Prolonged Isolated Thrombocytopenia After Allo-SCT : N-acetyl-L-cysteine (NAC) Versus Supportive Therapy

NCT ID: NCT03391856

Last Updated: 2020-09-25

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-03-01
• Study Completion Date: 2018-12-30

Brief Summary

Isolated prolonged thrombocytopenia (PT) is a common complication after allogeneic stem cell transplantation with significant poor prognosis. No standard treatment is available. The current study assigned PT randomly to 2 arms: intervention arm with N-acetyl-L-cysteine (NAC) and control arm with supportive therapy.This is a prospective randomized controlled study.

Detailed Description

1. patients diagnosed with PT at day 60 post transplant will be randomized assigned to intervention arm (NAC) or controlled arm (supportive therapy: prophylactic platelet transfusion was given when platelet count <20000/ul.)

2. Response will be evaluated at day 90. Response was defined as platelet recovery to >= 20000/ul for 7 consecutive days without transfusion support during the enrollment period. All the other patients not achieved above criteria was defined as no response.

3. For those without response in both arms, patients will received NAC plus recombinant human thrombopoietin (rhTPO) for an additional 30 days. rhTPO was given at 300IU/kg/d for 28 consecutive days or platelet > 50000/ul independent of platelet transfusion.

Conditions

Thrombocytopenia; Stem Cell Transplant Complications

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

intervention arm

NAC 400mg p.o tid from day 60 to day 90 post transplant

Group Type: EXPERIMENTAL

N-acetyl-L-cysteine (NAC)

Intervention Type: DRUG

NAC treatment for PT: 400mg p.o tid from day 60 to 90 post transplant

controlled arm

Supportive therapy including platelet infusion:prophylactic platelet transfusion was given when platelet count \<20000/ul

Group Type: OTHER

supportive therapy

Intervention Type: OTHER

prophylactic platelet transfusion was given when platelet count \<20000/ul

Interventions

N-acetyl-L-cysteine (NAC)

NAC treatment for PT: 400mg p.o tid from day 60 to 90 post transplant

Intervention Type: DRUG

supportive therapy

prophylactic platelet transfusion was given when platelet count \<20000/ul

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

• age>= 14 years
• diagnosed as PT (defined as platelet count in peripheral blood never achieve 20000/miro-liter for 7 consecutive days without transfusion at day 60 post transplantation)
• serum creatine level < ULN (upper limit of normal), serum ALT/AST /TBil<=2 ULN
• without active CMV/EBV/ADV reactivation, active GVHD, without relapse or minimal residual disease at enrollment

Exclusion Criteria

• history of asthma
• allergy to NAC
• refuse to participate

• Minimum Eligible Age: 14 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

The First Affiliated Hospital of Soochow University

OTHER

Sponsor Role: collaborator

Nanfang Hospital, Southern Medical University

OTHER

Sponsor Role: collaborator

Peking University People's Hospital

OTHER

Sponsor Role: lead

Responsible Party

Xiaojun Huang,MD

Head of Peking University Institute of Hematology

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Xiao-Jun Huang, MD

Role: PRINCIPAL_INVESTIGATOR

Peiking University

Locations

Peking University People's Hospital

Beijing, Beijing Municipality, China

Countries

China

Other Identifiers

2017PHB220

Identifier Type: -

Identifier Source: org_study_id