up to 100 days
platelet count evaluating above 30*10^9/L but below 50*10^9/L independent of platelet transfusion
From date of randomization until the date of platelet count between 30*10^9/L and 50*10^9/L, assessed up to 100 days

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
infection
Time Frame: From date of randomization until the date of diagnose of infection, assessed up to 100 days
clinical diagnose of infection or evidence of next generation of sequencing（NGS）of body fluid or CT or culture of samples collected from patients
From date of randomization until the date of diagnose of infection, assessed up to 100 days
Severe adverse events
Time Frame: From date of randomization until the date of occurrence of any severe adverse event, assessed up to 100 days
any events that prevent the clinical trial continue besides hepatic injury renal function impairment, et al.
From date of randomization until the date of occurrence of any severe adverse event, assessed up to 100 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

The First Affiliated Hospital of Soochow University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Anticipated)

December 1, 2021

Primary Completion (Anticipated)

June 30, 2024

Study Completion (Anticipated)

June 30, 2026

Study Registration Dates

First Submitted

July 30, 2021

First Submitted That Met QC Criteria

November 8, 2021

First Posted (Actual)

November 18, 2021

Study Record Updates

Last Update Posted (Actual)

November 18, 2021

Last Update Submitted That Met QC Criteria

November 8, 2021

Last Verified

April 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SOOCHOW-HY-2021-2

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Stem Cell Transplant Complications

Clinical Trials on Azacitidine

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Malaysia

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe