AZA Combined With NAC for PIT After HSCT

ConditionsThrombocytopenia, Isolated Stem Cell Transplant Complications

Trial statusNot yet recruiting

Trial phasePhase 2

Trial typeInterventional

Biological sexAll

Age18-65

SponsorThe First Affiliated Hospital of Soochow University

About this trial

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.

Eligibility criteria

This trial accepts healthy volunteers

Qualifiers

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;

Disqualifiers

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;

Trial design

Design model

Parallel

Treatments tested in this trial

Azacitidine
Drug
28 days for one cycle, evaluation post 3 cycles of treatment. Continue if response exists, otherwise, quit the trial and seek for other therapies.
N Acetyl L Cysteine
Drug
28 days for one cycle, evaluation post 3 cycles of treatment. Continue if response exists, otherwise, quit the trial and seek for other therapies.

Treatment groups

100 Participants

are divided into 2 treatment groups

Group A: Control groupNo intervention 0 interventions

Group B: AZA+NAC groupExperimental treatment 2 interventions

Trial outcomes

Primary outcomes

platelet reconstruction

platelet count above 50\*10\^9/L independent of transfusion

Time frame

From date of randomization until the date of platelet reconstruction, assessed up to 100 days

Secondary outcomes

overall survival

the time from the date of day 1 post HSCT to the date of death due to any cause

Time frame

From date of randomization until the date of death from any cause, assessed up to 1 year

overall response rate

platelet count evaluating above 30\*10\^9/L but below 50\*10\^9/L independent of platelet transfusion

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

Other outcomes

infection

clinical diagnose of infection or evidence of next generation of sequencing（NGS）of body fluid or CT or culture of samples collected from patients

Time frame

From date of randomization until the date of diagnose of infection, assessed up to 100 days

Severe adverse events

any events that prevent the clinical trial continue besides hepatic injury renal function impairment, et al.

Time frame

From date of randomization until the date of occurrence of any severe adverse event, assessed up to 100 days

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

The First Affiliated Hospital of Soochow University

Lead sponsor

Source ClinicalTrials.gov

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